StemCell Therapy

With the focus of personal privacy increasing, it is unsurprising that additional laws are being proposed to increase privacy rights, including the California Privacy Rights Act initiative on the ballot this upcoming November. More immediately, the California legislature passed, and Governor Newsom signed, the Genetic Information Privacy Act ("GIPA"). GIPA specifically targets biometric information, due to the increase of genetic tracing services, like 23andMe and Ancestry.com. This law pertains to adding more protections to genetic privacy. Many questions arise following the passage of GIPA, such as what businesses are affected? What, if any, penalties or causes of action exist under this new law? How does this law work alongside the CCPA?

WHAT IS IN THE LAW?

The law requires notices and actual, express consent from consumers for direct-to-consumer genetic testing companies, and any other company that collects, uses, maintains, or discloses information collected from biometric samples, or from any other element concerning genetic material (i.e. genes). Regarding the express consent provision in particular, this requires that consent is provided for: (1) the use of data through the genetic testing product being provided, for those specific purposes; (2) the storage of the consumer's biometric sample after testing is complete; (3) each use of the genetic data or sample beyond what was originally intended; (4) each transfer or disclosure to a third party other than service providers, including that third party's name; and (5) any marketing based on the genetic data. In essence, unless a consumer explicitly opts in, these companies cannot store, use, or market based on the genetic information.

WHAT ARE THE PENALTIES?

The penalties for not following GIPA are akin to those for the CCPA, with a $1,000 fine, plus court costs for negligent violations, and $10,000 for willful violations. Furthermore, unlike the CCPA, this law does include a private right of action, as it allows a person who has suffered injury in fact, or has lost money or property, as a result of a violation. In essence, this increases the damages for a company which fails to reasonably secure genetic information from data breaches, though plaintiffs may have difficulty showing that money or property was lost due to the exposure of their genetic information, unlike the CCPA, which implements statutory damages just for the breach occurring.

HOW DOES THIS INTERACT WITH THE CCPA?

Regarding the CCPA, both laws will be in effect, and these companies would be obliged to provide additional notices in addition to those required under the CCPA. Furthermore, they both would protect the same information, as the CCPA does protect biometric data, which would largely overlap with the protections of the genetic information under GIPA. GIPA and the CCPA also both require that reasonable security is utilized to protect the consumer's genetic information. However, GIPA also goes further, in requiring that reasonable security is taken to prevent unauthorized destruction as well. Furthermore, it is noteworthy that GIPA relies on the same "reasonable security" language as the CCPA.

Ultimately, GIPA places stricter requirements on genetic testing companies, such that they will have to be more transparent with consumers, and it may serve as a model for future changes to the CCPA. For instance, GIPA will require more click-wrap agreements and additional changes to items like Terms of Use agreements, to ensure that users agree to each use of the genetic data.

WHAT SHOULD A BUSINESS DO?

For businesses subject to GIPA, reviewing, adjusting and implementing additional consent measures to a website should be paramount, as well as reviewing and updating the privacy policy once more in order to make sure that all notices are present. Furthermore, reviewing current security measures and processes is equally important, due to the more stringent requirements on the restrictions against unauthorized destruction of information.

Ultimately, the biggest change to genetic testing companies under GIPA may be an increased reliance on consumer accounts permitting consumers to login, see, and manage their data in order to give the individualized consents, an increased reliance on click-wrap agreements requiring that consumers scroll through, read, and accept actions by the company before the company takes them, or a combination of the two.

For other businesses, GIPA provides an opportunity to implement and utilize stricter privacy guidelines, and implement them as a potential benefit to consumers, as GIPA requirements largely surpass those under the CCPA.

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California Enacts the Genetic Privacy Information Act | Newmeyer Dillion - JD Supra

The Borofka family celebrated their son JTs second birthday by throwing him a drive-by birthday parade Sunday. JT Borofka has already achieved more obstacles than most kids his age. Suffering with Triosephosphate Isomerase Deficiency, or TPI, this rare genetic disease has been challenging to say the least.Hes weaker, but at the moment hes stable, explained JTs mom, Tara Borofka. We still work with physical therapy. Hes got a little bit better head control. JT is extremely strong and doesnt give up. If theres a toy that is a little too far, he will reach for it even if he has to fall over.And just like JT, his doctors in Pittsburgh arent giving up either.The next step is to go through all the compounds they have found that could possibly be a cure, explained Jason Borofka, JTs dad.Michael Palladino, Professor of Pharmacology and Chemical Biology at the University of Pittsburgh School of Medicine said those compounds will need to be tested.We can test them first in a mouse model, explained Palladino. If you can show that not only did it work in JTs cell. We have JTs cells to test these drugs in, but when we put it in an animal model with his same mutations, that that animal model improves as well.The process can take anywhere from 8 months to 3 years, but while the Borofkas wait for the cure, theyre focusing on celebrating another year with their son.If you would like more information about JT and the researching being done in Pittsburgh, you can visit their website.

The Borofka family celebrated their son JTs second birthday by throwing him a drive-by birthday parade Sunday. JT Borofka has already achieved more obstacles than most kids his age. Suffering with Triosephosphate Isomerase Deficiency, or TPI, this rare genetic disease has been challenging to say the least.

Hes weaker, but at the moment hes stable, explained JTs mom, Tara Borofka. We still work with physical therapy. Hes got a little bit better head control. JT is extremely strong and doesnt give up. If theres a toy that is a little too far, he will reach for it even if he has to fall over.

And just like JT, his doctors in Pittsburgh arent giving up either.

The next step is to go through all the compounds they have found that could possibly be a cure, explained Jason Borofka, JTs dad.

Michael Palladino, Professor of Pharmacology and Chemical Biology at the University of Pittsburgh School of Medicine said those compounds will need to be tested.

We can test them first in a mouse model, explained Palladino. If you can show that not only did it work in JTs cell. We have JTs cells to test these drugs in, but when we put it in an animal model with his same mutations, that that animal model improves as well.

The process can take anywhere from 8 months to 3 years, but while the Borofkas wait for the cure, theyre focusing on celebrating another year with their son.

If you would like more information about JT and the researching being done in Pittsburgh, you can visit their website.

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The Borofka family celebrates another year with their son, who is suffering from a rare genetic disease - KSBW Monterey

MedicalResearch.com Interview with:

Dr Mario FalchiHead of Bioinformatics for the School of Life Course SciencesDepartment of Twin Research & Genetic EpidemiologyKings College London

MedicalResearch.com: What is the background for this study?

Response: The relationship between sun exposure and health is a double-edged sword, on one side there is the beneficial effect of vitamin D production and on the other the increased risk of skin cancer, depending on length and frequency of exposure, and on the individual skin type.

Despite public health campaigns, changing sun-seeking behaviour seems to be challenging for some people, even for those with a familial or personal history of skin cancer. Previous investigations have suggested that exposure to UV could be addictive.

MedicalResearch.com: What are the main findings?

Response: We investigated the sun seeking behaviour of 2,500 twins from the TwinsUK cohort, finding that identical twins tend to share a more similar attitude towards sun exposure compared to fraternal twins. Sun seeking seems to be heritable and significantly influenced by genetics. To identify the genes involved, we performed a genome-wide association study of sun seeking behaviour in 260,000 volunteers from the UK Biobank and the US Nurses Health Studies and Health Professionals Follow-up Study, which highlighted five significantly-associated genetic loci. These loci are enriched for genes expressed in the central nervous system, and that have been previously associated with behavioural traits, cognitive function, and addiction. Interestingly, one of these genes has also recently been associated with vitamin D levels

MedicalResearch.com: What should readers take away from your report?

Response: Sun seeking behaviour is influenced by genes involved in neuropsychological traits and addiction. This should be taken into account to improve the efficiency of public health campaigns aimed at reducing sun exposure and incidence of skin cancer.

MedicalResearch.com: What recommendations do you have for future research as a result of this work?

Response: Further investigations aimed at understanding the mechanisms of addiction more generally, and the biological pathways involved will help in identifying potential drug targets to tackle and help controlling a number of risky behaviours that have a negative impact on public health.

Citation:

Sanna Marianna, Li Xin, Visconti Alessia, Freidin Maxim B, Sacco Chiara, Ribero Simone, Hysi Pirro, Bataille Veronique, Han Jiali, Falchi Mario.Looking for Sunshine: Genetic Predisposition to Sun-Seeking in 265,000 Individuals of European Ancestry.Journal of Investigative Dermatology, 2020; DOI:10.1016/j.jid.2020.08.014

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Last Modified: Sep 14, 2020 @ 11:12 am

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Craving the Sun? Twin Study Finds It May Be Genetic - MedicalResearch.com

Sept. 14, 2020 10:45 UTC

BOTHELL, Wash. & KENILWORTH, N.J.--(BUSINESS WIRE)-- Seattle Genetics, Inc. (Nasdaq: SGEN) and Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced two new strategic oncology collaborations.

This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20200914005237/en/

The companies will globally develop and commercialize Seattle Genetics ladiratuzumab vedotin, an investigational antibody-drug conjugate (ADC) targeting LIV-1, which is currently in phase 2 clinical trials for breast cancer and other solid tumors. The collaboration will pursue a broad joint development program evaluating ladiratuzumab vedotin as monotherapy and in combination with Mercks anti-PD-1 therapy KEYTRUDA (pembrolizumab) in triple-negative breast cancer, hormone receptor-positive breast cancer and other LIV-1-expressing solid tumors. Under the terms of the agreement, Seattle Genetics will receive a $600 million upfront payment and Merck will make a $1.0 billion equity investment in 5.0 million shares of Seattle Genetics common stock at a price of $200 per share. In addition, Seattle Genetics is eligible for progress-dependent milestone payments of up to $2.6 billion.

