header logo image


Page 300«..1020..299300301302..310320..»

Animal Stem Cell Therapy Market Size, Drivers, Potential Growth Opportunities, Competitive Landscape, Trends And Forecast To 2027 – Eurowire

November 11th, 2020 11:55 am

Animal Stem Cell Therapy Market Overview

The Global Animal Stem Cell Therapy Market is showing positive signs of growth. With the current COVID-19 pandemic scenario, new business opportunities are sprouting in the market. Organizations must explore new markets to expand their business globally and locally. For getting a deeper understanding of the emerging trends, the Global Animal Stem Cell Therapy Market report showcases various factors that drive the economy worldwide. Moreover, the companies will get to know the market landscape for the next decade 2020-2027.

The Global Animal Stem Cell Therapy Market report has been uniquely designed to cater to the needs of the businesses of the 21st century. Going digital is the new normal. Moreover, companies can get to understand their strengths and weaknesses after assessing the market. The next decade is going to be ruled by customer-centric services. To align the business operations, the management team can utilize the actionable recommendations offered at the end of the Global Animal Stem Cell Therapy Market report. Factors that can lift or reduce the business are termed as the external factors that also govern the functioning of the market or industries as a whole.

Before designing the blueprint, every business group can go through the Global Animal Stem Cell Therapy Market report to understand the key business areas. For shaping a new business venture or expanding into a new market, every company must look into the opportunities and threats that are lurking in the current market. To make an efficient business plan, corporations need to understand the market dynamics that will shape the market in the forecast period (2020-2027).

Following key players have been profiled with the help of proven research methodologies:

Animal Stem Cell Therapy Market: Competitive Landscape

To get a head start in a new market, every enterprise needs to understand the competitive landscape and the basic rules that have kept the specific market afloat. The global Animal Stem Cell Therapy Market report unravels the secret ingredients used by competitors to meet the demands of their target audience. For specifically understanding the need to balance the capital invested with profits, organizations must use specific indicators. These indicators will not only help in pointing towards growth but also act as an alert to the upcoming threats in the near future. A proper business plan and approach can guarantee a smooth path ahead for every organization.

If the firms believe in offering a memorable experience to their prospective customers, the Global Animal Stem Cell Therapy Market report is going to be very useful. Facts and figures are churned into this investigative report to share the strengths and weaknesses of the company. With new technologies being introduced every day, many new entrants have started their business in the market. So, to understand their approach towards the market, the Global Animal Stem Cell Therapy Market report has a dedicated section. From the financial aspect to legal, the market report covers all the major things required to study the market and put the business plan in action. Not only this, the competitors added in the report can be altered as per the clients needs and expectations. Furthermore, the companies get a basic outline of moves, in the Global Animal Stem Cell Therapy Market report, that can push the business to emerald heights, both in terms of sales and customer generation for the estimated time frame (2020-2027).

Animal Stem Cell Therapy Market Segmentation:

The Animal Stem Cell Therapy Market has been examined into different global market segments such as type, applications, and global geographies. Each and every global market segment has been studied to get informative insights into various global regions.

Animal Stem Cell Therapy Market Segment by Type:

Animal Stem Cell Therapy Market Segment by Application:

Animal Stem Cell Therapy Market Segment by Global Presence:

North America Latin America Middle East Asia-Pacific Africa Europe

The report has been aggregated by using a couple of research methodologies such as primary and secondary research techniques. It helps in collecting informative pieces of professional information for deriving effective insights into the market. This informative report helps in making well informed and strategic decisions throughout the forecast period.

Animal Stem Cell Therapy Market: Scope of the Report

To properly get a deeper understanding of the Global Animal Stem Cell Therapy Market, this detailed report is the best choice for businesses. To boost the business along with gaining an edge over the competition, every enterprise needs to focus on the pain points of the market (under investigation). Our experienced professionals have collated facts and figures.

This in-depth analysis has revealed many fascinating facts for organizations. For smooth functioning, every business needs to be flexible towards the latest market trends. For this, the framework must be designed to adapt to the trends running at the moment. An end-to-end examination done on the target crowd helped in building the fundamental segment of the investigation, namely the external factors. These have a high tendency to push or pull the industries. Entire industries can either flourish or wipe out due to these uncontrollable factors. Global Animal Stem Cell Therapy market report shows the most affordable options for new as well as established business players to gain market share.

With the highly experienced and motivated team at your service, the team also provides the impact of major factors such as Porters five forces. In the Global Animal Stem Cell Therapy Market, every business runs on the image that is generated digitally in the current decade. Hence, companies need to understand the legal hurdles also. Moreover, with the in-depth study conducted across the various market verticals, it is crystal clear that stakeholders also play a significant role in running the business. Get all the details in the Global Animal Stem Cell Therapy Market report and understand your competitors.

Key questions answered through this analytical market research report include:

What are the latest trends, new patterns and technological advancements in the Animal Stem Cell Therapy Market? Which factors are influencing the Animal Stem Cell Therapy Market over the forecast period? What are the global challenges, threats and risks in the Animal Stem Cell Therapy Market? Which factors are propelling and restraining the Animal Stem Cell Therapy Market? What are the demanding global regions of the Animal Stem Cell Therapy Market? What will be the global market size over the coming future? What are the different effective business strategies followed by global companies?

If you have any custom requirements, please let us know and we will offer you the customized report as per your requirements.

About Us:

Market Research Intellect provides syndicated and customized research reports to clients from various industries and organizations with the aim of delivering functional expertise. We provide reports for all industries including Energy, Technology, Manufacturing and Construction, Chemicals and Materials, Food and Beverage, and more. These reports deliver an in-depth study of the market with industry analysis, the market value for regions and countries, and trends that are pertinent to the industry.

Contact us:

Mr. Steven Fernandes

Market Research Intellect

New Jersey ( USA )

Tel: +1-650-781-4080

Website https://www.marketresearchintellect.com/

See more here:
Animal Stem Cell Therapy Market Size, Drivers, Potential Growth Opportunities, Competitive Landscape, Trends And Forecast To 2027 - Eurowire

Read More...

Mesenchymal stem cells in the treatment of COVID-19-progress and challenges – DocWire News

November 11th, 2020 11:55 am

This article was originally published here

Sheng Wu Gong Cheng Xue Bao. 2020 Oct 25;36(10):1970-1978. doi: 10.13345/j.cjb.200216.

ABSTRACT

At present, SARS-CoV-2 is raging, and novel coronavirus pneumonia (COVID-19) has caused more than 35 million confirmed patients and more than 500 000 cases death, which seriously endanger human health, socioeconomic development, as well as global medical and public health systems. COVID-19 is highly contagious, has a long incubation period, and causes many death cases due to lack of effective specific treatment. Mesenchymal stem cells have powerful anti-inflammatory and immunoregulatory functions, and can effectively reduce the cytokine storm caused by coronavirus in patients, and improve the pulmonary fibrosis of patients, promote the repair of damaged lung tissue, and reduce the mortality. Currently, a number of related clinical trials of mesenchymal stem cell treatment of COVID-19 have been conducted, and have confirmed the safety and efficacy, suggesting a good clinical application prospect. While progress has been made in mesenchymal stem cell therapy for COVID-19, we should also catch sight of the problems and challenges faced by mesenchymal stem cell clinical trials under severe epidemic situation, including clinical trials design, stem cell quality management, and ethics in treatment. Only by paying attention to these can we guarantee the safe and effective development of mesenchymal stem cell clinical trials in the treatment of COVID-19.

PMID:33169563 | DOI:10.13345/j.cjb.200216

Original post:
Mesenchymal stem cells in the treatment of COVID-19-progress and challenges - DocWire News

Read More...

Mesenchymal stem cells in therapy of coronavirus disease 2019 – a review – DocWire News

November 11th, 2020 11:55 am

This article was originally published here

Sheng Wu Gong Cheng Xue Bao. 2020 Oct 25;36(10):1979-1991. doi: 10.13345/j.cjb.200355.

ABSTRACT

Coronavirus disease 2019 (COVID-19) has spread widely on a large scale in the whole world at present, seriously endangering human health. There are still no effective and specific drugs, so it is urgent to find safe and effective therapeutic methods. Mesenchymal stem cells (MSCs) have many biological functions of powerful immunomodulation and tissue repair and regeneration. As a stem cell therapy, it has the potential to reduce the tissue injury and mortality in severe patients infected with novel coronavirus. At present, many research institutions in China and abroad have started a number of clinical research projects about MSCs in the treatment of COVID-19. In addition, those projects have initially confirmed the safety and effectiveness of this therapy. Therefore, this research field has been proved to have a very good clinical therapy prospect.

PMID:33169564 | DOI:10.13345/j.cjb.200355

See the original post:
Mesenchymal stem cells in therapy of coronavirus disease 2019 - a review - DocWire News

Read More...

Global Stem Cell Therapy Market Growth Factor with Regional Forecast, Segmentation, Investment Opportunities, Industry Research and End User Analysis…

November 11th, 2020 11:55 am

The Stem Cell Therapy Market study describes the current market size and market forecast, market prospects, main drivers and constraints, regulatory scenario, industry trend, PESTLE analysis, PORTER analysis, new product approvals / launch, promotion and marketing campaigns, pricing analysis , competitive environment to assist companies in decision-making. The data from the study is focused on current and historical market dynamics that assist in decisions related to investment.

Stem Cell Therapy offers fundamental industry overview representing market trends, company profiles, growth drivers, market scope and Stem Cell Therapy size estimation. The valuable Stem Cell Therapy industry insights, type, application, deployment status and research regions are studied. A thorough analysis of gross margin view, trade news, industry plans and policies, constraints are explained. A complete Stem Cell Therapy industry scenario is explained from 2014 to 2019 and forecast estimates are presented from 2020-2027. The productions, industry chain analysis, gross margin structure and deployment models are stated in detail. Top regions analysed in the report include North America, South America, Europe, Asia-Pacific, Middle East & Africa and the rest of the world. The Stem Cell Therapy industry presence and maturity analysis will lead to investment feasibility and development scope.

Get A Free Sample Copy Here:

https://www.reportspedia.com/report/business-services/2015-2027-global-stem-cell-therapy-industry-market-research-report,-segment-by-player,-type,-application,-marketing-channel,-and-region/57925#request_sample

Stem Cell Therapy Market Leading Players (2019-2027):

Celgene CorporationOsiris Therapeutics, Inc.Pharmicell Co., LtdMEDIPOST Co., Ltd.Promethera BiosciencesFibrocell Science, Inc.Holostem Terapie Avanzate S.r.l.Cytori TherapeuticsNuvasive, Inc.RTI Surgical, Inc.Anterogen Co., Ltd.RTI Surgical, Inc

Market Segment Analysis

By Types:

Adult Stem CellsHuman EmbryonicInduced Pluripotent Stem CellsVery Small Embryonic Like Stem Cells

By Applications:

Regenerative MedicineDrug Discovery and Development

By Region

North America

Europe

Asia-Pacific

LAMEA

Ask for Discount @:

https://www.reportspedia.com/discount_inquiry/discount/57925

Stem Cell Therapy Industry Report addresses different regions like North America, Europe, Asia-Pacific, Middle East & Africa and Latin America. The production value, gross margin analysis, development trend, and Stem Cell Therapy market positioning is explained. The industrial chain study, potential buyers, distributors and traders details are explained. The challenges to the growth and market restraints are explained. The market maturity study, investment scope and gross margin study are profiled. The production process structure, market share, manufacturing cost and Stem Cell Therapy saturation analysis is covered. This will helps the industry aspirants to analysis growth feasibility and development plans.

A special highlight on cost structure, import-export scenario and sales channels of Stem Cell Therapy industry is presented. The benchmarking products, dynamic market changes, upstream raw material and downstream buyers analysis are presented. The business trends, key players analysis and product segment study are explained. The regional SWOT analysis, gross margin analysis, application analysis and industry barriers are explained. The value, volume and consumption from 2019-2027 is portrayed. All the essential details like pricing structure of raw materials, labour cost, sales channels and downstream buyers are presented.

Inquire Before Buying Or Ask For Custom Requirement:

https://www.reportspedia.com/report/business-services/2015-2027-global-stem-cell-therapy-industry-market-research-report,-segment-by-player,-type,-application,-marketing-channel,-and-region/57925#inquiry_before_buying

In the next segment, the forecast Stem Cell Therapy industry perspective is covered. Under forecast statistics, the market value, volume and consumption forecast from 2019-2027 is explained. Stem Cell Therapy regional analysis for major regions and countries in this region is stated. The study of new Stem Cell Therapy industry aspirants and analysts opinions for this industry is presented. The limitations to the industry growth, market risks, Stem Cell Therapy growth opportunities and market trends are viewed. The revenue, Stem Cell Therapy market status, past market performance and product details are presented.

Salient Features Of The Report:

The Stem Cell Therapy report serves as a vital guide in portraying present and forecast industry statistics and market size. The supply/ demand situation, gross margin view and competitive profile of top Stem Cell Therapy players are presented. The Stem Cell Therapy market breakdown by product, type, application and regions will provide sophisticated and precise analysis. Recent developments in Stem Cell Therapy industry, growth opportunities, constraints are studied completely. Also, new product launch events, mergers & acquisitions of Stem Cell Therapy, and industry plans and policies are covered.

The revenue estimates of Stem Cell Therapy market based on top industry players, their product type, applications and regions is studied. The cost structures, gross margin view, sales channel analysis and value chain is explained. In the next segment, the SWOT analysis of players, cost structures, traders, distributors and dealers are listed. The forecast study on Stem Cell Therapy industry will be useful for business plans and growth analysis.

To know More Details About Stem Cell Therapy Market research Report @:

https://www.reportspedia.com/report/business-services/2015-2027-global-stem-cell-therapy-industry-market-research-report,-segment-by-player,-type,-application,-marketing-channel,-and-region/57925#table_of_contents

Contact Us:

Name: Alex White

Phone: US: +1(806)4400782/ UK: +44 33 3303 4979

Email: [emailprotected]

See the original post here:
Global Stem Cell Therapy Market Growth Factor with Regional Forecast, Segmentation, Investment Opportunities, Industry Research and End User Analysis...

Read More...

Stem Cell Therapy Market 2020 Trends, Growth Insight, Share, Competitive Analysis, Statistics, Regional and Global Industry Forecast To 2025 – PRnews…

November 11th, 2020 11:55 am

Adroit Market Research delivers well-researched industry-wide information on the Stem Cell Therapy market. It provides information on the markets essential aspects such as top participants, factors driving Stem Cell Therapy market growth, precise estimation of the Stem Cell Therapy market size, upcoming trends, changes in consumer behavioral pattern, markets competitive landscape, key market vendors, and other market features to gain an in-depth analysis of the market. Additionally, the report is a compilation of both qualitative and quantitative assessment by industry experts, as well as industry participants across the value chain. The report also focuses on the latest developments that can enhance the performance of various market segments.

This report strategically examines the micro-markets and sheds light on the impact of technology upgrades on the performance of the Stem Cell Therapy market. The report presents a broad assessment of the market and contains solicitous insights, historical data, and statistically supported and industry-validated market data. The report offers market projections with the help of appropriate assumptions and methodologies. The research report provides information as per the market segments such as geographies, products, technologies, applications, and industries.

A sample of report copy could be downloaded by visiting the site: https://www.adroitmarketresearch.com/contacts/request-sample/691?utm_source=PT

Competitive Landscape:The global Stem Cell Therapy market is highly consolidated due to the presence of a large number of companies across this industry. The report discusses the current market standing of these companies, their past performances, demand and supply graphs, production and consumption patterns, sales network, distribution channels, and growth opportunities in the market. Moreover, it highlights the strategic approaches of the key players towards expanding their product offerings and reinforcing their market presence.

The report analyzes the key elements such as demand, growth rate, cost, capacity utilization, import, margin, and production of the global market players. A number of the factors are considered to analyze the global Stem Cell Therapy Market. The global Stem Cell Therapy Market report demonstrates details of different sections and sub-sections of the global Stem Cell Therapy Market on the basis of topographical regions. The report provides a detailed analysis of the key elements such as developments, trends, projections, drivers, and market growth of the global Stem Cell Therapy Market. It also offers details of the factors directly impacting on the growth of the global Stem Cell Therapy Market. It covers the fundamental ideas related to the growth and the management of the global Stem Cell Therapy Market.

Enquire for in-depth information before buying this report @ https://www.adroitmarketresearch.com/contacts/enquiry-before-buying/691?utm_source=PT

Global Stem Cell Therapy market is segmented based by type, application and region.Based on Type, the market has been segmented into:

Based on cell source, the market has been segmented into,

Adipose Tissue-Derived Mesenchymal SCsBone Marrow-Derived Mesenchymal SCsEmbryonic SCsOther Sources

Based on Application, the market has been segmented into:

Based on therapeutic application, the market has been segmented into,

Musculoskeletal DisordersWounds & InjuriesCardiovascular DiseasesGastrointestinal DiseasesImmune System DiseasesOther Applications

Stem Cell Therapy Industry Report addresses different regions like North America, Europe, Asia-Pacific, Middle East & Africa and Latin America. The production value, gross margin analysis, development trend, and Stem Cell Therapy market positioning is explained. The industrial chain study, potential buyers, distributors and traders details are explained. The challenges to the growth and market restraints are explained. The market maturity study, investment scope and gross margin study are profiled. The production process structure, market share, manufacturing cost and Stem Cell Therapy saturation analysis is covered. This will helps the industry aspirants to analysis growth feasibility and development plans. A special highlight on cost structure, import-export scenario and sales channels of Stem Cell Therapy industry is presented. The benchmarking products, dynamic market changes, upstream raw material and downstream buyers analysis are presented. The business trends, key players analysis and product segment study are explained. The regional SWOT analysis, gross margin analysis, application analysis and industry barriers are explained. The value, volume and consumption from 2019-2024 is portrayed. All the essential details like pricing structure of raw materials, labour cost, sales channels and downstream buyers are presented.

Browse complete Stem Cell Therapy market report description and TOC @ https://www.adroitmarketresearch.com/industry-reports/stem-cell-therapy-market?utm_source=PT

Key Questions Answered in the Report:1.What is the size of the overall Stem Cell Therapy Market and its segments?2.What are the key segments and sub-segments in the market?3.What are the key drivers, restraints, opportunities, and challenges of the Stem Cell Therapy Market and how they are expected to impact the market?4.What are the attractive investment opportunities within the Market?5.What is the Stem Cell Therapy Market size at the regional and country-level?6.Who are the key market players and their key competitors?7.Market value- chain and key trends impacting every node with reference to companies8.What are the strategies for growth adopted by the key players in Stem Cell Therapy Market?9.How does a particular company rank against its competitors with respect to revenue, profit comparison, operational efficiency, cost competitiveness, and market capitalization?10.How financially strong are the key players in Stem Cell Therapy Market (revenue and profit margin, market capitalization, expenditure analysis, investment analysis)?11.What are the recent trends in Stem Cell Therapy Market? (M&A, partnerships, new product developments, expansions)

Table of ContentsChapter 1 Stem Cell Therapy Market OverviewChapter 2 Global Economic Impact on IndustryChapter 3 Global Market Competition by ManufacturersChapter 4 Global Production, Revenue (Value) by RegionChapter 5 Global Supply (Production), Consumption, Export, Import by RegionsChapter 6 Global Production, Revenue (Value), Price Trend by TypeChapter 7 Global Market Analysis by ApplicationChapter 8 Manufacturing Cost AnalysisChapter 9 Industrial Chain, Sourcing Strategy and Downstream BuyersChapter 10 Marketing Strategy Analysis, Distributors/TradersChapter 11 Market Effect Factors AnalysisChapter 12 Global Stem Cell Therapy Market Forecast

Adroit Market Research is an India-based business analytics and consulting company. Our target audience is a wide range of corporations, manufacturing companies, product/technology development institutions and industry associations that require understanding of a markets size, key trends, participants and future outlook of an industry. We intend to become our clients knowledge partner and provide them with valuable market insights to help create opportunities that increase their revenues. We follow a code Explore, Learn and Transform. At our core, we are curious people who love to identify and understand industry patterns, create an insightful study around our findings and churn out money-making roadmaps.

