StemCell Therapy

Professor Glenn Cohen is a Professor of Law at Harvard Law School. He is also the director of Harvard Law School's Petrie-Flom Center for Health Law Policy, Biotechnology, and Bioethics, and one of the world's leading experts on the intersections of bioethics and the law. Cohen's current projects relate to big data, health information technologies, reproduction/ reproductive technology, research ethics, organ rationing in law and medicine, health policy, FDA law, translation medicine, and medical tourism. The utilization of CRISPR technology as a gene editing tool has spurred significant debate across the globe. In this interview, we gain insight of Cohen's perspectives on the "CRISPR revolution" and learn about the basic ethical issues surrounding the manipulation of the genome for enhancement.

Molly Campbell (MC): You are one of the leading experts on the intersection of bioethics and the law. Please can you tell us more about this field and the types of cases it addresses?Glenn Cohen (GC): Wherever law, medicine, and ethics intersect, thats where the field and I are. Whether it is the ethics of research, reproductive technologies, genetics, end of life decision-making, mental health, neuroscience, rationing, AI, clinical practice, etc. It is a robust and very exciting field.

MC: Currently, what restrictions apply to the use of CRISPR technology in different cell types and organisms? What applications are scientists not allowed to use CRISPR or other gene-editing technologies for? GC: In lay terms, in the United States an appropriations rider prohibits FDA from considering the use of germline gene editing in human beings. Thus, it is not possible to do a clinical trial or the like of this. Many (perhaps all, it is not clear everywhere) other countries across the world also prohibit in one way or another, but not all regulatory regimes may be as effective.

MC: The work of Jiankui He arguably startled the scientific community. In your opinion, do you think the publication of He's work prompted authorities to address regulating CRISPR technology? Or was there already a conversation taking place?GC: There was very robust conversation long before Dr. Hes terrible (and in my view completely unethical) experiments. For example, this report from the National Academies. While CRISPR is relatively new in terms of technology, in fact bioethicists have been talking about the basic issues surrounding manipulating the genome for enhancement for at least 40 years if not longer.

MC: There are concerns that the CRISPR tool could be used for enhancement purposes. In recent opinion article you say, "Anyone who has a position on enhancement has not thought deeply enough on the question." Please can you expand on what you mean by this?

GC: My claim is that enhancement is not a single monolithic thing, so it is hard to have a single position on it. Some enhancements would be wonderful and perhaps the state should subsidize them. Others would be terrible and perhaps the state should prohibit. Only when we think about it with some specificity can we know what we think the answer should be. In the article you mention I draw the following distinctions, for example, though others are possible:

1. Biological vs. Non-Biological Enhancement

a. Genetic enhancements vs. non-genetic biological enhancements

2. Choosing for Ourselves vs. Choosing for Others Who Cannot Choose for Themselvesa. Enhancing after birth vs. enhancing before birthi. Enhancing by selection vs. enhancing by manipulation of already fertilized embryos or implanted fetuses

3. Enhancements Compatible with Expanding Life Plans vs. Enhancements That Will Limit Options

4. Reversible vs. Irreversible Enhancement

5. Some would distinguish enhancement from treatment (though others are skeptical about this distinction)a. Enhancements to the upper bounds of what people already have vs. enhancements that add beyond human nature as it now stands

6. Enhancements for Absolute vs. Positional Goods

MC: A novel community of gene-editing "biohackers" has emerged in the rise of CRISPR technology. What are your opinions of biohackers conducting gene-editing experiments from their homes, from a legal and ethical perspective?GC:I think the community is very interesting. I am a huge fan of open science and the building of intellectual communities. I think the key question is whether/when the work undertaken by this community could pose significant externalities for others. Thats probably where I would start to get concerned.

MC: How do we approach implementing a global legal and ethical framework for using gene-editing technologies? What progress has been made thus far?GC: The WHO has chartered an advisory committee which has recommended a registry of all those doing gene editing work and has advised that it is irresponsible at this time for anyone to proceed with clinical applications of human germline genome editing." I think the existence of this committee (alongside the NASEM, Nuffield Council) and others working on these issues is a great step.

My own view is that we ought to be looking for a responsible translational pathway that might allow some clinical work to be reviewed and approved by regulators like the FDA in the future, but certainly there is nothing there yet. The international aspect makes this very, very difficult. Some have suggested we ought to go for an international treaty, like what we have on landmines and chemical weapons but also recognition of adoption, while others think this is infeasible.

MC: What challenges exist when looking to create laws surrounding a novel scientific technology?GC: There are quite a few. The first is uncertainty whenever you move to first-in-humans, whatever pre-clinical work you have done, there is always open questions. The same was true with IVF. The second is the politicization of science and the reduction of difficult and nuanced questions to talking points. The third is deep philosophical disagreement on some key points (for example, some take quite literally the idea of "man created in Gods image" and view altering the human genome as a rejection of that. If thats what someone believes for religious reasons then it is very hard to talk about these issues at a more policy level). Fourth, is the importance but difficult of public engagement. The UK in its public consultation on mitochondrial replacement therapy (that ultimately paved the way for permitting that technology to be used in a limited way) was a very good recent model, but quite difficult and expensive. Moreover, some felt it didnt go far enough in the direction of deliberative democracy. The hope is we will see more such initiatives for gene editing and other novel technologies.

Professor Glenn Cohen, Haravard Law School, was speaking with Molly Campbell, Science Writer, Technology Networks.

Catch up on the previous instalment of Technolology Networks Explores the CRISPR Revolution, an interview with Professor George Church, here.

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Technology Networks Explores the CRISPR Revolution: An Interview With Professor Glenn Cohen, World-leading Expert on Bioethics - Technology Networks

The University of Vermont Health Network has begun a pilot project to offer Genomic DNA Testing to patients as part of their clinical care. The pilot program is the beginning of an effort to increase the integration of genetic disease risks into routine medical care, which holds promise for providing Vermonters with valuable information to guide their health decisions.

"Our overall health and longevity are determined about 30 percent by genetics," said Debra Leonard, MD, PhD, Chair, Pathology and Laboratory Medicine. "But until now, most of our clinical health care decisions have been made without understanding the differences in each individual's DNA that could help guide those decisions."

Patients who choose to get the Genomic DNA Test can learn about differences in their DNA that make certain diseases more likely, such as cancer and heart disease. Knowing these genetically-determined disease risks may help patients and health care providers adjust their care to keep people as healthy as possible. While genetic testing to identify the cause of a patient's symptoms to reach a diagnosis is now common in health care, proactive genomic testing to identify health risks across a population is just beginning to be considered, and most projects are being done only in the research setting.

The UVM Health Network is partnering with Invitae and LunaPBC on the pilot project. Invitae will provide information for 147 genes that are well-established indicators of increased risk for certain diseases for which clinical treatment guidelines are established. The test also screens for carrier status for other diseases. Follow-up testing for family members will be provided when appropriate.

"Nearly 1 in 6 healthy individuals exhibits a genetic variant for which instituting or altering medical management is warranted," said Robert Nussbaum, MD, Chief Medical Officer of Invitae. "Genetic screening like the Genomic DNA Test in a population health setting can help identify these risk factors so clinicians can better align disease management and prevention strategies for each patient."

The UVM Health Network is offering the Genomic DNA Test as part of clinical care, but health and genomic data can also help researchers learn more about health and disease. Patients who get the test can consent to securely share their data with researchers through LunaDNA, partner LunaPBC's sharing platform. LunaDNA provides patients with the opportunity to share their genomic and electronic health record information to advance health and disease management research. In the future, patients will also be able to share lifestyle, environment, and nutrition data.Shared data is de-identified and aggregatedduring studiesto protect the privacy of each patient while being used to answer important medical research questions.

"Vermonters who choose to share their genomic data for research will play a leading role in the advancement of precision medicine," said Dawn Barry, LunaPBC President and Co-founder. "This effort puts patients first to create a virtuous cycle for research that doesn't sacrifice patients' control or privacy.We are proud to bring our values as a public benefit corporation and community-owned platform to this partnership."

Dr. Leonard spoke about the project, the UVM Health Network's partnership with LunaPBC and Invitae, and the role of genomics in population health on Monday at the Santa Fe Foundation's Clinical Lab 2.0 Workshop in Chicago, a national conference at which pathologists and healthcare leaders from across the country share ways that pathology can be integral to improving population health.

"Vermont and other states are moving away from 'fee-for-service' health care and toward a system that emphasizes prevention, keeping people healthy and treating illness at its earliest stages," Dr. Leonard said. "Integrating genetic risks into clinical care will help patients and providers in their decision-making."

The pilot project began on Friday, November 1, when the first patient agreed to have the test. During the pilot stage of the project over the next year, the Genomic DNA Test will be offered to approximately 1,000 patients over the next year who: are at least 18 years old; receive their primary care from a participating UVM Health Network Family Medicine provider; are not currently pregnant or the partner of someone who is currently pregnant; and are part of the OneCare Vermont Accountable Care Organization (ACO), a care coordination and quality improvement organization.

Patients do not have to pay for the test or for discussions with the UVM Health Network's Genomic Medicine Resource Center's genetic counselors before and after testing. The test uses a small amount of blood, and focuses on the parts of a patient's DNA that most affect health and health care. Results will go into each patient's medical record, protected like all medical information, and available to the patient and all of their health care providers.

"Much work has gone into getting ready to start this project and it has taken an entire team," Dr. Leonard said. "Providers from Family Medicine, Cardiology, the Familial Cancer Program, Medical Genetics and Pathology, patient and family advisors, ethics and regulatory compliance leaders, Planning, Finance and OneCare Vermont have all worked together to get us across the start line for this initiative."

Patients should be aware that the UVM Health Network will never call them on the phone to ask them to get this test. Testing is arranged through a patient's primary health care provider and only if the patient agrees to have the test.

THE FUTUREIt's understanding technology that gets us an edge to find the "next Apple," or the "next Amazon."

This is what CML Pro does. We are members of Thomson First Call -- our research sits side by side with Goldman Sachs, Morgan Stanley and the rest, but we are the anti-institution and break the information asymmetry.

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Thanks for reading, friends. The author is long shares of Invitae at the time of this writing.

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The University of Vermont Initiates Genomic DNA Testing in Partnership With Genomics Leader Invitae (NYSE:NVTA) as Cigna Joins Invitae's Covered Lives...

These Five Life Science Organizations are Striving to Cure HIV

For those that lived through the devastation and horror of the HIV/AIDS epidemic of the early 1980s, effective treatment, let alone a cure for Human Immunodeficiency Virus (HIV), seemed unimaginable.

Some three decades later, a host of Maryland life science companies and research organizations are getting closer to making what was once unthinkable, real.

So little was known about this devastating immune disorder in the early phases of the HIV/AIDS epidemic.

In the early days of the HIV/AIDS crisis, the BioHealth Capital Region was the epicenter of HIV/AIDS research, with much of this groundbreaking research occurring within the lab of the now famed NIH researcher, Dr. Robert Gallo. In 1983 and 1984 Gallo and his collaborators co-discovered and confirmed that the virus responsible for the killer disease known as AIDS was human T lymphotropic virus type III (HTLV-III). Gallo and the company went on to develop the first test that identified the virus in humansthe HIV-antibody blood test.

By 1983 the disease had started to spread globally. By 1999, approximately 33 million people across the globe were living with HIV and an estimated 14,000,000 million people had died from AIDS since the epidemic began.

The 1995 approval of Highly Active Antiretroviral Treatment (HAART), which was the result of the remarkable, collaborative efforts of the scientific community, led to the reduction of AIDS-related deaths and hospitalizations by 60-80%. A short time later what was once a three-drug cocktail had been transformed into a pill taken once daily by HIV sufferers.

As of 2017, 19.5 million people are estimated to be receiving antiretroviral treatment globally. While one of the greatest achievements in medical history, HAART and subsequent treatment forms do not cure HIV. Within just weeks of stopping treatment, the virus returns to full strength and chronic inflammation caused by suppressed HIV can lead to adverse health effects over the long term. Current HIV treatments control it but do not cure it; in fact, research shows that those being treated for HIV are more susceptible to other diseases and health risks at an earlier age.

Despite the amazing advancements in HIV/AIDS treatments, HIV/AIDS continues to be a major global health threat.

It would be a fitting conclusion for an HIV cure to emerge from the state where the virus was first linked to AIDS and where the first human diagnostic was developed.

Multiple Maryland companies and research institutions are on the leading-age of HIV research and development, making the state a hotbed of potential next-generation HIV therapies and, possibly, the source of a cure for this devastating global health issue. Some of the most promising cure candidates are coming out of Marylands thriving cell and gene therapy cluster.

Lets take a look at some of the amazing progress thats happening right now across Maryland and take a deeper dive into five of the leading organizations that are on a mission to develop the first HIV cure.

AGT is a gene and cell therapy company with a proprietary gene-delivery platform to rapidly develop gene and cell therapies to cure infectious diseases, cancers and monogenic disorders.

One of its lead gene therapy products is a potential functional cure for HIV. AGT just announced that it has submitted the IND to the FDA for a Phase I trial of its autologous cell therapy for HIV.

While HIV has become a manageable chronic virus for many, in less developed countries HIV/AIDS is still a devastating illness. Developing an HIV cure would relieve millions from the side effects of antiretrovirals used to suppress HIV and prevent AIDS, avoid the serious quality-of-life issues of long-term treatment, and potentially save the lives of countless others.

AGT is currently developing a highly innovative HIV treatment strategy that uses the tools of genetic medicine for immunotherapy to potentially create a functional cure for HIV.

If we are successful, patients will be able to throw away their medication, will not progress to AIDS, and will be immune to future HIV exposures.

The potential single-dose treatment would be delivered as a genetically-modified cell product made from a patients own cells. AGTs strategy is unique because it focuses on the key immune cells responsible for catalyzing strong immunity against a virus. AGTs treatment strategy seeks to protect these cells; one of the first cell subsets to be disabled by HIV. This subset of cells is understood to be critical to building an immune response to any virus. If achieved, the cells natural process of immunity is restored and any future rise of HIV in the body will be attacked by an individuals own immune system.