Separately, Seattle Genetics has granted Merck an exclusive license to commercialize TUKYSA (tucatinib), a small molecule tyrosine kinase inhibitor, for the treatment of HER2-positive cancers, in Asia, the Middle East and Latin America and other regions outside of the U.S., Canada and Europe. Seattle Genetics will receive $125 million from Merck as an upfront payment and is eligible for progress-dependent milestones of up to $65 million.

Collaborating with Merck on ladiratuzumab vedotin will allow us to accelerate and broaden its development program in breast cancer and other solid tumors, including in combination with Mercks KEYTRUDA, while also positioning us to leverage our U.S. and European commercial operations, said Clay Siegall, Ph.D., President and Chief Executive Officer of Seattle Genetics. The strategic collaboration for TUKYSA will help us reach more patients globally and benefit from the established commercial strength of one of the worlds premier pharmaceutical companies.

These two strategic collaborations will enable us to further diversify Mercks broad oncology portfolio and pipeline, and to continue our efforts to extend and improve the lives of as many patients with cancer as possible, said Dr. Roger M. Perlmutter, President, Merck Research Laboratories. We look forward to working with the team at Seattle Genetics to advance the clinical program for ladiratuzumab vedotin, which has shown compelling signals of efficacy in early studies, and to bring TUKYSA to even more patients with cancer around the world.

Ladiratuzumab Vedotin Collaboration Details

Under the terms of the agreement, Seattle Genetics and Merck will collaborate and equally share costs on the global development of ladiratuzumab vedotin and other LIV-1-targeting ADCs. The companies have agreed to jointly develop and share future costs and profits for ladiratuzumab vedotin on a 50:50 basis worldwide. Merck will pay Seattle Genetics $600 million upfront and make a $1.0 billion equity investment in 5.0 million shares of Seattle Genetics common stock at a price of $200 per share. In addition, Seattle Genetics will be eligible to receive up to $2.6 billion in milestone payments, including $850 million in development milestones and $1.75 billion in sales milestones.

The companies will jointly develop and commercialize ladiratuzumab vedotin and equally share profits worldwide. The companies will co-commercialize in the U.S. and Europe. Seattle Genetics will be responsible for marketing applications for approval in the U.S. and Canada, and will record sales in the U.S., Canada and Europe. Merck will be responsible for marketing applications for approval in Europe and in countries outside the U.S. and Canada, and will record sales in countries outside the U.S., Europe and Canada. Including the upfront payment, equity investment proceeds and potential milestone payments, Seattle Genetics is eligible to receive up to $4.2 billion.

The closing of the equity investment is contingent on completion of review under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 (HSR Act).

TUKYSA Collaboration Details

Under the terms of the agreement, Merck has been granted exclusive rights to commercialize TUKYSA in Asia, the Middle East and Latin America and other regions outside of the U.S., Canada and Europe. Seattle Genetics retains commercial rights and will record sales in the U.S., Canada and Europe. Merck will be responsible for marketing applications for approval in its territory, supported by the positive results from the HER2CLIMB clinical trial.

Merck will also co-fund a portion of the TUKYSA global development plan, which encompasses several ongoing and planned trials across HER2-positive cancers, including breast, colorectal, gastric and other cancers set forth in a global product development plan. Seattle Genetics will continue to lead ongoing TUKYSA global development planning and operational execution. Merck will solely fund and conduct country-specific clinical trials necessary to support anticipated regulatory applications in its territory.

Seattle Genetics will receive from Merck $125 million as an upfront payment and is eligible to receive progress-dependent milestones of up to $65 million. Seattle Genetics will also receive $85 million in prepaid research and development payments to be applied to Mercks global development funding obligations. In addition, Seattle Genetics would receive tiered royalties on sales of TUKYSA in Mercks territory.

The financial impact of these collaborations is not included in Seattle Genetics 2020 guidance.

Seattle Genetics Conference Call Details

Seattle Genetics management will host a conference call to discuss these collaborations today at 6:00 a.m. Pacific Time (PT); 9:00 a.m. Eastern Time (ET). The event will be simultaneously webcast and available for replay from the Seattle Genetics website at http://www.seattlegenetics.com, under the Investors section. Investors may also participate in the conference call by calling 844-763-8274 (domestic) or +1 412-717-9224 (international). The conference ID is 10147850.

About Ladiratuzumab Vedotin

Ladiratuzumab vedotin is a novel investigational ADC targeted to LIV-1. Most metastatic breast cancers express LIV-1, which also has been detected in several other cancers, including lung, head and neck, esophageal and gastric. Ladiratuzumab vedotin utilizes Seattle Genetics proprietary ADC technology and consists of a LIV-1-targeted monoclonal antibody linked to a potent microtubule-disrupting agent, monomethyl auristatin E (MMAE) by a protease-cleavable linker. This novel ADC is designed to bind to LIV-1 on cancer cells and release the cell-killing agent into target cells upon internalization. Ladiratuzumab vedotin may also cause antitumor activity through other mechanisms, including activation of an immune response by induction of immunogenic cell death.

About TUKYSA (tucatinib)

TUKYSA is an oral, small molecule tyrosine kinase inhibitor (TKI) of HER2, a protein that contributes to cancer cell growth. TUKYSA in combination with trastuzumab and capecitabine was approved by the U.S. Food and Drug Administration (FDA) in April 2020 for adult patients with advanced unresectable or metastatic HER2-positive breast cancer, including patients with brain metastases, who have received one or more prior anti-HER2-based regimens in the metastatic setting. In addition, TUKYSA received approval in Canada, Singapore, Australia and Switzerland under the Project Orbis initiative of the FDA Oncology Center of Excellence that provides a framework for concurrent submission and review of oncology products among international partners. A marketing application is under review in the European Union.

TUKYSA is being evaluated in several ongoing clinical trials and additional studies are planned. Current trials include the following:

For additional information, visit http://www.clinicaltrials.gov.

TUKYSA Important Safety Information

Warnings and Precautions

If diarrhea occurs, administer antidiarrheal treatment as clinically indicated. Perform diagnostic tests as clinically indicated to exclude other causes of diarrhea. Based on the severity of the diarrhea, interrupt dose, then dose reduce or permanently discontinue TUKYSA.

Monitor ALT, AST, and bilirubin prior to starting TUKYSA, every 3 weeks during treatment, and as clinically indicated. Based on the severity of hepatoxicity, interrupt dose, then dose reduce or permanently discontinue TUKYSA.

Adverse Reactions

Serious adverse reactions occurred in 26% of patients who received TUKYSA. Serious adverse reactions in 2% of patients who received TUKYSA were diarrhea (4%), vomiting (2.5%), nausea (2%), abdominal pain (2%), and seizure (2%). Fatal adverse reactions occurred in 2% of patients who received TUKYSA including sudden death, sepsis, dehydration, and cardiogenic shock.

Adverse reactions led to treatment discontinuation in 6% of patients who received TUKYSA; those occurring in 1% of patients were hepatotoxicity (1.5%) and diarrhea (1%). Adverse reactions led to dose reduction in 21% of patients who received TUKYSA; those occurring in 2% of patients were hepatotoxicity (8%) and diarrhea (6%).

The most common adverse reactions in patients who received TUKYSA (20%) were diarrhea, palmar-plantar erythrodysesthesia, nausea, fatigue, hepatotoxicity, vomiting, stomatitis, decreased appetite, abdominal pain, headache, anemia, and rash.

Lab Abnormalities

In HER2CLIMB, Grade 3 laboratory abnormalities reported in 5% of patients who received TUKYSA were: decreased phosphate, increased ALT, decreased potassium, and increased AST. The mean increase in serum creatinine was 32% within the first 21 days of treatment with TUKYSA. The serum creatinine increases persisted throughout treatment and were reversible upon treatment completion. Consider alternative markers of renal function if persistent elevations in serum creatinine are observed.

Drug Interactions

Use in Specific Populations

For more information, please see the full Prescribing Information for TUKYSA here.

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 and pediatric patients with refractory classical Hodgkin lymphoma (cHL), or who have relapsed after 3 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 the confirmatory trials.

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).

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.

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.

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Seattle Genetics and Merck Announce Two Strategic Oncology Collaborations - BioSpace

BOSTON & BORDENTOWN, N.J.--(BUSINESS WIRE)--Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) and Caelum Biosciences today announced the initiation of the Cardiac Amyloid Reaching for Extended Survival (CARES) Phase 3 clinical program to evaluate CAEL-101, a first-in-class amyloid fibril targeted therapy, in combination with standard-of-care (SoC) therapy in AL amyloidosis. The CARES clinical program includes two parallel Phase 3 studies one in patients with Mayo stage IIIa disease and one in patients with Mayo stage IIIb disease and will collectively enroll approximately 370 patients globally. Enrollment is underway in both studies. The primary objective of the clinical program is to assess overall survival.

In AL amyloidosis, misfolded amyloid proteins can build up in many organs throughout the body, including the heart and kidneys, causing significant damage to these organs and impairing their function. While current treatments address the bone marrow disorder that creates the misfolded amyloid proteins, there are no approved therapies for the significant organ damage the disease causes, said John Orloff, M.D., Executive Vice President and Head of Research and Development at Alexion. CAEL-101 has the potential to be the first treatment to target and remove the amyloid deposits from these organs. Data from Phase 1 studies suggest that this treatment approach may improve organ function and long-term survival. We look forward to investigating this further in the Phase 3 clinical program.

AL amyloidosis is particularly devastating when it affects the heart, with median survival in these patients of less than one year following diagnosis, said Michael Spector, President and Chief Executive Officer of Caelum. Long-term survival data from AL amyloidosis patients treated with CAEL-101 in the Phase 1a/1b study showed that 78 percent were still alive after a median follow-up time of more than three years. We recognize the urgent need for new treatments that address the organ damage caused by AL amyloidosis and are working together with the AL amyloidosis community and Alexion to advance the Phase 3 clinical program as quickly as possible.