Ryan JohnsonAccount Manager Global3131 McKinney Ave Ste 600, Dallas,TX 75204, U.S.APhone No.: USA: +1 972-362 -8199 / +91 9665341414

Read the original here:
Stem Cell Therapy Market 2020 Trends, Growth Insight, Share, Competitive Analysis, Statistics, Regional and Global Industry Forecast To 2025 - PRnews...

Read More...

Canine Stem Cell Therapy Market Analysis, Latest Trends and Regional Growth Forecast by Types and Applications 2020 – TechnoWeekly

November 11th, 2020 11:55 am

The globalCanine Stem Cell Therapy marketwas valued at US$ 118.5 Mn in 2018 and is expected to reach US$ 240.7 Mn in 2026, growing at a CAGR of 9.3% during the forecast period. With COVID-19 pandemic, many industries are transforming rapidly. The Global Canine Stem Cell Therapy Market is one of the major industries undergoing changes. This year many industries have vanished entirely from the market and many industries have risen.

Moreover, the government-backed schemes throughout the globe are offering many advantages to businesses. As the governing bodies are supporting the industries, it is a strong pillar to support the market growth of Canine Stem Cell Therapy in the upcoming decade (2020-2026). Organizations planning to move into new market segments can take the help of market indicators to draw a business plan. With the technological boom, new markets are blossoming across the globe, making it a breeding ground for new businesses.

Request for Sample with Complete TOC and Figures & Graphs @ https://www.alltheresearch.com/sample-request/206

Global Canine Stem Cell Therapy Market 2020: Covering both the industrial and the commercial aspects of the Global Canine Stem Cell Therapy Market, the report encircles several crucial chapters that give the report an extra edge. The Global Canine Stem Cell Therapy Market report deep dives into several parts of the report that plays a crucial role in getting the holistic view of the report. The list of such crucial aspects of the report includes company profile, industry analysis, competitive dashboard, comparative analysis of the key players, regional analysis with further analysis country wise.

Global Canine Stem Cell Therapy Market Analysis by Key Players:

Any questions, Lets discuss with the analyst @ https://www.alltheresearch.com/speak-to-analyst/206

Moreover, one of the uniqueness in the report is that it also covers the country-level analysis of the regulatory scenario, technology penetration, predictive trends, and prescriptive trends. This not only gives the readers of the report the actual real-time insights but also gives country-wise analysis, that plays a vital role in decision making. The inclusion of the report is not limited to the above mention key pointers. The report also emphasizes on the market opportunities, porters five forces, and analysis of the different types of products and application of the Global Canine Stem Cell Therapy Market.

The report splits by major applications:

Then report analyzed by types:

Global Canine Stem Cell Therapy Market Report is a professional and in-depth research report on the worlds major regional market conditions of the Canine Stem Cell Therapy industry, focusing on the main regions and the main countries as Follows:

COVID-19 Impact on Canine Stem Cell Therapy Market:

The outbreak of COVID-19 has brought along a global recession, which has impacted several industries. Along with this impact COVID Pandemic has also generated few new business opportunities for Canine Stem Cell Therapy Market. Overall competitive landscape and market dynamics of Canine Stem Cell Therapy has been disrupted due to this pandemic. All these disruptions and impacts has been analysed quantifiably in this report, which is backed by market trends, events and revenue shift analysis. COVID impact analysis also covers strategic adjustments for Tier 1, 2 and 3 players of Canine Stem Cell Therapy Market.

Get Brief Information on Pre COVID-19 Analysis and Post COVID-19 Opportunities in Canine Stem Cell Therapy Market @ https://www.alltheresearch.com/impactC19-request/206

Table of Contents Includes Major Pointes as follows:

Browse Full Research report along with TOC, Tables & Figures: https://www.alltheresearch.com/report/206/Canine Stem Cell Therapy

About AllTheResearch:

AllTheResearch was formed with the aim of making market research a significant tool for managing breakthroughs in the industry. As a leading market research provider, the firm empowers its global clients with business-critical research solutions. The outcome of our study of numerous companies that rely on market research and consulting data for their decision-making made us realise, that its not just sheer data-points, but the right analysis that creates a difference. While some clients were unhappy with the inconsistencies and inaccuracies of data, others expressed concerns over the experience in dealing with the research-firm. Also, same-data-for-all-business roles was making research redundant. We identified these gaps and built AllTheResearch to raise the standards of research support.

FOR ALL YOUR RESEARCH NEEDS, REACH OUT TO US AT:

Contact Name: Rohan S.

Email: [emailprotected]

Phone: +1 (407) 768-2028

See the article here:
Canine Stem Cell Therapy Market Analysis, Latest Trends and Regional Growth Forecast by Types and Applications 2020 - TechnoWeekly

Read More...

Does Talaris’ Investigational Cell Therapy Have Potential to Be a ‘Pipeline in a Product’? – BioSpace

November 11th, 2020 11:55 am

Photo courtesy of Talaris.

Talaris Chief Executive Officer Scott Requadt sees FCR001, the companys investigational cell therapy, as a potential pipeline in a product (an experimental treatment that could have multiple uses across a number of indications).

In fact, it was that versatility of the products potential that caused Requadt to join the company as CEO in 2018 after the company, known then as Regenerex, secured $100 million in financing. At the time, Requadt helmed the venture capital group Claris that backed the Series A.

I was really enthusiastic about the company and FCR001, Requadt told BioSpace in an interview.

FCR001, an investigational, allogeneic cell therapy, was previously part of Novartis gene and cell therapy unit, until it was dissolved in 2016. When that unit dissolved, rights to FCR001 reverted to Regenerex. With the return of those rights came multiple opportunities in organ transplant and severe autoimmune disorders. Cell therapies can address complex, multi-pathway diseases and Talaris has big plans for the future of FCR001 to help patients acquire or restore immune tolerance.

Our goal is to basically do for immune tolerance what CAR-T has done for oncology, Requadt said. The same product and the same basic biology will be used. We can treat organ transplant and autoimmune diseases in the same manner.

Through FCR001, Talaris will be able to change the underlying pathology of the disease so the immune system no longer sees it as a threat, Requadt said. The companys lead program is in kidney donor transplant, but FCR001, a one-time stem cell therapy, is also being explored as a treatment for scleroderma, a multi-system autoimmune disease. If FCR001 is effective in these areas, Requadt said those successes will pave the way for use in other indications.

This is a pipeline from a single product, Requadt said of FCR001, which has received a Regenerative Medicine Advanced Therapy designation from the U.S. Food and Drug Administration (FDA).

In July, Kentucky-based Talaris dosed the first patient in its Phase III FREEDOM-1 study of FCR001 in living donor kidney transplant (LDKT) recipients. The trial is expected to enroll 120 adult patients who will receive kidney donations from living donors. The primary endpoint of the study will be the proportion of FCR001 recipients who are free from necessary drugs to maintain immunosuppression without biopsy proven acute rejection at 24 months post-transplant.

Organ transplant patients are required to continue taking drugs to suppress their immune systems to protect the new organ from immune system responses. However, those immunosuppressant treatments drugs are toxic to the kidney and can ultimately kill the transplanted organ in 10 to 15 years. The drugs can also lead to metabolic disorders and cardiovascular issues, Requadt said.

In 2018, Talaris posted positive Phase II data in LDKT recipients, with 70% of patients who received the treatment able to discontinue the use of immunosuppressant drugs. The Phase II data showed that every tolerized patient has been able to remain free of the use of chronic immunosuppressants for up to 10 years. The median follow-up following transplant was five years, with the longest case being a decade. Additionally, Requadt said the company has also seen better kidney functions in recipients who received FCR100 due to the lack of toxicity issues.

While there are other companies exploring similar approaches, Requadt said to his knowledge, no other group has a 10-year data set that demonstrates the safety and efficacy of a treatment like FRC001.

Not only has Talaris seen the impressive results in removing patients from the anchor of immunosuppressant drugs, Requadt said the use of FCR001 decreased the risk of rejection in patients whose biomarkers did not have as high a match with the kidney donors.

In addition to the Phase III study in LDKT patients, Talaris also has plans to conduct a Phase II study in LDKT Delayed Tolerance Induction and will begin research in Deceased Donor Kidney Transplant patients.

Talaris will also use FCR001 in the treatment of diffuse systemic sclerosis (SSc), a severe form of the rare autoimmune disease scleroderma, a rare and potentially fatal chronic autoimmune disease which causes progressive scarring, or fibrosis, of the bodys connective tissues. Autologous hematopoietic stem cell transplant has been shown to halt organ damage and induce remission of the disease. However, with the use of a patients own stem cells, there is a risk of disease recurrence. Also, some patients must undergo full myeloablative conditioning with or without total body irradiation, which is associated with direct organ toxicity and increased risk of future cancers. Talaris aims to harness the power of FCR001 and use stem cells from donors to lower those risks and provide an opportunity for these patients, Requadt said.

For all of these indications were using the same product and were using the same protocols with patients and were treating the patients in the same way. This has the potential to be paradigm shifting, Requadt said.

Excerpt from:
Does Talaris' Investigational Cell Therapy Have Potential to Be a 'Pipeline in a Product'? - BioSpace

Read More...

Cancer Stem Cell Therapy Market: Industry Trends and Developments 2020-2025 – Zenit News

November 11th, 2020 11:55 am

Market Overview of Cancer Stem Cell Therapy Market

The Cancer Stem Cell Therapy market report provides a detailed analysis of global market size, regional and country-level market size, segmentation market growth, market share, competitive Landscape, sales analysis, impact of domestic and global market players, value chain optimization, trade regulations, recent developments, opportunities analysis, strategic market growth analysis, product launches, area marketplace expanding, and technological innovations.

The global Cancer Stem Cell Therapy market size is expected to gain market growth in the forecast period of 2020 to 2025, with a CAGR of xx%% in the forecast period of 2020 to 2025 and will expected to reach USD xx million by 2025, from USD xx million in 2019.

Get PDF Sample Copy of this Report to understand the structure of the complete report: (Including Full TOC, List of Tables & Figures, Chart) @ https://www.marketresearchhub.com/enquiry.php?type=S&repid=2802065&source=atm

Market segmentation

Cancer Stem Cell Therapy market is split by Type and by Application. For the period 2015-2025, the growth among segments provide accurate calculations and forecasts for sales by Type and by Application in terms of volume and value. This analysis can help you expand your business by targeting qualified niche markets.

segment by Type, the product can be split intoAutologous Stem Cell TransplantsAllogeneic Stem Cell TransplantsSyngeneic Stem Cell TransplantsOther

Market segment by Application, split intoHospitalClinicMedical Research InstitutionOther

Based on regional and country-level analysis, the Cancer Stem Cell Therapy market has been segmented as follows:North AmericaUnited StatesCanadaEuropeGermanyFranceU.K.ItalyRussiaNordicRest of EuropeAsia-PacificChinaJapanSouth KoreaSoutheast AsiaIndiaAustraliaRest of Asia-PacificLatin AmericaMexicoBrazilMiddle East & AfricaTurkeySaudi ArabiaUAERest of Middle East & Africa

Regional analysis is another highly comprehensive part of the research and analysis study of the global Cancer Stem Cell Therapy market presented in the report. This section sheds light on the sales growth of different regional and country-level Cancer Stem Cell Therapy markets. For the historical and forecast period 2015 to 2025, it provides detailed and accurate country-wise volume analysis and region-wise market size analysis of the global Cancer Stem Cell Therapy market.

Do You Have Any Query Or Specific Requirement? Ask to Our Industry [emailprotected] https://www.marketresearchhub.com/enquiry.php?type=E&repid=2802065&source=atm

The report offers in-depth assessment of the growth and other aspects of the Cancer Stem Cell Therapy market in important countries (regions), including:

North America (United States, Canada and Mexico)

Europe (Germany, France, UK, Russia and Italy)

Asia-Pacific (China, Japan, Korea, India and Southeast Asia)

South America (Brazil, Argentina, etc.)

Middle East & Africa (Saudi Arabia, Egypt, Nigeria and South Africa)

Cancer Stem Cell Therapy competitive landscape provides details by vendors, including company overview, company total revenue (financials), market potential, global presence, Cancer Stem Cell Therapy sales and revenue generated, market share, price, production sites and facilities, SWOT analysis, product launch. For the period 2015-2020, this study provides the Cancer Stem Cell Therapy sales, revenue and market share for each player covered in this report.

In the competitive analysis section of the report, leading as well as prominent players of the global Cancer Stem Cell Therapy market are broadly studied on the basis of key factors. The report offers comprehensive analysis and accurate statistics on revenue by the player for the period 2015-2020. It also offers detailed analysis supported by reliable statistics on price and revenue (global level) by player for the period 2015-2020.The key players covered in this studyAVIVA BioSciencesAdnaGenAdvanced Cell DiagnosticsSilicon Biosystems

You can Buy This Report from Here @ https://www.marketresearchhub.com/checkout?rep_id=2802065&licType=S&source=atm

The content of the study subjects, includes a total of 15 chapters:

Chapter 1, to describe Cancer Stem Cell Therapy product scope, market overview, market opportunities, market driving force and market risks.

Chapter 2, to profile the top manufacturers of Cancer Stem Cell Therapy , with price, sales, revenue and global market share of Cancer Stem Cell Therapy in 2018 and 2019.

Chapter 3, the Cancer Stem Cell Therapy competitive situation, sales, revenue and global market share of top manufacturers are analyzed emphatically by landscape contrast.

Chapter 4, the Cancer Stem Cell Therapy breakdown data are shown at the regional level, to show the sales, revenue and growth by regions, from 2015 to 2020.

Chapter 5, 6, 7, 8 and 9, to break the sales data at the country level, with sales, revenue and market share for key countries in the world, from 2015 to 2020.

Chapter 10 and 11, to segment the sales by type and application, with sales market share and growth rate by type, application, from 2015 to 2020.

Chapter 12, Cancer Stem Cell Therapy market forecast, by regions, type and application, with sales and revenue, from 2020 to 2025.

Chapter 13, 14 and 15, to describe Cancer Stem Cell Therapy sales channel, distributors, customers, research findings and conclusion, appendix and data source.

Contact Us:

marketresearchhub

Tel: +1-518-621-2074

USA-Canada Toll Free: 866-997-4948

Email: [emailprotected]

About marketresearchhub

marketresearchhub is the one stop online destination to find and buy market research reports & Industry Analysis. We fulfil all your research needs spanning across industry verticals with our huge collection of market research reports. We provide our services to all sizes of organisations and across all industry verticals and markets. Our Research Coordinators have in-depth knowledge of reports as well as publishers and will assist you in making an informed decision by giving you unbiased and deep insights on which reports will satisfy your needs at the best price.

Read more:
Cancer Stem Cell Therapy Market: Industry Trends and Developments 2020-2025 - Zenit News

Read More...

Stem Cell Therapy Market Insights Global Analysis and Forecast by (2020 2027) – The Think Curiouser

November 11th, 2020 11:55 am

Emergen Research has published its latest report, titled Global Stem Cell Therapy Market, which is a unique compilation of the vital elements of the Stem Cell Therapy industry, including the key market growth drivers, constraints, opportunities, limitations, and threats. The micro- and macro-economic factors claimed to bolster the global market expansion in the near future have been encased in this report. The report consists of a broad database of the market dynamics intended to facilitate market estimation over the projected timeline. The latest report is highly beneficial for businesses and stakeholders looking forward to setting a robust footing in this industry.

The Global Stem Cell Therapy Market size was valued at USD 6.16 Billion in 2019 and is anticipated to reach USD 11.97 Billion by 2027 at a CAGR of 8.7%.

Request Sample copy of this [emailprotected] https://www.emergenresearch.com/request-sample-form/83

The top market competitors profiled in the report include Virgin Health Bank, Celgene Corporation, ReNeuron Group plc, Biovault Family, Precious Cells International Ltd., Mesoblast Ltd., Opexa Therapeutics, Inc., Caladrius, Neuralstem, Inc., and Pluristem, among others.

The Global Stem Cell Therapy Market report is dubbed as the first study encompassing the current situation of the Stem Cell Therapy market that is gravely impacted by the COVID-19 outbreak. The global health emergency has drastically affected the global economy, causing significant repercussions on this particular business sphere. The deadly viral outbreak and the subsequent global lockdown enforcement have beleaguered the Stem Cell Therapy business landscape. Moreover, it has impeded the developmental scope of various manufacturers and buyers involved in this sector. Thus, the report offers insightful speculations about the post-COVID-19 scenario of the Stem Cell Therapy industry.

Type Outlook (Revenue, USD Million; 2017-2027)

Application Outlook (Revenue, USD Million; 2017-2027)

End-User Outlook (Revenue, USD Million; 2017-2027)

The report thoroughly scrutinizes the Stem Cell Therapy market on the basis of the global market reach and consumer bases of the key geographical segments. Under the regional analysis, the report authors have studied the presence of the global Stem Cell Therapy market across the major regions and further highlighted their respective market shares, market sizes, revenue contributions, sales networks, distribution channels, and numerous other significant aspects.

To know more about the report, visit @ https://www.emergenresearch.com/industry-report/stem-cell-therapy-market

Stem Cell Therapy Market Segmentation by Region:

Competitive Outlook:

The latest research report involves an exhaustive study of the leading companies functioning mechanisms participating in this industry. According to our team of analysts, these companies hold a considerable portion of the overall Stem Cell Therapy market share. Under this section of the report, the principal strategic initiatives led by these companies, including mergers & acquisitions, joint ventures, new business deals, new product launches, collaborations, technological upgradation, and several others, have been emphasized.

Request customization of the report @ https://www.emergenresearch.com/request-for-customization-form/83

Key Report Coverage:

Frequently asked questions addressed in the report:

Request a discount on the report @ https://www.emergenresearch.com/request-discount-form/83

Read the original:
Stem Cell Therapy Market Insights Global Analysis and Forecast by (2020 2027) - The Think Curiouser

Read More...