AGTs treatment includes the production of an autologous cell product that is highly enriched for HIV-specific CD4+ T cells that are then transduced with a lentivirus vector known as AGT103 to protect against HIV-mediated T cell depletion. The combination of these enriched cells and the lentiviral vector forms a cell product AGT has dubbed AGT103-T. This cell product is delivered intravenously to HIV patients. AGT103-T should control viremia and work to remove infected cells from the body, thus eliminating the need for lifelong antiretroviral treatment.

AGT is currently collaborating with the Institute of Human Virology, University of Maryland Baltimore to collect leukapheresis specimens from HIV positive individuals for an ongoing observational study performing and qualifying the cell process, which is explained in greater detail here. The company expects its potential HIV cure to move into clinical trials in the next six months.

IHV is part of the University of Maryland School of Medicine and is a recognized leader in the virology field. IHV was founded by Dr. Robert C. Gallo who co-discovered HIV and developed the first HIV blood test.

IHV is heavily focused on HIV/AIDS research and the organization is currently progressing a promising HIV/AIDS vaccine through its pipeline. IHV01 (FLSC-001) has completed a Phase I trial and was supported, in part, from funding provided by the Bill and Melinda Gates Foundation.

This potential HIV/AIDS treatment seeks to neutralize the different strains of HIV found across the globe from the moment of infection. IHVs HIV/AIDS research is focused on the CCR5 chemokine receptor that plays a crucial role in HIV-1 infection and as such offers an important potential therapeutic target. (IHV Website). IHV is striving to develop biological HIV/AIDS treatments that are less expensive, have fewer adverse impacts and are more accessible to patients around the globe.

Lentigen is a leading provider of custom lentiviral vectors used in cell and gene therapy research and development. For HIV, Lentigen is at the forefront of efforts to use Chimeric Antigen Receptors (CAR) T-Cell therapy to improve the treatment of HIV and possibly cure it.

Lentigen, along with researchers at the University of Pittsburgh in Pennsylvania and the Albert Einstein School of Medicine, has been conducting a promising study of the use of CAR T in the treatment of HIV. The researchers developed duoCAR T cells that were able to kill white blood cells infected with a range of HIV variants. Testing in mice also produced promising results. Mice with humanized immune systems were simultaneously injected with CAR T cell and HIV-infected human cells into their spleens. When the spleens were examined a week later, five of the six mice had no identifiable HIV DNA and their viral levels had decreased by 97.5% (source: Science).

The study hopes to test the duoCAR T approach in HIV-infected people in the near future.

IBBR is a joint research enterprise of the University of Maryland, College Park and the National Institute of Standards and Technology (NIST). Last year IBBR received $3.9M from the National Institutes of Health (NIH) to develop a multi-specific, single-agent antibody therapeutic against HIV-1 to block virus infection and to clear the reservoir of HIV-infected cells from the body, according to an IBBR press release from November 2018.

The project is led by Dr. Yuxing Li, Associate Professor, Department of Microbiology and Immunology, University of Maryland School of Medicine, and Fellow at the Institute for Bioscience and Biotechnology Research (IBBR), in collaboration with Dr. Qingsheng Li, University of Nebraska-Lincoln, and Dr. Keith Reeves, Harvard Medical School/Beth Israel Deaconess Medical Center.

IBBRs research has focused on overcoming some of the limitations of existing antiretroviral (ARV) HIV treatments, including adverse side effects, ARV treatment drug resistance and how HIV integrates into the human genome, creating pockets of HIV that ARV cannot eliminate. Dr. Li and his group have produced bi and tri-specific antibodies that demonstrated neutralization of 95% of circulating HIV-1 viruses. These bi and tri-specific antibodies can also bind to multiple locations on the HIV-1 surface glycoprotein Env, which could potentially thwart treatment resistance via mutation. The team is now working to optimize their multivalent antibody constructs to recognize Env proteins on the surface of latently infected host cells, and to signal other immune system components to destroy those cells that contain the hard-to-reach viral pockets, or as the team calls them, a viral reservoir. (IBBR press release)

NIAID has been at the forefront of HIV research for decades and continues to be a major player in the research and development of possible HIV treatments and potential cures. NIAIDs research into HIV played a critical role in developing ARV drugs that transformed HIV into a chronic condition rather than a fatal infection.

NIAID-supported research has led to many ARV drug improvements, including reducing the number of pills required, diminishing adverse impacts and identifying the best drug combinations. The organization works with many leading global HIV/AIDS research organizations to identify and develop better HIV treatments.

NIAID is focused on both developing new HIV treatments as well as supporting other researchers and research organizations investigating new therapies. The ultimate goal is to potentially make HIV treatment a single dose, lifetime treatment, and, eventually, the complete eradication of HIV. NIAID is involved in many research and development projects focused on HIV and there are too many to dig into in a single article. Some of their current HIV research and development efforts are focused on investigational long-acting HIV drugs, rilpivirine LA and cabotegravir LA, for patients that have had difficulty following conventional antiretroviral therapy programs. Another NIAID study will test combining monthly injections of cabotegravir LA and infusions of an NIAID-discovered broadly neutralizing antibody called VRC01LS to see if the combination can keep HIV suppressed in people whose infection was previously controlled by antiretroviral therapy.

The organizations support has also helped in the discovery of the experimental drug islatravir (also known as EFdA or MK-8591) and maturation inhibitors. NIAID also has partnered with the Maryland industry, including a research collaboration agreement with AGT for research studies on the companys cell and gene therapy for HIV/AIDS.

A partnership between NIAID, Frederick National Labs for Cancer Research (operated by Leidos Biomedical Research, Inc.) and a team of collaborators recently developed 38 new simian/human immunodeficiency viruses (SHIVs) for prevention and treatment studies. These new SHIVs have closed a gap that previously existed in HIV research. These SHIVs are pathogens engineered in the lab that can help in the investigation of potential new HIV therapies as well as other treatments and vaccines.

These SHIVs target HIV subtype C, which causes approximately half of all HIV infections, and were created using HIV samples from people recently infected, allowing better modeling of more current forms of HIV subtype C circulating globally. The stronger modeling will increase pre-clinical researchs ability to predict effectiveness. Other SHIVs had used samples acquired from patients that had been infected long before the sample was pulled, limiting the SHIVs effectiveness against more current strains of HIV. While improvements are still needed, including challenges with replication, these new tools for HIV research and discovery hold tremendous promise.

In the late 1970s and early 1980s finding a cure for HIV/AIDS wasnt even on the radar. The scientific community was racing to understand the fundamentals of a virus that was rapidly spreading devastation and death across the globe. The speed with which the medical community came to understand the disease and to develop treatments like HAART is one of the truly amazing stories of the 20th century.

One or several of these Maryland companies and research institutions have a real chance to achieve what was once unthinkablefinding a cure for HIV that could help tens of millions of people across the globe live better, healthier and longer lives.

If an HIV cure emerges from Maryland, the BHCR community will have helped write the final chapter of HIV/AIDS terrible yet hopeful story.

Steve has over 20 years experience in copywriting, developing brand messaging and creating marketing strategies across a wide range of industries, including the biopharmaceutical, senior living, commercial real estate, IT and renewable energy sectors, among others. He is currently the Principal/Owner of StoryCore, a Frederick, Maryland-based content creation and execution consultancy focused on telling the unique stories of Maryland organizations.

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Heres Why the First Cure for HIV Could Emerge from Maryland - BioBuzz

CAMBRIDGE, Mass., Nov. 05, 2019 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that senior management will present at the Credit Suisse 28th Annual Healthcare Conference on Tuesday, Nov. 12, 2019 at 11:10 a.m. E.T. / 9:10 a.m. M.T. The fireside chat will be held at the at The Phoenician in Scottsdale, Ariz.

The presentation will be webcast live under the investor relations section of Sareptas website at http://www.sarepta.com and will be archived there following the presentation for 90 days. Please connect to Sarepta's website several minutes prior to the start of the broadcast to ensure adequate time for any software download that may be necessary.

AboutSarepta TherapeuticsSarepta is at the forefront of precision genetic medicine, having built an impressive and competitive position in Duchenne muscular dystrophy (DMD) and more recently in gene therapies for Limb-girdle muscular dystrophy diseases (LGMD), MPS IIIA, Pompe and other CNS-related disorders, totaling over 20 therapies in various stages of development. The Companys programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. Sarepta is fueled by an audacious but important mission: to profoundly improve and extend the lives of patients with rare genetic-based diseases. For more information, please visit http://www.sarepta.com.

Internet Posting of InformationWe routinely post information that may be important to investors in the 'For Investors' section of our website atwww.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us.

Source: Sarepta Therapeutics, Inc.

Sarepta Therapeutics, Inc.Investors:Ian Estepan, 617-274-4052iestepan@sarepta.com

Media:Tracy Sorrentino, 617-301-8566tsorrentino@sarepta.com

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Sarepta Therapeutics to Present at the Credit Suisse 28th Annual Healthcare Conference - GlobeNewswire

GW also welcomes the positive guideline recommendation for Sativex (nabiximols) for the treatment of spasticity due to multiple sclerosis as part of NICEs evaluation of cannabis-based medicinal products (CBMPs)

Cannabidiol oral solution and nabiximols are the first and only plant-derived cannabis-based medicines to be routinely reimbursed on the NHS

LONDON and CARLSBAD, Calif., Nov. 11, 2019 (GLOBE NEWSWIRE) -- GW Pharmaceuticals plc (Nasdaq: GWPH) (GW, the Company or the Group), world leader in discovering, developing and commercialising cannabinoid prescription medicines, today announces that two of its medicines, EPIDYOLEX (cannabidiol) oral solution and Sativex (nabiximols), have been recommended by the UKs National Institute for Health and Care Excellence (NICE) to receive routine reimbursement from NHS England.

This represents the first-time any plant-derived cannabis-based medicine has been recommended by NICE for use on the NHS. Cannabidiol oral solution is recommended as an adjunctive therapy for seizures associated with Lennox Gastaut syndrome (LGS) or Dravet syndrome, in conjunction with clobazam, for patients two years of age and older. Nabiximols, reviewed as part of NICEs evaluation of cannabis-based medicinal products (CBMPs), has been considered cost effective for the treatment of spasticity due to multiple sclerosis.

This is a momentous occasion for UK patients and families who have waited for so many years for rigorously tested, evidenced and regulatory approved cannabis-based medicines to be reimbursed by the NHS, said Chris Tovey, GWs Chief Operating Officer. This is proof that cannabis-based medicines can successfully go through extensive randomised placebo-controlled trials and a rigorous NICE evaluation process to reach patients. I am hugely proud of the entire GW team for achieving this milestone in the country where the company was founded and where both of these medicines were developed and are manufactured.

Commenting on the NICE recommendation for cannabidiol oral solution, Dr Rhys Thomas, Consultant Neurologist at the Royal Victoria Hospital in Newcastle, said: This is a significant moment for adults and children with the most difficult to treat epilepsies. NICEs recommendation of cannabidiol oral solution follows a period of great anticipation and enthusiasm for patients and their clinicians. The European Medicines Agency (EMA) licence and availability through the NHS is welcome as we badly need additional effective treatments for Dravet and Lennox Gastaut syndromes.

We welcome the addition of cannabidiol oral solution as a new medicine to add to the Dravet syndrome treatment armamentarium. Dravet syndrome is a devastating condition and having a new treatment option offers potential new hope to patients and their families searching for better seizure control, said Galia Wilson, Chair, Dravet Syndrome UK. Many families come to us asking about the potential of cannabis-based medicines, particularly cannabidiol (CBD), and we are thrilled that one is now available on the NHS.

When added to other anti-epileptic therapies, GWs cannabidiol oral solution significantly reduced the frequency of seizures in patients with LGS and Dravet syndrome.The most common adverse reactions that occurred in patients treated with the medicine were somnolence, decreased appetite, diarrhoea, pyrexia, fatigue and vomiting.GWs development programme represents the only well-controlled clinical evaluation of a cannabinoid medication for patients with refractory epilepsy.

GWs cannabidiol oral solution was approved by the EMA and received marketing authorisation in September 2019 under the trade name EPIDYOLEX as an adjunctive therapy for seizures associated with LGS or Dravet syndrome, in conjunction with clobazam, for patients two years of age and older. Following this approval, GW has been working with the relevant bodies in the UK, Germany, Spain, France and Italy to secure reimbursement ahead of the anticipated launch of the medicine in these countries.

The inclusion of nabiximols in NICE guidelines comes as part of the comprehensive evaluation of the clinical and cost-effectiveness of CBMPs. Nabiximols has been approved by medicines regulators in more than 25 countries around the world. Nabiximols was approved in the UK by the Medicines and Healthcare products Regulatory Agency (MHRA) in 2010 and is marketed in the UK by GWs commercial partner, Bayer.

About GW Pharmaceuticals plc and Greenwich Biosciences, Inc.Founded in 1998, GW is a biopharmaceutical company focused on discovering, developing and commercialising novel therapeutics from its proprietary cannabinoid product platform in a broad range of disease areas. GWs lead product, EPIDIOLEX (cannabidiol oral solution) is commercialised in the US by its U.S. subsidiary Greenwich Biosciences for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS) or Dravet syndrome in patients two years of age or older. This product has received approval in Europe under the tradename EPIDYOLEX. The Company continues to evaluate EPIDIOLEX in additional rare conditions including Tuberous Sclerosis Complex (TSC) and Rett syndrome. GW commercialised the worlds first plant-derived cannabinoid prescription medicine, Sativex (nabiximols), which is approved for the treatment of spasticity due to multiple sclerosis in numerous countries outside the United States and for which the Company is now advancing a late stage programme in order to seek FDA approval. The Company has a deep pipeline of additional cannabinoid product candidates which includes compounds in Phase 1 and 2 trials for epilepsy, autism, glioblastoma, and schizophrenia. For further information, please visit http://www.gwpharm.com.