About the CARES Phase 3 Clinical Program

The CARES clinical program consists of two parallel double-blind, randomized, event-driven global Phase 3 studies, which are evaluating the efficacy and safety of CAEL-101 in AL amyloidosis patients who are newly diagnosed and nave to standard of care (SoC) treatment (cyclophosphamide-bortezomib-dexamethasone (CyBorD) chemotherapy). One study is enrolling approximately 260 patients with Mayo stage IIIa disease and one study is enrolling approximately 110 patients with Mayo stage IIIb disease. The studies will be conducted at approximately 70 sites across North America, the United Kingdom, Europe, Israel, Japan, and Australia.

In each study, participants are being randomized in a 2:1 ratio to receive either CAEL-101 plus SoC or placebo plus SoC once weekly for four weeks. This will be followed by a maintenance dose administered every two weeks until the last patient enrolled completes at least 50 weeks of treatment. Patients will continue follow-up visits every 12 weeks.

The primary study objectives are overall survival and the safety and tolerability of CAEL-101. Key secondary objectives will assess functional improvement in the six-minute walk test (6MWT), quality of life measures (Kansas City Cardiomyopathy Questionnaire Overall Score & Short Form 36 version 2 Physical Component Score) and cardiac improvement (Global Longitudinal Strain, or GLS).

Phase 2 Study Results

The Phase 2 open-label dose escalation study was conducted to investigate higher doses of CAEL-101 than had been evaluated in Phase 1 studies with a primary objective to identify the best dose to advance into Phase 3 development. The study evaluated the safety and tolerability of CAEL-101 in 13 AL amyloidosis patients at three study sites who received up to 1000 mg/m2 of CAEL-101 (two times the Phase 1 dose) administered in combination with SoC treatment. The study met its primary objectives, supporting the safety and tolerability of CAEL-101 and the selection of the 1000 mg/m2 dose for the Phase 3 study.

Phase 1a/1b Long-Term Follow-Up Results Presented at ISA 2020

As previously reported, the Phase 1a/1b study of CAEL-101 was the first clinical trial to demonstrate improvement in cardiac function via GLS after treatment with an amyloid fibril targeted therapy in AL amyloidosis patients with amyloid cardiac involvement. New long-term follow-up data from the Phase 1a/1b study will be presented at the virtual International Symposium on Amyloidosis (ISA), September 14 to 18, 2020, in the poster titled, Long term follow-up of patients with AL amyloidosis treated on a phase 1 study of Anti-Amyloid Monoclonal Antibody CAEL-101 (Abstract #342, Divaya Bhutani, M.D., et. al, Columbia University Medical Center). These data demonstrate 78 percent survival (15/19) at a median follow-up of more than three years (37 months) in AL amyloidosis patients treated with CAEL-101 as well as durable organ response among evaluable patients, further supporting the advancement of CAEL-101 into Phase 3 development.

About CAEL-101

CAEL-101 is a first-in-class monoclonal antibody (mAb) designed to improve organ function by reducing or eliminating amyloid deposits in the tissues and organs of patients with AL amyloidosis. The antibody is designed to bind to misfolded light chain protein and amyloid and shows binding to both kappa and lambda subtypes. In a Phase 1a/1b study, CAEL-101 demonstrated improved organ function, including cardiac and renal function, in 27 patients with relapsed and refractory AL amyloidosis who had previously not had an organ response to standard of care therapy. CAEL-101 has received Orphan Drug Designation from both the U.S. Food and Drug Administration and European Medicine Agency as a therapy for patients with AL amyloidosis.

About AL Amyloidosis

AL amyloidosis is a rare systemic disorder caused by an abnormality of plasma cells in the bone marrow. Misfolded immunoglobulin light chains produced by plasma cells aggregate and form fibrils that deposit in tissues and organs. This deposition can cause widespread and progressive organ damage and high mortality rates, with death most frequently occurring as a result of cardiac failure. Current standard of care includes plasma cell directed chemotherapy and autologous stem cell transplant, but these therapies do not address the organ dysfunction caused by amyloid deposition, and up to 80 percent of patients are ineligible for transplant.

AL amyloidosis is a rare disease but is the most common form of amyloidosis. There are approximately 22,000 patients across the United States, France, Germany, Italy, Spain and the United Kingdom. AL amyloidosis has a one-year mortality rate of 47 percent, 76 percent of which is caused by cardiac amyloidosis.

About Alexion

Alexion is a global biopharmaceutical company focused on serving patients and families affected by rare diseases and devastating conditions through the discovery, development and commercialization of life-changing medicines. As a leader in rare diseases for more than 25 years, Alexion has developed and commercializes two approved complement inhibitors to treat patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS), as well as the first and only approved complement inhibitor to treat anti-acetylcholine receptor (AchR) antibody-positive generalized myasthenia gravis (gMG) and neuromyelitis optica spectrum disorder (NMOSD). Alexion also has two highly innovative enzyme replacement therapies for patients with life-threatening and ultra-rare metabolic disorders, hypophosphatasia (HPP) and lysosomal acid lipase deficiency (LAL-D) as well as the first and only approved Factor Xa inhibitor reversal agent. In addition, the company is developing several mid-to-late-stage therapies, including a copper-binding agent for Wilson disease, an anti-neonatal Fc receptor (FcRn) antibody for rare Immunoglobulin G (IgG)-mediated diseases and an oral Factor D inhibitor as well as several early-stage therapies, including one for light chain (AL) amyloidosis, a second oral Factor D inhibitor and a third complement inhibitor. Alexion focuses its research efforts on novel molecules and targets in the complement cascade and its development efforts on the core therapeutic areas of hematology, nephrology, neurology, metabolic disorders and cardiology. Headquartered in Boston, Massachusetts, Alexion has offices around the globe and serves patients in more than 50 countries. This press release and further information about Alexion can be found at: http://www.alexion.com.

[ALXN-P]

About Caelum Biosciences

Caelum Biosciences, Inc. (Caelum) is a clinical-stage biotechnology company developing treatments for rare and life-threatening diseases. Caelums lead asset, CAEL-101, is a novel antibody for the treatment of patients with amyloid light chain (AL) amyloidosis. In 2019, Caelum entered a collaboration agreement with Alexion under which Alexion acquired a minority equity interest in Caelum and an exclusive option to acquire the remaining equity in the company based on Phase 3 CAEL-101 data. Caelum was founded by Fortress Biotech, Inc. (NASDAQ: FBIO). For more information, visit http://www.caelumbio.com.

Forward-Looking Statement

This press release contains forward-looking statements that involve risks and uncertainties relating to future events and the future performance of Alexion and Caelum, including statements related to: the safety and efficacy CAEL-101 as a treatment for AL amyloidosis; CAEL-101 has the potential to be the first treatment to target and remove the amyloid deposits from the heart, kidney and other organs; data from the Phase 1 studies suggest that the treatment approach may improve organ function and long-term survival and enrollment of the Phase 3 trials. Forward-looking statements are subject to factors that may cause Alexion's and Caelums results and plans to differ materially from those expected by these forward looking statements, including for example: the anticipated safety profile and the benefits of the CAEL-101 may not be realized (and the results of the clinical trials may not be indicative of future results); the inability to enroll and complete the Phase 3 trial; results of clinical trials may not be sufficient to satisfy regulatory authorities; results in clinical trials may not be indicative of results from later stage or larger clinical trials (or in broader patient populations); the possibility that results of clinical trials are not predictive of safety and efficacy and potency of our products (or we fail to adequately operate or manage our clinical trials) which could cause us to discontinue sales of the product (or halt trials, delay or prevent us from making regulatory approval filings or result in denial of approval of our product candidates); the severity of the impact of the COVID-19 pandemic on Alexions or Caelums business, including on commercial and clinical development programs; unexpected delays in clinical trials; unexpected concerns regarding products and product candidates that may arise from additional data or analysis obtained during clinical trials or obtained once used by patients following product approval; future product improvements may not be realized due to expense or feasibility or other factors; delays (expected or unexpected) in the time it takes regulatory agencies to review and make determinations on applications for the marketing approval of our products; inability to timely submit (or failure to submit) future applications for regulatory approval for our products and product candidates; inability to timely initiate (or failure to initiate) and complete future clinical trials due to safety issues, IRB decisions, CMC-related issues, expense or unfavorable results from earlier trials (among other reasons); future competition from biosimilars and novel products; decisions of regulatory authorities regarding the adequacy of our research, marketing approval or material limitations on the marketing of our products; delays or failure of product candidates to obtain regulatory approval; delays or the inability to launch product candidates due to regulatory restrictions, anticipated expense or other matters; interruptions or failures in the manufacture and supply of our products and our product candidates; failure to satisfactorily address matters raised by regulatory agencies regarding our products and product candidates; uncertainty of long-term success in developing, licensing or acquiring other product candidates or additional indications for existing products; the adequacy of our pharmacovigilance and drug safety reporting processes; failure to protect and enforce our data, intellectual property and proprietary rights and the risks and uncertainties relating to intellectual property claims, lawsuits and challenges against us; the risk that third party payors (including governmental agencies) will not reimburse for the use of our products at acceptable rates or at all; delay of collection or reduction in reimbursement due to adverse economic conditions or changes in government and private insurer regulations and approaches to reimbursement; adverse impacts on supply chain, clinical trials, manufacturing operations, financial results, liquidity, hospitals, pharmacies and health care systems from natural disasters and global pandemics, including COVID-19 and a variety of other risks set forth from time to time in Alexion's filings with the SEC, including but not limited to the risks discussed in Alexion's Quarterly Report on Form 10-Q for the period ended June 30, 2020 and in their other filings with the SEC. Alexion disclaims any obligation to update any of these forward-looking statements to reflect events or circumstances after the date hereof, except when a duty arises under law.