Merck Announces KEYNOTE-598 Trial Evaluating KEYTRUDA in Combination With Ipilimumab Versus KEYTRUDA Monotherapy in Certain Patients With Metastatic…

November 11th, 2020 11:54 am

Merck Announces KEYNOTE-598 Trial Evaluating KEYTRUDA (pembrolizumab) in Combination With Ipilimumab Versus KEYTRUDA Monotherapy in Certain Patients With Metastatic Non-Small Cell Lung Cancer To Stop for Futility and Patients to Discontinue Ipilimumab

Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced that it will be stopping KEYNOTE-598, a Phase 3 trial investigating KEYTRUDA, Mercks anti-PD-1 therapy, in combination with ipilimumab (Yervoy ), compared with KEYTRUDA monotherapy, for the first-line treatment of patients with metastatic non-small cell lung cancer (NSCLC) whose tumors express PD-L1 (tumor proportion score [TPS] 50%) with no EGFR or ALK genomic tumor aberrations. Merck is discontinuing the study following the recommendation of an independent Data Monitoring Committee (DMC), which determined the benefit/risk profile of the combination did not support continuing the trial. At an interim analysis, the combination of KEYTRUDA and ipilimumab showed no incremental benefit in overall survival (OS) or progression-free survival (PFS), the studys dual primary endpoints, compared with KEYTRUDA alone and crossed futility boundaries. No new safety signals for KEYTRUDA monotherapy were observed, however the combination of KEYTRUDA and ipilimumab was associated with a higher incidence of grade 3-5 adverse events (AEs), serious AEs, and AEs leading to discontinuation or death, compared with KEYTRUDA monotherapy. Merck will inform study investigators of the recommendation from the DMC and the DMC is advising that patients in the study discontinue treatment with ipilimumab/placebo. Data from this study will be submitted for presentation at an upcoming scientific congress and communicated to regulatory agencies.

We conducted KEYNOTE-598 in order to explicitly explore whether combining our anti-PD-1 therapy, KEYTRUDA, with ipilimumab provided additional benefits beyond treatment with KEYTRUDA alone in the metastatic non-small cell lung cancer setting, said Dr. Roy Baynes, senior vice president and head of global clinical development, chief medical officer, Merck Research Laboratories. It is very clear that in this study, the addition of ipilimumab did not add clinical benefit but did add toxicity. KEYTRUDA monotherapy remains a standard of care for the treatment of certain patients with metastatic non-small cell lung cancer whose tumors express PD-L1.

While the combination of an anti-PD-1 therapy plus ipilimumab has been approved in certain indications, studies supporting these approvals have, for the most part, not compared the combination directly with anti-PD-1 monotherapy. Bristol Myers Squibb has reported topline results of CheckMate-915, a Phase 3 study in adjuvant melanoma that directly compared treatment with ipilimumab in combination with an anti-PD-1 therapy versus the anti-PD-1 therapy alone. In two separate news releases issued over the last year, the company announced the study did not meet its co-primary endpoints in the all-comer population or in patients whose tumors expressed PD-L1

Merck has an extensive clinical development program in lung cancer and is advancing multiple registration-enabling studies with KEYTRUDA in combination with other treatments and as monotherapy. The lung program is evaluating KEYTRUDA across all stages of disease and lines of therapy in over 200 trials with more than 10,000 patients.

About KEYNOTE-598

KEYNOTE-598 (ClinicalTrials.gov, NCT03302234 ) is a randomized, double-blind, Phase 3 trial investigating KEYTRUDA in combination with ipilimumab compared to KEYTRUDA monotherapy for the first-line treatment of patients with metastatic NSCLC whose tumors express PDL1 (TPS 50%) with no EGFR or ALK genomic tumor aberrations. The dual primary endpoints are OS and PFS. Secondary endpoints include objective response rate, duration of response and safety. The study enrolled 568 patients who were randomized (1:1) to receive:

About Lung Cancer

Lung cancer, which forms in the tissues of the lungs, usually within cells lining the air passages, is the leading cause of cancer death worldwide. Each year, more people die of lung cancer than die of colon and breast cancers combined. The two main types of lung cancer are non-small cell and small cell. Non-small cell lung cancer (NSCLC) is the most common type of lung cancer, accounting for about 85% of all cases. Small cell lung cancer (SCLC) accounts for about 10% to 15% of all lung cancers. Before 2014, the five-year survival rate for patients diagnosed in the U.S. with NSCLC and SCLC was estimated to be 5% and 6%, respectively.

About KEYTRUDA (pembrolizumab) Injection, 100 mg

KEYTRUDA is an anti-PD-1 therapy that works by increasing the ability of the bodys immune system to help detect and fight tumor cells. KEYTRUDA is a humanized monoclonal antibody that blocks the interaction between PD-1 and its ligands, PD-L1 and PD-L2, thereby activating T lymphocytes which may affect both tumor cells and healthy cells.

Merck has the industrys largest immuno-oncology clinical research program. There are currently more than 1,200 trials studying KEYTRUDA across a wide variety of cancers and treatment settings. The KEYTRUDA clinical program seeks to understand the role of KEYTRUDA across cancers and the factors that may predict a patients likelihood of benefitting from treatment with KEYTRUDA, including exploring several different biomarkers.

Selected KEYTRUDA (pembrolizumab) Indications

Melanoma

KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic melanoma.

KEYTRUDA is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph node(s) following complete resection.

Non-Small Cell Lung Cancer

KEYTRUDA, in combination with pemetrexed and platinum chemotherapy, is indicated for the first-line treatment of patients with metastatic nonsquamous non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.

KEYTRUDA, in combination with carboplatin and either paclitaxel or paclitaxel protein-bound, is indicated for the first-line treatment of patients with metastatic squamous NSCLC.

KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with NSCLC expressing PD-L1 [tumor proportion score (TPS) 1%] as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations, and is stage III where patients are not candidates for surgical resection or definitive chemoradiation, or metastatic.

KEYTRUDA, as a single agent, is indicated for the treatment of patients with metastatic NSCLC whose tumors express PD-L1 (TPS 1%) as determined by an FDA-approved test, with disease progression on or after platinum-containing chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving KEYTRUDA.

Small Cell Lung Cancer

KEYTRUDA is indicated for the treatment of patients with metastatic small cell lung cancer (SCLC) with disease progression on or after platinum-based chemotherapy and at least 1 other prior line of therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

Head and Neck Squamous Cell Cancer

KEYTRUDA, in combination with platinum and fluorouracil (FU), is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent head and neck squamous cell carcinoma (HNSCC).

KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent HNSCC whose tumors express PD-L1 [combined positive score (CPS) 1] as determined by an FDA-approved test.

KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent or metastatic head and neck squamous cell carcinoma (HNSCC) with disease progression on or after platinum-containing chemotherapy.

Classical Hodgkin Lymphoma

KEYTRUDA is indicated for the treatment of adult patients with relapsed or refractory classical Hodgkin lymphoma (cHL).

KEYTRUDA is indicated for the treatment of pediatric patients with refractory cHL, or cHL that has relapsed after 2 or more lines of therapy.

Primary Mediastinal Large B-Cell Lymphoma

KEYTRUDA is indicated for the treatment of adult and pediatric patients with refractory primary mediastinal large B-cell lymphoma (PMBCL), or who have relapsed after 2 or more prior lines of therapy. KEYTRUDA is not recommended for treatment of patients with PMBCL who require urgent cytoreductive therapy.

Urothelial Carcinoma

KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who are not eligible for cisplatin-containing chemotherapy and whose tumors express PD-L1 [combined positive score (CPS) 10], as determined by an FDA-approved test, or in patients who are not eligible for any platinum-containing chemotherapy regardless of PD-L1 status. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who have disease progression during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.

KEYTRUDA is indicated for the treatment of patients with Bacillus Calmette-Guerin (BCG)-unresponsive, high-risk, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors who are ineligible for or have elected not to undergo cystectomy.

Microsatellite Instability-High or Mismatch Repair Deficient Cancer

KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR)

This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with MSI-H central nervous system cancers have not been established.

Microsatellite Instability-High or Mismatch Repair Deficient Colorectal Cancer

KEYTRUDA is indicated for the first-line treatment of patients with unresectable or metastatic MSI-H or dMMR colorectal cancer (CRC).

Gastric Cancer

KEYTRUDA is indicated for the treatment of patients with recurrent locally advanced or metastatic gastric or gastroesophageal junction (GEJ) adenocarcinoma whose tumors express PD-L1 (CPS 1) as determined by an FDA-approved test, with disease progression on or after two or more prior lines of therapy including fluoropyrimidine- and platinum-containing chemotherapy and if appropriate, HER2/neu-targeted therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Esophageal Cancer

KEYTRUDA is indicated for the treatment of patients with recurrent locally advanced or metastatic squamous cell carcinoma of the esophagus whose tumors express PD-L1 (CPS 10) as determined by an FDA-approved test, with disease progression after one or more prior lines of systemic therapy.

Cervical Cancer

KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy whose tumors express PD-L1 (CPS 1) as determined by an FDA-approved test. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Hepatocellular Carcinoma

KEYTRUDA is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Merkel Cell Carcinoma

KEYTRUDA is indicated for the treatment of adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC). This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Renal Cell Carcinoma

KEYTRUDA, in combination with axitinib, is indicated for the first-line treatment of patients with advanced renal cell carcinoma (RCC).

Tumor Mutational Burden-High

KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic tumor mutational burden-high (TMB-H) [10 mutations/megabase (mut/Mb)] solid tumors, as determined by an FDA-approved test, that have progressed following prior treatment and who have no satisfactory alternative treatment options. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with TMB-H central nervous system cancers have not been established.

Cutaneous Squamous Cell Carcinoma

KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cutaneous squamous cell carcinoma (cSCC) that is not curable by surgery or radiation.

Selected Important Safety Information for KEYTRUDA

Immune-Mediated Pneumonitis

KEYTRUDA can cause immune-mediated pneumonitis, including fatal cases. Pneumonitis occurred in 3.4% (94/2799) of patients with various cancers receiving KEYTRUDA, including Grade 1 (0.8%), 2 (1.3%), 3 (0.9%), 4 (0.3%), and 5 (0.1%). Pneumonitis occurred in 8.2% (65/790) of NSCLC patients receiving KEYTRUDA as a single agent, including Grades 3-4 in 3.2% of patients, and occurred more frequently in patients with a history of prior thoracic radiation (17%) compared to those without (7.7%). Pneumonitis occurred in 6% (18/300) of HNSCC patients receiving KEYTRUDA as a single agent, including Grades 3-5 in 1.6% of patients, and occurred in 5.4% (15/276) of patients receiving KEYTRUDA in combination with platinum and FU as first-line therapy for advanced disease, including Grades 3-5 in 1.5% of patients. Pneumonitis occurred in 8% (31/389) of patients with cHL receiving KEYTRUDA as a single agent, including Grades 3-4 in 2.3% of patients.

Monitor patients for signs and symptoms of pneumonitis. Evaluate suspected pneumonitis with radiographic imaging. Administer corticosteroids for Grade 2 or greater pneumonitis. Withhold KEYTRUDA for Grade 2; permanently discontinue KEYTRUDA for Grade 3 or 4 or recurrent Grade 2 pneumonitis.

Immune-Mediated Colitis

KEYTRUDA can cause immune-mediated colitis. Colitis occurred in 1.7% (48/2799) of patients receiving KEYTRUDA, including Grade 2 (0.4%), 3 (1.1%), and 4 (

Immune-Mediated Hepatitis (KEYTRUDA) and Hepatotoxicity (KEYTRUDA in Combination With Axitinib)

Immune-Mediated Hepatitis

KEYTRUDA can cause immune-mediated hepatitis. Hepatitis occurred in 0.7% (19/2799) of patients receiving KEYTRUDA, including Grade 2 (0.1%), 3 (0.4%), and 4 (

Hepatotoxicity in Combination With Axitinib

KEYTRUDA in combination with axitinib can cause hepatic toxicity with higher than expected frequencies of Grades 3 and 4 ALT and AST elevations compared to KEYTRUDA alone. With the combination of KEYTRUDA and axitinib, Grades 3 and 4 increased ALT (20%) and increased AST (13%) were seen. Monitor liver enzymes before initiation of and periodically throughout treatment. Consider more frequent monitoring of liver enzymes as compared to when the drugs are administered as single agents. For elevated liver enzymes, interrupt KEYTRUDA and axitinib, and consider administering corticosteroids as needed.

Immune-Mediated Endocrinopathies

KEYTRUDA can cause adrenal insufficiency (primary and secondary), hypophysitis, thyroid disorders, and type 1 diabetes mellitus. Adrenal insufficiency occurred in 0.8% (22/2799) of patients, including Grade 2 (0.3%), 3 (0.3%), and 4 (

Monitor patients for signs and symptoms of adrenal insufficiency, hypophysitis (including hypopituitarism), thyroid function (prior to and periodically during treatment), and hyperglycemia. For adrenal insufficiency or hypophysitis, administer corticosteroids and hormone replacement as clinically indicated. Withhold KEYTRUDA for Grade 2 adrenal insufficiency or hypophysitis and withhold or discontinue KEYTRUDA for Grade 3 or Grade 4 adrenal insufficiency or hypophysitis. Administer hormone replacement for hypothyroidism and manage hyperthyroidism with thionamides and beta-blockers as appropriate. Withhold or discontinue KEYTRUDA for Grade 3 or 4 hyperthyroidism. Administer insulin for type 1 diabetes, and withhold KEYTRUDA and administer antihyperglycemics in patients with severe hyperglycemia.

Immune-Mediated Nephritis and Renal Dysfunction

KEYTRUDA can cause immune-mediated nephritis. Nephritis occurred in 0.3% (9/2799) of patients receiving KEYTRUDA, including Grade 2 (0.1%), 3 (0.1%), and 4 (

Immune-Mediated Skin Reactions

Immune-mediated rashes, including Stevens-Johnson syndrome (SJS), toxic epidermal necrolysis (TEN) (some cases with fatal outcome), exfoliative dermatitis, and bullous pemphigoid, can occur. Monitor patients for suspected severe skin reactions and based on the severity of the adverse reaction, withhold or permanently discontinue KEYTRUDA and administer corticosteroids. For signs or symptoms of SJS or TEN, withhold KEYTRUDA and refer the patient for specialized care for assessment and treatment. If SJS or TEN is confirmed, permanently discontinue KEYTRUDA.

Other Immune-Mediated Adverse Reactions

Immune-mediated adverse reactions, which may be severe or fatal, can occur in any organ system or tissue in patients receiving KEYTRUDA and may also occur after discontinuation of treatment. For suspected immune-mediated adverse reactions, ensure adequate evaluation to confirm etiology or exclude other causes. Based on the severity of the adverse reaction, withhold KEYTRUDA and administer corticosteroids. Upon improvement to Grade 1 or less, initiate corticosteroid taper and continue to taper over at least 1 month. Based on limited data from clinical studies in patients whose immune-related adverse reactions could not be controlled with corticosteroid use, administration of other systemic immunosuppressants can be considered. Resume KEYTRUDA when the adverse reaction remains at Grade 1 or less following corticosteroid taper. Permanently discontinue KEYTRUDA for any Grade 3 immune-mediated adverse reaction that recurs and for any life-threatening immune-mediated adverse reaction.

The following clinically significant immune-mediated adverse reactions occurred in less than 1% (unless otherwise indicated) of 2799 patients: arthritis (1.5%), uveitis, myositis, Guillain-Barr syndrome, myasthenia gravis, vasculitis, pancreatitis, hemolytic anemia, sarcoidosis, and encephalitis. In addition, myelitis and myocarditis were reported in other clinical trials, including classical Hodgkin lymphoma, and post-marketing use.

Treatment with KEYTRUDA may increase the risk of rejection in solid organ transplant recipients. Consider the benefit of treatment vs the risk of possible organ rejection in these patients.

Infusion-Related Reactions

KEYTRUDA can cause severe or life-threatening infusion-related reactions, including hypersensitivity and anaphylaxis, which have been reported in 0.2% (6/2799) of patients. Monitor patients for signs and symptoms of infusion-related reactions. For Grade 3 or 4 reactions, stop infusion and permanently discontinue KEYTRUDA.

Complications of Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)

Fatal and other serious complications can occur in patients who receive allogeneic hematopoietic stem cell transplantation (HSCT) before or after being treated with a PD-1/PD-L1 blocking antibody. Transplant-related complications include hyperacute graft-versus-host disease (GVHD), acute GVHD, chronic GVHD, hepatic veno-occlusive disease (VOD) after reduced intensity conditioning, and steroid-requiring febrile syndrome (without an identified infectious cause). These complications may occur despite intervening therapy between PD-1/PD-L1 blockade and allogeneic HSCT. Follow patients closely for evidence of transplant-related complications and intervene promptly. Consider the benefit versus risk of treatment with a PD-1/PD-L1 blocking antibody prior to or after an allogeneic HSCT.

Increased Mortality in Patients With Multiple Myeloma

In trials in patients with multiple myeloma, the addition of KEYTRUDA to a thalidomide analogue plus dexamethasone resulted in increased mortality. Treatment of these patients with a PD-1 or PD-L1 blocking antibody in this combination is not recommended outside of controlled trials.

Embryofetal Toxicity

Based on its mechanism of action, KEYTRUDA can cause fetal harm when administered to a pregnant woman. Advise women of this potential risk. In females of reproductive potential, verify pregnancy status prior to initiating KEYTRUDA and advise them to use effective contraception during treatment and for 4 months after the last dose.

Adverse Reactions

In KEYNOTE-006, KEYTRUDA was discontinued due to adverse reactions in 9% of 555 patients with advanced melanoma; adverse reactions leading to permanent discontinuation in more than one patient were colitis (1.4%), autoimmune hepatitis (0.7%), allergic reaction (0.4%), polyneuropathy (0.4%), and cardiac failure (0.4%). The most common adverse reactions (20%) with KEYTRUDA were fatigue (28%), diarrhea (26%), rash (24%), and nausea (21%).

In KEYNOTE-002, KEYTRUDA was permanently discontinued due to adverse reactions in 12% of 357 patients with advanced melanoma; the most common (1%) were general physical health deterioration (1%), asthenia (1%), dyspnea (1%), pneumonitis (1%), and generalized edema (1%). The most common adverse reactions were fatigue (43%), pruritus (28%), rash (24%), constipation (22%), nausea (22%), diarrhea (20%), and decreased appetite (20%).

In KEYNOTE-054, KEYTRUDA was permanently discontinued due to adverse reactions in 14% of 509 patients; the most common (1%) were pneumonitis (1.4%), colitis (1.2%), and diarrhea (1%). Serious adverse reactions occurred in 25% of patients receiving KEYTRUDA. The most common adverse reaction (20%) with KEYTRUDA was diarrhea (28%).

In KEYNOTE-189, when KEYTRUDA was administered with pemetrexed and platinum chemotherapy in metastatic nonsquamous NSCLC, KEYTRUDA was discontinued due to adverse reactions in 20% of 405 patients. The most common adverse reactions resulting in permanent discontinuation of KEYTRUDA were pneumonitis (3%) and acute kidney injury (2%). The most common adverse reactions (20%) with KEYTRUDA were nausea (56%), fatigue (56%), constipation (35%), diarrhea (31%), decreased appetite (28%), rash (25%), vomiting (24%), cough (21%), dyspnea (21%), and pyrexia (20%).

In KEYNOTE-407, when KEYTRUDA was administered with carboplatin and either paclitaxel or paclitaxel protein-bound in metastatic squamous NSCLC, KEYTRUDA was discontinued due to adverse reactions in 15% of 101 patients. The most frequent serious adverse reactions reported in at least 2% of patients were febrile neutropenia, pneumonia, and urinary tract infection. Adverse reactions observed in KEYNOTE-407 were similar to those observed in KEYNOTE-189 with the exception that increased incidences of alopecia (47% vs 36%) and peripheral neuropathy (31% vs 25%) were observed in the KEYTRUDA and chemotherapy arm compared to the placebo and chemotherapy arm in KEYNOTE-407.