About EPIDIOLEX/EPIDYOLEX (cannabidiol) oral solutionEPIDIOLEX/EPIDYOLEX (cannabidiol), the first prescription, plant-derived cannabis-based medicine approved by the FDA for use in the U.S., is an oral solution which contains highly purified cannabidiol (CBD). The medicine is for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS) or Dravet syndrome in patients two years of age or older and is the first in a new class of anti-epileptic medications with a novel mechanism of action. EPIDYOLEX received a positive opinion from the European Medicines Agencys (EMA) Committee for Medicinal Products for Human Use (CHMP) in July 2019 and the European Commission (EC) granted the marketing authorisation on 23 September 2019 for adjunctive use in conjunction with clobazam. The medicine was granted an Orphan Drug Designation from the EMA for the treatment of seizures associated with LGS, Dravet syndrome, and Tuberous Sclerosis Complex (TSC).

About Sativex (nabiximols)Sativex (nabiximols), the first cannabinoid medicine derived from the cannabis plant, is an oromucosal spray which contains a complex mixture of cannabinoids, including delta-9-tetrahydrocannabinol (THC) and cannabidiol (CBD) and specific minor cannabinoids and other non-cannabinoid components. Nabiximols is approved in over 25 countries around the world for the treatment of spasticity due to multiple sclerosis (MS) in people who have not responded adequately to other anti-spasticity medication and who demonstrate clinically significant improvement in spasticity related symptoms during an initial trial of therapy. Nabiximols is currently licensed to Almirall in Europe (excluding the UK), to Bayer in the UK and Canada, Neopharm in Israel, IDS Medical in UAE, Al-Mojil in Kuwait, Ipsen in Latin America (excluding Mexico and Islands of Caribbean), and Emerge Healthcare in New Zealand and Australia.

About Dravet syndrome Dravet syndrome is a severe infantile-onset and highly treatment-resistant epileptic encephalopathy frequently associated with genetic mutations in the sodium channel gene SCN1A. Onset of Dravet syndrome typically occurs during the first year of life in previously healthy and developmentally normal infants. Initial seizures are often body temperature related, severe, and long-lasting. Over time, patients with Dravet syndrome often develop multiple types of seizures, including tonic-clonic, myoclonic and atypical absences and are prone to bouts of prolonged seizures including status epilepticus, which can be life threatening. Risk of premature death including SUDEP (sudden unexpected death in epilepsy) is elevated in patients with Dravet syndrome. Additionally, the majority of patients will develop moderate to severe intellectual and development disabilities and require lifelong supervision and care.

About Lennox-Gastaut syndrome (LGS) The onset of LGS typically occurs between the ages of 3 to 5 years and can be caused by a number of conditions, including brain malformations, severe head injuries, central nervous system infections and genetic neuro-degenerative or metabolic conditions. In up to 30 percent of patients, no cause can be found. Patients with LGS commonly have multiple seizure types including drop and convulsive seizures, which frequently lead to falls and injuries, and non-convulsive seizures. Resistance to anti-epileptic drugs (AEDs) is common in patients with LGS. Most patients with LGS experience some degree of intellectual impairment, as well as developmental delays and aberrant behaviours.

About Multiple Sclerosis (MS)Multiple sclerosis (MS) is a chronic neurological condition characterized by progressive and disabling loss of motor and sensory nervous system functions. In Europe, the prevalence rate of MS is estimated to be 83 per 100,000 and is most commonly diagnosed between the ages of 20 and 40, although it can affect younger and older people too. In the UK, it affects around 100,000 people. Spasticity related to MS is an involuntary increase in muscle tone affecting more than 80% of MS patients across their disease evolution, and being moderate or severe in one third of them despite physiotherapy and first line drug treatments. The burden of spasticity grows as the MS evolves. When the muscle is moved externally, there is more resistance to this movement than there normally would be and the muscle feels stiff or rigid. Increased muscle tone also means that muscles are slow to relax, and this causes stiffness. Spasticity, beyond the continuous stiffness, may also cause muscles to jerk suddenly in an uncontrolled way.

Forward-looking statementsThis news release contains forward-looking statements that reflect GW's current expectations regarding future events, including statements regarding financial performance, the timing of clinical trials, the timing and outcomes of regulatory or intellectual property decisions, the relevance of GW products commercially available and in development, the clinical benefits of EPIDIOLEX/EPIDYOLEX (cannabidiol) oral solution and Sativex (nabiximols), and the safety profile and commercial potential of both medicines. Forward-looking statements involve risks and uncertainties. Actual events could differ materially from those projected herein and depend on a number of factors, including (inter alia), the success of GWs research strategies, the applicability of the discoveries made therein, the successful and timely completion and uncertainties related to the regulatory process, and the acceptance of EPIDIOLEX/EPIDYOLEX, Sativex and other products by consumer and medical professionals. A further list and description of risks and uncertainties associated with an investment in GW can be found in GWs filings with the U.S. Securities and Exchange Commission. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. GW undertakes no obligation to update or revise the information contained in this press release, whether as a result of new information, future events or circumstances or otherwise.

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GW Pharmaceuticals receives positive NICE recommendation for EPIDYOLEX (cannabidiol) oral solution for the treatment of seizures in patients with two...

BOTHELL Nov 11, 2019 (Thomson StreetEvents) -- Edited Transcript of Sarepta Therapeutics Inc earnings conference call or presentation Thursday, November 7, 2019 at 9:30:00pm GMT

* Alexander G. Cumbo

Sarepta Therapeutics, Inc. - Executive VP & Chief Commercial Officer

* Douglas S. Ingram

Sarepta Therapeutics, Inc. - President, CEO & Director

Sarepta Therapeutics, Inc. - Executive VP of R&D and Chief Medical Officer

* Ian M. Estepan

Sarepta Therapeutics, Inc. - Senior VP of Corporate Affairs & Chief of Staff

Sarepta Therapeutics, Inc. - SVP of Gene Therapy

Sarepta Therapeutics, Inc. - Executive VP, CFO & Chief Business Officer

Robert W. Baird & Co. Incorporated, Research Division - Senior Research Analyst

* Christopher N. Marai

Nomura Securities Co. Ltd., Research Division - MD & Senior Analyst of Biotechnology

* Debjit D. Chattopadhyay

H.C. Wainwright & Co, LLC, Research Division - MD of Equity Research & Senior Healthcare Analyst

BTIG, LLC, Research Division - MD and Specialty Pharmaceutical & Biotechnology Research Analyst

Janney Montgomery Scott LLC, Research Division - Equity Research Analyst & Director of Biotechnology Research

Good day, ladies and gentlemen, and welcome to the Sarepta Therapeutics Third Quarter 2019 Earnings Call. (Operator Instructions) As a reminder, today's call is being recorded.

And now I'd like to introduce your host for today's program, Ian Estepan, Senior Vice President, Chief of Staff and Corporate Affairs.

Ian M. Estepan, Sarepta Therapeutics, Inc. - Senior VP of Corporate Affairs & Chief of Staff [2]

Thank you, Michelle, and thank you all for joining today's call. Earlier today, we released our financial results for the third quarter of 2019. The press release is available on our website at http://www.sarepta.com, and our 10-Q was filed with the SEC earlier this afternoon. Joining us on the call today are Doug Ingram, Sandy Mahatme; Bo Cumbo, Dr. Gilmore O'Neill; and Dr. Rodino-Klapac. After our formal remarks, we'll open up the call for questions.

I'd like to note that during this call, we'll be making a number of forward-looking statements. Please take a moment to review our slide on the webcast which contains our forward-looking statements. These forward-looking statements involve risks and uncertainties, many of which are beyond Sarepta's control. Actual results could materially differ from these forward-looking statements, and any such risks can materially and adversely affect the business, the results of operations and the trading prices of Sarepta's common stock.

For a detailed description of applicable risks and uncertainties, we encourage you to review the company's most recent quarterly report on Form 10-Q filed with the Securities and Exchange Commission as well as the company's other SEC filings. The company does not undertake any obligation to publicly update its forward-looking statements, including any financial projections provided today, based on subsequent events or circumstances.

And with that, let me turn the call over to our CEO, Doug Ingram, who will provide an overview on our recent progress. Doug?

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Douglas S. Ingram, Sarepta Therapeutics, Inc. - President, CEO & Director [3]

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Thank you, Ian. Good afternoon and evening, and thank you all for joining us for Sarepta Therapeutics Third Quarter 2019 Conference Call.

Our ambitious strategy involving one of the deepest multi-platform genetic medicine pipelines in biotech has required focused execution over the course of 2019. To remind you, we have more than 25 active programs across our RNA and gene therapy platforms, and we're either actively in or in late-stage planning for some 9 human clinical trials to advance our plans. I am pleased to say that over the course of 2019 and in the third quarter specifically, we have made very significant strides in advancing our programs and our strategic vision, and I'm excited to discuss those advancements. However, while doing so, I must also acknowledge what we all know that we had a setback in the third quarter. And rather than burying it among or after a discussion of our successes, I will begin by commenting on our CRL disappointment that occurred in August.

Having worked diligently on our submission for VYONDYS 53, the generic name of that is golodirsen, for well over a year and based on all of our interactions with the Division of Neurology Products, we were very confident that we would obtain an approval on our PDUFA date, which was August 19. Instead, as you know, we were surprised to have received a complete response letter, also known as a CRL, signed by the Office of Drug Evaluation I. Our disappointment extends beyond Sarepta to the 8% of exon 53 amenable DMD patients in the United States who degenerate every day while they await access to this therapy.

When I joined Sarepta, I made some commitments externally and to the Division of Neurology, that we intended to build a positive relationship with the Division of Neurology, one founded on transparency and on solid evidence-based science. And consistent with that commitment, we will work with the agency to address the reasons for the CRL and determine a pathway for a potential approval if one is possible.

I've heard from those who would prefer that I speak more often and more publicly on this issue and/or that I would attempt to engage the patient community or others to assist, for instance, in applying external pressure to bring this therapy along faster. I have no intention of doing either of those things. If we can win the day with this therapy and with this issue, we will have done so on the science and on the regulations and in collaborative evidence-based discussions with our reviewers at the FDA.

Now I've also heard some speculation about the implications of the CRL. So let me take a moment to address these as well. First, the VYONDYS CRL does have implications for our submission for our next PMO, casimersen. As they are closely related, we will await clarity on the VYONDYS matter before we submit for casimersen in the United States. But let me disabuse anyone who might have concerns for our other programs. The CRL does not have any read-through to our micro-dystrophin gene therapy program. The CRL involved 2 safety signals in connection with an application for an accelerated approval. Our micro-dystrophin program is overseen by a different part of the FDA, CBER, and we are not seeking accelerated approval there. There is simply no overlap in either substance or personnel.

Secondly, to those who may believe that the CRL suggests some sort of bias on behalf of the Division of Neurology towards Sarepta, I would unequivocally and emphatically disagree. Let me reiterate that I remain convinced that we were treated very fairly and professionally by the Division of Neurology. Also, I'm very proud of the Sarepta team and how they comported themselves during this review. From my perspective, we have gone a long way in the last 2.5 years in forging a positive evidence-based working relationship with the division. We will work diligently to address the VYONDYS CRL. But with that, I do not intend to provide a prediction on outcome or on timing or to provide interim views during the process. However, I will provide an update to the patient, physician and investment communities once we have definitive clarity on the outcome of those discussions.

Now moving to our positive achievements in the quarter. We have made some enormous amount of progress in this third quarter. EXONDYS continues to perform well with third quarter sales above consensus at $99 million. That is a 26% increase over same quarter last year. Commenting for a moment on our confirmatory trial for EXONDYS, to remind you, this trial comprises 3 arms: one with EXONDYS at 100 mg per kg and another at 200 mg per kg versus our current dose at 30 mg per kg. The trial design, which was an FDA requirement, will answer whether higher doses of EXONDYS provide even more benefit than the currently approved dose. Now since the comparator arms involve higher doses than the currently approved dose, we were required to begin our confirmatory trial with a healthy human volunteer study. We have completed this trial, and based on the results, we have initiated the main confirmatory trial. We will begin dosing this quarter.

Staying on our RNA franchise. We have moved to our multi-ascending dose trial for our next-generation RNA platform, the PPMO, and we are dosing trial participants now. We will have safety and dosing insight in 2020. If our PPMO shows encouraging results, in addition to SRP-5051, that's the construct that we're currently in a multi-ascending dose regarding, we have 5 additional constructs that have already been built, which in total have the potential to treat as much as 43% of the DMD community. We are also conducting research now on new therapeutic targets that could be served by our PPMO platform.

Moving next to our gene therapy platform. As you know, we are spending enormous resource and energy to build out our vision of an enduring gene therapy engine. Between our research and clinical-stage programs, we have more than 14 therapeutic candidates advancing through research and development. We have made great progress thus far this year and quarter, led by our most advanced program, SRP-9001, for DMD, which, at least to my knowledge, is the highest-potential late-stage gene therapy program currently in biotech. As you should be aware, our double-blind, placebo-controlled SRP-9001 micro-dystrophin trial, the trial that we call Study 2, was fully dosed by midyear, but we took advantage of the availability of additional study material and previously announced that we had increased the study n from 24 patients to 40 patients, significantly increasing the study power and confidence in this study. In addition to our initial site with Dr. Jerry Mendell at Nationwide Children's Hospital, we have added a second site at UCLA with Dr. Perry Shieh. And I'm very proud to be associated with that clinician and investigator. Both sites are actively dosing patients, and we remain on target to complete our dosing by year-end.

Micro-dystrophin manufacturing is progressing well. From a capacity perspective, Brammer has now completed the buildout of our single-use micro-dystrophin manufacturing facility in Lexington, Massachusetts. We also have dedicated suites with Paragon in Maryland with actually substantially greater capacity than our dedicated Lexington facility, which means we have robustly secured capacity well in advance of launch.

Our analytical development work proceeds well, and we continue to make progress on process development and yield optimization. Given our recent capacity, analytical development and process development progress, we remain on track to commence our next trial, Study 301, with commercial development supply by mid-2020. Now Study 2 is being conducted with clinical material from Nationwide Children's Hospital. Study 301 will be a multicenter, multi-country, placebo-controlled trial using commercial process material from our hybrid manufacturing model with Brammer and Paragon. The main study will include DMD patients ages 4 to 7, but we are also planning a separate study for older and non-ambulatory patients as well.