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Alexion and Caelum Biosciences Announce Start of Phase 3 Studies of CAEL-101 in AL Amyloidosis - Business Wire

The pursuit of new medical treatments often hinges on the balance between the efficacy of the drug and the adverse effects it causes. A key part of that balance is deciding on recommended dosing for a given treatment.

Opioids provide an excellent example of the challenge in maintaining that balance and the risks of non-ideal dosing. 20% of patients experiencing pain are prescribed opioids even though the recommended dose of the drugs will cause 50-80% of patients to experience some serious adverse effects. OxyContins standard dose was discovered to wear off early for many leading some users to take dangerously high amounts.

The problem with dosing is well known in the medical community. For instance, some doctors have advocated using much lower doses than recommended on product monographs in non-severe situations. Similarly, for cancer treatments, high dosing levels are being challenged as advanced treatments like immunotherapy gain popularity.

Problems exist on the other end of the spectrum as well. Nonsteroidal Anti-inflammatory Drugs NSAIDs are a common over-the-counter pain treatment and a 2018 study found that 15% of people took dangerously high amounts to reduce their pain. One explanation for the overuse was the lack of sufficient pain relief provided by the recommended dose. Taken in large quantities, the drugs can cause serious stomach issues.

A major reason for these dosing issues is that decisions are made on a statistical basis for large sample patient populations. The variability of these populations, including differences in height, weight, and ethnic background, make it unlikely that recommended doses will be ideal for everyone. Fortunately, as medicine is opening to non-pharmaceutical options, personalization is becoming easier. As a result, a new paradigm for dosing is emerging.

Personalized MedicinePersonalized medicine, a medical approach that optimizes both response and safety for individual patients, provides an alternative model to dosing that considers individual patient needs. For instance, 5% of the population do not get pain relief from codeine while others have it stay in the body too long due to their genetics. The ability to identify a patients genetics can help doctors make dosing decisions.

Unfortunately, genetics-based personalized medicine is costly and complicated for various reasons such as increased training and adapting electronic health records. An alternative approach is to allow patients to try different doses in cooperation with their physicians to discover the ideal balance. With pharmaceuticals though, this can mean delaying an effective treatment or exposing patients to adverse reactions and drug interactions.

Personalization Through DevicesOne solution that is gaining popularity is to turn to non-pharmaceutical medical devices when possible. These devices allow for a far greater range of safe treatment options that a patient can adjust even during a single treatment. For example, several medical devices using electrical neuromodulation have been developed to treat pain, spinal injuries, epilepsy, migraine, and other conditions. The devices stimulate nerves through electrical pulses delivered at different intensities to treat the given problem. Essentially, these intensities are the doses, but because they are not interacting with the body as pharmaceuticals do, they allow for a wider range of treatment options.

More than just a new technological solution, the possibility presented by these and similar medical devices is significant. User-controlled customization of the intensity of each treatment will both engage users and create a far higher chance of finding an ideal dose for each patient. Chronic pain patients, who may be more reliant on frequent use of pharmaceuticals, could especially benefit as they are most susceptible to having to limit dosing even if it makes the therapy less successful.

Back to the NSAID issue mentioned above, it is very easy for patients to take a third Advil pill for pain relief without sensing the danger of overdosing. With devices delivering electronic stimulation, the feeling of intensity is an immediate sensational signal that places a natural limit on the dose, without any serious long-term negative effects. Patients can trust that they can take a high yet tolerable dose safely. If their issue is still not solved, they can go back to their doctor to try different treatments.

ConclusionPersonalizing dosing is the future of treatment development. Allowing for individual patients to select their ideal dose will improve outcomes and reduce adverse events. Some aspects of personalization, such as seamlessly incorporating a patients genetics into dosing decisions, is still too costly to apply widely. However, we can create new treatments that give patients more control and safe choices, such as those provided by medical devices.

Pains ubiquity and its ability to be treated through medical devices make it a perfect target for this new approach. As with any new paradigm though, we need to educate patients and medical professionals about this new possibility. Furthermore, we need to lower costs and improve insurance processes to make sure that they are available to everyone.

If we do so, we will not only support patients (or customize treatment), but we will also improve outcomes and save money by avoiding adverse events.

Photo: Olena Agapova, Getty Images

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Moving beyond one-dose-fits-all: How medtech and personalized dosing can improve outcomes - MedCity News

BIRMINGHAM, Ala. (PRWEB) September 14, 2020

CerFlux, Inc., creators of advanced personalized medicine technology for specifically matching treatments to each cancer patients tumor before treatment, announced today the receipt of a $249,148 National Cancer Institute R43 grant to help fund continuing development of its low-cost, rapid Personalized Oncology Efficacy Test (POET) for Pancreatic Ductal Adenocarcinoma.

What makes this endeavor particularly noteworthy is that it will not only expand the body of knowledge but, if successful, transform cancer treatment around the world. Today, less than 3% of funded research makes such a leap across the valley of death from new knowledge to innovation.

Pancreatic ductal adenocarcinoma (PDAC) is among the deadliest cancers, with a less than 9% five-year survival rate. Without advances in technology like POET, the death rate from PDAC is estimated to increase to 60,000 deaths/year within the next 10 years. What makes PDAC particularly insidious is that a large number of patients arent diagnosed until the disease is at an advanced stage. While research and discovery are making new treatment options available, the lack of tools to quickly select the right treatment for each patient on a personalized basis creates a frustrating paradox in clinical decision-making. This is because every tumor is unique in its makeup and its response to treatment.

Without personalized predictive tools like POET, treatment is based on generalized parameters such as age, disease stage, etc. often leading to a mismatch between treatments and tumors. Consequently, about 75% of patients nearly 3 out of 4 have to endure first line chemotherapy that turns out to be ineffective because the treatment regimen did not match the patients tumor, imposing a substantial physical, emotional, and financial burden.

CerFlux POET addresses this critical, urgent, and unmet need for accessible and affordable predictive technologies that identify optimal therapy regimens and strategically eliminate ineffective options.

Our goal for this phase of the project is to identify best practices for using POET in personalized therapy and to measure the degree to which the effectiveness of the treatment observed in POET approximates the response in the corresponding patient. The idea is to not only predict which ones will be effective but also identify those which would be potentially ineffective for that specific patient to reduce the pain and time, explains CerFlux CEO Dr. Karim Budhwani.

This endeavor is further enhanced by a commercial-academic collaboration between CerFlux, Inc. and the James Comprehensive Cancer Center at the Ohio State University. This NCI funded project will build on recent work by the CerFlux team including a patented (US 10,114,010B1) biomimetic in vitro platform for pharmacological transport and pancreatic microtissue tumor models.

Dr. Allan Tsung, Director of the Gastrointestinal Disease Specific Research Group, adds, "I truly believe that the best cancer care is multidisciplinary care and similarly, advances in cancer research requires team science with multidisciplinary collaboration. The most innovative answers to problems are when you bring together ideas from different fields. Our team working together with CerFlux, Inc., embraces the belief at the Ohio State James Comprehensive Cancer Center that 'there is no routine cancer'. With advances in technology like POET, we strive to offer groundbreaking, personalized treatment options for all our patients battling cancer."

Predictive technologies like POET that match each patient with the right treatment regimen BEFORE treatment is administered will transform pancreatic cancer treatment in the near-term and make a difference in the lives of patients and their families, as well as providers around the world.

About CerFlux, Inc.CerFlux, Inc., a Birmingham, Alabama, based biotech company, is creating advanced, personalized medicine solutions to rapidly identify the most effective cancer treatments for each patient on an individualized basis. While breakthroughs in biopharma discovery are helping to produce a library of treatment options, many new therapeutics are becoming more specialized and therefore effective for smaller populations. This underscores an even greater need for matching treatments to tumors on an individualized basis; ironically, cancer is a ubiquitous rare disease. Our goal is to build solutions that quickly and clearly separate optimal from ineffective options, so clinical teams can successfully treat the disease thereby improving treatment outcomes and quality of life for cancer patients.

Additional InformationTo request collateral materials for publication or to schedule interviews with principals from CerFlux, Inc., please contact Rebecca Dobrinski at rsd@CerFlux.com or 205.335.3434 for availability.

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CERFLUX Announces National Cancer Institute Award - PR Web

A policy initiative was set up 5 years ago with the goal of spreading the adoption of a therapeutic strategy to maximize the brain health of every person with MS. The principle idea was that MS should be treated as effectively as early as possible, said Gavin Giovannoni, MBBCh, PhD, FCP (Neurol., SA), FRCP, FRCPath, chair of neurology at the Blizard Institute in the Barts and The London School of Medicine and Dentistry at Queen Mary University of London.

This means intervening early and effectively to affect the trajectory of MS over time. Im convinced we are doing this now, he said.

In the predisease-modifying therapy (DMT) era, by age 65, more than 80% of patients with MS needed a walking stick1. However, by the era of low-efficacy DMTs, that proportion was down to 60%, and now, in the high-efficacy era of DMTs, that proportion has fallen further, to 20% to 30% of patients needing a walking stick by the age of 65.

So, theres no doubt that by adopting this early, effective treatment approach, we have a major impact on the overall outcome, Giovannoni explained.

However, the challenge remains: how do we determine which patients with MS will respond to treatments? If there are 100 patients, its not clear who will or wont respond to therapy, and the only real option is to use statistical modeling.

Providers have to decide if they want to gamble on low- or high-efficacy therapies when they initiate treatment. Should they start with low-efficacy and move through the tiers or go right to the top tier from the outset?