In KEYNOTE-042, KEYTRUDA was discontinued due to adverse reactions in 19% of 636 patients with advanced NSCLC; the most common were pneumonitis (3%), death due to unknown cause (1.6%), and pneumonia (1.4%). The most frequent serious adverse reactions reported in at least 2% of patients were pneumonia (7%), pneumonitis (3.9%), pulmonary embolism (2.4%), and pleural effusion (2.2%). The most common adverse reaction (20%) was fatigue (25%).

In KEYNOTE-010, KEYTRUDA monotherapy was discontinued due to adverse reactions in 8% of 682 patients with metastatic NSCLC; the most common was pneumonitis (1.8%). The most common adverse reactions (20%) were decreased appetite (25%), fatigue (25%), dyspnea (23%), and nausea (20%).

Adverse reactions occurring in patients with SCLC were similar to those occurring in patients with other solid tumors who received KEYTRUDA as a single agent.

In KEYNOTE-048, KEYTRUDA monotherapy was discontinued due to adverse events in 12% of 300 patients with HNSCC; the most common adverse reactions leading to permanent discontinuation were sepsis (1.7%) and pneumonia (1.3%). The most common adverse reactions (20%) were fatigue (33%), constipation (20%), and rash (20%).

In KEYNOTE-048, when KEYTRUDA was administered in combination with platinum (cisplatin or carboplatin) and FU chemotherapy, KEYTRUDA was discontinued due to adverse reactions in 16% of 276 patients with HNSCC. The most common adverse reactions resulting in permanent discontinuation of KEYTRUDA were pneumonia (2.5%), pneumonitis (1.8%), and septic shock (1.4%). The most common adverse reactions (20%) were nausea (51%), fatigue (49%), constipation (37%), vomiting (32%), mucosal inflammation (31%), diarrhea (29%), decreased appetite (29%), stomatitis (26%), and cough (22%).

In KEYNOTE-012, KEYTRUDA was discontinued due to adverse reactions in 17% of 192 patients with HNSCC. Serious adverse reactions occurred in 45% of patients. The most frequent serious adverse reactions reported in at least 2% of patients were pneumonia, dyspnea, confusional state, vomiting, pleural effusion, and respiratory failure. The most common adverse reactions (20%) were fatigue, decreased appetite, and dyspnea. Adverse reactions occurring in patients with HNSCC were generally similar to those occurring in patients with melanoma or NSCLC who received KEYTRUDA as a monotherapy, with the exception of increased incidences of facial edema and new or worsening hypothyroidism.

In KEYNOTE-204, KEYTRUDA was discontinued due to adverse reactions in 14% of 148 patients with cHL. Serious adverse reactions occurred in 30% of patients; those 1% included pneumonitis, pneumonia, pyrexia, myocarditis, acute kidney injury, febrile neutropenia, and sepsis. Three patients died from causes other than disease progression. The most common adverse reactions (20%) were upper respiratory tract infection (41%), musculoskeletal pain (32%), diarrhea (22%), and pyrexia, fatigue, and cough (20% each).

Go here to see the original:
Merck Announces KEYNOTE-598 Trial Evaluating KEYTRUDA in Combination With Ipilimumab Versus KEYTRUDA Monotherapy in Certain Patients With Metastatic...

Read More...

KEYTRUDA (pembrolizumab) Plus LENVIMA (lenvatinib) Demonstrated Statistically Significant Improvement in Progression-Free Survival (PFS), Overall…

November 11th, 2020 11:54 am

KENILWORTH, N.J., & WOODCLIFF LAKE, N.J.--(BUSINESS WIRE)--Merck (NYSE: MRK):

KEYTRUDA (pembrolizumab) Plus LENVIMA (lenvatinib) Demonstrated Statistically Significant Improvement in Progression-Free Survival (PFS), Overall Survival (OS) and Objective Response Rate (ORR) Versus Sunitinib as First-Line Treatment for Patients With Advanced Renal Cell Carcinoma

LENVIMA Plus Everolimus Also Showed Statistically Significant Improvement in PFS and ORR Endpoints Versus Sunitinib

Results of Investigational Phase 3 KEYNOTE-581/CLEAR Trial (Study 307) to be Presented at Upcoming Medical Meeting

Merck (NYSE: MRK), known as MSD outside the United States and Canada, and Eisai today announced new investigational data demonstrating positive top-line results from the pivotal Phase 3 KEYNOTE-581/CLEAR trial (Study 307). In the trial, the combinations of KEYTRUDA, Mercks anti-PD-1 therapy, plus LENVIMA, the orally available multiple receptor tyrosine kinase inhibitor discovered by Eisai, and LENVIMA plus everolimus were evaluated versus sunitinib for the first-line treatment of patients with advanced renal cell carcinoma (RCC). KEYTRUDA plus LENVIMA met the trials primary endpoint of progression-free survival (PFS) and its key secondary endpoints of overall survival (OS) and objective response rate (ORR), demonstrating a statistically significant and clinically meaningful improvement in PFS, OS and ORR versus sunitinib in the intention-to-treat (ITT) study population. LENVIMA plus everolimus also met the trials primary endpoint of PFS and a key secondary endpoint of ORR, demonstrating a statistically significant and clinically meaningful improvement in PFS and ORR versus sunitinib in the ITT study population. The ITT population included patients across all Memorial Sloan Kettering Cancer Center (MSKCC) risk groups (favorable, intermediate and poor). The safety profiles of both KEYTRUDA plus LENVIMA and LENVIMA plus everolimus were consistent with previously reported studies. Merck and Eisai will discuss these data with regulatory authorities worldwide, with the intent to submit marketing authorization applications based on these results, which will be presented at an upcoming medical meeting.

The results for KEYTRUDA plus LENVIMA versus sunitinib, which showed a statistically significant improvement in progression-free survival, overall survival and objective response rate, build on the growing scientific evidence that supports the investigation of KEYTRUDA-based combinations for the first-line treatment of advanced renal cell carcinoma, said Dr. Gregory Lubiniecki, Associate Vice President, Oncology Clinical Research, Merck Research Laboratories. Merck and Eisai are committed to working together to continue to explore the potential of the KEYTRUDA plus LENVIMA combination, particularly in areas of great unmet need such as renal cell carcinoma.

The results from KEYNOTE-581/CLEAR (Study 307) support the potential use of KEYTRUDA plus LENVIMA for the first-line treatment of advanced RCC. These data also support the potential first-line use of LENVIMA plus everolimus, which is already approved in advanced RCC following prior antiangiogenic therapy, said Dr. Takashi Owa, Vice President, Chief Medicine Creation and Chief Discovery Officer, Oncology Business Group at Eisai. These findings energize our efforts as we continue to advance our understanding and address the unmet needs of patients with difficult-to-treat cancers.

Merck and Eisai are continuing to study the KEYTRUDA plus LENVIMA combination through the LEAP (LEnvatinib And Pembrolizumab) clinical program across 19 trials in 13 different tumor types (endometrial carcinoma, hepatocellular carcinoma, melanoma, non-small cell lung cancer, RCC, squamous cell carcinoma of the head and neck, urothelial cancer, biliary tract cancer, colorectal cancer, gastric cancer, glioblastoma, ovarian cancer and triple-negative breast cancer).

About KEYNOTE-581/CLEAR (Study 307)

KEYNOTE-581/CLEAR (Study 307) is a multi-center, randomized, open-label, Phase 3 trial (ClinicalTrials.gov, NCT02811861) evaluating LENVIMA in combination with KEYTRUDA or in combination with everolimus versus sunitinib for the first-line treatment of patients with advanced RCC. The primary endpoint is PFS by independent review per RECIST v1.1 criteria. Key secondary endpoints include OS, ORR and safety. The study enrolled approximately 1,050 patients who were randomized to one of three treatment arms to receive:

About Renal Cell Carcinoma (RCC)

Worldwide, it is estimated there were more than 403,000 new cases of kidney cancer diagnosed and more than 175,000 deaths from the disease in 2018. In the U.S. alone, it is estimated there will be nearly 74,000 new cases of kidney cancer diagnosed and almost 15,000 deaths from the disease in 2020. Renal cell carcinoma is by far the most common type of kidney cancer; about nine out of 10 kidney cancers are RCCs. Renal cell carcinoma is about twice as common in men as in women. Most cases of RCC are discovered incidentally during imaging tests for other abdominal diseases. Approximately 30% of patients with RCC will have metastatic disease at diagnosis, and as many as 40% will develop metastases after primary surgical treatment for localized RCC. Survival is highly dependent on the stage at diagnosis, and with a five-year survival rate of 12% for metastatic disease, the prognosis for these patients is poor.

About KEYTRUDA (pembrolizumab) Injection, 100 mg

KEYTRUDA is an anti-PD-1 therapy that works by increasing the ability of the bodys immune system to help detect and fight tumor cells. KEYTRUDA is a humanized monoclonal antibody that blocks the interaction between PD-1 and its ligands, PD-L1 and PD-L2, thereby activating T lymphocytes which may affect both tumor cells and healthy cells.

Merck has the industrys largest immuno-oncology clinical research program. There are currently more than 1,200 trials studying KEYTRUDA across a wide variety of cancers and treatment settings. The KEYTRUDA clinical program seeks to understand the role of KEYTRUDA across cancers and the factors that may predict a patient's likelihood of benefitting from treatment with KEYTRUDA, including exploring several different biomarkers.

Selected KEYTRUDA (pembrolizumab) Indications

Melanoma

KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic melanoma.

KEYTRUDA is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph node(s) following complete resection.

Non-Small Cell Lung Cancer

KEYTRUDA, in combination with pemetrexed and platinum chemotherapy, is indicated for the first-line treatment of patients with metastatic nonsquamous non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.

KEYTRUDA, in combination with carboplatin and either paclitaxel or paclitaxel protein-bound, is indicated for the first-line treatment of patients with metastatic squamous NSCLC.

KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with NSCLC expressing PD-L1 [tumor proportion score (TPS) 1%] as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations, and is stage III where patients are not candidates for surgical resection or definitive chemoradiation, or metastatic.

KEYTRUDA, as a single agent, is indicated for the treatment of patients with metastatic NSCLC whose tumors express PD-L1 (TPS 1%) as determined by an FDA-approved test, with disease progression on or after platinum-containing chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving KEYTRUDA.

Small Cell Lung Cancer

KEYTRUDA is indicated for the treatment of patients with metastatic small cell lung cancer (SCLC) with disease progression on or after platinum-based chemotherapy and at least 1 other prior line of therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

Head and Neck Squamous Cell Cancer

KEYTRUDA, in combination with platinum and fluorouracil (FU), is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent head and neck squamous cell carcinoma (HNSCC).

KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent HNSCC whose tumors express PD-L1 [combined positive score (CPS) 1] as determined by an FDA-approved test.

KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent or metastatic head and neck squamous cell carcinoma (HNSCC) with disease progression on or after platinum-containing chemotherapy.

Classical Hodgkin Lymphoma

KEYTRUDA is indicated for the treatment of adult patients with relapsed or refractory classical Hodgkin lymphoma (cHL).

KEYTRUDA is indicated for the treatment of pediatric patients with refractory cHL, or cHL that has relapsed after 2 or more lines of therapy.

Primary Mediastinal Large B-Cell Lymphoma

KEYTRUDA is indicated for the treatment of adult and pediatric patients with refractory primary mediastinal large B-cell lymphoma (PMBCL), or who have relapsed after 2 or more prior lines of therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials. KEYTRUDA is not recommended for treatment of patients with PMBCL who require urgent cytoreductive therapy.

Urothelial Carcinoma

KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who are not eligible for cisplatin-containing chemotherapy and whose tumors express PD-L1 [combined positive score (CPS) 10], as determined by an FDA-approved test, or in patients who are not eligible for any platinum-containing chemotherapy regardless of PD-L1 status. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who have disease progression during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.

KEYTRUDA is indicated for the treatment of patients with Bacillus Calmette-Guerin (BCG)-unresponsive, high-risk, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors who are ineligible for or have elected not to undergo cystectomy.

Microsatellite Instability-High or Mismatch Repair Deficient Cancer

KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR)

This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with MSI-H central nervous system cancers have not been established.

Microsatellite Instability-High or Mismatch Repair Deficient Colorectal Cancer

KEYTRUDA is indicated for the first-line treatment of patients with unresectable or metastatic MSI-H or dMMR colorectal cancer (CRC).

Gastric Cancer

KEYTRUDA is indicated for the treatment of patients with recurrent locally advanced or metastatic gastric or gastroesophageal junction (GEJ) adenocarcinoma whose tumors express PD-L1 (CPS 1) as determined by an FDA-approved test, with disease progression on or after two or more prior lines of therapy including fluoropyrimidine- and platinum-containing chemotherapy and if appropriate, HER2/neu-targeted therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Esophageal Cancer

KEYTRUDA is indicated for the treatment of patients with recurrent locally advanced or metastatic squamous cell carcinoma of the esophagus whose tumors express PD-L1 (CPS 10) as determined by an FDA-approved test, with disease progression after one or more prior lines of systemic therapy.

Cervical Cancer

KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy whose tumors express PD-L1 (CPS 1) as determined by an FDA-approved test. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Hepatocellular Carcinoma

KEYTRUDA is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Merkel Cell Carcinoma

KEYTRUDA is indicated for the treatment of adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC). This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Renal Cell Carcinoma

KEYTRUDA, in combination with axitinib, is indicated for the first-line treatment of patients with advanced renal cell carcinoma (RCC).

Endometrial Carcinoma

KEYTRUDA, in combination with LENVIMA, is indicated for the treatment of patients with advanced endometrial carcinoma that is not MSI-H or dMMR, who have disease progression following prior systemic therapy and are not candidates for curative surgery or radiation. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trial.

Tumor Mutational Burden-High

KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic tumor mutational burden-high (TMB-H) [10 mutations/megabase (mut/Mb)] solid tumors, as determined by an FDA-approved test, that have progressed following prior treatment and who have no satisfactory alternative treatment options. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with TMB-H central nervous system cancers have not been established.

Cutaneous Squamous Cell Carcinoma

KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cutaneous squamous cell carcinoma (cSCC) that is not curable by surgery or radiation.

Selected Important Safety Information for KEYTRUDA (pembrolizumab)

Immune-Mediated Pneumonitis

KEYTRUDA can cause immune-mediated pneumonitis, including fatal cases. Pneumonitis occurred in 3.4% (94/2799) of patients with various cancers receiving KEYTRUDA, including Grade 1 (0.8%), 2 (1.3%), 3 (0.9%), 4 (0.3%), and 5 (0.1%). Pneumonitis occurred in 8.2% (65/790) of NSCLC patients receiving KEYTRUDA as a single agent, including Grades 3-4 in 3.2% of patients, and occurred more frequently in patients with a history of prior thoracic radiation (17%) compared to those without (7.7%). Pneumonitis occurred in 6% (18/300) of HNSCC patients receiving KEYTRUDA as a single agent, including Grades 3-5 in 1.6% of patients, and occurred in 5.4% (15/276) of patients receiving KEYTRUDA in combination with platinum and FU as first-line therapy for advanced disease, including Grades 3-5 in 1.5% of patients.

Monitor patients for signs and symptoms of pneumonitis. Evaluate suspected pneumonitis with radiographic imaging. Administer corticosteroids for Grade 2 or greater pneumonitis. Withhold KEYTRUDA for Grade 2; permanently discontinue KEYTRUDA for Grade 3 or 4 or recurrent Grade 2 pneumonitis.

Immune-Mediated Colitis

KEYTRUDA can cause immune-mediated colitis. Colitis occurred in 1.7% (48/2799) of patients receiving KEYTRUDA, including Grade 2 (0.4%), 3 (1.1%), and 4 (<0.1%). Monitor patients for signs and symptoms of colitis. Administer corticosteroids for Grade 2 or greater colitis. Withhold KEYTRUDA for Grade 2 or 3; permanently discontinue KEYTRUDA for Grade 4 colitis.

Immune-Mediated Hepatitis (KEYTRUDA) and Hepatotoxicity (KEYTRUDA in Combination With Axitinib)

Immune-Mediated Hepatitis

KEYTRUDA can cause immune-mediated hepatitis. Hepatitis occurred in 0.7% (19/2799) of patients receiving KEYTRUDA, including Grade 2 (0.1%), 3 (0.4%), and 4 (<0.1%). Monitor patients for changes in liver function. Administer corticosteroids for Grade 2 or greater hepatitis and, based on severity of liver enzyme elevations, withhold or discontinue KEYTRUDA.

Hepatotoxicity in Combination With Axitinib

KEYTRUDA in combination with axitinib can cause hepatic toxicity with higher than expected frequencies of Grades 3 and 4 ALT and AST elevations compared to KEYTRUDA alone. With the combination of KEYTRUDA and axitinib, Grades 3 and 4 increased ALT (20%) and increased AST (13%) were seen. Monitor liver enzymes before initiation of and periodically throughout treatment. Consider more frequent monitoring of liver enzymes as compared to when the drugs are administered as single agents. For elevated liver enzymes, interrupt KEYTRUDA and axitinib, and consider administering corticosteroids as needed.

Immune-Mediated Endocrinopathies

KEYTRUDA can cause adrenal insufficiency (primary and secondary), hypophysitis, thyroid disorders, and type 1 diabetes mellitus. Adrenal insufficiency occurred in 0.8% (22/2799) of patients, including Grade 2 (0.3%), 3 (0.3%), and 4 (<0.1%). Hypophysitis occurred in 0.6% (17/2799) of patients, including Grade 2 (0.2%), 3 (0.3%), and 4 (<0.1%). Hypothyroidism occurred in 8.5% (237/2799) of patients, including Grade 2 (6.2%) and 3 (0.1%). The incidence of new or worsening hypothyroidism was higher in 1185 patients with HNSCC (16%) receiving KEYTRUDA, as a single agent or in combination with platinum and FU, including Grade 3 (0.3%) hypothyroidism. Hyperthyroidism occurred in 3.4% (96/2799) of patients, including Grade 2 (0.8%) and 3 (0.1%), and thyroiditis occurred in 0.6% (16/2799) of patients, including Grade 2 (0.3%). Type 1 diabetes mellitus, including diabetic ketoacidosis, occurred in 0.2% (6/2799) of patients.

Monitor patients for signs and symptoms of adrenal insufficiency, hypophysitis (including hypopituitarism), thyroid function (prior to and periodically during treatment), and hyperglycemia. For adrenal insufficiency or hypophysitis, administer corticosteroids and hormone replacement as clinically indicated. Withhold KEYTRUDA for Grade 2 adrenal insufficiency or hypophysitis and withhold or discontinue KEYTRUDA for Grade 3 or Grade 4 adrenal insufficiency or hypophysitis. Administer hormone replacement for hypothyroidism and manage hyperthyroidism with thionamides and beta-blockers as appropriate. Withhold or discontinue KEYTRUDA for Grade 3 or 4 hyperthyroidism. Administer insulin for type 1 diabetes, and withhold KEYTRUDA and administer antihyperglycemics in patients with severe hyperglycemia.