Commenting on a few of our other gene therapy programs. Following exceptional expression and biomarker results in our first 3-patient cohort dosed with our construct for limb-girdle 2E, in October, we announced positive 9-month functional results in that same cohort. Consistent with robust expression of the native beta-sarcoglycan protein, that is the cause of the disease, all patients improved on every functional endpoint by the 9-month time point. Consistent with the protocol, we will treat an additional 3-patient cohort with a higher dose, and then in early 2020, we will decide on the dose for what we hope to be the pivotal trial. These results will help inform dosing not only of our 2E program but also on the other limb-girdle programs in our pipeline. We will also meet with the FDA in the near term to discuss the development pathway for our limb-girdle programs. And informed by this and further work on manufacturing, we will provide an update on the clinical pathway and the timing for our limb-girdle portfolio in 2020.

Next, led by our partner Lysogene, the AAVance gene therapy study for MPS IIIA, also known as Sanfilippo Syndrome Type A, is proceeding well with 13 patients having been dosed to date. MPS IIIA is a rare autosomal recessive lysosomal storage disease that primarily affects the brain and the spinal cord, causing severe cognitive decline, motor disease, behavioral decline and unfortunately death at a young age. AAVance is a single-arm trial evaluating the safety and efficacy of an rh10-mediated gene therapy to deliver the missing SGSH gene with the goal of robustly expressing the missing enzyme in the brain that is the cause of MPS IIIA.

Moving to Charcot-Marie-Tooth, or CMT. Dr. Zarife Sahenk of Nationwide Children's Hospital intends to commence dosing of the proof-of-concept study for CMT 1A subject only to obtaining final release of trial material for that study. CMT is the largest inherited neuromuscular disease in the world. And CMT 1A, a devastating peripheral nerve disease, is also the most prevalent form of CMT. Dr. Sahenk's gene therapy is an AAV 1-mediated construct to deliver the neurotrophic factor-3, NT-3. In animal models, NT-3 has been shown to promote nerve regeneration, improved motor function, histopathology and electrophysiology of peripheral nerves. And in early proof-of-principle studies, NT-3 has shown markers of clinical benefits in patients with CMT 1A when administered subcutaneously.

In summary, we have made great progress in the third quarter and over the course of 2019 toward our ambitions, advancing our RNA and gene therapy platforms, advancing our many development programs, building out our gene therapy manufacturing capacity and building out our tower. As with any ambitious strategy, our progress this quarter was met with an obstacle in the form of the VYONDYS CRL. The breadth of our ambition inevitably comes with challenges and obstacles to address and to overcome. But to those who might at times feel discouraged or disheartened by the need to overcome the occasional barrier, we should keep top of mind what we are doing with all of this. If we are successful in our mission, we will not merely be among the most significant gene therapy and genetic medicine biotechnology companies in existence, but we will have, more importantly, extended, improved and saved the lives of countless patients who would otherwise have been left hopeless.

And with that, I will turn the call over to Sandy to provide an update on the financials. Sandy?

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Sandesh Mahatme, Sarepta Therapeutics, Inc. - Executive VP, CFO & Chief Business Officer [4]

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Thanks, Doug. Good afternoon, everyone. Let me start by saying that we had another strong quarter both in terms of financial performance and in progress towards the pipeline and manufacturing capabilities. With a current top line run-rate of approximately $400 million and a cash balance over $1 billion, we are in a strong position to continue to accelerate our strategic imperatives and invest in the growth of Sarepta. Net product revenue for the third quarter of 2019 was $99 million compared to $78.5 million for the same period of 2018. The increase primarily reflects higher demand for EXONDYS 51.

On a GAAP basis, the company reported a net loss of $126.3 million and $76.4 million or approximately $1.70 and $1.15 per share for the third quarter of 2019 and 2018, respectively. We reported a non-GAAP net loss of $84.4 million or $1.14 per share compared to non-GAAP net loss of $37.1 million or $0.56 per share in the third quarter of 2018.

In the third quarter of 2019, we recorded approximately $13 million in cost of sales compared to $8.7 million in the same period of 2018. The increase was primarily driven by inventory costs related to higher demand for EXONDYS 51 during the third quarter of 2019 as well as accrued royalty payments to BioMarin and the University of Western Australia.

On a GAAP basis, we recorded $133.9 million and $86.6 million of R&D expenses for the third quarters of 2019 and 2018, respectively, which is a year-over-year increase of $47.3 million. R&D expenses were $110.5 million for the third quarter of 2019 compared to $64.2 million for the same period of 2018, an increase of $46.3 million. The year-over-year growth in non-GAAP R&D expense was driven primarily due to continuing ramp-up of our micro-dystrophin program, our ESSENCE program and initiation of certain post-marketing studies for EXONDYS 51.

Turning to SG&A. On a GAAP basis, we recorded $75.4 million and $53 million of expenses for the third quarters of 2019 and '18, respectively, a year-over-year increase of $22.4 million. On a non-GAAP basis, the SG&A expenses were $59.6 million for the third quarter of 2019 compared to $42.5 million for the same period of 2018, an increase of $17.1 million. The year-over-year increase was primarily driven by significant organizational growth and continued expansion to support a commercial launch -- to support our commercial launch plans globally and almost 30 therapies in various stages of development across several therapeutic modalities.

On a GAAP basis, we recorded $2.5 million in other expenses for the third quarter of 2019 compared to $7 million for the same period of 2018. The favorable change is primarily driven by the payoff of certain debt instruments during the third quarter of 2018 as well as a higher return on investments over the third quarter of 2019.

We had approximately $1.1 billion in cash, cash equivalents and investments as of September 30, 2019.

With that, I'd like to turn the call over to Bo for a commercial update. Bo?

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Alexander G. Cumbo, Sarepta Therapeutics, Inc. - Executive VP & Chief Commercial Officer [5]

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Thank you, Sandy. Good afternoon, everyone. To begin, we are pleased with the continued strong performance of EXONDYS 51 in the third quarter. Total revenues reached $99 million. We were also pleased to be in a position to increase our 2019 revenue guidance range from $365 million to $375 million to a range of $370 million to $380 million for EXONDYS 51. Sales have increased quarter-over-quarter for over 3 years now, and we continue to see consistent demand for EXONDYS 51 as we speak today.

Compliance and adherence have remained high and stable since launch and to-date continue to remain steady. It should be noted that in the past 2 years, we've experienced ordering volatility at the end of the year and suspect that we could see a change in ordering patterns with both Christmas and New Year's falling in the middle of the week. Internally, we are assuming the pattern from previous years could be more extreme this year due to both holidays falling midweek. With that said, we feel comfortable with the guidance provided.

The success we achieved this year reflects the impact EXONDYS 51 continues to have on patient lives. We remain the leading voice with KOLs and payers across the world in support of Duchenne patients and are recognized as the leader in RNA and gene therapies within the Duchenne field. Our strategy to advance the very best science, build awareness and appreciation for Duchenne and pave new pathways so Duchenne patients gain access to therapy have resulted in the successful trajectory of EXONDYS 51 since its approval just over 3 years ago and will play a role for future therapies.

As for golodirsen, if approved, we will be ready to launch, leveraging our knowledge and experience to facilitate rapid access to individuals amenable to exon 53. Our work is focused on delivering, and grounding us in all we do is the patient. That journey begins with identifying patients in our core therapeutic areas: Duchenne, the limb-girdle muscular dystrophy and MPS IIIA. Patient identification will be central to the commercial organization for the balance of 2019 and leading into 2020 and beyond. The genetic testing program, Decode Duchenne, which we started with PPMD many years ago, consistently identifies patients. We are also in the process of building genetic testing programs for our other disease states we are working on as well. We believe patient identification will always be one of our primary commercial goals, and we will continue to place resources on these programs.

Another important goal will be gene therapy site readiness. We are already working on global site readiness for our DMD micro-dystrophin program and working with many of the Zolgensma and Spinraza sites treating SMA. Based on the very strong results Novartis demonstrated with their recent launch of Zolgensma and understanding the label and the differences in patient population sizes between the 2 disease states, we believe having a strong network of sites ready and trained to handle gene therapies will be critical. We will continue to focus on this as we move through worldwide development and, if successful, commercialization.

We also believe it is critical to focus on access and reimbursement as early as possible. We're already speaking to and educating large to midsized insurance plans as well as CMS and Medicaid providers on the differences between chronic therapies and onetime gene therapies and the importance of quickly gaining access to these therapies for diseases like Duchenne. We have built constructive relationships with payers over time and look forward to continuing to work with them to support broad access.

In the limb-girdle muscular dystrophy, we are focused on disease education and identifying patients. The limb-girdle muscular dystrophies are a family of diseases, over 30 subtypes in all. Therefore, patient identification is of critical importance. Our plan is to leverage our knowledge and experience to ensure that we're able to serve these communities as we have in Duchenne. We've already attended limb-girdle muscular dystrophy conferences, held educational symposiums at major neuromuscular conferences, held advisory boards to understand how physicians identify and treat patients and already have a digital presence within the community. All of this will help us prepare for the potential to support multiple launches in the years to come.

Sarepta's prospects to transform the lives of patients with rare diseases is unparalleled in the industry. We have the largest neuromuscular RNA and gene therapy pipeline in the industry, and we understand the responsibility that comes with such an important mission.

With that, I will turn the call back to Doug for closing remarks.

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Douglas S. Ingram, Sarepta Therapeutics, Inc. - President, CEO & Director [6]

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Thank you, Bo. So looking forward, we have a number of significant milestones to achieve over the rest of 2019 and through 2020. First, we intend to complete dosing of our SRP-9001 Study 2, that's our micro-dystrophin study, by year-end with functional readout 48 weeks thereafter. We soon intend to launch process development for SRP-9001, not manufacturing for purposes of conducting our next clinical trial, gain insight from the agency on CMC and on our trial itself and then to commence Study 301 by mid-2020. We intend to dose an additional high-dose cohort for limb-girdle 2E and then make a dose selection. We intend to gain regulatory and manufacturing insight and to present an update on the development pathway and time line for our entire limb-girdle program in 2020. Dr. Sahenk intends to commence a proof-of-concept study for CMT gene therapy, NT-3. And we intend to obtain safety and dosing insight for our PPMO program in the first half of 2020. So we obviously have a lot to do but a lot of milestones as well over the coming months and quarters.

Thank you all for joining us tonight, and I'll open up the line for questions now.

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Questions and Answers

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Operator [1]

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(Operator Instructions) Our first question comes from Alethia Young of Cantor Fitzgerald.

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Alethia Rene Young, Cantor Fitzgerald & Co., Research Division - Head of Healthcare Research [2]

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Congrats on all the progress over the quarter. This may be a simple one, but I was just curious to get your perspective around Zolgensma partial hold. And like should we -- is there any -- are there any reads to potentially make thinking about other gene therapy programs?

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Douglas S. Ingram, Sarepta Therapeutics, Inc. - President, CEO & Director [3]

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Thank you for that question, Alethia. Okay. So well, first, let me say this. Let's make sure we're all on the same page. For those of you maybe unaware, I expect everyone is aware, Novartis recently announced that their clinical trial for their AAV9-mediated SMA gene therapy for intrathecal administration was placed on a partial clinical hold due to neurotoxicity that was seen in animal models. So first, understand this, we do not have a unique insight into the Zolgensma clinical hold itself or the Zolgensma program. Certainly, one should look at Novartis to gain accurate insight on that program and those issues.

So with that said, I should tell you, we see no read-through to our program, and there's a host of reasons for that. First, understand that we are dosing peripherally with IV administration. We're not dosing intrathecally as was the issue, as announced by Novartis, regarding that partial clinical hold. And second of all, understand that we're not using AAV9. Dr. Louise Rodino-Klapac who is with us tonight and Dr. Jerry Mendell chose rh74 for a number of specific attributes. One of the significant ones was that rh74, unlike AAV9 as an example, does not promiscuously cross the blood-brain barrier. And unlike SMA where that would be of value, there is absolutely no value to these micro-dystrophin constructs in the CNS at all. They have promoters that wouldn't turn on in the CNS, so there would be no value there. So this seems to have been a very wise choice.

And also note this, that we have an enormous amount of preclinical and animal model evidence with respect to rh74. And even at doses that are multiples higher than we're using in our clinical trial, we have never seen evidence of neurotoxicity as relates to AAVrh74.

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Operator [4]

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Our next question comes from Whitney Ijem of Guggenheim.

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Whitney Glad Ijem, Guggenheim Securities, LLC, Research Division - Senior Analyst of Biotechnology [5]

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Congrats on all the progress. I'll ask a question on the original 4 micro-dystrophin patients. Curious if we'll get an update on them in 2020 either in an update from you or possibly a publication from Dr. Mendell.

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Douglas S. Ingram, Sarepta Therapeutics, Inc. - President, CEO & Director [6]

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Yes. Thanks for that question. Thank you for your comments. So yes, Dr. Mendell has always had a keen interest in publishing the 1-year data on the 4 patients, and he is working on the manuscript even as we speak. So I feel very confident that we'll have a publication in 2020 on the first 4 patients.

Read this article:
Edited Transcript of SRPT earnings conference call or presentation 7-Nov-19 9:30pm GMT - Yahoo Finance

SEATTLE--(BUSINESS WIRE)--Shape Therapeutics, Inc. (ShapeTx), a development-stage biotechnology company leading the field of RNA-editing gene therapy, announces $35.5M in Series A financing, led by New Enterprise Associates (NEA), with additional participation from CureDuchenne Ventures. The new capital will enable the company to extend its growing portfolio of intellectual property, recruit and hire top scientific talent and advance its groundbreaking RNA and protein targeting platforms focused on curing human diseases.

These platforms include the proprietary ShapeTx RNAfix technology that enables direct in vivo targeting and modification of RNA by leveraging proteins such as Adenosine Deaminases Acting on RNA (ADARs), suppressor tRNAs, and engineered adeno-associated viruses (AAVs). The RNAfix platform differentiates from other contemporary genome engineering technologies by engaging natural human cellular machinery to modify RNA.