Research has shown that early access to high-efficacy DMTs results in better outcomes. For fingolimod, natalizumab, and alemtuzumab the response rates are much higher, and they are associated with a lower risk of conversion to secondary progressive MS compared with therapies like interferon beta or glatiramer acetate.2 In addition, patients who started on high-efficacy therapies within 2 years of disease onset had less disability after 6 to 10 years than patients who started high-efficacy therapy later in the disease course.3

Theres little doubt in my mindearly access is whats important, Giovannoni said. Early access not late access.

However, treatment is not only about efficacypatients are going to be exposed to serious adverse events on these therapies. This is when patient choice becomes important. Providers will need to take into account issues like family planning and pregnancy when personalizing treatments.

Thats why safety comes into it, Giovannoni said. We always worry about the serious adverse event when we are customizing treatment strategies for patients with multiple sclerosis.

At baseline, the provider needs to look for ways to de-risk the high-efficacy DMTs to make them more palatable for patients to try as a first-line therapy.

Understanding the individuals disease is crucial, including profiling the patient in terms of understanding their risk aversion, adherence, comorbidities, and other factors before customizing treatment However, once that is done, it must be layered onto the health system and economic factors, he pointed out.

So, there is not a simple solution to the customized medicine in relation to our disease-modifying therapy, Giovannoni said.

Subgroup analyses to understand response rates

Predicting a better response to treatments is very important in MS now that there are multiple treatment options to choose from, said Maria Pia Sormani, professor of biostatistics at the University of Genoa, Italy.

However, it is not easy to define response to treatment in MS. For instance, in solid tumors, a reduction in the tumor mass after chemotherapy is defined as a response to treatment because there is no spontaneous mass reduction without treatment. In relapsing-remitting MS, simply having no relapses after an immunomodulatory drug cannot be defined as having a response to treatment, because in the disease, patients can have stable disease course even without treatment.

A subgroup analysis can help better understand patients who are responders to therapy, she explained. One study of cladribine tablets showed that while the overall risk reduction versus placebo was 50%, there were differences in response depending on disease activity.4 In patients with high disease activity, the probability of disease progression was reduced by more than 80% among patients who took cladribine; the reduction was less than 20% in patients without high disease activity.

Sometimes, when many subgroups are tested, researchers will find by chance that some subgroups have a higher response to treatment, but that finding might not be confirmed in a follow-up study, Sormani noted.

Another analysis showed that in the DEFINE study,5 patients with no prior MS treatment had a relapse risk reduction of 67% when treated with oral BG-12 (dimethyl fumarate), but in the CONFIRM study,6 the opposite was true: the reduction was only 36% in patients with no MS treatment, and the risk reduction was larger in patients who had previous treatment (54%).

A new approach that Sormani and her colleagues crafted would create a personalized profile of patients who respond better using a patient-specific continuous score.7 They applied the analysis to 3 trials: ALLEGRO was used to define the score; BRAVO was used to test the score; and CONCERTO was used to externally validate the findings.

The score was able to define responders and nonresponders, and Sormani provided an example of how it might be applied in clinical practice. First instance, take a patient with MS with 1 relapse in the previous year, 1 gadolinium-enhancing lesion on the baseline scan, and a normalized brain volume of 1600 cm3:

In conclusion, Sormani noted that there are now statistical techniques based on post-hoc analyses of clinical trials that can help identify which patients will benefit the most from which treatments in order to choose the best MS therapy for them.

We must try to push on pharmaceutical companies to allow re-analysis of their clinical trials to identify subgroup responders to every drug, she said.

References

1. Capra R, Cordioli C, Rasia S, Gallo F, Signori A, Sormani MP. Assessing long-term prognosis improvement as a consequence of treatment pattern changes in MS. Mult Scler. 2017;23(13):1757-1761. doi:10.1177/1352458516687402

2. Brown JWL, Coles A, Horakova D, et al. Association of initial disease-modifying therapy with later conversion to secondary progressive multiple sclerosis. JAMA. 2019;321(2):175-187. doi:10.1001/jama.2018.20588

3. He A, Merkel B, Brown JWL, et al. Timing of high-efficacy therapy for multiple sclerosis: a retrospective observational cohort study. Lancet Neurol. 2020;19(4):307-316. doi:10.1016/S1474-4422(20)30067-3

4. Giovannoni G, Soelberg Sorensen P, Cook S, et al. Efficacy of cladribine tablets in high disease activity subgroups of patients with relapsing multiple sclerosis: a post hoc analysis of the CLARITY study. Mult Scler. 2019;25(6):819-827. doi:10.1177/1352458518771875

5. Bar-Or A, Gold R, Kappos L, et al. Clinical efficacy of BG-12 (dimethyl fumarate) in patients with relapsing-remitting multiple sclerosis: subgroup analyses of the DEFINE study. J Neurol. 2013;260(9):2297-305. doi:10.1007/s00415-013-6954-7

6. Hutchinson M, Fox RJ, Miller DH, et al. Clinical efficacy of BG-12 (dimethyl fumarate) in patients with relapsing-remitting multiple sclerosis: subgroup analyses of the CONFIRM study. J Neurol. 2013;260(9):2286-96. doi:10.1007/s00415-013-6968-1

7. Pellegrini F, Copetti M, Bovis F, et al. A proof-of-concept application of a novel scoring approach for personalized medicine in multiple sclerosis. Mult Scler. 2020;26(9):1064-1073. doi:10.1177/1352458519849513

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Choosing the Right MS Treatment by Customizing Therapy to the Individual - AJMC.com Managed Markets Network

NEW YORK, Sept. 14, 2020 /PRNewswire/ --PulsePoint, the leading technology company using real-time data to accelerate health outcomes, is set to host their second bi-annual Digital Health Forum in October. Themed "The Future was Today," the event is designed around discussions aiming to disentangle many of the challenges within healthcare and rewrite an ideal vision that capitalizes on the opportunities technology and health data present.

PulsePoint prides itself in being a forward moving company, a sentiment captured in its corporate tagline, 'Leading Health Forward'. With this event, the company is leaning into the change we're currently going through and enlisting other forward-oriented minds to collectively re-envision how we use data and technology to help people make better health decisions.

Many big minds are trying to work through the current business challenges created by the pandemic. PulsePoint wants to look beyond, to address key issues facing healthcare, specifically around what health data will look like in five years, how rules are being re-written and what new behaviors are being formed. While no one can see that far ahead, PulsePoint is crafting the event to stimulate an exchange of ideas and creative thinking, with the ultimate goal of making us all more purposeful in charting a smart, productive and compassionate way forward.

The forum will feature an eclectic mix of multidisciplinary speakers including doctors, health policy officials, futurists, disruptors, technologists, journalists, non-profits, media personalities, and even teens representing the voices of tomorrow. Key headliners include Dr. Scott Gottlieb, former commissioner of the FDA, Bob Garfield, co-host of On the Media and Dr. Amy Zalman, CEO of Prescient and Professor of Foresight at Georgetown University.

PulsePoint's last Digital Health Forum, at Lincoln Center in 2018, focused on 'Radical Health Personalization' and explored topics such as consumer privacy, health data security, personalized medicine and humanitarianism in digital health.

"PulsePoint's Digital Health Forum is a foundational event for the health industry as data and technology intercepts with the huge transformational shift we are seeing in the market. The year 2020 will be viewed as a point in time when the health industry fundamentally embraced digital technology and finally turned their backs on the analog world that has slowed down its progress for so many years," said PulsePoint CEO, Sloan Gaon. "We're bringing experts together from different parts of health, technology and media to dig into what's new, what's next and what's actionable."

The event will be held virtually on October 6th. Register at TheFutureWasToday.com

About PulsePoint

We are a technology company using real time data to transform healthcare. Through machine learning and programmatic automation, we interpret the hard-to-read signals of the health journey to understand the connection points between relevance and engagement. We do this by unifying real-time Digital Determinants of Health, offline and clinical data to create a unique and precise view of health audiences that refines, improves and increases its view over time. Visit http://www.pulsepoint.com for more about the company's technology and award winning culture.

Contact: Maria Simeone [emailprotected]

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PulsePoint's Digital Health Forum 'The Future was Today' to Explore the Rapid Transformational Change Occurring Within Healthcare - PRNewswire

Robots welding at the assembly line of the French car maker Peugeot/Citroen in Brazil. Artificial intelligence offers a chance for the Latin America's economies to leapfrog to greater innovation and economic progress.

Photo: Antonio Scorza/AFP/ Getty Images

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E-commerce firms have faced a conundrum in Latin America: How can they deliver packages in a region where 25% of urban populations live in informal, squatterneighborhoods with no addresses?

Enter Chazki, a logistics startup from Peru, which partnered with Arequipas Universidad San Pablo to build an artificial intelligence robot to generate new postal maps across the country. The company has now expanded to Argentina, Mexico and Chile, introducing remote communities and city outskirts to online deliveries.

Thats just one example of how machine learning is bringing unique Latin American solutions to unique Latin American challenges. Artificial intelligence and its correlated technologies could prove a major boon to the regions public and private sectors. In turn, its policymakers and business leaders have to better prepare to take full advantage while warding off potential downsides.

Latin America has long been the victim of low productivity and the COVID-19 pandemic is predictably making matters worse. Now, artificial intelligence is a chance for the regions economies to leapfrog to greater innovation and economic progress. Research suggests that AI will add a full percentage point of GDP to five of South Americas biggest economies Argentina, Brazil, Chile, Colombia and Peru by 2035.

Artificial intelligence could play transformative roles in Latin America for just about every sector, according to the Inter-American Development Bank (IADB). That means using AI to predict trade negotiation outcomes, commodity prices and consumer trends, or developing algorithms for use in factories, personalized medicine, infrastructure prototyping, autonomous transportation and energy consumption.