Immune-Mediated Nephritis and Renal Dysfunction

KEYTRUDA can cause immune-mediated nephritis. Nephritis occurred in 0.3% (9/2799) of patients receiving KEYTRUDA, including Grade 2 (0.1%), 3 (0.1%), and 4 (<0.1%) nephritis. Nephritis occurred in 1.7% (7/405) of patients receiving KEYTRUDA in combination with pemetrexed and platinum chemotherapy. Monitor patients for changes in renal function. Administer corticosteroids for Grade 2 or greater nephritis. Withhold KEYTRUDA for Grade 2; permanently discontinue for Grade 3 or 4 nephritis.

Immune-Mediated Skin Reactions

Immune-mediated rashes, including Stevens-Johnson syndrome (SJS), toxic epidermal necrolysis (TEN) (some cases with fatal outcome), exfoliative dermatitis, and bullous pemphigoid, can occur. Monitor patients for suspected severe skin reactions and based on the severity of the adverse reaction, withhold or permanently discontinue KEYTRUDA and administer corticosteroids. For signs or symptoms of SJS or TEN, withhold KEYTRUDA and refer the patient for specialized care for assessment and treatment. If SJS or TEN is confirmed, permanently discontinue KEYTRUDA.

Other Immune-Mediated Adverse Reactions

Immune-mediated adverse reactions, which may be severe or fatal, can occur in any organ system or tissue in patients receiving KEYTRUDA and may also occur after discontinuation of treatment. For suspected immune-mediated adverse reactions, ensure adequate evaluation to confirm etiology or exclude other causes. Based on the severity of the adverse reaction, withhold KEYTRUDA and administer corticosteroids. Upon improvement to Grade 1 or less, initiate corticosteroid taper and continue to taper over at least 1 month. Based on limited data from clinical studies in patients whose immune-related adverse reactions could not be controlled with corticosteroid use, administration of other systemic immunosuppressants can be considered. Resume KEYTRUDA when the adverse reaction remains at Grade 1 or less following corticosteroid taper. Permanently discontinue KEYTRUDA for any Grade 3 immune-mediated adverse reaction that recurs and for any life-threatening immune-mediated adverse reaction.

The following clinically significant immune-mediated adverse reactions occurred in less than 1% (unless otherwise indicated) of 2799 patients: arthritis (1.5%), uveitis, myositis, Guillain-Barr syndrome, myasthenia gravis, vasculitis, pancreatitis, hemolytic anemia, sarcoidosis, and encephalitis. In addition, myelitis and myocarditis were reported in other clinical trials, including classical Hodgkin lymphoma, and postmarketing use.

Treatment with KEYTRUDA may increase the risk of rejection in solid organ transplant recipients. Consider the benefit of treatment vs the risk of possible organ rejection in these patients.

Infusion-Related Reactions

KEYTRUDA can cause severe or life-threatening infusion-related reactions, including hypersensitivity and anaphylaxis, which have been reported in 0.2% (6/2799) of patients. Monitor patients for signs and symptoms of infusion-related reactions. For Grade 3 or 4 reactions, stop infusion and permanently discontinue KEYTRUDA.

Complications of Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)

Immune-mediated complications, including fatal events, occurred in patients who underwent allogeneic HSCT after treatment with KEYTRUDA. Of 23 patients with cHL who proceeded to allogeneic HSCT after KEYTRUDA, 6 (26%) developed graft-versus-host disease (GVHD) (1 fatal case) and 2 (9%) developed severe hepatic veno-occlusive disease (VOD) after reduced-intensity conditioning (1 fatal case). Cases of fatal hyperacute GVHD after allogeneic HSCT have also been reported in patients with lymphoma who received a PD-1 receptorblocking antibody before transplantation. Follow patients closely for early evidence of transplant-related complications such as hyperacute graft-versus-host disease (GVHD), Grade 3 to 4 acute GVHD, steroid-requiring febrile syndrome, hepatic veno-occlusive disease (VOD), and other immune-mediated adverse reactions.

In patients with a history of allogeneic HSCT, acute GVHD (including fatal GVHD) has been reported after treatment with KEYTRUDA. Patients who experienced GVHD after their transplant procedure may be at increased risk for GVHD after KEYTRUDA. Consider the benefit of KEYTRUDA vs the risk of GVHD in these patients.

Increased Mortality in Patients With Multiple Myeloma

In trials in patients with multiple myeloma, the addition of KEYTRUDA to a thalidomide analogue plus dexamethasone resulted in increased mortality. Treatment of these patients with a PD-1 or PD-L1 blocking antibody in this combination is not recommended outside of controlled trials.

Embryofetal Toxicity

Based on its mechanism of action, KEYTRUDA can cause fetal harm when administered to a pregnant woman. Advise women of this potential risk. In females of reproductive potential, verify pregnancy status prior to initiating KEYTRUDA and advise them to use effective contraception during treatment and for 4 months after the last dose.

Adverse Reactions

In KEYNOTE-006, KEYTRUDA was discontinued due to adverse reactions in 9% of 555 patients with advanced melanoma; adverse reactions leading to permanent discontinuation in more than one patient were colitis (1.4%), autoimmune hepatitis (0.7%), allergic reaction (0.4%), polyneuropathy (0.4%), and cardiac failure (0.4%). The most common adverse reactions (20%) with KEYTRUDA were fatigue (28%), diarrhea (26%), rash (24%), and nausea (21%).

In KEYNOTE-002, KEYTRUDA was permanently discontinued due to adverse reactions in 12% of 357 patients with advanced melanoma; the most common (1%) were general physical health deterioration (1%), asthenia (1%), dyspnea (1%), pneumonitis (1%), and generalized edema (1%). The most common adverse reactions were fatigue (43%), pruritus (28%), rash (24%), constipation (22%), nausea (22%), diarrhea (20%), and decreased appetite (20%).

In KEYNOTE-054, KEYTRUDA was permanently discontinued due to adverse reactions in 14% of 509 patients; the most common (1%) were pneumonitis (1.4%), colitis (1.2%), and diarrhea (1%). Serious adverse reactions occurred in 25% of patients receiving KEYTRUDA. The most common adverse reaction (20%) with KEYTRUDA was diarrhea (28%).

In KEYNOTE-189, when KEYTRUDA was administered with pemetrexed and platinum chemotherapy in metastatic nonsquamous NSCLC, KEYTRUDA was discontinued due to adverse reactions in 20% of 405 patients. The most common adverse reactions resulting in permanent discontinuation of KEYTRUDA were pneumonitis (3%) and acute kidney injury (2%). The most common adverse reactions (20%) with KEYTRUDA were nausea (56%), fatigue (56%), constipation (35%), diarrhea (31%), decreased appetite (28%), rash (25%), vomiting (24%), cough (21%), dyspnea (21%), and pyrexia (20%).

In KEYNOTE-407, when KEYTRUDA was administered with carboplatin and either paclitaxel or paclitaxel protein-bound in metastatic squamous NSCLC, KEYTRUDA was discontinued due to adverse reactions in 15% of 101 patients. The most frequent serious adverse reactions reported in at least 2% of patients were febrile neutropenia, pneumonia, and urinary tract infection. Adverse reactions observed in KEYNOTE-407 were similar to those observed in KEYNOTE-189 with the exception that increased incidences of alopecia (47% vs 36%) and peripheral neuropathy (31% vs 25%) were observed in the KEYTRUDA and chemotherapy arm compared to the placebo and chemotherapy arm in KEYNOTE-407.

In KEYNOTE-042, KEYTRUDA was discontinued due to adverse reactions in 19% of 636 patients with advanced NSCLC; the most common were pneumonitis (3%), death due to unknown cause (1.6%), and pneumonia (1.4%). The most frequent serious adverse reactions reported in at least 2% of patients were pneumonia (7%), pneumonitis (3.9%), pulmonary embolism (2.4%), and pleural effusion (2.2%). The most common adverse reaction (20%) was fatigue (25%).

Read the original here:
KEYTRUDA (pembrolizumab) Plus LENVIMA (lenvatinib) Demonstrated Statistically Significant Improvement in Progression-Free Survival (PFS), Overall...

Read More...

Gilead and Kite to Share Latest Scientific Advances in Hematologic Malignancies at ASH 2020 – Investing News Network

November 11th, 2020 11:54 am

16 Abstracts, Including Three Oral Presentations, Highlight Breadth of Companys Innovation in Immuno-Oncology for Patients with Blood Cancers

Kite Data Highlight Yescarta Long-Term Efficacy in Relapsed/Refractory Large B-Cell Lymphoma, its Potential as An Earlier Line of Therapy in DLBCL, as well as Results in Other Cancers, and One-Year Follow-up Results for Tecartus in Relapsed Mantle Cell Lymphoma

Magrolimab Demonstrates Continued Response Rates in Updated Results of Phase 1b Study of Acute Myeloid Leukemia Patients, Including Those with TP53 Mutation

Gilead Sciences, Inc. (Nasdaq: GILD) and Kite, a Gilead Company, today announced that 16 abstracts, including three oral presentations from the companies combined immuno-oncology research and development programs, have been accepted for presentation at the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition. The meeting, which is being held virtually on December 5-8, 2020, will feature presentations on Yescarta (axicabtagene ciloleucel), Tecartus (brexucabtagene autoleucel, KTE-X19) and other ongoing research from Kites chimeric antigen receptor (CAR) T cell therapy development program, as well as magrolimab, Gileads first-in-class, investigational anti-CD47 monoclonal antibody.

This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20201105005130/en/

The evidence supporting our innovation in hematologic malignancies continues to grow, providing assurance of the lasting and positive impact our diverse oncology pipeline could achieve over time, said Merdad Parsey, MD, PhD, Chief Medical Officer, Gilead Sciences. We continue to see broad potential across our oncology portfolio anchored by Kite in cell therapy and Gileads anti-CD47 monoclonal antibody to transform care for patients with hard-to-treat blood cancers.

New Long-Term Efficacy Data and the Potential of CAR T Therapy for More Patients

Building on three-year data presented at ASH 2019, overall survival results at four years from the pivotal ZUMA-1 trial of Yescarta in patients with refractory large B-cell lymphoma will be presented (Abstract #1187). Additionally, new data include one-year follow-up results from the ZUMA-2 study evaluating KTE-X19 in relapsed or refractory mantle cell lymphoma (Abstract #1120), as well as several studies evaluating the potential of Yescarta in new indications include an interim analysis of ZUMA-12 in first-line large B-cell lymphoma among patients with high-risk features (Abstract #405) and the ZUMA-5 primary analysis in relapsed or refractory indolent non-Hodgkin lymphoma (NHL), including follicular lymphoma (FL) and marginal zone lymphoma (MZL; Abstract #700).

Data from the ZUMA-5 primary analysis form the basis for the supplemental Biologics License Application (sBLA) for Yescarta currently under review by the U.S. Food & Drug Administration (FDA). Yescarta has previously been granted a Breakthrough Therapy Designation by the FDA for relapsed or refractory FL or MZL after at least two prior therapies and has been granted a Priority Review with a target action date, under the Prescription Drug User Fee Act (PDUFA), of March 5, 2021.

Our data at ASH build on the established strengths of our CAR T franchise, including practice-changing potential in new cancers, said Ken Takeshita, MD, Kites Global Head of Clinical Development. As we become the first company to present four-year CAR T data from a pivotal study in large B-cell lymphoma and continue to expand our leadership in cell therapy across different hematologic malignancies and into earlier lines of therapy, we remain committed to bringing the benefits of cell therapies to as many patients as possible.

Harnessing Potential First-in-Class Anti-CD47 Antibody in Difficult-to-Treat Malignancies

Researchers will give an oral presentation of updated results from the Phase 1b study of magrolimab in patients with previously-untreated acute myeloid leukemia (AML) who cannot undergo treatment with intensive chemotherapy, including patients with TP53 -mutant AML (Abstract #330). The FDA recently assigned Breakthrough Designation to magrolimab, in combination with azacitidine for the treatment of adult patients with newly-diagnosed MDS including intermediate-, high-, or very high-risk tumor types to expedite the development and regulatory review of this investigational treatment. Magrolimab also received PRIME Designation for treatment of MDS from the European Medicines Agency (EMA).

Dates and times for all accepted abstracts are as follows:

Area of Focus, Presentation Number and Date/Time

Abstract Title

Oral Presentations

Acute Myeloid Leukemia

Abstract #330

Sunday, Dec 6

(12:30pm ET / 9:30am PT)

The First-in-Class Anti-CD47 Antibody Magrolimab Combined with Azacitidine Is Well-Tolerated and Effective in AML Patients: Phase 1b Results

Large B-cell Lymphoma

Abstract #405

Sunday, Dec 6

(4:15pm ET / 1:15pm PT)

Interim Analysis of ZUMA-12: A Phase 2 Study of Axicabtagene Ciloleucel (Axi-Cel) as First-Line Therapy in Patients (Pts) with High-Risk Large B Cell Lymphoma (LBCL)

Non-Hodgkin Lymphoma Abstract #700

Monday, Dec 7

(4:30pm ET / 1:30pm PT)

Primary Analysis of ZUMA-5: A Phase 2 Study of Axicabtagene Ciloleucel (Axi-Cel) in Patients With Relapsed/Refractory (R/R) Indolent Non-Hodgkin Lymphoma (iNHL)

Poster Presentations

Follicular Lymphoma

Abstract #1145

Saturday, Dec 5

(10:00am ET / 7:00am PT)

Safety Profile of Idelalisib in Patients with Refractory Follicular Lymphoma: Interim Analysis of a Noninterventional Study

Large B-cell Lymphoma Abstract #1187

Saturday, Dec 5

(10:00am ET / 7:00am PT)

Long-Term Survival and Gradual Recovery of B Cells in Patients With Refractory Large B Cell Lymphoma Treated With Axicabtagene Ciloleucel (Axi-Cel)

Large B-cell Lymphoma Abstract #2100

Sunday, Dec 6

(10:00am ET / 7:00am PT)

Outcomes of Patients (Pts) in ZUMA-9, A Multicenter, Open-Label Study of Axicabtagene Ciloleucel (Axi-Cel) in Relapsed/Refractory Large B Cell Lymphoma (R/R LBCL) for Expanded Access and Commercial Out-of-Specification (OOS) Product

Large B-cell Lymphoma

Abstract #1224

Saturday, Dec 5

(10:00am ET / 7:00am PT)

The First Retrospective Commercial Claims-Based Analysis of CAR T Treated Patients With Relapsed or Refractory Large B-Cell Lymphoma (R/R LBCL)

Large B-cell Lymphoma

Abstract #2500

Sunday, Dec 6

(10:00am ET / 7:00am PT)

Cost and Healthcare Utilization in Relapsed/Refractory Diffuse Large B-Cell Lymphoma: A Real-World Analysis of Medicare Beneficiaries Receiving Chimeric Antigen Receptor T-Cell Vs. Autologous and Allogeneic Hematopoietic Stem Cell Transplants

Large B-cell Lymphoma

Abstract #2548

Sunday, Dec 6

(10:00am ET / 7:00am PT)

Burden of Illness and Outcomes in the 2nd Line Treatment of Large B-Cell Lymphoma: A Real-World Comparison of Medicare Beneficiaries with and without Stem Cell Transplants

Large B-cell Lymphoma

Abstract #1646

Saturday, Dec 5

(10:00am ET / 7:00am PT)

Lines of Therapy in Patients with Relapsed or Refractory Large B-Cell Lymphoma and Stem Cell Transplant-Intended Treatment

Mantle Cell Lymphoma

Abstract #1120

Saturday, Dec 5

(10:00am ET / 7:00am PT)

One-Year Follow-Up of ZUMA-2, the Multicenter, Registrational Study of KTE-X19 in Patients With Relapsed/Refractory Mantle Cell Lymphoma

Mantle Cell Lymphoma

Abstract #1126

Saturday, Dec 5

(10:00am ET / 7:00am PT)

Pharmacological Profile and Clinical Outcomes of KTE-X19 by Prior Bruton Tyrosine Kinase Inhibitors (BTKi) Exposure or Mantle Cell Lymphoma (MCL) Morphology in Patients With Relapsed/Refractory (R/R) MCL in the ZUMA-2 Trial

Non-Hodgkin Lymphoma

Abstract #2036

Sunday, Dec 6

(10:00am ET / 7:00am PT)

Retreatment With Axicabtagene Ciloleucel (Axi-Cel) in Patients With Relapsed/Refractory Indolent Non-Hodgkin Lymphoma in ZUMA-5

Trials-In-Progress

Acute Lymphoblastic Leukemia & Non-Hodgkin Lymphoma

Abstract #1896

Sunday, Dec 6

(10:00am ET / 7:00am PT)

ZUMA-4: A Phase 1/2 Multicenter Study of KTE-X19 in Pediatric and Adolescent Patients With Relapsed/Refractory B Cell Acute Lymphoblastic Leukemia or Non-Hodgkin Lymphoma

Large B-cell Lymphoma

Abstract #2103

Sunday, Dec 6

(10:00am ET / 7:00am PT)

ZUMA-19: A Phase 1/2 Multicenter Study of Lenzilumab Use with Axicabtagene Ciloleucel (Axi-Cel) in Patients (Pts) With Relapsed or Refractory Large B Cell Lymphoma (R/R LBCL)

Online Publication

Follicular Lymphoma

Efficacy Outcomes of Treatments for Double Relapsed/Refractory Follicular Lymphoma (R/R FL): A Systematic Literature Review

For more information, including a complete list of abstract titles at the meeting, please visit: https://ash.confex.com/ash/2020/webprogram/start.html .

Yescarta was the first CAR T cell therapy to be approved by the FDA for the treatment of adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, primary mediastinal large B-cell lymphoma, and high grade B-cell lymphoma and DLBCL arising from FL. Yescarta is not indicated for the treatment of patients with primary central nervous system lymphoma. In July, Tecartus became the first CAR T cell therapy to receive accelerated approval from the FDA for the treatment of relapsed or refractory mantle cell lymphoma, based on overall response rate and durability of response. Continued approval for this indication may be contingent upon additional data from a confirmatory trial. The U.S. Prescribing Information for Yescarta and Tecartus each have BOXED WARNINGS for the risks of CRS and neurologic toxicities, and Yescarta and Tecartus are each approved with a risk evaluation and mitigation strategy (REMS) due to these risks; see below for Important Safety Information.

The uses of Yescarta in relapsed or refractory FL or MZL or as a first-line treatment for patients with large B-cell lymphoma and high-risk genetics are investigational and not approved anywhere globally. Its efficacy and safety have not been established for these indications.

Magrolimab is investigational and not approved anywhere globally. Its efficacy and safety have not been established. More information about clinical trials with magrolimab is available at http://www.clinicaltrials.gov .

ABOUT MAGROLIMAB

Magrolimab is a first-in-class, investigational monoclonal antibody against CD47 and macrophage checkpoint inhibitor that is designed to interfere with recognition of CD47 by the SIRP receptor on macrophages, thus blocking the dont eat me signal used by cancer cells to avoid being ingested by macrophages. Magrolimab is being developed in several hematologic and solid tumor malignancies, including MDS. Magrolimab has been granted Fast Track Designation for the treatment of MDS, AML, DLBCL and FL. Magrolimab has also been granted Orphan Drug Designation by the FDA and EMA for treatment of MDS and AML.