ShapeTx was founded on the work of Dr. Prashant Mali, Assistant Professor of Bioengineering at UCSD, who during his postdoctoral fellowship in the George Church laboratory at Harvard Medical School pioneered the use of CRISPR in human cells. ShapeTx RNAfix platform is built upon his lab's most recent work demonstrating in vivo use of guide RNAs to recruit native ADARs and to fix mutations in multiple rare genetic disease mouse models.

Our technology can correct mutations or target specific genes in neurodegenerative, oncology, metabolic and rare genetic disorders by hijacking naturally occurring proteins such as ADARs present in our cells using just a short guide RNA. Our proprietary new platform avoids the risk of in vivo immunogenicity and permanent off-target damages commonly associated with CRISPR-based approaches, explained Francois Vigneault, Ph.D., President and CEO, who was previously VP of Research at Juno Therapeutics after a successful co-acquisition of AbVitro, Inc. by Juno and Celgene.

Ed Mathers, Partner at NEA and Board member at ShapeTx, said, One rarely comes across a proprietary technology platform with such transformative potential led by a focused and data-driven scientific group with a successful track record in pre-clinical and clinical development. The team has shown us an exciting demonstration of the technology in multiple in vivo models, alongside one of the strongest IP estates we have seen in the field. NEA looks forward to backing the company in future rounds as they move the technology toward the clinic.

While the ShapeTx platform will be enabling for many other genetic diseases, Dr. Malis in vivo proof of concept in Duchenne Muscular Dystrophy was quite exciting and could potentially lead to a cure for families suffering from such a debilitating disorder, said Debra Miller, CEO and Founder of CureDuchenne and CureDuchenne Ventures.

The ShapeTx Series A financing coincides with the formation of a world-class Scientific Advisory Board comprised of foremost global experts in genomics, bioengineering, and gene editing, including George Church Ph.D., James Collins Ph.D., and Don Cleveland Ph.D. The scientific advisory board will serve as strategic advisors and ensure that the research and development of its platforms meet the highest standards of scientific merit.

Prashant and Francois are some of the most innovative and brilliant individuals that have come through my lab over the years, and it will be impressive to see these two disrupt the field of gene therapy with this paradigm-shifting technology, said Dr. George Church, Professor in Genetics at Harvard Medical School and member of the ShapeTx Scientific Advisory board.

Shape Therapeutics Scientific Advisory Board Members:

George Church, Ph.D.

George Church Ph.D., world-famous geneticist, molecular engineer, and chemist. He developed the methods used for the first genome sequence & million-fold cost reductions since, as well as pioneered many of the CRISPR advances in genome editing. He is currently a Professor of Genetics at Harvard Medical School and Professor of Health Sciences and Technology at Harvard and the Massachusetts Institute of Technology (MIT). He is Director of the U.S. Department of Energy Technology Center and Director of the National Institutes of Health Center of Excellence in Genomic Science. He has received numerous awards, including the 2011 Bower Award and Prize for Achievement in Science from the Franklin Institute and election to the National Academy of Sciences and Engineering.

James Collins, Ph.D.

James Collins Ph.D., is one of the pioneers of the field of synthetic biology and has made multiple synthetic biology and bioengineering breakthroughs in biotechnology and biomedicine. He serves as the Termeer Professor of Medical Engineering & Science and Professor of Biological Engineering at MIT, as well as a member of the Harvard-MIT Health Sciences & Technology Faculty, and core member of the Wyss Institute. His many awards include a Rhodes Scholarship, a MacArthur Genius Award, a National Institutes of Health Directors Pioneer Award. Jim is also an elected member of the National Academy of Sciences, the National Academy of Engineering, the National Academy of Medicine, the American Academy of Arts & Sciences, as well as a charter fellow of the National Academy of Inventors.

Don Cleveland Ph.D.

Don Cleveland Ph.D. is an award-winning inventor and pioneer in the field of Antisense Oligonucleotide (ASO) and their uses in gene therapy. He was recently awarded the Breakthrough Prize in Life Sciences for his work on the pathogenesis of disease and ASO-mediated treatment approaches in ALS and Huntingtons disease. Don is currently Professor of Medicine and Department Chair of Cellular and Molecular Medicine and Neurosciences at the University of California at San Diego, and Head, Laboratory for Cell Biology at the San Diego branch of Ludwig Cancer Research. He has made pioneering discoveries on the mechanisms of chromosome movement and cell-cycle control during normal cellular division, as well as the principles of neuronal cell development and the relationship to defects that contribute to inherited neurodegenerative disease.

About Shape Therapeutics, Inc.

Shape Therapeutics, Inc. is creating the worlds leading RNA and protein targeting platforms focused on the cure of human diseases. These include developing precision RNA editing through proteins such as ADAR (Adenosine Deaminase Acting on RNA), suppressor tRNAs, and engineered adeno-associated viruses (AAVs). The RNAfix technology allows for the editing of RNA using natural human cellular machinery, limiting the risk associated with immunogenicity, cellular toxicity, or off-target DNA editing. The teams founders include Prashant Mali, Ph.D., Francois Vigneault, Ph.D., and John Suliman. ShapeTx is headquartered in Seattle, Washington, with a satellite site opening in Cambridge, Massachusetts. For additional information, visit http://www.ShapeTx.com.

About NEA

New Enterprise Associates, Inc. (NEA) is a global venture capital firm focused on helping entrepreneurs build transformational businesses across multiple stages, sectors, and geographies. With more than $20 billion in cumulative committed capital since the firm's founding in 1978, NEA invests in technology and healthcare companies at all stages in a company's lifecycle, from seed stage through IPO. The firm's long track record of successful investing includes more than 225 portfolio company IPOs and more than 375 acquisitions. For additional information, visit http://www.nea.com.

About CureDuchenne Ventures

CureDuchenne Ventures supports Duchenne research by using philanthropic donations to encourage the development of new Duchenne drugs. Through an impact financing model, we can provide equity or royalty financing to biotech and pharmaceutical companies. CureDuchennes portfolio includes 16 wide-ranging projects with several successful exits. Investments from CureDuchenne Ventures have successfully de-risked and leveraged more than $2.3 billion in follow-on financing from venture capital, biotech, and pharmaceutical companies to fund emerging projects to find treatments for Duchenne. For additional information, visit https://www.cureduchenne.org/ventures/.

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Shape Therapeutics, Inc. Raises $35.5M Series A Financing, Led by NEA and Announces the Formation of a World-Class Scientific Advisory Board, to...

Have you ever wondered why you wash your rice or soak it overnight before cooking it? Perhaps you wash your rice grains to enhance taste, reduce starch levels, or maybe that's just the way your family has always prepped rice. Thanks to a tip from science communicator Samantha Yammine who came across Dr. Nausheen Sadiq's neat finding while live-tweeting a forum on Diversity and Excellence in Science it turns out there is another reason why, as washing rice actually helps reduce the concentration of heavy metals, like chromium, cadmium, arsenic, and lead.

Heavy metal contamination in crops can be caused by human activities, such as mining, fertilizers, pesticides, and sewage sludge. Compared to most cereal crops though, rice (Oryza sativa L.) actually accumulates more heavy materials, like cadmium or arsenic, where long-term heavy metal intake can cause health risks. For example, long-term arsenic exposure leads to skin disease, high blood pressure, and neurological effects. This is especially important to consider as rice is a staple food across the globe.

Heavy metal contamination in crops can be caused by human activities, such as mining, fertilizers, pesticides, and sewage sludge.

Photo by TUAN ANH TRAN on Unsplash

In a recent study, researchers investigated the effects of different cooking methods (normal, high-pressure and microwave cooking) on the concentration, bio-accessibility and health risks posed by three heavy metals (cadmium, arsenic and lead) in two strains of brown rice. After cooking 100 grams of brown rice grains, researchers evaluated bioaccessibility (i.e. how much of the heavy metal is released for absorption) by mixing rice samples with simulated gastric fluid, and then used spectrometery to measure heavy metal concentration. Lastly, the researchers calculated the health risk posed by the heavy metals by calculating values such as the average daily dose.

Overall, the researchers found that instead of the three different cooking methods, it was the washing process which significantly reduced concentrations of cadmium, arsenic and lead, suggesting that the reduction may be due to rice morphology. For example, lead is found largely in the outer compartments of rice kernels, so lead is more likely to be removed during rice washing.

In contrast, the three cooking methods did impact bioaccessibility i.e. how much of the heavy metal would be released for absorption by the body. Here, washing and soaking isn't enough as rice absorbs water poorly at 25C. This finding was also reflected in calculated values: the average daily doses of cadmium, arsenic and lead were lower in washed and cooked rice, compared to raw rice.

It's worth noting that the European Commission has enforced limits on heavy metal levels - for example, arsenic is currently limited to 200 parts per billion (ppb) for adults and 100 ppb for infants. Both the U.S. and Canada currently have no limits in place for arsenic in food though Canada is currently reviewing a proposal to add maximum levels for arsenic found in white and brown rice, while the U.S. FDA has previously released a (non-binding) risk assessment, suggesting the same 100 ppb levels as Europe.

So the takeaway here is that yes, your family and all those professional chefs have been right all along. Yes, washing rice involves sacrificing some of its nutritional value, but doing so means you can reduce the levels of heavy metals present in grains, and still enjoy dishes like rice cakes. And returning back to Yammine's reporting, Saudiq actually shared that by soaking and washing rice for ~5 mins, you can get rid of 50-100% of these elements. (Thanks Sam!)

Link:
Reddit's r/science community is one of science writing's biggest outlets, with the stats to prove it - Massive Science

The late Gary Middleton, founder of Little Rock design, marketing and print-solutions company Magna IV, was one of the first chairmen for the Arkansas Arthritis Foundation's Jingle Bell Run/Walk for Arthritis.

As a result, Magna IV has remained a strong supporter of the annual fundraiser, and so has Steven Schilling, the company's director of sales.

Schilling, a longtime Arthritis Foundation board member, has taken over as the Jingle Bell Run chairman for the fourth year in a row. That follows four years as the event's volunteer coordinator.

As chairman, his job, besides the actual raising of the money, is to find sponsors, vendors and volunteers and watch over the logistics, which, considering the scope of the event, can be formidable. It incorporates 10K and 5K runs, a one-mile walk and an Elf Run for kids.

"I've been with Magna IV for 26 years," Schilling says. "The owner was involved, so that's how I got involved."

This year's run is on Dec. 7, starting and ending at the Clinton Presidential Center, 1200 President Clinton Ave., Little Rock. The course takes runners and walkers to the Broadway Bridge, which they traverse into North Little Rock, returning south of the river over the Junction Bridge.

Arthritis Foundation chapters stage an annual Jingle Bell Run in more than 100 cities nationwide. The foundation bills it as the longest-running, holiday-theme 5K race in the United States and "the original festive race for charity, bringing people from all walks of life together to champion arthritis research and resources."

Register at jbr.org/LittleRock. Participants are encouraged to wear a seasonal costume and tie jingle bells to their shoelaces. "Every runner gets jingle bells, so as the race progresses, there's a lot of jingling going on," Schilling says.

They're also encouraged to sign up in teams, although individual runners and walkers are welcome. Last year's event drew 800, most of them forming about 50 teams, Schilling says. He's hoping to have at least that many taking part this year, and to raise as much or more than last year's $75,000 take.

The money comes from sponsorships and registration fees, which range from $40 in advance, $45 day of race, for the timed 10K (which includes T-shirt, timing chip, gear check and bells) to $30 for what the foundation calls "Jingle in Your Jammies" ("Can't attend the event, but still want to be part of the fun? Choose this option to receive a shirt and [raise funds] for a cure!") -- and, Schilling adds "You get to stay in bed."

In addition to T-shirts, participants get goody bags (L'Oreal is a national sponsor, so they usually include makeup and lipsticks) and a small amount of free food, primarily, at least in years past, sliced fruit. Ben E. Keith is supplying water.

The event requires 30-40 volunteers for setup, post-race tear-down and keeping things running in between. Schilling says about two-thirds of those come each year from the Central High ROTC.

"They're required to have community service hours," he explains, and because their mission is military, "they're on time and eager to work." During the race they're out on the course, operating water stations and "making sure runners are where they're supposed to be."

Things kick off around 9:15 a.m., as participants register and pick up packets and timing chips, the Elf Village (kids zone) and vendor booths open, choral groups sing Christmas carols and attendees can take pictures with Santa. The Kids Run begins at 10:15, followed by the 10K at 10:30, the 5K at 11 and the Joe Cook 1 Mile Memorial Walk, named for the chairman who was Schilling's predecessor, at 11:10. An awards ceremony is slated for 11:40.

There are also costume contests for humans -- and pets.

"Some people show up that have nothing to do with the run," Schilling says. "They just want to enter their pet in the contest." It doesn't draw too many unusual pets -- mostly dogs and cats, although Schilling says if you have, say, a monitor lizard, it's welcome. "But you get some crazy costumes," he says. For example: "We've had some very small dogs dressed as very big reindeer."

Humans often show up in very ugly Christmas sweaters. "I've seen some pretty horrendous sweaters," he adds.

It's all part of what he describes as the event's family-friendly, laid-back approach and fun vibe.

More than 54 million Americans live with arthritis, including 673,000 in Arkansas, according to foundation statistics. Schilling says he's one of those.

"The Jingle Bell Run is a 28-year tradition in Little Rock and known nationally as the original festive race for charity," says Angela Harris, the state chapter's executive director.

"Our honorees and volunteers are what make this event successful and memorable every year, and this year we're humbled to honor Sherry Little, who is a true Arthritis Warrior and continually commits her time to raising awareness and funds for our cause year after year." Little has walked more than 15 years in honor of her daughter, Michelle, and to "spread awareness [that] arthritis can strike at any age."

Lindee and Lola Throckmorton are this year's young honorees; other laureates include Brian Barnett of the University of Arkansas for Medical Sciences Spine Institute, corporate chair; Dr. Joel Smith, medical honoree; Martin Orthopedics; and presenting sponsor BKD.

Schilling's volunteer credentials include four years as a coach for Resurrecting Baseball in the Inner City, based at Little Rock's Lamar Porter Field, aimed at giving at-risk teens something to do in the summers. He also volunteered for more than 20 years as a coach for his three kids' youth soccer, baseball, basketball and football teams, but that's over -- his youngest is turning 21.