AIs applications in Latin America are already becoming reality. Argentinian Banco Galicia, Colombian airline Avianca, and Brazilian online shopping platform Shop Fcil have all adopted chatbots as virtual customer service assistance to help people. Chiles The Not Company developed an algorithm that analyzes animal-based food products and a database of 400,000 plants to generate recipes for vegan alternatives, and Perus National University of Engineering built machines to autonomously detect dangerous gases.

Expect the trend to continue in the near future. An MIT global survey of senior business executives worldwide found that, by the end of 2019, 79% of companies in Latin America had launched AI programs. The results have been positive; fewer than 2% of respondents reported that the initiatives made lower-than expected returns.

Another key factor is public acceptance of AI and automation. Thus far, Latin Americans are ahead of the curve in embracing the future, with another recent poll showing that 83% of Brazilian consumers said they would trust banking advice entirely generated by a computer, compared to a global average of 71%.

In a region that suffers from endemic corruption, pervasive violence, weak institutions and challenging socioeconomic conditions, governments, policymakers and organizations can use AI to tackle critical issues in the region, including food security, smart cities, natural resources and unemployment.

In Argentina, for example, artificial intelligence is being used to predict and prepare for teenage pregnancy and school dropout, as well as to outline unseen business opportunities in city neighborhoods. In Colombia and Uruguay, software has been developed to predict where crimes are likely to occur. In Brazil, the University of So Paulo is developing machine-learning technology that will rapidly assess the likelihood that patients have dengue fever, Zika or chikungunya when they arrive at a medical center.

At a time when public support for democracy in Latin America is flailing, AI could help come to the rescue. Congressional bodies across the region could use AI to boost the transparency and input of the legislative process. Indeed, the Hacker Laboratory, an innovation lab within Brazils Chamber of Deputies, is using AI platforms to facilitate interactions between lawmakers and citizens.

AI is not risk-free, of course. Elon Musk called AI humanitys biggest existential threat, and Stephen Hawking said it could spell the end of the human race.

Apocalyptic scenarios aside, the immediate danger of AI in Latin America is unemployment and inequality. The Inter-American Development Bank (IADB) warned in a 2018 study that between 36% and 43% of jobs could be lost due to artificial intelligence as a result of automatization. Indeed, Latin Americas governments must be prepared to set up guardrails and implement best practices for the implementation of AI.

Several governments in the region have already announced AI public policy plans. Mexico was one of the first 10 countries in the world to create a national AI strategy. Meanwhile, Brazil launched anational Internet of Things plan, which includes the countrys commitment to a network of AI laboratories across strategic areas including cybersecurity and defense. Chile is coordinating with civil society groups and experts to adopt its own AI plan.

Move aside, Mercosur! Governments in Latin America might also find that machine learning strengthens regional ties. That means harnessing AI to crunch data on trade flows and rules, find areas of consensus in multilateral negotiations, or create algorithms for regional trade. After all, AI models have a 300% greater predictive capacity than traditional econometric models, according to the Inter-American Development Bank.

Beyond competing national plans for AI, Latin American leaders should be drafting a strategy that is specific to its region much like the European Union is doing. A key takeaway from the recent UNESCO Regional Forum on AI in Latin America and the Caribbean was that the technology must develop with respect to universally recognized human rights and values.

In 2021, artificial intelligence could generate almost $3 trillion in business value and 6.2 billion hours of productivity worldwide. Latin America is rightfully jumping onto the bandwagon and has the potential to lead the parade in some areas.

To make full use of what could be a transformational productivity revolution for the region, government and business leaders must pump more resources into technology planning and education. The implementation of AI must improve, not accelerate, the regions inequity.

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Latin America's Growing Artificial Intelligence Wave BRINK News and Insights on Global Risk - BRINK

SAN FRANCISCO, Sept. 14, 2020 /PRNewswire/ -- The global artificial intelligence in drug discovery marketsize is expected to reach USD 3.5 billion by 2027, expanding at a CAGR of 28.8%, according to a new report by Grand View Research, Inc. The growing adoption of artificial intelligence (AI) platforms aimed at reducing the overall cost associated with drug discovery and to enhance results is expected to propel the demand for these platforms in the pharma market.

Key suggestions from the report:

Read 80 page research report with ToC on "Artificial Intelligence In Drug Discovery Market Size, Share & Trends Analysis Report By Therapeutic Area (Oncology, Cardiovascular Disease, Metabolic Diseases), By Application, By Region, And Segment Forecasts, 2020 - 2027'' at: https://www.grandviewresearch.com/industry-analysis/artificial-intelligence-drug-discovery-market

Based on the application, the drug optimization and repurposing segment accounted for the largest revenue share in 2019. AI platforms help in identifying alternative applications for existing medicines. This can help pharma companies diversify their portfolio of offerings and assist in producing alternative therapies through minor alterations in the pharmaceutical product. Furthermore, AI platforms also enable researchers to identify the toxic effects of the medicine on the body and thus reduce the chances of adverse effects.

In 2019, oncology application accounted for the largest share whereas, the infectious disease application segment is expected to emerge as the fastest-growing application segment over the forecast period. Artificial intelligence already plays a vital role in the early detection of cancer. Furthermore, as treatments for cancer may vary for each patient, personalized medicine has proven to be an effective alternative.

North America dominated the market with a revenue share of 59.4% in 2019 owing to the increasing adoption of artificial intelligence and the presence of a large number of market players. Asia Pacific is expected to emerge as the fastest-growing region in the market over the forecast period. The growing adoption of new technology in India and China for new drug development and a focus towards boosting pharmaceutical capacities within the countries is expected to drive the market in the region.

Grand View Research has segmented the global artificial intelligence in drug discovery market based on application, therapeutic area, and region:

List of Key Players of Artificial Intelligence In Drug Discovery Market

Find more research reports on Healthcare IT Industry, by Grand View Research:

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About Grand View Research

Grand View Research, U.S.-based market research and consulting company, provides syndicated as well as customized research reports and consulting services. Registered in California and headquartered in San Francisco, the company comprises over 425 analysts and consultants, adding more than 1200 market research reports to its vast database each year. These reports offer in-depth analysis on 46 industries across 25 major countries worldwide. With the help of an interactive market intelligence platform, Grand View Research helps Fortune 500 companies and renowned academic institutes understand the global and regional business environment and gauge the opportunities that lie ahead.

Contact:Sherry JamesCorporate Sales Specialist, USAGrand View Research, Inc.Phone: +1-415-349-0058Toll Free: 1-888-202-9519Email: [emailprotected]Web: https://www.grandviewresearch.comFollow Us: LinkedIn| Twitter

SOURCE Grand View Research, Inc.

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Artificial Intelligence In Drug Discovery Market Size Worth $3.5 Billion By 2027: Grand View Research, Inc. - PRNewswire

DGAP-News: Evotec SE / Key word(s): Miscellaneous14.09.2020 / 07:30 The issuer is solely responsible for the content of this announcement.

Hamburg, Germany, 14 September, 2020: Evotec SE (Frankfurt Stock Exchange: EVT, MDAX/TecDAX, ISIN: DE0005664809) and the French-based BIOASTER Technological Research Institute ("BIOASTER") today announced that they have entered into a partnership to advance research for infectious diseases. BIOASTER is a technology and innovation hub located in Lyon, France that has created a new model to address the latest challenges in the microbiology field, including antimicrobial resistance and vaccine safety and efficacy.

The organisations have been working together since the beginning of 2020 and Evotec represents the first global research and development company to locate staff in BIOASTER's premises in Lyon.

Under the terms of the collaboration agreement, the two organisations aim to put forward new research projects against infectious diseases and antimicrobial resistance, potentially creating new therapies and technologies. This collaboration had already started through the European research projects and development consortium ERA4TB (European Regimen Accelerator for Tuberculosis) and GNA NOW (Novel Gram-negative antibiotic now) within the AMR accelerator supported by the Innovative Medicines Initiative IMI2, aimed at reducing resistance to antibiotics and developing new therapeutic solutions.

"BIOASTER is proud to have support from the French and European leaders in diagnostics, vaccines, and animal health, and is pleased to formalise such a partnership with Evotec at the Lyon site. This allows BIOASTER to strengthen its agility and its capacity for technological innovation, in particular on antimicrobial resistance. BIOASTER now has a high-value network of close partners, which covers our four fields of application: antimicrobials, diagnostics, vaccines and microbiota understanding are key for manufacturers and patients alike," said Dr Philippe Archinard, President of IRT BIOASTER.

"The agreement will nurture the research ecosystem of Lyon Metropole and its Biodistrict, and impact on both national and European levels. The fact that Evotec, a company headquartered in Hamburg, Germany, chose to locate its unit dedicated to infectious diseases together with BIOASTER in the Biodistrict Lyon, reinforces the reputation of this French health ecosystem, while offering a more complete and more powerful technological toolbox to speed up industrialisation of innovations," said Emeline Baume, first vice president of Lyon Metropole.

"We are very glad to be partnering with BIOASTER, bringing Evotec's proven global resources for anti-infective research and drug development to Lyon," said Dr Werner Lanthaler, Chief Executive Officer of Evotec. "Both Evotec and BIOASTER have made a long-term commitment to tackle the challenge of antimicrobial resistance and we are confident that together we will be able to efficiently drive forward the progress in this field with high and rising unmet medical need."

The two entities gather more than 120 researchers in total, share the same advanced infrastructures, including five BSL3 laboratories, with easy access to diverse equipments: this co-location creates a new pole of attraction, particularly suited to the expectations of industrials to accelerate and de-risk their product developments in infectious diseases.