About Yescarta

Original post:
Gilead and Kite to Share Latest Scientific Advances in Hematologic Malignancies at ASH 2020 - Investing News Network

Read More...

The great reset: new danger on the horizon – Amandala

November 11th, 2020 11:54 am

Belize City, Nov. 2, 2020 Most people in Belize are either taken up these days with finding a job/income, with fears of COVID-19, or with anticipation of the General Elections of Nov. 11, 2020. But lurking in the shadows is a much more dangerous foe.

In the past it was called The New World Order, but that has been so discredited, that the wizard behind the curtain had to change the name to The Great Reset. What is this Great Reset?

The Great Reset is a new social contract that ties you to it through an electronic ID linked to your bank account and health records, and a social credit ID that will dictate every facet of your life. While the COVID-19 pandemic is being used as a justification for the Great Reset movement, the agenda has nothing to do with health and everything to do with a long-term plan to monitor and control the world through digital surveillance and artificial intelligence.

The Great Reset and the Fourth Industrial Revolution are rebranded terms for the old New World Order, melded with the trans-humanist movement. Technocracy (which is the new name for Fascism) is an economic system of resource allocation that revolves around computer technology in particular artificial intelligence, digital surveillance, and Big Data (5G) collection and the digitization of industry and government, which in turn allows for the automation of social engineering and social rule, thereby doing away with the need for democratically elected leadership.

While the real plan is to usher in a tech-driven dystopia free of democratic controls, the elites speak of this plan as a way to bring us back into harmony with nature the Green New Deal. Importantly, the pandemic is being used to destroy local economies around the world, which will then allow the World Economic Forum to come in through the IMF and rescue debt-ridden countries through facilitated financial bailouts.

However, the price for this salvation is your personal freedom and liberty. And, again, one of the aspects of the Fascist plan is to eliminate national borders and nationalism in general.

Who are the main actors behind the Great Reset?

Bill Gates and the World Economic Forum, along with the United Nations (which keeps a relatively low profile), appear to be at the heart of the big boys agenda. Gates is also the largest money-bag for the World Health Organization the medical branch of the U.N. Other key partners that play important roles in the implementation of the elites/globalists agenda include foundations such as the Rockefeller Foundation, the Rockefeller Brothers Fund, the Ford Foundation, Bloomberg Philanthropies, the UN Foundation, and George Soros Open Society Foundation; companies such as: Avanti Communications, a British provider of satellite technology with global connectivity, and 2030 Vision, a partnership of technology giants that is aimed at providing the infrastructure and technology solutions needed to realize the U.N.s 2030 Sustainable Development Goals. 2030 Vision is also partnered with Frontier 2030, which is a partnership of organizations under the helm of the World Economic Forum.

These organizations include the major Wall Street bankers/financiers; Google, the No. 1 Big Data collector in the world and a leader in AI services; MasterCard, which is leading the globalist charge to develop digital IDs and banking services, and Salesforce, a global leader in cloud computing, the internet of things and artificial intelligence.

Incidentally, Salesforce is led by Marc Benioff, who is also on the World Economic Forums board of directors, and Professor Klaus Schwab, chairman of the World Economic Forum.

Most Belizeans know little or nothing about the trans-humanist movement, or Human 2.0, which is geared at transcending biology through computer technology. Or, as Dr. Carrie Madej of USA explains in a blog, their goal is to meld human biology with computer technology and artificial intelligence. Two visible proponents of trans-humanism are Ray Kurzweil (director of engineering at Google since 2012) and Elon Musk (founder of SpaceX, Tesla and Neuralink). According to Dr. Madej, today we may be standing at the literal crossroads of trans-humanism, thanks to the fast approaching release of one or more mRNA COVID-19 vaccines.

Many of the COVID 19 vaccines https://articles.mercola.com/sites/articles/archive/2020/05/22/coronavirus-vaccine-timetable.aspx are not conventional vaccines. Their design is aimed at manipulating your very biology, and therefore have the potential to alter the biology of the entire human race. Conventional vaccines train your body to recognize and respond to the proteins of a particular virus by injecting a small amount of the actual viral protein into your body, thereby triggering an immune response from your body and the development of antibodies.This is not what happens with an mRNA vaccine. The theory behind these vaccines is that when you inject the mRNA into your cells, it will stimulate your cells to manufacture their own viral protein. The mRNA COVID-19 vaccine will be the first of its kind. No mRNA vaccine has ever been licensed before. And, to add insult to injury, theyre forgoing all animal safety testing.

Madej has reviewed the background of certain individuals participating in the race for a COVID-19 vaccine, which include Moderna co-founder Derrick Rossi, a Harvard researcher who successfully reprogrammed stem cells using modified RNA, thus changing the function of the stem cells. Moderna was founded on this concept of being able to modify human biological function through genetic engineering.

The mRNA vaccines are designed to instruct your cells to make the SARS-CoV-2 spike protein, the glycoprotein that attaches to the ACE2 receptor of the cell. The idea is that by creating the SARS-CoV-2 spike protein, your immune system will mount a response to it and begin producing antibodies to the virus.

However, as we now know, Moderna is having problems, because both the CEO and CFO have, according to the Wall Street Journal, dumped their shares and sold everything, making some $350 million + dollars.

But the biggest insult by the globalists to our intelligence is the censorship of the news about the research done by genetic analysis using the Oak Ridge National Lab supercomputer called the Summit which has revealed an interesting new hypothesis that helps explain the disease progression of COVID-19. A September 1, 2020 Medium article1 by Thomas Smith reviewed the findings of what is now referred to as the Bradykinin hypothesis.

As reported by Smith, the computer crunched data on more than 40,000 genes obtained from 17,000 genetic samples.

Summit is the second-fastest computer in the world, but the process which involved analysing 2.5 billion genetic combinations still took more than a week. When Summit was done, researchers analysed the results. It was, in the words of Dr. Daniel Jacobson, lead researcher and chief scientist for computational systems biology at Oak Ridge, a eureka moment.

Bradykinin is a chemical that helps regulate your blood pressure and is controlled by your renin-angiotensin system (RAS). As explained in the Academic Press book on vitamin D (which has a significant impact on the RAS):

The renin-angiotensin system (RAS) is a central regulator of renal and cardiovascular functions. Over-activation of the RAS leads to renal and cardiovascular disorders, such as hypertension and chronic kidney disease, the major risk factors for stroke, myocardial infarction, congestive heart failure, progressive atherosclerosis, and renal failure.

The Bradykinin hypothesis provides a model that helps explain some of the more unusual symptoms of COVID-19, including its bizarre effects on the cardiovascular system. It also strengthens the hypothesis that vitamin D plays a really important role in the disease.

Your ACE2 receptors are the primary gateways of the virus, as the virus spike protein binds to the ACE2 receptor. As explained2 by Smith:

COVID-19 infection generally begins when the virus enters the body through ACE2 receptors in the nose The virus then proceeds through the body, entering cells in other places where ACE2 is also present But once Covid-19 has established itself in the body, things start to get really interesting The data Summit analysed shows that COVID-19 isnt content to simply infect cells that already express lots of ACE2 receptors. Instead, it actively hijacks the bodys own systems, tricking it into up-regulating ACE2 receptors in places where theyre usually expressed at low or medium levels, including the lungs.

In this sense, COVID-19 is like a burglar who slips in your unlocked second-floor window and starts to ransack your house. Once inside, though, they dont just take your stuff they also throw open all your doors and windows so their accomplices can rush in and help pillage more efficiently.

The end result is a Bradykinin storm, and according to the researchers, this appears to be an important factor in many of COVID-19s lethal effects, even more so than the Cytokine storms associated with the disease. As Bradykinin accumulates, the more serious COVID-19 symptoms appear. Mounting clinical data suggest COVID-19 is actually primarily a vascular disease rather than a respiratory one, and runaway Bradykinin build-up help explain this.

The good news is that since Bradykinin storms are to blame, there are a number of already existing drugs (Icatibant, Danazol, Stanozolol) that can help prevent Bradykinin storms, and there are many other safe, inexpensive strategies like nebulized peroxide, ozone, molecular hydrogen, steroids, exogenous ketones, and Quercetin with zinc, vitamin D, and high-dose vitamin C.

And there are two reports by the American CDC. One says that 70.6% of COVID-19 patients always wore a mask3. The other says only 6% of all COVID-19 deaths were due ONLY to coronavirus4. And yet another said that the common seasonal flu caused more deaths than COVID-19.

So, if COVID-19 deaths are not what is being reported by the mass media, if the SAR CoV-2 virus is not as deadly to humans, then why the lockdowns, the face masks, the social distancing, the destruction of the way we live, of our economies? Why? Why?

But not all men are blind. On Oct 25, 2020, the Archbishop of Ulpiana, former Apostolic Nuncio to the United States of America, Carlo Maria Vigano, wrote an open letter (which over 100 million Americans have read) to President Donald Trump. The letter is long and is all over the internet. This is some of it:

at this hour in which the fate of the whole world is being threatened by a global conspiracy against God and humanityin the midst of the silence of both civil and religious authoritiesthis historical moment sees the forces of Evil aligned in a battle against the children of Lightwe see heads of nations and religious leaders pandering to this suicide of Western culture and its Christian soul, while the fundamental rights of citizens and believers are denied in the name of a health emergency that is revealing itself more and more fully as instrumental to the establishment of an inhuman faceless tyranny.

A global plan called the Great Reset is underway. Its architect is a global lite that wants to subdue all of humanity, imposing coercive measures with which to drastically limit individual freedoms and those of entire populations Behind the world leaders who are the accomplices and executors of this infernal project, there are unscrupulous characters who finance the World Economic Forum and Event 201, promoting their agenda.

The purpose of the Great Reset is the imposition of a health dictatorship aiming at the imposition of liberticidal measures, hidden behind tempting promises of ensuring a universal income and cancelling individual debt. The price of these concessions from the International Monetary Fund will be the renunciation of private property and adherence to a program of vaccination against Covid-19 and Covid-21 promoted by Bill Gates with the collaboration of the main pharmaceutical groups. Beyond the enormous economic interests that motivate the promoters of the Great Reset, the imposition of the vaccination will be accompanied by the requirement of a health passport and a digital ID, with the consequent contact tracing of the population of the entire world. Those who do not accept these measures will be confined in detention camps or placed under house arrest, and all their assets will be confiscated.

Mr President, I imagine that you are already aware that in some countries the Great Reset will be activated between the end of this year and the first trimester of 2021. For this purpose, further lockdowns are planned, which will be officially justified by a supposed second and third wave of the pandemic. But this world, Mr. President, includes people, affections, institutions, faith, culture, traditions, and ideals: people and values that do not act like automatons, who do not obey like machines, because they are endowed with a soul and a heart, because they are tied together by a spiritual bond that draws its strength from above, from that God that our adversaries want to challenge, just as Lucifer did at the beginning of time with his non serviam.

Until a few months ago, it was easy to smear as conspiracy theorists those who denounced these terrible plans, which we now see being carried out down to the smallest detail. No one, up until last February, would ever have thought that, in all of our cities, citizens would be arrested simply for wanting to walk down the street, to breathe, to want to keep their business open, to want to go to church on Sunday. Yet, now it is happening all over the world.

Mr. President, you have clearly stated that you want to defend the nation One Nation under God, fundamental liberties, and non-negotiable values that are denied and fought against today. It is you, dear President, who are the one who opposes the deep state, the final assault of the children of darkness.

For this reason, it is necessary that all people of goodwill be persuaded of the epochal importance of the imminent election Your adversary is also our adversary: it is the Enemy of the human race, He who is a murderer from the beginning (Jn 8:44).

And yet, in the midst of this bleak picture, this apparently unstoppable advance of the Invisible Enemy, an element of hope emerges. The adversary does not know how to love, and it does not understand that it is not enough to assure a universal income or to cancel mortgages in order to subjugate the masses and convince them to be branded like cattle. This people, which for too long has endured the abuses of a hateful and tyrannical power, is rediscovering that it has a soul; it is understanding that it is not willing to exchange its freedom for the homogenization and cancellation of its identity; it is beginning to understand the value of familial and social ties, of the bonds of faith and culture that unite honest people.

This Great Reset is destined to fail because those who planned it do not understand that there are still people ready to take to the streets to defend their rights, to protect their loved ones, to give a future to their children and grandchildren. The levelling inhumanity of the globalist project will shatter miserably in the face of the firm and courageous. To be an instrument of Divine Providence is a great responsibility, for which you will certainly receive all the graces of state that you need, since they are being fervently implored for you by the many people who support you with their prayers.

Meanwhile, here in Belize, we kill our so-called COVID-19 patients. Ventilators will kill you. Doctors of Belize, read the report of the US Oak Ridge National Lab on COVID-19. NO one needs to die anymore from COVID-19. US President Trump, who is 74 years old, was cured after 3 days of COVID-19.

And by the time you read this article, the world will know who won the elections in the United States.

Curfew on Nov. 11, election night in Belize, is part of the Globalist agenda. Let the people celebrate their victory. Open the churches, the schools, the bars; open the society. Send the globalist/elites back to Hell with Lucifer.

(Footnotes)1https://elemental.medium.com/a-supercomputer-analyzed-covid-19-and-an-interesting-new-theory-has-emerged-31cb8eba9d63

2ibid3https://www.cdc.gov/mmwr/volumes/69/wr/mm6936a5.htmRead the table at the end.4https://www.cdc.gov/nchs/nvss/vsrr/covid_weekly/index.html

Link:
The great reset: new danger on the horizon - Amandala

Read More...

Teladoc Is A Strong Buy: A Radical Healthcare Change Will Come – Seeking Alpha

November 11th, 2020 11:54 am

Introduction

Last week, the Teladoc (TDOC) and Livongo (LVGO) merger was completed. That means that Livongo doesn't trade anymore. If you still had your Livongo shares, you got (or will get if your broker is a bit slower) 0.5920 per share of Livongo in Teladoc shares plus cash of $11.33, paid with a special dividend of $7.09 on October 29th, and the rest when your shares were changed to Teladoc shares.

(Source)

As a former shareholder of Livongo, you may not be completely familiar with how Teladoc is positioned now. If there is a buy-out or a merger, that always generates mixed feelings, or at least it should. If it doesn't, it means that you had a bad stock in your portfolio.

With Potential Multibaggers, my marketplace service here on Seeking Alpha, I try to find multibaggers early on. I picked Livongo as a Potential Multibagger on December 26, 2019. The stock then traded at $24.86 and had a market cap of just $2.5B. The stock returned 462.2%, so it's more than a fivebagger in less than a year. But still, quite a few shareholders, from both companies, didn't feel great about the merger.

I think Teladoc could still be a multibagger at this point for patient investors. I think most investors underestimate that this combined company could represent the future of our healthcare system. In this article, written from a bird's eye perspective, I will try to explain why.

I think a lot of people know that the American healthcare system (and that of most Western countries) is unsustainable. It's too expensive but nobody seems to find a way out to cut costs.

The problem is that our healthcare is one of the last sectors that has not been disrupted by tech yet. The system originates from a time when bigger was better because it was more affordable to have standard procedures. Long ago, there was a family doctor and he knew you and you knew him and you had a personal relationship with that man (female doctors almost didn't exist back then).

After the Second World War, two evolutions emerged that made this system unsustainable: people reached a higher and higher age and the Baby Boomers were born. That put pressure on both ends of the healthcare system and the solutions were more scale and introducing standard procedures, so the productivity of healthcare workers became higher. Specialized care also contributed to more efficiency. And it worked.

But there was a side effect. People don't feel connected to the people that should care for them. They often feel treated like numbers, like patients at best, but mostly not like individual people. I'm not throwing a stone here at doctors, nurses and other healthcare workers. They often share that feeling. They don't have the time to deeply care about people. Their time is limited, they have to reach the quota. A doctor is not paid for listening to you. He's paid per patient that he handles. In other words, the less he or she listens, the more the doctor is incentivized. And that wears out a lot of healthcare workers.

Normally, such a market would correct itself. If you are not properly served in Lowe's (LOW), you go to Home Depot (HD) or the other way around. If one is really not giving enough attention to its clients, the company will go bankrupt eventually. That's where the efficiency of the market plays its role.

But in healthcare, the patients are not the customers. They are the goods, as it were. Customers are the paying party. And who pays for healthcare costs? Exactly, the insurance companies. Their only objective is as little costs as possible and that's why they pay per visit, for example.

But this creates the strange effect that a doctor that treats you very well is only paid once, while a bad doctor, who follows the wrong procedure and has to repair the damage or gives the wrong diagnosis, is rewarded each time he or she treats you and so earns more money than the good doctor.

To fix the healthcare system, a little reparation here and there won't help. We saw in the last decades that tech has entered almost every industry and has disrupted industries completely. Think of how Amazon (AMZN) made Sears obsolete. The same thing should (and probably will) happen in healthcare.

Disruption comes from the Latin verb 'disrumpere', which means to break apart and that is what healthcare needs: breaking things apart to build them up again. The bottom line of every healthcare reform should be to use tech and turn the system upside down. Health should be rewarded and paid for, not sickness.

All insurance is meant for emergency cases, except for healthcare insurance. Doctors are incentivized to do as many consults and tests as possible because someone makes money on that: the doctor himself or the hospital, mostly both. A hospital, for example, makes 10 or 20 times more money if you go to the emergency room than if you use an online platform to talk to a telehealth doctor.

As a patient, if you see a doctor, most of that little time you spend with him or her is dedicated to tests, collecting data. But suppose the doctor already would have all the data when you come in and he or she has already been able to look into your case, your history, and tens of up-to-date data points before you came in, that doctor could have time for that which we all crave when we visit a doctor: talking about what you exactly have and what it means, what we can do to get better, a discussion about what the underlying cause could be, talking about the psychological effects that your condition brings with it, what the best plan of action would be for you, etc. In other words, the doctor could become some kind of health coach, a professional that, with the help of precise data, could prescribe a trajectory to better health, hold you accountable, help you when sticking to the plan is tough etc. A doctor could partly become a real healthcare worker, not just a sickcare worker.

That would mean that you wouldn't have to visit a doctor as often. If you have a chronic condition, you could be monitored 24/7 by sensors, assisted by AI, as Livongo does for diabetes. You are not only monitored but you also get health nudges. That means that you would know exactly what is the right path for you. And that path is much more individualized than most people can imagine.

Hemant Taneja, a venture capitalist of General Catalyst who founded Livongo with Glen Tullman, calls this new space in healthcare 'health assurance'.

(Hemant Taneja, right, together with Livongo founder Glen Tullman, source)

Taneja is convinced that health assurance industry will see several $100B+ companies. I suspect that he was the great driving force behind the Livongo/Teladoc merger, together with Glen Tullman. They realized that Livongo can become so much more combined with Teladoc than on its own.

This is how health assurance is defined:

Health assurance is an emerging category of consumer-centric, data-driven healthcare services that are designed to bend the cost curve of care and help us stay well. Built on the principles of open technology standards, these services employ empathetic user design and responsible AI. This is the future of your health experience.

If you want to know more about this, you could read Taneja's interesting book Unhealthcare.

(Source)

Now that you know the basics of the concept of health assurance, I think you can see the potential for Teladoc and Livongo in this market. There is no company that is as well-positioned as the combination of Teladoc and Livongo. Livongo brings measuring, data collection and especially AI to the table, Teladoc has a worldwide network of telehealth.