He stepped away from the foundation for a while but returned to the board eight years ago.

"The race was so important to Mr. Middleton, and out of my relationship to and respect for him, it became important to me. And my employer, Magna IV, has been very supportive of my involvement."

Photo by Eric E. HarrisonSteven Schilling credits his relationship with and respect for Gary Middleton, founder of Magna IV and a strong supporter of the Arkansas Arthritis Foundation and its Jingle Bell Run, for his involvement with the foundation.

High Profile on 11/10/2019

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Magna IV true to tradition in jingling run for arthritis - Arkansas Online

Graph 1

Vectra Predicts Risk of Radiographic Progression in 1 Year

Myriad Genetics, Inc.

Graph 2

Vectra Predicts Risk of Cardiovascular Events in Patients with RA

Myriad Genetics, Inc.

SALT LAKE CITY, Nov. 09, 2019 (GLOBE NEWSWIRE) -- Myriad Genetics, Inc. (NASDAQ: MYGN), a global leader in precision medicine, announced that its Myriad Autoimmune business unit will present new data on the Vectra test at the 2019 ACR/ARP Annual Meeting being held Nov. 8-13, 2019 in Atlanta, GA. The key findings are that the Vectra test predicts the risk of radiographic progression (RP) within one year, and the Vectra score, in combination with other clinical measures, predicts the risk of a cardiovascular (CV) event in people with rheumatoid arthritis (RA).

A hallmark feature ofrheumatoid arthritisisinflammation, which increases the risk of joint damage, cardiovascular disease and other comorbidities, said Elena Hitraya, M.D., Ph.D., rheumatologist and chief medical officer at Myriad Autoimmune. The data being presented by our academic collaborators at ACR show that the Vectra test accurately measures inflammation and can help predict patients risk of adverse health outcomes, enabling clinicians to tailor precision treatment plans to achieve better outcomes.

Vectra Posters

Title:Predicting Risk of Radiographic Progression for Patients with Rheumatoid Arthritis.Presenter:Jeff Curtis, M.D., M.S., MPH, University of Alabama at Birmingham.Date:Sunday, Nov. 10, 2019. 9:00-11:00 a.m.Location:Poster 466.

This study evaluated the ability of the Vectra test to predict patients individual percentage risk of RP within one year. The analysis included combined data from 973 patients in four cohorts. The results demonstrate that the adjusted Vectra score was a superior predictor of RP within one year compared to DAS28-CRP, CRP, CDAI and swollen joint count. Additionally, the risk of permanent joint damage increased continuously with the adjusted Vectra score, meaning patients with a low adjusted Vectra score had a one to three percent risk of RP in one year, while patients with a moderate-to-high score had between seven and 47 percent risk (Graph 1). Based on these new data, the company is working to enhance the Vectra test report to provide patients with their individual risk of radiographic progression in one year.

To view Graph 1: Vectra Predicts Risk of Radiographic Progression in 1 Year,please visit the following link:https://www.globenewswire.com/NewsRoom/AttachmentNg/514919cd-81ca-4084-81df-682fedc1784b

Too often people with RA are over- or under-treated because it is difficult for clinicians to accurately measure inflammation and determine the long-term prognosis of RA patients. As a result, some people are at increased risk of rapid radiographic progression, said Jeff Curtis, M.D., M.S., MPH, lead investigator, rheumatologist and Professor of medicine in the Division of Clinical Immunology and Rheumatology at the University of Alabama at Birmingham. It is critical that clinicians have reliable information when making treatment decisions. Our study demonstrated that the Vectra score was the strongest predictor of radiographic progression, which may help inform treatment plans and prevent future joint damage.

Title:Derivation and Validation of a Biomarker-Based Cardiovascular Risk Prediction Score in Rheumatoid Arthritis.Presenter:Jeff Curtis, M.D., M.S., MPH; University of Alabama at Birmingham.Date:Tuesday, Nov. 12, 2019. 9:00-11:00 a.m.Location:Poster 2350.

This study evaluated 30,751 Medicare patients with RA to develop and validate the Vectra CVD score, which predicts risk for a first cardiovascular (CV) event by combining data from Vectra and clinical measures. The primary CV outcome was a composite of three types of CV events heart attack, stroke, and CV death occurring within 3 years from testing. When the performance of the Vectra CVD score was compared to four other CV prediction models, the Vectra CVD score was a significant predictor of CV risk and was superior to all four other models. Importantly, when risk scores were converted to 3-year percentage risk for having a CV event, approximately 80 percent of patients were found to have a moderate or high risk of a CV event over 3 years, based on risk categories analogous to those of the American College of Cardiology/American Heart Association 2018 guidelines (Graph 2).

To view Graph 2: Vectra Predicts Risk of Cardiovascular Events in Patients with RA, please visit the following link:https://www.globenewswire.com/NewsRoom/AttachmentNg/c902b4ec-a3c8-439f-9557-0a9b05631a1f

People with rheumatoid arthritis have almost double the risk of heart attack, stroke and atherosclerosis. Traditional CV risk factors alone do not fully explain the increased rates of CV events in RA, and inflammation is a missing component that is measured by the Vectra test, said Dr. Curtis. In this study, the Vectra CVD score effectively predicted CV risk in people with RA. We believe the Vectra CVD score may assist clinicians to more quickly identify patients at high risk for CV events and target interventions that can be potentially life-saving.

The company plans to publish these new data in peer reviewed medical journal and make the Vectra CVD score available to clinicians in fiscal year 2020. Please visit Myriad Autoimmune at booth #1419 to learn more about Vectra. Follow Myriad on Twitter via @myriadgenetics and follow meeting news by using the hashtag #ACR19.

About VectraVectra is a multi-biomarker molecular blood test that provides an objective and personalized measure of inflammatory disease activity in patients with rheumatoid arthritis. Vectra provides unsurpassed ability to predict radiographic progression and can help guide medical management decisions with the goal of improving patient outcomes. Vectra testing is performed at a state-of-the-art CLIA (Clinical Laboratory Improvement Amendments) facility. Test results are reported to the physician five to seven days from shipping of the specimen. Physicians can receive test results by fax or the private web portal, VectraView. For more information on Vectra, please visit: http://www.vectrascore.com.

About Myriad GeneticsMyriad Genetics Inc. is a leading precision medicine company dedicated to being a trusted advisor transforming patient lives worldwide with pioneering molecular diagnostics. Myriad discovers and commercializes molecular diagnostic tests that: determine the risk of developing disease, accurately diagnose disease, assess the risk of disease progression, and guide treatment decisions across six major medical specialties where molecular diagnostics can significantly improve patient care and lower healthcare costs. Myriad is focused on five critical success factors: building upon a solid hereditary cancer foundation, growing new product volume, expanding reimbursement coverage for new products, increasing RNA kit revenue internationally and improving profitability with Elevate 2020. For more information on how Myriad is making a difference, please visit the Company's website: http://www.myriad.com.

Myriad, the Myriad logo, BART, BRACAnalysis, Colaris, Colaris AP, myPath, myRisk, Myriad myRisk, myRisk Hereditary Cancer, myChoice, myPlan, BRACAnalysis CDx, Tumor BRACAnalysis CDx, myChoice HRD, EndoPredict, Vectra, GeneSight, riskScore, Prolaris, Foresight and Prequel are trademarks or registered trademarks of Myriad Genetics, Inc. or its wholly owned subsidiaries in the United States and foreign countries. MYGN-F, MYGN-G.

Safe Harbor StatementThis press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements relating to the Company presenting new data on the Vectra test at the 2019 ACR Annual Meeting; the Vectra test enabling clinicians to tailor precision treatment plans to achieve better outcomes; the Vectra score helping inform treatment plans and prevent future joint damage; the Vectra CVD score assisting clinicians to more quickly identify patients at high risk for CV events and target interventions that can be potentially life-saving; publishing these new data in peer reviewed medical journal; making the Vectra CVD score available to clinicians in fiscal year 2020; and the Company's strategic directives under the caption "About Myriad Genetics." These "forward-looking statements" are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by forward-looking statements. These risks and uncertainties include, but are not limited to: the risk that sales and profit margins of our molecular diagnostic tests and pharmaceutical and clinical services may decline; risks related to our ability to transition from our existing product portfolio to our new tests, including unexpected costs and delays; risks related to decisions or changes in governmental or private insurers reimbursement levels for our tests or our ability to obtain reimbursement for our new tests at comparable levels to our existing tests; risks related to increased competition and the development of new competing tests and services; the risk that we may be unable to develop or achieve commercial success for additional molecular diagnostic tests and pharmaceutical and clinical services in a timely manner, or at all; the risk that we may not successfully develop new markets for our molecular diagnostic tests and pharmaceutical and clinical services, including our ability to successfully generate revenue outside the United States; the risk that licenses to the technology underlying our molecular diagnostic tests and pharmaceutical and clinical services and any future tests and services are terminated or cannot be maintained on satisfactory terms; risks related to delays or other problems with operating our laboratory testing facilities and our healthcare clinic; risks related to public concern over genetic testing in general or our tests in particular; risks related to regulatory requirements or enforcement in the United States and foreign countries and changes in the structure of the healthcare system or healthcare payment systems; risks related to our ability to obtain new corporate collaborations or licenses and acquire new technologies or businesses on satisfactory terms, if at all; risks related to our ability to successfully integrate and derive benefits from any technologies or businesses that we license or acquire; risks related to our projections about our business, results of operations and financial condition; risks related to the potential market opportunity for our products and services; the risk that we or our licensors may be unable to protect or that third parties will infringe the proprietary technologies underlying our tests; the risk of patent-infringement claims or challenges to the validity of our patents or other intellectual property; risks related to changes in intellectual property laws covering our molecular diagnostic tests and pharmaceutical and clinical services and patents or enforcement in the United States and foreign countries, such as the Supreme Court decision in the lawsuit brought against us by the Association for Molecular Pathology et al; risks of new, changing and competitive technologies and regulations in the United States and internationally; the risk that we may be unable to comply with financial operating covenants under our credit or lending agreements; the risk that we will be unable to pay, when due, amounts due under our credit or lending agreements; and other factors discussed under the heading "Risk Factors" contained in Item 1A of our most recent Annual Report on Form 10-K for the fiscal year ended June 30, 2019, which has been filed with the Securities and Exchange Commission, as well as any updates to those risk factors filed from time to time in our Quarterly Reports on Form 10-Q or Current Reports on Form 8-K. All information in this press release is as of the date of the release, and Myriad undertakes no duty to update this information unless required by law.

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New Studies Demonstrate the Predictive Value of the Vectra Test in People Diagnosed with Rheumatoid Arthritis - GlobeNewswire

The report analyzes the leading players of the global Cell Expansion Technologies market by inspecting their market share, recent developments, new product launches, partnerships, mergers, or acquisitions, and their target markets. This report also includes an exhaustive analysis of their product profiles to explore the products and applications their operations are concentrated on in the global Cell Expansion Technologies market. Additionally, the report gives two distinct market forecasts, one from the perspective of the producer and another from that of the consumer. It also offers valuable recommendations for new as well as established players of the global Cell Expansion Technologies market. It also provides beneficial insights for both new as well as established players of the global Cell Expansion Technologies market.

In a word, the report provides major statistics on the state of the industry and is a valuable source of guidance and direction for companies and individuals interested in the market.

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Leading vendors in the market are included based on profile, business performance etc. Vendors mentioned as follows:

CYTOMATRIXDANAHER CORP.CORNING INC.LIFE TECHNOLOGIES LTD.MERCK MILLIPOREPLURISTEM THERAPEUTICS INC.GE HEALTHCARECELLPROTHERAEarthrise Nutritionals LLCOthers

We provide detailed product mapping and investigation of various market scenarios. Our expert analysts provide a thorough analysis and breakdown of the market presence of key market leaders. We strive to stay updated with the recent developments and follow the latest company news related to the industry players operating in the global Cell Expansion Technologies market. This helps us to comprehensively analyze the individual standing of the companies as well as the competitive landscape. Our vendor landscape analysis offers a complete study to help you gain the upper hand in the competition.

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CLINICAL DIAGNOSTICSDRUG DISCOVERY AND DEVELOPMENTREGENERATIVE MEDICINE AND STEM CELL RESEARCH

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The report on global Cell Expansion Technologies market is intended to offer business owners, stakeholders and field marketing executives a broad overview of the business they should be focussing on for the estimated period. The research further holds vital information on the size of market and data on the prominent leaders product owners have to compete with, in the coming years. Assessments of the broad strengths, as well as weaknesses too, add value to the overall research. Products details not only cover the popular applications and its performance, but it also unveils certain trends and value of specific products within specific regions.

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Cell Expansion Technologies Market with Report In Depth Industry Analysis on top manufacturers and forecasts 2019-2026 - Chronicle Voice

Looking for broad exposure to the Healthcare - Biotech segment of the equity market? You should consider the Invesco Dynamic Biotechnology & Genome ETF (PBE), a passively managed exchange traded fund launched on 06/23/2005.

Passively managed ETFs are becoming increasingly popular with institutional as well as retail investors due to their low cost, transparency, flexibility and tax efficiency. They are excellent vehicles for long term investors.

Sector ETFs also provide investors access to a broad group of companies in particular sectors that offer low risk and diversified exposure. Healthcare - Biotech is one of the 16 broad Zacks sectors within the Zacks Industry classification. It is currently ranked 2, placing it in top 13%.

Index Details

The fund is sponsored by Invesco. It has amassed assets over $227.92 M, making it one of the average sized ETFs attempting to match the performance of the Healthcare - Biotech segment of the equity market. PBE seeks to match the performance of the Dynamic Biotechnology & Genome Intellidex Index before fees and expenses.

This is comprised of stocks of 30 U.S. biotechnology and genome companies. These are companies that are principally engaged in the research, development, manufacture and marketing and distribution of various biotechnological products, services and processes and companies that benefit significantly from scientific and technological advances in biotechnology and genetic engineering and research.

Costs

Expense ratios are an important factor in the return of an ETF and in the long term, cheaper funds can significantly outperform their more expensive counterparts, other things remaining the same.