ABOUT BIOASTER TECHNOLOGICAL RESEARCH INSTITUTECreated in 2012, following the French initiative of Technological Research Institutes, BIOASTER is a non-for-profit foundation developing a unique technological and innovative model to support the latest challenges in microbiology. In particular, BIOASTER uses and develops high value technological innovations that accelerate development of medical solutions for populations and personalized medicine.The aim of BIOASTER is to bring together academic, industry and its capacities and specific knowledge to develop and execute high impact collaborative projects requiring industry compatible innovative technologies.Key figures:* 4 fields of expertise: antimicrobials, diagnostics, microbiota, vaccines* BSL2 & BSL3 laboratories in Lyon and Paris* 100+ employees, including 80% of scientific experts, 17 nationalities* 66+ collaborative projects, involving 27 private partners, 29 public partners.www.bioaster.org

ABOUT EVOTEC SEEvotec is a drug discovery alliance and development partnership company focused on rapidly progressing innovative product approaches with leading pharmaceutical and biotechnology companies, academics, patient advocacy groups and venture capitalists. We operate worldwide and our more than 3,300 employees provide the highest quality stand-alone and integrated drug discovery and development solutions. We cover all activities from target-to-clinic to meet the industry's need for innovation and efficiency in drug discovery and development (EVT Execute). The Company has established a unique position by assembling top-class scientific experts and integrating state-of-the-art technologies as well as substantial experience and expertise in key therapeutic areas including neuronal diseases, diabetes and complications of diabetes, pain and inflammation, oncology, infectious diseases, respiratory diseases, fibrosis, rare diseases and women's health. On this basis, Evotec has built a broad and deep pipeline of approx. 100 co-owned product opportunities at clinical, pre-clinical and discovery stages (EVT Innovate). Evotec has established multiple long-term alliances with partners including Bayer, Boehringer Ingelheim, Bristol-Myers Squibb, CHDI, Novartis, Novo Nordisk, Pfizer, Sanofi, Takeda, UCB and others. For additional information please go to http://www.evotec.com and follow us on Twitter @Evotec.

FORWARD-LOOKING STATEMENTSInformation set forth in this press release contains forward-looking statements, which involve a number of risks and uncertainties. The forward-looking statements contained herein represent the judgement of Evotec as of the date of this press release. Such forward-looking statements are neither promises nor guarantees, but are subject to a variety of risks and uncertainties, many of which are beyond our control, and which could cause actual results to differ materially from those contemplated in these forward-looking statements. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any such statements to reflect any change in our expectations or any change in events, conditions or circumstances on which any such statement is based.

14.09.2020 Dissemination of a Corporate News, transmitted by DGAP - a service of EQS Group AG.The issuer is solely responsible for the content of this announcement.

The DGAP Distribution Services include Regulatory Announcements, Financial/Corporate News and Press Releases. Archive at http://www.dgap.de

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EVOTEC AND BIOASTER PARTNER TO BUILD A TECHNOLOGY AND INNOVATION HUB IN LYON - PharmiWeb.com

Debut Bio

SAN DIEGO (PRWEB) September 15, 2020

Debut Biotechnology, a UC-Irvine spinout advancing scalable cell-free biomanufacturing, today announced that it has been awarded $1M from the Air Force Research Labs Biotechnology Grand Challenge. Debut Bio is the inaugural recipient of the award, which honors the companys proposal on biosynthesis of monomers for aerospace applications.

In the proposal, Debut Bio details its ability to leverage the companys scalable biomanufacturing platform for bio- and metal-free synthesis of polymer precursors relevant for the aerospace industry. Debut Bios platform will provide AFRL with the ability to effectively and efficiently biomanufacture specialty ingredients needed for aerospace applications. In doing so, Debut Bio expects to reduce aerospace material costs and improve vehicle maintenance processes by extending service lifetimes.

The Air Force Research Lab has a well-documented history in discovering and leveraging breakthrough technologies. By offering a cell-free, continuous biomanufacturing process, we are excited to continue that legacy of innovation through our work with them, said Dr. Joshua Britton, CEO and Founder of Debut Biotechnology. Cell-free biomanufacturing enables the quality manufacturing of high-value molecules at a price point that scales far beyond what is possible today. In bringing this approach to the AFRL, we hope to usher in a new era of biomanufacturing for high-value molecules that is scalable, economically viable, and results in solutions that are efficient and effective.

Debut Bio has applied its technology to the pharmaceutical and specialty ingredient industries, allowing customers to manufacture high-value molecules by combining custom-designed immobilized enzymes with continuous biomanufacturing processes. Through the utilization of plug-and-play enzyme cartridges, the companys cell-free platform has the ability to transform low-value bio-renewable materials into high-value specialty chemicals.

The Debut Biotechnology proposal provides a path to a scalable, bio- and metal free synthesis of key aerospace polymer precursors, explained Dr. Davide Simone, AFRLs technology advisor for biosynthesis of monomers for aerospace thermosets and senior research chemist. Expected benefits for the Air Force include a significant savings in composite aerospace structural material costs and the elimination of oxidation promoting metal catalysts from structural components, extending service lifetimes.

Debut Bios enzyme cartridges are sequentially arranged in a manner which mimics pathways found in plants and other organisms. Each cartridge is finely-tuned to provide the ideal temperature, pH, concentration, solvent, and reaction time for each individual step to maximize enzymatic productivity and product titer levels. The platform requires far fewer steps than traditional materials manufacturing and avoids the use of environmentally harmful chemical synthesis. Importantly, it overcomes many challenges typically associated with relying on cells for biomanufacturing or extraction of ingredients from plants and other sources.

In Debut Bios system, materials are passed from one enzyme reactor to another to build in-vitro or cell-free biosynthetic pathways. The companys platform uses a sustainable approach by leveraging advanced enzymes outside of the cell to maximize performance and avoid issues with cell-based fermentation systems. The platform technology operates under a continuous manufacturing regime, allowing Debut Bio to scale beyond the capabilities of other manufacturing processes currently on the market, which are constrained by the precipitation of enzymes, the need for multiple large reactors, and the specific requirements of enzymes. By leveraging continuous manufacturing, Debut Bio is able to drive down the cost of cell-free manufacturing to provide scale, consistency, novelty, and ultimately the ability to produce a biomolecule with the click of a button.

Debut Bio serves organizations across the defense and specialty ingredient industries, with commitments from several large scale manufacturers to integrate its platform into their existing pipelines.

To learn more, please visit: https://debutbiotech.com.

About Debut Biotechnology

Debut Biotechnology is commercializing cell-free biomanufacturing solutions by creating advanced enzyme-based manufacturing processes for high-value molecules. Our platform combines immobilized enzymes with highly controlled and automated continuous manufacturing systems for the pharmaceutical and specialty chemical industries.

Debut Biotechnology was founded by Joshua Britton, Greg Weiss, and Brady Beauchamp and is based in sunny San Diego. To learn more, please visit: https://debutbiotech.com/.

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Debut Biotechnology Awarded $1M from Air Force Research Lab to Leverage Cell-Free Continuous Biomanufacturing in Aerospace Applications - PR Web

Nantkwest Inc (NK) is around the top of the Biotechnology industry according to InvestorsObserver. NK received an overall rating of 62, which means that it scores higher than 62 percent of all stocks. Nantkwest Inc also achieved a score of 79 in the Biotechnology industry, putting it above 79 percent of Biotechnology stocks. Biotechnology is ranked 31 out of the 148 industries.

Analyzing stocks can be hard. There are tons of numbers and ratios, and it can be hard to remember what they all mean and what counts as good for a given value. InvestorsObserver ranks stocks on eight different metrics. We percentile rank most of our scores to make it easy for investors to understand. A score of 62 means the stock is more attractive than 62 percent of stocks.

Our proprietary scoring system captures technical factors, fundamental analysis and the opinions of analysts on Wall Street. This makes InvestorsObservers overall rating a great way to get started, regardless of your investing style. Percentile-ranked scores are also easy to understand. A score of 100 is the top and a 0 is the bottom. Theres no need to try to remember what is good for a bunch of complicated ratios, just pay attention to which numbers are the highest.

Nantkwest Inc (NK) stock is trading at $7.94 as of 1:12 PM on Monday, Sep 14, a gain of $1.34, or 20.3% from the previous closing price of $6.60. The stock has traded between $6.84 and $7.97 so far today. Volume today is above average. So far 1,740,191 shares have traded compared to average volume of 1,370,495 shares.

Click Here to get the full Stock Score Report on Nantkwest Inc (NK) Stock.

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Where Does Nantkwest Inc (NK) Stock Fall in the Biotechnology Field? - InvestorsObserver

Evelo Biosciences Inc (EVLO) is around the middle of the Biotechnology industry according to InvestorsObserver. EVLO received an overall rating of 44, which means that it scores higher than 44 percent of all stocks. Evelo Biosciences Inc also achieved a score of 45 in the Biotechnology industry, putting it above 45 percent of Biotechnology stocks. Biotechnology is ranked 31 out of the 148 industries.

Finding the best stocks can be tricky. It isnt easy to compare companies across industries. Even companies that have relatively similar businesses can be tricky to compare sometimes. InvestorsObservers tools allow a top-down approach that lets you pick a metric, find the top sector and industry and then find the top stocks in that sector.

Our proprietary scoring system captures technical factors, fundamental analysis and the opinions of analysts on Wall Street. This makes InvestorsObservers overall rating a great way to get started, regardless of your investing style. Percentile-ranked scores are also easy to understand. A score of 100 is the top and a 0 is the bottom. Theres no need to try to remember what is good for a bunch of complicated ratios, just pay attention to which numbers are the highest.

Evelo Biosciences Inc (EVLO) stock is trading at $5.08 as of 2:35 PM on Monday, Sep 14, a rise of $0.39, or 8.32% from the previous closing price of $4.69. The stock has traded between $4.81 and $5.22 so far today. Volume today is less active than usual. So far 65,585 shares have traded compared to average volume of 145,817 shares.