Investors who just see what there is now could get scared away from Teladoc. They look at the number of telehealth visits that could slow down after the peak of the coronavirus and they are afraid that Teladoc will just have been a COVID-19-induced fad. But if you look at the future, you see that this company could be in the sweet spot when healthcare will be disrupted over the next decade.

Insurance companies will help them with this. As we have already seen, they just want to pay as little as possible. Livongo saved insurance companies $88 per patient per month. The reason: the monitoring of diabetes patients, the fact that they have a diabetes coach that is always available and the health nudges reduce the medical costs dramatically. $88 per patient per month means more than $1,000 per year per patient. I think you see the potential.

Besides that, the patients also feel freer than before. Their diabetes doesn't control their lives as much. They need less medication and if they need it, the data will indicate it before the attack.

This is just for diabetes. That's already a big market. But Livongo doesn't just focus on diabetes but also on hypertension and obesity. Those are two huge markets as well. The hypertension market is estimated to be $23B in 2026 and the obesity treatment market is estimated to be around $20B in 2026. But suppose you add the weight loss market to this, which is worth about $70B in the US alone, and you can see the potential.

The obesity-related healthcare costs are estimated at around $147B annually, so this might mean big savings for healthcare spending.

There will certainly be other companies in this space than just Livongo and Teladoc, companies that will also focus on other domains, but so far, I don't see any competitor that is as advanced as Livongo/Teladoc is.

When I pick a Potential Multibagger, I turn a company inside out and that means that I know Livongo, its founders Glen Tullman and Hemant Taneja and what the company exactly does really well.

I didn't know Teladoc as well before the merger was announced. I had it on my watchlist, but I had not done a really deep dive. When I did, I found a lot that I liked. This statement by Teladoc's CEO Jason Gorevic on the closing of the merger is worth reading word for word if you want to understand the combination of the companies:

Both Teladoc Health and Livongo were founded with the same mission: to create a new kind of healthcare experience, one that empowers people everywhere to live their healthiest life. Today's news (the merger, FGTV) dramatically accelerates our ability to make this a reality for the tens of millions of consumers and healthcare professionals we serve around the world. Together, our team will achieve the full promise of whole-person virtual care, leveraging our combined applied analytics, expert guidance and connected technology to deliver, enable and empower better health outcomes."

There are a few critical phrases here. Let's look at them one by one.

"a new kind of healthcare experience" This is the disruption that I mentioned. Not just fine-tuning the current system, but a completely fresh start.

"one that empowers people everywhere to live their healthiest life".

This is the health assurance that I was talking about. Helping people to live their healthiest life is the reverse of what the current healthcare system thrives on. Empowerment means that people will be able to decide for themselves and take their health into their own hands. The 'everywhere' in this phrase refers to the global footprint Teladoc has.

"whole-person virtual care"

This refers to everything from health assurance to data, treatment plans, health nudges, and specialized diagnosis and surgery. Teladoc/Livongo will be the only one-stop-shop for taking your healthcare into your own hands. Healthcare is one of the few paternalistic sectors left. Paternalistic here means: "We know everything, you don't. Your only function is to give us the money and shut up."

Again, that's not throwing a stone to healthcare workers. I have a really, really deep respect for the people working in healthcare and this pandemic has highlighted even more how crucial they are. Several of my friends work in the sector.

They actually often feel the same frustration as patients. They don't have enough time to really listen, which would help them to diagnose more accurately, they have to do too much administration, they lose precious time gathering simple data and there are no efficient follow-up programs. Most healthcare workers would love to have the time to establish a human connection with each of their patients and listen to every detail that could count. But there is just no time. That's why a lot of people seek help from all kinds of coaches.

This is Teladoc's representation of whole-person care:

(Source)

Let's look into this in a bit more detail.

As you see from the graph, and I think this is really important, the category Wellness and Prevention is included in that care too. It is explained as 'Complete regular screenings and improve nutrition, exercise and well-being'. That means planning your health.

No company flies blindly and every company has a clearly-defined goal for the future often with step-by-step roadmaps. But for health, the advice is often: "Eat well and exercise." That's like saying to a company: "Execute well and make money." In other words, although it's true, it's too general.

A personal note here to illustrate what I mean. For years, I was very tired and I had trouble staying awake after meals or after drinking coffee. The advice I got from my doctors? Eat healthily, do regular exercise.

I found out 5 years ago that I have a milk allergy but I had to find out all about what it meant for my life (dairy-free cooking, avoiding almost all cookies, but also potato chips etc.) on my own. It would have been great to have a specialist that could have coached me there.

The example of my own life is just to illustrate the truth about healthcare that we all intuitively know but that is not acknowledged enough under this system: we are all individuals, with our differences, our unique needs. In stock terms, we are as different as a steel producer stock and a SaaS stock. I'm a man of 6 feet and 5 inches and almost always, I get the same dose of medicine as a woman of barely 5 feet high.

We are on the brink of other breakthroughs in healthcare: stem cell therapy, gene therapy, cheap genome sequencing, CRISPR, and many more. All these trends point in one direction: individualized healthcare. Medicine will not be a mass-produced, mass-prescribed drug anymore. We will evolve to personalized medicine.

Initially, people will be split into different groups based on certain data points (age, weight, condition...) and later it will be really about you, the individual. Your genome, your microbiome, your allergies, your reactions to certain drugs, everything will be known and taken into account for your prescription. Lots of medicines contain milk, for example, as a filling agent. Each time again, I have to say this to a doctor and sometimes there are even no medicines on the market without milk. These will be produced in the individualized healthcare that will come, if only for me. Medicines will be prepared on who you are, not on who the masses are.

But it will be much more than just medicines. Which supplements should you take? What is the perfect exercise regimen for your type of body? How could you build up your condition for that marathon or triathlon you always dreamed of without the risk of an injury because of your specific body composition? What are the best shoes for your feet so they can be 3D-printed? What is the best diet for the specific microbes that you have in your gut? What are the diseases you are genetically susceptible to and what can you change in your lifestyle to prevent them?

Don't get me wrong, I'm not a pie-in-the sky thinker. This will not be for the first years, of course not. And at first, it could be unevenly distributed, as a lot has been throughout healthcare's history. The first individualized programs could come with a hefty price ticket. But probably that will democratize, as a lot of procedures have in healthcare. Or maybe it will democratize from the start but with different degrees of quality, a bit like smartwatches.

The medical know-how of Teladoc, its wide network of doctors and health specialists, combined with Livongo's AI, data gathering and processing capabilities, make that this company is, like no other that I know, prepared for the future of healthcare.

I haven't seen any other AI healthcare platform that even comes close to that of Livongo at this point. With its AI+AI approach (aggregate, interpret + apply, iterate) it has already learned a lot, both from the whole pool of patients as from individual patients. With more patients because of the merger, there will be more data and more data means more insights and new products over time.

And Livongo will double down on its AI and data analysis. Revealera.com is a website that looks at jobs, job openings and it tries to find relevant information from these data.

It showed that of all publicly-traded companies, Livongo had the highest percentage of job openings that require data science and machine learning. 16% of Livongo's jobs openings ask for experience in those fields.

This clearly shows to me that Teladoc/Livongo is skating where the puck will be, not just where it is.

If you look at the combination of the two companies that merged, you can see that they are very complementary. These are Teladoc's key growth strategies and in blue, Livongo could help to accelerate Teladoc's strategy:

(Source)

The companies estimate that there will be $500M in synergies.

(Source)

Now, I know that it's all too common in an acquisition or a merger to overestimate these synergies by a wide margin. But in this case, I think the synergy opportunities are actually very conservative. The companies even acknowledge that in their merger presentation:

There will be a lot of cross-selling, as there is only a 25% overlap in the customers of the companies. Even before the merger officially was closed, Livongo was cross-sold in two deals already by Teladoc.

The first deal was Fresenius Medical Care, a company specialized in working with patients that suffer from CKD (chronic kidney disease). Partnerships and distribution are quintessential in healthcare and in its field, Fresenius is a big player. It provides dialysis for 347,000 kidney patients. The press release of Fresenius explains:

This marks the first time Livongo will use its robust virtual care solutions to specifically support those with CKD and is a significant step forward in Fresenius Medical Care's efforts to provide a more coordinated care experience. With earlier intervention, Fresenius Health Partners also seeks to increase optimal dialysis starts, as well as offer earlier evaluation of transplantation and home dialysis options.

This shows that the possibilities for Livongo to branch out are numerous.

The second deal was with Florida Blue, part of GuideWell Mutual Holding. Together with its merging partner Teladoc, Livongo will offer Florida Blue members with diabetes virtual care, including connected devices, advanced data science, and telehealth.

Being the only one-stop-shop for digital healthcare will provide Teladoc/Livongo with a competitive advantage that others simply don't have at the moment. The companies shared an example about Claire, an imaginary future client. You can see the different stages and situations in which she can be helped by Teladoc after the merger with Livongo:

As you can see, there is not a single moment in the whole process that Claire has to leave the platform. In this way, Teladoc and Livongo show that they are very complimentary. And the data component of Livongo, combined with preventive healthcare, gives Teladoc a lot of flexibility to introduce even more products, each one more and more targeted and eventually personalized.

There are risks to every investment, of course, although I generally believe that too many investors overemphasize risk. What is risk? Risk is not the same as volatility, no matter what some want you to believe. Volatility is risky if you are a short-term investor. If you need the money in 2 years, volatility is a risk. But if you need the money in, let's say, 20 years, why would it matter if a certain stock is up or down 50% this year or the next?

If you read the great book 100 baggers by Chris Mayer, you'll see that ALL (!) of the 100 baggers (stocks that turn your $10K into $1M) saw drops of 50% and more at least once. Most several times, and often they dropped substantially more than 50%.

Risk is the chance that you will lose your money permanently, not volatility. That also means that you should look at risks in their context. All companies make mistakes and if you sell because of a mistake, you'll never find multibaggers. Do I need to remind you of the Amazon Fire phone, the Netflix Quikster failure, the Windows phone, Google Plus and so many more mistakes? Don't let one failed product mislead you. The company as a whole is much more important.

Having said that, what will I keep my eyes on for Teladoc?

First, I want to see that Livongo really has an impact on Teladoc because health assurance is more a part of what Livongo does right now. There is a risk that Teladoc doesn't leverage Livongo's capacities enough.

The second element that I will watch is how the two companies work together when it comes to company culture. Teladoc has a good tracking record when it comes to acquisitions and giving them a place where they feel good inside of the company but this merger with Livongo is on a whole different level. I see some good signs because Teladoc CEO Jason Gorevic and Livongo's founder and executive chairman have already come out together several times and the two seem to share the same vision.

The third and final element of risk that I want to touch on is competition. At this moment, I don't see any competitor that is even close to Teladoc after the merger with Livongo but that can always change fast, of course. On the other hand, this market is so big that there will be several winners. And the size of the market, that's the next topic of this article.

The market cap of the combined company is around $30B at this moment. For Potential Multibagger stocks, I want to see the possibility that the stock could be a tenbagger in the next 10 years. For Teladoc, I think this is still possible, despite its already substantial market cap. The company has everything it needs to start a new era in healthcare as I showed, and it's in a gigantic market. This is the title of recent research:

Global Digital Health Market was Valued at USD 111.4 billion in 2019 and is Expected to Reach USD 510.4 billion by 2025, Observing a CAGR of 29.0% during 2020-2025

Teladoc operates in a TAM (total addressable market) of $510B in 2025 and at this moment it is the only 360 digital health company. That's a great position to be in. For those who wouldn't know: TAM is the yearly total addressable market. The fact that Teladoc projects a CAGR (compound annual growth rate) of 30% to 40% seems conservative to me. If you would add the synergies, it will be at least to the higher end of that margin, in my opinion.

If Teladoc could just bring in 3% of that TAM of 2025, that would already mean $15.3B. If you slap a P/S ratio of 20 on that, you already have a company with a market cap of more than $300B. A P/S of 20 might seem aggressive but for a company growing at more than 30% per year and gross margins which will probably be in the mid-70s, I think it's very reasonable. You can tinker with the numbers but the conclusion to me is always that if Teladoc executes well, it has the potential to become a giant.

I'm not saying that the company will already have 3% of the global digital health market by 2025, mind you. I think revenue of 1% of the TAM, about $5.1B, is possible at that moment, though, and much more growth will be in the pipeline.

There are always a lot of ifs but when I look at Teladoc, I can see the potential to become really big, ten times or more bigger than today.

With a lot of healthcare disruption knocking at the door, such as cheap genome sequencing, CRISPR, personalized medicines and much more, data will become more and more important for healthcare. Livongo's AI will add that to Teladoc.

The combination of Teladoc and Livongo definitely has the first-mover advantage in a very important and big emerging market because it can provide a 360 degrees one-stop-shop for personalized digital healthcare.

If you have enjoyed this article, feel free to hit the "Follow" button next to my name.

In the meantime, keep growing!

Potential Multibaggers focuses on finding multibaggers early on.

Potential Multibaggers is not for those who trade in and out of stocks but for long-term investors who want life-changing returns.

More here:
Teladoc Is A Strong Buy: A Radical Healthcare Change Will Come - Seeking Alpha

Read More...

How genetics can help predict risks of cancer recurrence and improve treatment – Euronews

November 11th, 2020 11:53 am

A biobank is a storage facility for biological samples including blood, human tissue and/or DNA. They can then be used at any time for future medical research or pioneering methods.

The Managing Director of Estonian Biobank, Andres Metspalu, gives us some insight:

"I started the Estonian Biobank about 20 years ago. Our biobank is pretty large for a small country. We have around 20% of the entire Estonian population over the age of 18 included in our biobank; which equates to more than 200,000 individuals.

"They have all been analysed genetically, which is really remarkable. That is why we can do this genetic medicine not only for cancer, but also for other diseases.

"More than 3,000 people have already received their genetic risk (result) from the biobank.

"This is what keeps me busy every day, doing research and also facilitating the use of this information in healthcare".

"We are mainly talking about (predicting the risks of developing diseases like) cancer, cardiovascular diseases and type-2 diabetes. We also study melanoma, prostate cancer and lung cancer.

"We are also doing pharmacogenomics, drug response (how our bodies respond to drug intake).

"Not all drugs work on everyone as (pharmaceutical) companies believe or expect. Some drugs (can be) pretty harmful. You (can) get reactions and you (can) get side effects. You may end up in hospital after taking prescription drugs.

"Genetics can predict some serious events. It (genetics) should be used. This is what we are trying to introduce into everyday medical practice in Estonia".

Read the original post:
How genetics can help predict risks of cancer recurrence and improve treatment - Euronews

Read More...

Biopharma Money on the Move: November 4-10 – BioSpace

November 11th, 2020 11:53 am

Here's a look at which companies are raking in cash this week in the biopharma industry.

Apollomics, Inc.

Committed tocombatting cancer with precision,Apollomics plans to use the$124.2 million Series Cfinancing to focusclinicalefforts on its lead programs: APL-101 and APL-106. APL-101 is an oral c-MET inhibitorcurrently involved in several ongoing clinical trials. TheSPARTA trialis in Phase II, targeting non-small cell lung cancer,glioblastomamultiforme and solid tumors with MET amplifications.APL-106 is a first-in-class targeted inhibitordesignedtoblockE-selectin, an adhesion molecule on cells in bone marrow,from binding with blood cancer cells.It has received Breakthrough Therapy Designation from the FDA in relapsed and refractory acute myeloid leukemia.In 2019,Apollomicsraised$100 million in a Series B round.

Decibel Therapeutics

Decibel Therapeutics made some noise this week with an oversubscribed Series D financing, raking in$82.2 million. The company will use the funds to advance DM-OTO, a gene therapy to restore hearing in children with congenital deafness due to a deficiency in the otoferlin gene.Clinical testing is expected to initiate in2022.DB-020 is already in a phase Ib study with cancer patients as apreventative treatment for the ototoxicityassociated with cisplatin-based chemotherapy.Hearing and balance disorders have historically been overlooked by the biopharma industry, even though they exact a devastating toll on the lives of hundreds of millions of people around the globe. At Decibel, we are dedicated to restoring hearing and balance with precision therapeutics designed to deliver the right genetic medicine specifically to the right cells in the ear, said Laurence Reid, Ph.D., Chief Executive Officer of Decibel.

Adagio Therapeutics

After launchingin Junewith $50 million, Adagio has pocketed another$80 million in a Series Bfinancing round to take its COVID-19 antibody into the clinic next year. Adagios neutralizing monoclonal antibodies are expected to provide broad protection against not onlyagainst SARS-CoV-2 and SARS-CoV-1, but also additional bat coronaviruses that have yet to cross the species barrier. Adagio got the green light from the FDA to proceed with their first-in-human study in early 2021.We were impressed by the thoughtful approach that Adagio took. By dealing with the broader coronavirus problem, we expect ADG20 to be more resistant to escape mutations and potentially cover future coronavirus pandemics, said Krishna Yeshwant, Managing Partner at GV. As a preventative agent, ADG20 holds the promise of providing the efficacy necessary to deliver greater protection against COVID-19. Given its unique combination of attributes, ADG20 could complement and supplement vaccines by providing rapid, durable antibody protection against current and future coronaviruses.

InmageneBiopharmaceuticals

Drug development companyInmagenehas its eye on being number one in immunology in China.The$21 million Series Bclosed this week will be pumped into conducting global clinical trials, research and development, and product in-licensing activities.Currently candidate IMG-020 is in a Phase II psoriasis trial, with a strong safety profile and clear clinical benefits giving it best-in-class potential. Manufactured inan E. coli system, IMG-020 is less than 1/20thof the average manufacturing cost of a typical antibody drug.The candidate is about to enter global registration trials formultiple indications.

Locus Biosciences

Its been a busy season for this CRISPR-engineer.In September,Locussigneda$144 million contract with BARDAto develop theirproduct targeting E. coli bacteria causing recurrent urinary tract infections. This week they closed a$14.4 million deal with CARB-Xto advance development of LBP-KP01, another CRISPR Cas3-enhanced bacteriophage (crPhage) product, targeting K. pneumoniae.The initial indication will be to target recurrent UTIs, then development for targeting lung infections (pneumonia), intra-abdominal infections (IAIs) and bacteremia.Together, the two cocktails have the potential to treat more than 90% of UTIs.Auniquedual mechanismof bacteria-hunting bacteriophages along with the DNA-targetingCRISPR-Cas3makesLocuscandidates significantly more effective at killing the targeted bacteria cells, regardless of whether they are resistant to antibiotics.Both the U.S. Centers for Disease Control and Prevention (CDC) and World Health Organization (WHO) have identified antibiotic-resistant K. pneumoniaeas an urgent and serious public health threat requiring development of new treatments.

Memo Therapeutics

Swiss innovator Memo Therapeutics has raised over$15.3 million in a Series Bprimarilyto advance its COVID-19 antibody treatment.The company entered into a partnership with NorthwayBiotechpharmain August to manufactureMTX-COVAB, which is currently going through a fast-tracked development path as an immunotherapy and a preventative of the novel coronavirus. Memo plans to begin clinical studies in 2021. Proceeds will also be used to advance its neutralizing antibody MTX-005 against BK virus infection in renal transplant patients into Phase II studies."We believe Memo Therapeutics AG has taken innovation in the field of antibody discovery to the next level. Their ability to exploit the power of microfluidic single-cell molecular cloning could not only serve to move one step closer to conquer the COVID pandemic but also potentially other infectious diseases and cancer, said Dr. Robert Schier, Investment Director atSwisscantoInvest.