Annual operating expenses for this ETF are 0.57%, making it on par with most peer products in the space.

Sector Exposure and Top Holdings

While ETFs offer diversified exposure, which minimizes single stock risk, a deep look into a fund's holdings is a valuable exercise. And, most ETFs are very transparent products that disclose their holdings on a daily basis.

This ETF has heaviest allocation in the Healthcare sector--about 100% of the portfolio.

Looking at individual holdings, Biogen Inc (BIIB) accounts for about 6.52% of total assets, followed by Vertex Pharmaceuticals Inc (VRTX) and Celgene Corp (CELG).

The top 10 holdings account for about 49.20% of total assets under management.

Performance and Risk

The ETF has added roughly 10.04% so far this year and is down about -3.11% in the last one year (as of 11/05/2019). In that past 52-week period, it has traded between $43.44 and $56.26.

The ETF has a beta of 1.43 and standard deviation of 23.63% for the trailing three-year period, making it a high risk choice in the space. With about 30 holdings, it has more concentrated exposure than peers.

Alternatives

Invesco Dynamic Biotechnology & Genome ETF carries a Zacks ETF Rank of 3 (Hold), which is based on expected asset class return, expense ratio, and momentum, among other factors. Thus, PBE is a reasonable option for those seeking exposure to the Health Care ETFs area of the market. Investors might also want to consider some other ETF options in the space.

SPDR S&P Biotech ETF (XBI) tracks S&P Biotechnology Select Industry Index and the iShares Nasdaq Biotechnology ETF (IBB) tracks Nasdaq Biotechnology Index. SPDR S&P Biotech ETF has $3.81 B in assets, iShares Nasdaq Biotechnology ETF has $7.02 B. XBI has an expense ratio of 0.35% and IBB charges 0.47%.

Bottom Line

To learn more about this product and other ETFs, screen for products that match your investment objectives and read articles on latest developments in the ETF investing universe, please visit Zacks ETF Center.

Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free reportInvesco Dynamic Biotechnology & Genome ETF (PBE): ETF Research ReportsiShares Nasdaq Biotechnology ETF (IBB): ETF Research ReportsSPDR S&P Biotech ETF (XBI): ETF Research ReportsCelgene Corporation (CELG) : Free Stock Analysis ReportVertex Pharmaceuticals Incorporated (VRTX) : Free Stock Analysis ReportBiogen Inc. (BIIB) : Free Stock Analysis ReportTo read this article on Zacks.com click here.Zacks Investment Research

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Should You Invest in the Invesco Dynamic Biotechnology & Genome ETF (PBE)? - Yahoo Finance

FORT LAUDERDALE, Fla., Nov. 6, 2019 /PRNewswire/ --Goodwin Biotechnology, Inc. announced that it has recently licensed an industry leading electronic content and document management solution to enhance the efficiency and effectiveness of its Quality systems and operations. This improvement continues to ensure that Goodwin Biotechnology remains uniquely positioned as aqualified and flexible biopharmaceutical Contract Development and Manufacturing Organization (CDMO) that offers an integrated Single Source Solution from Cell Line Development, Process Development and cGMP manufacturing including Scale-Up, cGMP Contract Manufacturing, and Aseptic Fill / Finish of mammalian cell-culture derived monoclonal antibodies, recombinant proteins, vaccines, and Antibody Drug Conjugates (ADCs).

"Acquisition and implementation of this sophisticated electronic solution demonstrates Goodwin's commitment to providing top-tier development and manufacturing services for our clients," noted Karl Pinto, Chief Executive Officer at Goodwin Biotechnology. "Goodwin's past successes supporting our clients through their early- and late-stage clinical trials, and regulatory compliant commercial manufacturing, will be strengthened through this recent investment. These strategic steps demonstrate our commitment to effectively support our clients through all stages of product development and cGMP manufacturing to assure conformance to worldwide regulatory standards." added Mr. Pinto.

About Goodwin Biotechnology, Inc.Goodwin Biotechnologyis a uniquely qualified and flexible, US-based CDMO that offers a Single Source Solution to enhance the value of complex biopharmaceuticals for our clients. With over 26 years of experience as an independent integrated contract manufacturer, Goodwin Biotechnology has worked as a strategic partner with companies of all sizes from small university spin-offs to major research institutes, government agencies and large, established and multi-national biopharmaceutical companies. Based on the impressive track record, Goodwin Biotechnology has received numerous industry awards that span from Frost & Sullivan's Customer Value and Leadership Award for Best Practices in Mammalian Contract Manufacturing to the Biologics cGMP Manufacturer of the Year 2018 by Global Health & Pharma News. Click here to view the press releases! Additional information may be found at http://www.GoodwinBio.com.

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Goodwin Biotechnology Launches a Digitization Initiative by Investing in a Sophisticated Electronic Quality Solution - Yahoo Finance

Biotech stocks extended their gains last week amid earnings news flow and the broader market strength. Large-cap pharma stocks saw particular strength.

Here are the key catalytic event a biotech investor should keep a tab on in the unfolding week.

The FDA is set to rule on Lipocine Inc (NASDAQ: LPCN)'s NDA for Tlando, or LPCN 1021 to treat hypogonadism in adult males. The PDUFA date is set for Saturday, Nov. 9.

Alkermes Plc (NASDAQ: ALKS) Phase 1/2 data for ALKS 4230 in solid tumors and ALKS 4230 in combination with Merck & Co., Inc. (NYSE: MRK)'s Keytruda in solid tumors.

Celldex Therapeutics, Inc. (NASDAQ: CLDX) Phase 1 data for CDX-1140 in solid tumors (Friday, Nov. 8).

Pieris Pharmaceuticals Inc (NASDAQ: PIRS) Phase 1/2 data for PRS-343 in HER2-positive solid tumors.

CELYAD SA/ADR (NASDAQ: CYAD) Phase 1 data for CYAD-01 and FOLFOX in colorectal cancer (Friday, Nov. 8) and Phase 1 data for CYAD-101 in colorectal cancer (Saturday, Nov. 9).

Heat Biologics Inc (NASDAQ: HTBX) Phase 2 data for HS-110 and Bristol-Myers Squibb Co (NYSE: BMY)'s Opdivo in non-small cell lung cancer (Friday).

Oncolytics Biotech, Inc. (NASDAQ: ONCY) - interim Phase 1b data for Pelareorep and Roche Holdings AG Basel ADR Common Stock (OTC: RHHBY)'s Tecentriq in breast cancer (Friday).

Nektar Therapeutics (NASDAQ: NKTR) updated Phase 1/2 data for NKTR-214 + Opdivo in multiple cancers (urothelial carcinoma, melanoma, renal cell carcinoma, and non-small cell lung cancer) (Saturday).

Mirati Therapeutics Inc (NASDAQ: MRTX) initial Phase 2 data for sitravatinib in urothelial carcinoma (Saturday).

Allena Pharmaceuticals Inc (NASDAQ: ALNA) Phase 2 data for reloxaliase, or ALLN-177, in primary hyperoxaluria and Phase 3 data for reloxaliase in enteric hyperoxaluria (Friday).

See Also: BTIG: Crispr Could Be A Takeover Target For Vertex

The earnings list is not comprehensive

Monday

Tuesday

Wednesday

Thursday

Friday

Galera Therapeutics has filed to offer 5 million shares in an IPO, with an estimated price range of $14-$16. The company has applied for listing its shares on the Nasdaq under the ticker symbol "GRTX."

89bio, a biotech company focusing on therapies for NASH and other metabolic disorders, is planning a 4.375-million IPO at an estimated price range of $15-17. The company has applied for listing its shares on the Nasdaq under the ticker symbol "ETNB."

Gene testing company Centogene is proposing to offer 4 million shares in an IPO, which are to be priced between $14 and $16. The company has applied to list the shares on the Nasdaq under the ticker symbol "CNTG."

CNS Pharmaceuticals, which develops therapies for brain cancer and other CNS tumors, has filed for a 2.125-million share IPO. The shares, which are likely to be priced in the range of $4-$5, are to be listed on the Nasdaq under the ticker symbol "CNSP."

Tela Bio, which sells soft tissue implants used in hernia repair and reconstructive surgery, is set to offer 4 million shares in an IPO, with each share to be priced between $14 and $16. The company seeks to list the shares on the Nasdaq under the ticker symbol "TELA."

BioNTech SE ADR (NASDAQ: BNTX)Vir Biotechnology Inc (NASDAQ: VIR)

2019 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.

Link:
The Week Ahead In Biotech: Smid-Cap Earnings Deluge, SITC Conference In The Spotlight - Benzinga

The company provides a detailed analysis of the market and future aspects of the Biotechnology Market. It focuses on critical and critical data that makes it a very important tool for research, experts, analysts, and managers to achieve ready-to-access analysis. The report provides an inclusive analysis of the Biotechnology market size forecast from 2018-2025.

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The report embraces the complete information of the key players involved in the worldwide Biotechnology market. In addition, it provides its market share by various regions with the company and product introduction and their position in the Biotechnology market. In addition, the report takes into account recent marketing developments as well as their marketing strategies along with an overall business overview. In addition, the report covers market growth factors and restraints of this market.

Prominent players of Biotechnology market:

Product Type Coverage (Market Size & Forecast, Major Company of Product Type etc.):

Application Coverage (Market Size & Forecast, Different Demand Market by Region, Main Consumer Profile etc.):

Regional Segmentation for Biotechnology market:

There are 10 chapters to put on view for Biotechnology market:

Chapter 1: Consumption by Regions

Chapter 2: Production, By Types, Revenue and Market share by Types

Chapter 3: Consumption, By Applications, Market share (%) and Growth Rate by Applications

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Chapter 5: Manufacturing cost analysis, Raw materials analysis, Region-wise manufacturing expenses

Chapter 6: Industrial Chain, Sourcing Strategy and Downstream Buyers

Chapter 7: Marketing Strategy Analysis, Distributors/Traders

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Chapter9: Market Forecast

Chapter 10: Biotechnology Research Findings and Conclusion, Appendix, methodology and data source

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Global Biotechnology Market 2019, Trend, CAGR Status, Growth, Analysis and Forecast to 2024 - BoundWatch

Analysts expect that Unity Biotechnology Inc (NASDAQ:UBX) will report earnings of ($0.49) per share for the current fiscal quarter, according to Zacks. Zero analysts have provided estimates for Unity Biotechnologys earnings. Unity Biotechnology posted earnings of ($0.43) per share in the same quarter last year, which indicates a negative year over year growth rate of 14%. The firm is expected to announce its next earnings report after the market closes on Wednesday, November 6th.

On average, analysts expect that Unity Biotechnology will report full year earnings of ($2.02) per share for the current financial year, with EPS estimates ranging from ($2.07) to ($1.96). For the next year, analysts expect that the company will post earnings of ($2.09) per share, with EPS estimates ranging from ($2.25) to ($1.93). Zacks Investment Researchs earnings per share averages are an average based on a survey of research firms that follow Unity Biotechnology.

Unity Biotechnology (NASDAQ:UBX) last released its earnings results on Wednesday, August 7th. The company reported ($0.54) earnings per share (EPS) for the quarter, missing analysts consensus estimates of ($0.44) by ($0.10).

Institutional investors and hedge funds have recently made changes to their positions in the company. Aperio Group LLC acquired a new position in shares of Unity Biotechnology during the 2nd quarter worth $50,000. Citadel Advisors LLC grew its stake in Unity Biotechnology by 449.0% in the 2nd quarter. Citadel Advisors LLC now owns 238,386 shares of the companys stock valued at $2,265,000 after buying an additional 194,965 shares during the last quarter. Ellington Management Group LLC acquired a new position in Unity Biotechnology in the 2nd quarter valued at $103,000. BlackRock Inc. grew its stake in Unity Biotechnology by 5.0% in the 2nd quarter. BlackRock Inc. now owns 1,992,613 shares of the companys stock valued at $18,929,000 after buying an additional 94,183 shares during the last quarter. Finally, Virtus ETF Advisers LLC grew its stake in Unity Biotechnology by 84.8% in the 2nd quarter. Virtus ETF Advisers LLC now owns 30,554 shares of the companys stock valued at $290,000 after buying an additional 14,021 shares during the last quarter. Institutional investors own 38.32% of the companys stock.

UBX stock opened at $6.88 on Friday. Unity Biotechnology has a 12-month low of $5.61 and a 12-month high of $17.46. The company has a market cap of $295.51 million, a price-to-earnings ratio of -2.71 and a beta of -0.19. The company has a 50 day moving average of $6.81 and a two-hundred day moving average of $7.63.

About Unity Biotechnology

Unity Biotechnology, Inc, a biotechnology company, engages in the research and development of therapeutics to extend human health span. The company's lead drug candidates include UBX0101 that is in Phase 1 clinical study for musculoskeletal disease; and UBX1967 for ophthalmologic diseases. It is also developing programs in pulmonary disorders.

Read More: Dollar Cost Averaging

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Brokerages Anticipate Unity Biotechnology Inc (NASDAQ:UBX) Will Announce Earnings of -$0.49 Per Share - Slater Sentinel

LOS ANGELES--(BUSINESS WIRE)--

Puma Biotechnology, Inc. (PBYI), a biopharmaceutical company, announced that Alan H. Auerbach, Chairman, Chief Executive Officer, President and Founder of Puma, will provide an overview of the Company at 10:55 a.m. MST (9:55 a.m. PST, 12:55 p.m. EST) on Tuesday, November 12, at the Credit Suisse 28th Annual Healthcare Conference. The conference will be held at The Phoenician Resort in Scottsdale, Arizona.

A live webcast of the presentation will be available on the Companys website at http://www.pumabiotechnology.com. The presentation will be archived on the website and available for 30 days.