Click Here to get the full Stock Score Report on Evelo Biosciences Inc (EVLO) Stock.

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Is Evelo Biosciences Inc (EVLO) Stock Near the Top of the Biotechnology Industry? - InvestorsObserver

The 70 rating InvestorsObserver gives to Avid Bioservices Inc (CDMO) stock puts it near the top of the Biotechnology industry. In addition to scoring higher than 90 percent of stocks in the Biotechnology industry, CDMOs 70 overall rating means the stock scores better than 70 percent of all stocks.

Searching for the best stocks to invest in can be difficult. There are thousands of options and it can be confusing on what actually constitutes a great value. Investors Observer allows you to choose from eight unique metrics to view the top industries and the best performing stocks in that industry. A score of 70 would rank higher than 70 percent of all stocks.

These scores are not only easy to understand, but it is easy to compare stocks to each other. You can find the best stock in an industry, or look for the sector that has the highest average score. The overall score is a combination of technical and fundamental factors that serves as a good starting point when analyzing a stock. Traders and investors with different goals may have different goals and will want to consider other factors than just the headline number before making any investment decisions.

Avid Bioservices Inc (CDMO) stock is trading at $7.69 as of 10:59 AM on Monday, Sep 14, a gain of $0.51, or 7.03% from the previous closing price of $7.18. The stock has traded between $7.27 and $7.78 so far today. Volume today is 347,046 compared to average volume of 413,400.

Click Here to get the full Stock Score Report on Avid Bioservices Inc (CDMO) Stock.

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Where Does Avid Bioservices Inc (CDMO) Stock Fall in the Biotechnology Field? - InvestorsObserver

Kolkata, Sep 14 (UNI) GlaxoSmithKline Pharmaceuticals Ltd. (GSK Pharmaceutical Ltd.) in collaboration with the Regional Centre for Biotechnology (RCB) has completed the first year of its Trust in Science Program and is marking it by hosting a six part Trust in Science webinar series. The first webinar was held today. Like the program, these webinars are also an academia-industry collaboration in which experts from the private sector and scientific community will provide insights on trends in bioinformatics and biostatistics and the future outlook of this valuable skill set. The Trust in Science program offers full PhD courses in Biostatistics and Bioinformatics to equip Indian students for a career in a data and AI-driven pharma research ecosystem.

Commenting on the progress of the program, Prof. Sudhanshu Vrati, Executive Director, Regional Centre for Biotechnology, said, Biostatistics and bioinformatics are rapidly emerging fields in biology and are set to change the way we approach scientific R&D. In the first year of this program, we got a good response from aspiring PhD candidates eager to explore scientific careers in these fields. Now, through our webinar series, we hope to reach out to even more Indian students and faculty to make them aware of the potential that lies in these fields and to realize the value of the Trust in Science PhD program.

In the keynote address, HE Jan Thompson, Acting British High Commissioner to India, said the Trust in Science initiative is a great example of public-private-partnership. She added, It is my hope that the Trust in Science initiative will give young researchers an opportunity to explore high-end expertise in drug discovery and the program becomes a spring-board for them to transition into a full-fledged pharma career in the future. The UK and Indian governments continue to work together in healthcare, exploring opportunities for some of the best UK companies to offer their products and services for improved healthcare delivery in India.

Dr. Renu Swarup, Secretary, Department of Biotechnology, Government of India, said, It is great to see that this RCB-GSK collaboration is more than a research budget collaboration between academia and industry, but one that looks to create a human resource foundation for new technology. I would like to see this partnership scaled up, with more partners included so that it becomes a national network for the country. I believe this webinar series can reach out to more people and draw in more institutes to see the value of this partnership..

Mr. Sridhar Venkatesh, Managing Director, GSK Pharmaceuticals Limited (India) said, The ongoing pandemic has highlighted the value of biostatistics and bioinformatics tools and methodologies to significantly reduce the time taken for a medicine to be brought from lab to market. This is the future of pharmaceuticals research and we believe that the Trust in Science programme is contributing to prepare a workforce that is future ready. We hope the webinar series will help aspiring PhD students to get a primer on the likely future of pharma research.

The launch webinar concluded with a learning session by Prof Partha Mazumdar, distinguished Professor, National Institute of Biomedical Genomics, who said, A confluence of statistical science and data science is the enabler of rapid and robust inferences in biology and medicine. He also spoke of the critical value of biostatistics and bioinformatics during the ongoing pandemic by referring to the study on the diversity and evolution of the coronavirus SARS-CoV-2 through the analysis of RNA sequences collected from 3636 infected patients across 55 countries. This is an exciting time in modern biology and medicine, he added, referring to the coming together of statics and big data analysis.

GSK Pharmaceutical Ltd.s Trust in Science initiative is a novel public-private partnership model to build R&D capacity for development and discovery in science. The pillars of this initiative are: scientific excellence, trust and talent development in line with GSKs Science, Technology and Culture values. The initiative was launched in 2011 initially to engage with leading researchers and institutions in Latin America, focused on Brazil and Argentina. GSK Pharmaceutical Ltd. is pleased to expand this initiative beyond Latin America to other emerging markets such as India.UNI SJC AND

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GSK Pharmaceuticals (India) in collaboration with Regional Centre for Biotechnology completes one year of 'Trust in Science' academia-industry...

A rating of 77 puts ChemoCentryx Inc (CCXI) near the top of the Biotechnology industry according to InvestorsObserver. ChemoCentryx Inc's score of 77 means it scores higher than 77% of stocks in the industry. ChemoCentryx Inc also received an overall rating of 60, putting it above 60% of all stocks. Biotechnology is ranked 31 out of the 148 industries.

Searching for the best stocks to invest in can be difficult. There are thousands of options and it can be confusing on what actually constitutes a great value. Investors Observer allows you to choose from eight unique metrics to view the top industries and the best performing stocks in that industry. A score of 60 would rank higher than 60 percent of all stocks.

These rankings allows you to easily compare stocks and view what the strengths and weaknesses are of a given company. This lets you find the stocks with the best short and long term growth prospects in a matter of seconds. The combined score incorporates technical and fundamental analysis in order to give a comprehensive overview of a stocks performance. Investors who then want to focus on analysts rankings or valuations are able to see the separate scores for each section.

ChemoCentryx Inc (CCXI) stock is trading at $53.41 as of 2:38 PM on Monday, Sep 14, an increase of $5.34, or 11.11% from the previous closing price of $48.07. The stock has traded between $48.54 and $53.69 so far today. Volume today is light. So far 346,181 shares have traded compared to average volume of 475,545 shares.

Click Here to get the full Stock Score Report on ChemoCentryx Inc (CCXI) Stock.

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Is ChemoCentryx Inc (CCXI) The Right Choice in Biotechnology? - InvestorsObserver

Denali Therapeutics Inc (DNLI) is near the top in its industry group according to InvestorsObserver. DNLI gets an overall rating of 65. That means it scores higher than 65 percent of stocks. Denali Therapeutics Inc gets a 84 rank in the Biotechnology industry. Biotechnology is number 31 out of 148 industries.

Analyzing stocks can be hard. There are tons of numbers and ratios, and it can be hard to remember what they all mean and what counts as good for a given value. InvestorsObserver ranks stocks on eight different metrics. We percentile rank most of our scores to make it easy for investors to understand. A score of 65 means the stock is more attractive than 65 percent of stocks.

Our proprietary scoring system captures technical factors, fundamental analysis and the opinions of analysts on Wall Street. This makes InvestorsObservers overall rating a great way to get started, regardless of your investing style. Percentile-ranked scores are also easy to understand. A score of 100 is the top and a 0 is the bottom. Theres no need to try to remember what is good for a bunch of complicated ratios, just pay attention to which numbers are the highest.

Denali Therapeutics Inc (DNLI) stock is trading at $36.01 as of 3:36 PM on Monday, Sep 14, a rise of $4.02, or 12.57% from the previous closing price of $31.99. The stock has traded between $31.75 and $36.12 so far today. Volume today is high. So far 1,773,161 shares have traded compared to average volume of 842,601 shares.

Click Here to get the full Stock Score Report on Denali Therapeutics Inc (DNLI) Stock.

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Is Denali Therapeutics Inc (DNLI) a Winner in the Biotechnology Industry? - InvestorsObserver

The 63 rating InvestorsObserver gives to Turning Point Therapeutics Inc (TPTX) stock puts it near the top of the Biotechnology industry. In addition to scoring higher than 81 percent of stocks in the Biotechnology industry, TPTXs 63 overall rating means the stock scores better than 63 percent of all stocks.

Finding the best stocks can be tricky. It isnt easy to compare companies across industries. Even companies that have relatively similar businesses can be tricky to compare sometimes. InvestorsObservers tools allow a top-down approach that lets you pick a metric, find the top sector and industry and then find the top stocks in that sector.

These scores are not only easy to understand, but it is easy to compare stocks to each other. You can find the best stock in an industry, or look for the sector that has the highest average score. The overall score is a combination of technical and fundamental factors that serves as a good starting point when analyzing a stock. Traders and investors with different goals may have different goals and will want to consider other factors than just the headline number before making any investment decisions.

Turning Point Therapeutics Inc (TPTX) stock is trading at $80.68 as of 2:39 PM on Monday, Sep 14, an increase of $9.58, or 13.47% from the previous closing price of $71.10. The stock has traded between $72.52 and $80.68 so far today. Volume today is 269,852 compared to average volume of 295,953.

Click Here to get the full Stock Score Report on Turning Point Therapeutics Inc (TPTX) Stock.

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Is Turning Point Therapeutics Inc (TPTX) a Winner in the Biotechnology Industry? - InvestorsObserver