Trailhead Biosystems

Pushing the boundaries of dimensional testing, Trailhead is increasing the speed, lowering the cost and reducing the risk of developing cell therapies. A$6.6 millioninfusion of cash will expand the companys High Dimensional Design of Experiments platform (HD-DoE) to support the generation of multiple specialized human cells with therapeutic properties and theirpilotscalemanufacturing.Trailheads aim is to rapidly develop the capability to create highly pure, specialized human cell types for regenerative medicine and therapeutic purposes at an industrial scale. "Biology is complex, but conventional science is not," saysJan Jensen, Ph.D., Chief Executive Officer and founder of Trailhead Biosystems. "We created Trailhead Biosystems to address key limitations in the scientific process, unlocking a deeper understanding of biology that will enable us to better control it."

More:
Biopharma Money on the Move: November 4-10 - BioSpace

Read More...

Yale scientists discover genes that could be COVID treatment targets – Yale Daily News

November 11th, 2020 11:52 am

Eric Wang, Senior Photographer

Researchers at the Yale School of Medicine have identified genes that could be future targets for COVID-19 treatments.

In a partnership with the Broad Institute of MIT and Harvard, researchers at the School of Medicines Department of Immunology performed a genome-wide CRISPR screen, which evaluated each of the 20,000 preselected genes in the African green monkey genome that could affect coronavirus infections. This technique allowed researchers to quickly and effectively evaluate the genetic information from over a million modified cells.

According to assistant professor of laboratory medicine and immunobiology Craig Wilen, using a genetically modified virus called a CRISPR library, certain genes of interest were knocked out in the monkey cells in order to stop their products from being made and used in the cell. The cells were then infected with the coronavirus, and those that survived were analyzed to detect what genes were knocked out and could be affecting viral infection. The results pointed to over 25 possible host genes related to infection, but two specific hits for receptor and enzyme encoding genes seemed most promising as treatment targets.

We think its possible that you could develop drugs that affect human targets, Wilen said. And the advantage there is it would be conserved and function across different coronaviruses.

Jin Wei, the studys primary author and a postdoctoral associate at Wilens lab, explained that he was directly involved in identifying the host genes critical to coronavirus infections.

According to Wei, the lab had prior experience in studying the modes of infections of RNA viruses such as MERS and other coronaviruses. This previous work meant they were uniquely prepared to study the genes that affect the SARS-CoV-2 virus infection which had never been done before.

We found there is no CRISPR screens for host genes for any coronaviruses, which may reveal novel therapeutic targets and inform our understanding of COVID-19 pathogenesis, Wei wrote in an email to the News. We leveraged our expertise with RNA virus pathogenesis and CRISPR screening to identify the host factors that are essential for SARS-CoV-2 infection.

Mia Madel Alfajaro, another postdoctoral associate at Wilens lab, explained that they found two important genes during their screening process that, when absent, helped cells survive the virus infection. One of them encodes the SARS-Cov-2 receptor, while the other is translated into an enzyme that aids the coronavirus in entering the cell.

Scientists at the Broad Institute provided the Yale researchers with the CRISPR library to be used in the monkey cells and the analyses they ran on the surviving cells genetic material.

Our group has significant expertise and capacity in terms of making CRISPR libraries, turning them from an idea into an actual test tube of particles, John Doench, an institute scientist at the Broad Institute, said.

Wei and Doench believe one of the main findings of the study comes from the comparison between SARS-CoV-2 and another coronavirus, MERS-CoV. These genetic hits that affect coronaviruses in general could be useful in finding pan-coronavirus treatments, according to Wilen.

According to Alfajaro, one of the limitations of this study is that there is no way to mimic exactly the behavior of a human beings lung cells, which means there are still many steps to be taken before a treatment is developed.

If we have [found] molecules, peptides or chemical inhibitors or COVID-19, that would be great, Alfajaro said. It will take time because some of the hits need to be developed.

Alfajaro believes drugs that are already approved by the FDA could be a possible focus for future research, since some of the drugs already on the market could affect the molecules found during the screening.

Doench does not believe that the main goal of the study was finding a drug that would end the pandemic. He argued that a future drug may be able to target the genes they found to create therapeutics for COVID-19, but that more work needs to be done.

From doing a genetic screen in a cell line in a monkey to having a drug target, there is so much science that needs to happen, he said.

According to Doench, the only way to stop the pandemic is through social distancing, wearing masks and eventually developing a vaccine.

The Broad Institute of MIT and Harvard was founded in 2004.

Beatriz Horta | beatriz.horta@yale.edu

Read the original:
Yale scientists discover genes that could be COVID treatment targets - Yale Daily News

Read More...

Generation Bio Reports Third Quarter 2020 Business Updates and Financial Results – GlobeNewswire

November 11th, 2020 11:52 am

CAMBRIDGE, Mass., Nov. 10, 2020 (GLOBE NEWSWIRE) -- Generation Bio Co. (Nasdaq: GBIO) is an innovative genetic medicines company creating a new class of non-viral gene therapy. Today the company reported recent business highlights and third quarter financial results.

2020 continues to be a year of progress and execution for Generation Bio as we advance our non-viral gene therapy approach, said Geoff McDonough, M.D., president and chief executive officer of Generation Bio.Despite the challenges of the COVID-19 pandemic, we remain on-track to advance our lead programs into IND-enabling preclinical development next year. We believe our strong cash balance positions us well to execute on our ambitions into 2023.

Recent Business Highlights

This period marks an expansion of our focus beyond our platform to include preclinical development and readiness for the clinic. To support this effort, I am pleased to announce the appointment of Tracy Zimmermann to chief development officer. Tracy will lead our pre-clinical development programs across the portfolio, building on the excellent foundation she has created since joining Generation Bio in 2018. Tracys new role allows for Doug Kerr to focus on building our clinical development capabilities as chief medical officer. Together with Matt Stanton, our chief scientific officer, Tracy and Doug make a terrific, complementary leadership team for our R&D work, Dr. McDonough said. A summary of the leadership appointments follows.

Dr. McDonough continued, Separately, Mark Angelino, our chief operating officer and co-founder, will undertake a planned transition from Generation Bio to return to early stage company formation work in early 2021. Although too soon for farewells, we are indebted to Mark for his vision and leadership in forming and building our community here.

Selected Anticipated Company Milestones

Upcoming Investor Conference Presentations

Management will present at two upcoming investor conferences:

Live webcasts of the presentation and the fireside chat will be available in the investor section of the company's website atwww.generationbio.com. The webcasts will be archived for 60 days following the presentations.

Financial Results

About Generation Bio

Generation Bio is an innovative genetic medicines company focused on creating a new class of non-viral gene therapy to provide durable, redosable treatments for people living with rare and prevalent diseases. The companys non-viral platform incorporates a proprietary, high-capacity DNA construct called closed-ended DNA, or ceDNA; a cell-targeted lipid nanoparticle delivery system, or ctLNP; and an established, scalable capsid-free manufacturing process. The platform is designed to enable multi-year durability from a single dose of ceDNA and to allow titration and redosing if needed. The ctLNP is designed to deliver large genetic payloads, including multiple genes, to specific tissues to address a wide range of indications. The companys efficient, scalable manufacturing process supports Generation Bios mission to extend the reach of gene therapy to more people, living with more diseases, in more places around the world.

For more information, please visit http://www.generationbio.com.

Forward-Looking Statements

Any statements in this press release about future expectations, plans and prospects for the Company, including statements about its strategic plans or objectives, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: uncertainties inherent in the identification and development of product candidates, including the conduct of research activities, the initiation and completion of preclinical studies and clinical trials and clinical development of the Companys product candidates; uncertainties as to the availability and timing of results from preclinical studies and clinical trials; whether results from preclinical studies will be predictive of the results of later preclinical studies and clinical trials; expectations for regulatory approvals to conduct trials or to market products; challenges in the manufacture of genetic medicine products; the Companys ability to obtain sufficient cash resources to fund the Companys foreseeable and unforeseeable operating expenses and capital expenditure requirements; the impact of the COVID-19 pandemic on the Companys business and operations; as well as the other risks and uncertainties set forth in the Risk Factors section of the Companys most recent quarterly report on Form 10-Q, and in subsequent filings the Company may make with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Companys views as of the date hereof. The Company anticipates that subsequent events and developments will cause the Companys views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing the Companys views as of any date subsequent to the date on which they were made.

Contacts:

InvestorsChelcie ListerTHRUST Strategic Communicationschelcie@thrustsc.com910-777-3049

MediaStephanie SimonTenBridge Communicationsstephanie@tenbridgecommunications.com617-581-9333

GENERATION BIO CO.CONSOLIDATED BALANCE SHEET DATA(unaudited)(in thousands)

GENERATION BIO CO.CONSOLIDATED STATEMENTS OF OPERATIONS(unaudited)(in thousands, except share and per share data)

Continue reading here:
Generation Bio Reports Third Quarter 2020 Business Updates and Financial Results - GlobeNewswire

Read More...

Exploring Genetic Variation and COVID-19 Clinical Variability – Technology Networks

November 11th, 2020 11:52 am

One of the biggest challenges that scientists and healthcare professionals are facing during the COVID-19 pandemic is the high rate of clinical variability. Whilst some patients present as asymptomatic, others are developing more severe symptoms such as pneumonia, and some cases are ultimately proving fatal. Why?The answer remains elusive; however, extensive research is exploring the possible contribution our genetics may be having. Genetic variation differences in the DNA sequences that make up our genome can impact our response to infectious diseases.

GoodCell uniquely measures and monitors inherited and acquired genetic variations in stem cells and other nucleated cells in our blood over time. Technology Networks recently spoke with Dr Salvatore Viscomi, chief medical officer at GoodCell, and attending physical at Baystate Health, to explore factors that might influence COVID-19 risk, and to discuss how the company is working to identify at-risk individuals through genetic variation analysis.

Molly Campbell (MC): For our readers that may be unfamiliar, can you discuss why medicine is moving towards a personalized approach, and why this is important considering genetic variation?Salvatore Viscomi (SV): Healthcare has traditionally taken the approach of one size fits all in defining individual risk for a disease and prescribing therapy for it. Understanding the differences between individuals on a molecular level optimizes assessment of an individuals susceptibility to a certain disease and predicting response to pharmacological therapy. Genomics plays the most important role in the emergence of personalized therapy. Identifying the inherited and acquired genetic variation will direct personalized screening and prevention plans and inform bespoke medical therapies.

MC: We know that there is high clinical variability across COVID-19 patients. How might genetic variation be contributing here, and what published evidence exists to support this?SV: Understanding immune response is critical to identifying individuals at high risk of severe morbidity and mortality. Emerging research suggests that accumulated genetic variation in our blood cells may be associated with a dysfunctional inflammatory response to COVID-19 leading to its pulmonary, cardiac and coagulopathic complications.

In a recent study published by JAMA Cardiology, researchers demonstrated an association between the presence of accumulated genetic change in our blood cells and a pro-inflammatory immune response that resembles the exaggerated cytokine release syndrome (CRS) manifested in COVID-19-positive patients. Direct evidence has emerged more recently; a study published in Cancers examined patients hospitalized with COVID-19 and found a significantly higher prevalence of accumulated genetic variation in all age groups compared to age-matched control groups.

MC: What impact might genetic variation in COVID-19 patients have on efforts to develop therapeutics or preventives, such as vaccines?SV: Identifying highly susceptible individuals through blood testing could have many applications. As an initial wave of vaccines move through Phase III trials and potentially come to market, we would have the data to determine prioritization of vaccinations when one is available. Business and government sectors need insight into risk factors that can inform inoculation strategies for societys most vulnerable, inform decisions around who should and should not be on the front lines, and give people more control when making personal decisions about how to mitigate individual risk. The broader field of genetics offers a window into the potential to correlate inherited and acquired gene mutations with immune response for the betterment of society, providing a more robust and accurate set of risk factors unique to every individual.

Furthermore, in high-risk individuals, targeting inflammation may be a clinical strategy to mitigate its clinical consequencesin COVID-19. For example, we may identify patients who are most responsive to pro-inflammatory inhibitors. Implementing measures intended to reduce subjects exposure to the infection or likelihood of contracting such infection through self-isolation, quarantine or social distancing may be advised.

MC: Can you explain the aims of GoodCell, and what the company does in terms of "banking blood for life"?SV: GoodCells mission is to extend and improve the quality of life through technology powered by our own cells. Blood is the author of our bodies, and can both cure as well as cause disease. Through our proprietary data aggregation and analytics technology platform, which aims to decode our blood cells and harness their insights to advance population and personal health, we empower individuals to identify, track and mitigate health risks. By getting ahead of their health risks, we enable the potential for a better life. In addition, through our personal biobanking service, long-term storage of your healthiest cells provides the opportunity for potential use in future therapeutics if you need them you are your best donor.

MC: Does GoodCell measure other "omics" parameters outside of genomics (DNA measurements and analysis), such as proteomics or metabolomics?SV: GoodCells platform leverages the power of blood to assess risk as such, we of course look at acquired and inherited genetic changes, but there are many more opportunities afforded by blood to understand and assess risk including routine blood chemistry tests, tests for biomarkers of disease, including emerging capabilities in liquid biopsy for earlier detection of solid tumor cancers. Ultimately, we are always looking to incorporate novel health and data insights into our product platform to better inform both an individuals health, as well as population-based health. Transcriptomics, epigenomics and metabolomics are but a few of the opportunities we are evaluating.

MC: What work is GoodCell currently conducting in the COVID-19 space?SV: GoodCell is currently engaged in a research collaboration with the New York Blood Center to evaluate how specific acquired and inherited genetic variation contribute to COVID-19 severity and recovery. We are analyzing genetic variation in asymptomatic/mildly symptomatic patients compared to hospitalized/ICU patients. GoodCell will evaluate the genetic variation in the collected samples using our proprietary assay platform to identify and validate their association with COVID-19 morbidity and mortality.

Salvatore Viscomi was speaking to Molly Campbell, Science Writer, Technology Networks.

Visit link:
Exploring Genetic Variation and COVID-19 Clinical Variability - Technology Networks

Read More...

Flaws emerge in modeling human genetic diseases in animals – The Conversation US

November 11th, 2020 11:52 am

My lab, based at the University of Southern California Keck School of Medicine, uses zebrafish to model human birth defects affecting the face. When I tell people this, they are often skeptical that fish biology has any relevance to human health.

But zebrafish have backbones like us, contain by and large the same types of organs, and, critically for genetic research, share many genes in common. My group has exploited these genetic similarities to create zebrafish models for several human birth defects, including Saethre-Chotzen Syndrome, in which the bones of the skull abnormally fuse together, and early-onset arthritis.

Similar to fish, our bodies develop under the control of about 25,000 genes. The trick is finding out what each gene does. Stunning advances such as CRISPR-based molecular scissors, for which the Nobel Prize in chemistry was just awarded, allow us to precisely change genes, and designer chemicals can silence particular genes. In a recent study from our group published in Nature, however, we find that these tools are still far from perfect. Although CRISPR now allows us to efficiently generate lab animals that can pass human disease mutations onto the next generation, claims that simply injecting CRISPR into embryos or silencing genes with designer chemicals can accurately model human genetic disease are being questioned.

Finding the precise mutation that causes a particular birth defect or a late-onset disease can be tedious work. The human genome is made up of 3 billion building blocks called DNA nucleotides, and changing just one of these can cause devastating birth defects.

To figure out if we have identified the right disease-causing mutation in humans, we typically engineer the same change into the genome of a lab animal. We then breed these animals to generate babies with the disease mutation and look for the appearance of defects similar to those in human patients.

We study zebrafish because they are small, which means we can grow thousands of different genetically modified animals. We routinely use CRISPR to engineer fish that pass on a gene-breaking mutation to the next generation.

We then study the appearance of defects similar to those in humans lacking these genes in essence creating personalized zebrafish avatars of genetic disease. As zebrafish embryos are transparent and develop rapidly outside the mother, they are particularly useful for understanding how human disease mutations disrupt normal development.

Even in zebrafish, engineering animals to lack particular genes can be a time-consuming process. In my lab, we first create gene mutations in embryos, grow these fish to adulthood and then breed fish together to look at defects in the next generation.

This whole process can take a year or longer. Unsurprisingly, many labs are attempting shortcuts. Some are injecting large quantities of CRISPR molecular scissors into animals and then looking for defects in these same animals. Others are using chemicals to turn off, or silence, genes in the embryo rather than permanently changing the genes.

More and more frequently studies like this are calling into question the accuracy of these shortcuts. In animals that have been injected with CRISPR molecular scissors, not every cell is changed in the same way. And the chemicals used to silence genes appear to have unintended consequences, poisoning the embryo in a generic way.

For example, researchers in Spain recently reported that a gene called prrx1a was critical for the proper development of the heart. To figure this out, they silenced prrx1a in zebrafish with chemicals. Then, in a second experiment, they injected CRISPR molecular scissors into zebrafish embryos and examined them just one day later for heart defects.

In contrast, we completely removed the prrx1a gene and looked at generations of fish lacking this gene. Hearts in these mutant fish developed perfectly normally, showing that prrx1a was not critical for heart development. Instead, we showed that the heart defects seen upon chemical treatment in the Spanish study were due to a general poisoning of the embryos unrelated to the prrx1a gene. Animals simply injected with CRISPR also showed defects not seen upon complete removal of the prrx1a gene, although the exact reasons for these differences remain a source of active debate.

And not just our group has noticed these flaws. Using similar gene removal as we reported, the group led by Didier Stainier refuted a study that had used CRISPR injection and gene silencing to link the tek gene to blood vessel development. Given the number of studies relying on gene silencing in lab animals, as opposed to engineering the DNA mutations, the causative genes for many human diseases may need to be reevaluated.

The desire for speed in research must not come at a cost of accuracy and reproducibility.

The good news is that, with the ease of CRISPR, we now know how to engineer the right types of mutations in lab animals to validate human disease mutations. By creating lab animals such as zebrafish that have the mutations engineered into their genomes and then observing whether their offspring develop the same diseases as patients with the mutations, we can be confident in having identified the right human disease gene.

[Deep knowledge, daily. Sign up for The Conversations newsletter.]

Getting it right is important for accurately counseling prospective parents of their genetic risks for certain birth defects, as well as identifying the relevant genes that can be targeted to prevent or even reverse disease.

Science is constantly evolving. While the ability to engineer the genome with CRISPR is opening up endless possibilities for human genetics, researchers must also recognize the limitations of new technologies. Although rapid, directly injecting CRISPR or silencing genes with chemicals gives misleading results too often. In order to confidently identify causative mutations linked to human disease, we will need to continue to study lab animals engineered to carry and pass on the same DNA changes as found in human patients.

See the original post here:
Flaws emerge in modeling human genetic diseases in animals - The Conversation US

Read More...

Page 300«..1020..299300301302..310320..»


2025 © StemCell Therapy is proudly powered by WordPress
Entries (RSS) Comments (RSS) | Violinesth by Patrick