About Puma Biotechnology

Puma Biotechnology, Inc. is a biopharmaceutical company with a focus on the development and commercialization of innovative products to enhance cancer care. Puma in-licenses the global development and commercialization rights to three drug candidates PB272 (neratinib, oral), PB272 (neratinib, intravenous) and PB357. Neratinib, oral was approved by the U.S. Food and Drug Administration in July 2017 for the extended adjuvant treatment of adult patients with early stage HER2-overexpressed/amplified breast cancer, following adjuvant trastuzumab-based therapy, and is marketed in the United States as NERLYNX (neratinib) tablets. NERLYNX was granted marketing authorization by the European Commission in September 2018 for the extended adjuvant treatment of adult patients with early stage hormone receptor-positive HER2-overexpressed/amplified breast cancer and who are less than one year from completion of prior adjuvant trastuzumab-based therapy. NERLYNX is a registered trademark of Puma Biotechnology, Inc.

Further information about Puma Biotechnology can be found at http://www.pumabiotechnology.com.

View source version on businesswire.com: https://www.businesswire.com/news/home/20191104005169/en/

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Puma Biotechnology to Present at the Credit Suisse Healthcare Conference - Yahoo Finance

MIRAMAR, Fla., Nov. 08, 2019 (GLOBE NEWSWIRE) -- Generex Biotechnology Corporation (GNBT) is pleased to announce that its 10k annual report will be filed Tuesday morning within the time afforded to public companies under the standard filing extension. Generex has received many calls in regard to this very important filing, and the company wants to assure everyone that the report has been completed, and the filing is definitive for Tuesday, November 12, 2019.

Joe Moscato, CEO and President commented, I appreciate everyones concern in this important filing as we march towards our NASDAQ listing. This annual report filing was much more complicated than last years report due to the many acquisitions the company made over the last year, which required that the financial audit be reviewed by multiple audit firms. Further, this is the first full audit that our new auditors have had to complete, so understanding all of Generex 23-year history of business, and the complexities of the MSO and healthcare initiatives that the company has initiated was no small task. Now that this first 10K audit has been completed for the NuGenerex family of companies, our financial accounting and auditing work will get easier, having been streamlined for the future with standard operating procedures.

About Generex Biotechnology Corp.

Generex Biotechnology is an integrated healthcare holding company with end-to-end solutions for patient centric care from rapid diagnosis through delivery of personalized therapies. Generex is building a new kind of healthcare company that extends beyond traditional models providing support to physicians in an MSO network, and ongoing relationships with patients to improve the patient experience and access to optimal care.

In addition to advancing a legacy portfolio of immune-oncology assets, medical devices, and diagnostics, the Company is focused on an acquisition strategy of strategic businesses that complement existing assets and provide immediate sources of revenue and working capital. Recent acquisitions include a management services organization, a network of pharmacies, clinical laboratory, and medical device companies with new and approved products.

Cautionary Note Regarding Forward-Looking Statements

This release and oral statements made from time to time by Generex representatives in respect of the same subject matter may contain "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. These statements can be identified by introductory words such as "expects," "plan," "believes," "will," "achieve," "anticipate," "would," "should," "subject to" or words of similar meaning, and by the fact that they do not relate strictly to historical or current facts. Forward-looking statements frequently are used in discussing potential product applications, potential collaborations, product development activities, clinical studies, regulatory submissions and approvals, and similar operating matters. Many factors may cause actual results to differ from forward-looking statements, including inaccurate assumptions and a broad variety of risks and uncertainties, some of which are known and others of which are not. Known risks and uncertainties include those identified from time to time in the reports filed by Generex with the Securities and Exchange Commission, which should be considered together with any forward-looking statement. No forward-looking statement is a guarantee of future results or events, and one should avoid placing undue reliance on such statements. Generex undertakes no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise. Generex claims the protection of the safe harbor for forward-looking statements that is contained in the Private Securities Litigation Reform Act.

Generex Contact:

Generex Biotechnology Corporation

Joseph Moscato 646-599-6222

Todd Falls 1-800-391-6755 Extension 222investor@generex.com

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Generex Biotechnology Provides Guidance: Form 10k Annual Report will be filed Tuesday Morning November 12th 2019 - Yahoo Finance

LOS ANGELES--(BUSINESS WIRE)--Puma Biotechnology, Inc. (Nasdaq: PBYI) announced that its licensing partner in Greater China, CANbridge Pharmaceuticals Inc., has received registration approval from the Department of Health in Hong Kong to market NERLYNX (neratinib) for the extended adjuvant treatment of adult patients with early stage hormone receptor positive HER2-overexpressed/amplified breast cancer and who have completed adjuvant trastuzumab-based therapy less than one year ago.

The rapid and smooth advancement of NERLYNX through the Hong Kong regulatory process to market approval is a testament to the quality of our regulatory expertise, and the CANbridge commitment to bring new treatments to underserved patient populations in greater China, said James Xue, PhD, Founder, Chairman and CEO, CANbridge Pharmaceuticals. With this first targeted therapy approved in our oncology platform, CANbridge is able to provide women in Hong Kong, with HER2-positive breast cancer at risk of recurrence, a new and vital treatment option shortly after approval in the West.

Alan H. Auerbach, Puma Biotechnologys Chairman, Chief Executive Officer and President, added, This registration approval through our partnership with CANbridge represents an important market entry for us as we execute on our global commercial strategy. CANbridge has the commercial infrastructure and resources to provide NERLYNX to patients in the region.

About HER2-Positive Breast Cancer

Approximately 20 to 25 percent of breast cancer tumors over-express the HER2 protein. HER2-positive breast cancer is often more aggressive than other types of breast cancer, increasing the risk of disease progression and death. Although research has shown that trastuzumab can reduce the risk of early stage HER2-positive breast cancer returning after surgery, up to 25% of patients treated with trastuzumab experience recurrence.

About CANbridge Pharmaceuticals

CANbridge Pharmaceuticals Inc. is a China-based biopharmaceutical company accelerating development and commercialization of specialty healthcare products for orphan diseases and targeted cancers, focusing on products that are unavailable or address medical needs that are underserved in the region. CANbridge has been widely recognized as a leader in orphan diseases in China. It has a global partnership with WuXi Biologics to develop and commercialize proprietary therapeutics for the treatment of rare genetic diseases. In addition, it has an exclusive licensing agreement to commercialize Hunterase, an enzyme replacement therapy for the treatment of Hunter syndrome, developed by GC Pharma and marketed in more than ten countries worldwide. CANbridge also has an oncology portfolio, which includes exclusive rights to develop and commercialize Puma Biotechnologys NERLYNX (neratinib), approved in the United States, and rights to other novel candidates.

About Puma Biotechnology

Puma Biotechnology, Inc. is a biopharmaceutical company with a focus on the development and commercialization of innovative products to enhance cancer care. Puma in-licenses the global development and commercialization rights to PB272 (neratinib, oral), PB272 (neratinib, intravenous) and PB357. Neratinib, oral was approved by the U.S. Food and Drug Administration in July 2017 for the extended adjuvant treatment of adult patients with early stage HER2-overexpressed/amplified breast cancer, following adjuvant trastuzumab-based therapy, and is marketed in the United States as NERLYNX (neratinib) tablets. NERLYNX was granted marketing authorization by the European Commission in August 2018 for the extended adjuvant treatment of adult patients with early stage hormone receptor-positive HER2-overexpressed/amplified breast cancer and who are less than one year from completion of prior adjuvant trastuzumab-based therapy. NERLYNX is a registered trademark of Puma Biotechnology, Inc.

Further information about Puma Biotechnology may be found at http://www.pumabiotechnology.com.

Important Safety Information Regarding NERLYNX (neratinib) U.S. Indication

NERLYNX (neratinib) tablets, for oral use

INDICATIONS AND USAGE: NERLYNX is a kinase inhibitor indicated for the extended adjuvant treatment of adult patients with HER2 overexpressed/amplified breast cancer, to follow adjuvant trastuzumab-based therapy.

CONTRAINDICATIONS: None

WARNINGS AND PRECAUTIONS:

ADVERSE REACTIONS: The most common adverse reactions ( 5%) were diarrhea, nausea, abdominal pain, fatigue, vomiting, rash, stomatitis, decreased appetite, muscle spasms, dyspepsia, AST or ALT increase, nail disorder, dry skin, abdominal distention, weight decreased and urinary tract infection.

To report SUSPECTED ADVERSE REACTIONS, contact Puma Biotechnology, Inc. at 1-844-NERLYNX (1-844-637-5969) and http://www.NERLYNX.com or FDA at 1-800-FDA-1088 or http://www.fda.gov/medwatch.

DRUG INTERACTIONS:

USE IN SPECIFIC POPULATIONS:

Please see Full Prescribing Information for additional safety information.

The recommended dose of NERLYNX is 240 mg (six 40 mg tablets) given orally once daily with food, continuously for one year. Antidiarrheal prophylaxis should be initiated with the first dose of NERLYNX and continued during the first 2 months (56 days) of treatment and as needed thereafter.

To help ensure patients have access to NERLYNX, Puma has implemented the Puma Patient Lynx support program to assist patients and healthcare providers with reimbursement support and referrals to resources that can help with financial assistance. More information on the Puma Patient Lynx program can be found at http://www.NERLYNX.com or 1-855-816-5421.

Forward-Looking Statements

This press release contains forward-looking statements, including statements regarding the worldwide expansion of NERLYNX. All forward-looking statements involve risks and uncertainties that could cause Pumas actual results to differ materially from the anticipated results and expectations expressed in these forward-looking statements. These statements are based on current expectations, forecasts and assumptions, and actual outcomes and results could differ materially from these statements due to a number of factors, which include, but are not limited to, the risk factors disclosed in the periodic and current reports filed by Puma with the Securities and Exchange Commission from time to time, including Pumas Annual Report on Form 10-K for the year ended December 31, 2018. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. Puma assumes no obligation to update these forward-looking statements, except as required by law.

Continued here:
Puma Biotechnology Licensing Partner CANbridge Pharmaceuticals Receives Registration Approval in Hong Kong for NERLYNX (neratinib) for Extended...

Dr. Lauren V. Wood, M.D. to present data from the companys phase 1 PDS0101 human trial demonstrating potent antigen-specific CD8+ T-cell responses in-vivo

PRINCETON, N.J., Nov. 06, 2019 (GLOBE NEWSWIRE) -- PDS Biotechnology Corporation (PDSB), a clinical-stage immuno-oncology company developing multiple therapies based on the companys proprietary Versamune T-cell activating technology , today announced that Lauren V. Wood, M.D., Chief Medical Officer of PDS Biotechnology, has been selected to make an oral presentation reporting on the study design and clinical data from its previously announced Phase 1 study of PDS0101 at the Society for Immunotherapy of Cancer (SITC) 34th Annual Meeting, taking place on November 7-10, 2019 in National Harbor, Maryland.

In addition to the immunogenicity and safety data from the Phase 1 study of PDS0101, we are very pleased to highlight follow-up data, demonstrating the translation of our Versamune-induced immunological mechanisms from animal to human studies in an oral presentation at the 34th annual SITC meeting, commented Lauren V. Wood, M.D., PDS Biotechnologys Chief Medical Officer. Based on this encouraging data, we are now focused on initiating three Phase 2 studies in the first quarter of 2020. These include our previously announced combination study to evaluate PDS0101 in combination with KEYTRUDA in the treatment of HPV16 positive head and neck cancer, a study to evaluate PDS0101 in combination with two immunotherapies in advanced HPV-associated cancers with the National Cancer Institute and a study, the details of which are included in our supplemental corporate presentation filed on October 29, 2019, to evaluate the combination of PDS0101 and chemoradiotherapy in patients with locally advanced cervical cancer.

Details for the oral presentation are below:

Abstract 017: A Novel Enantio-Specific Cationic Lipid R-DOTAP + HPV16 E6 & E7 Antigens Induces Potent Antigen-Specific CD8+ T Cell Responses In-Vivo in Subjects with CIN and High-Risk Human Papilloma Virus InfectionPresenter: Dr. Lauren V. Wood, M.D., Chief Medical Officer, PDS BiotechnologySession Title: Session 209: Virus Driven CancersDate: Friday, November 8th, 2019Time: 6:00 6:10pm ET

Full data from the Phase 1 study can be found in the supplemental corporate presentation filed on October 29, 2019.

About PDS Biotechnology

PDS Biotechnology is a clinical-stage immuno-oncology company developing multiple therapies based on the companys proprietary Versamune T-cell activating technology platform. The Versamune platform effectively delivers tumor-specific antigens for in-vivo uptake and processing, while also activating a critical immunological pathway, the type 1 interferon pathway, thus resulting in the production of potent tumor-specific killer T-cells. Using Versamune, PDS Biotechnology is engineering therapies designed to better recognize cancer cells and break down their defense systems to effectively attack and destroy tumors. PDS Biotechnologys pipeline combines the Versamune technology with tumor-specific antigens across several cancer types. To learn more, please visit http://www.pdsbiotech.com.

About PDS0101

PDS Biotechnologys lead candidate, PDS0101, combines the utility of the Versamune platform with targeted antigens in HPV-expressing cancers. In partnership with Merck and the National Cancer Institute (NCI), PDS Biotechnology is advancing PD0101 to Phase 2 studies in head and neck cancer and in HPV-related advanced cancer.

Forward Looking StatementsThis communication contains forward-looking statements (including within the meaning of Section 21E of the United States Securities Exchange Act of 1934, as amended, and Section 27A of the United States Securities Act of 1933, as amended) concerning PDS Biotechnology Corporation (the Company) and other matters. These statements may discuss goals, intentions and expectations as to future plans, trends, events, results of operations or financial condition, or otherwise, based on current beliefs of the Companys management, as well as assumptions made by, and information currently available to, management. Forward-looking statements generally include statements that are predictive in nature and depend upon or refer to future events or conditions, and include words such as may, will, should, would, expect, anticipate, plan, likely,believe,estimate,project,intend,and other similar expressions among others. Statements that are not historical facts are forward-looking statements. Forward-looking statements are based on current beliefs and assumptions that are subject to risks and uncertainties and are not guarantees of future performance. Actual results could differ materially from those contained in any forward-looking statement as a result of various factors, including, without limitation: the ability of the Company to integrate Edge and PDS Biotechnology following the merger; the Companys ability to protect its intellectual property rights; competitive responses to the completion of the merger; potential adverse reactions or changes to business relationships resulting from the completion of the merger;the Companys anticipated capital requirements, including the Companys anticipated cash runway and the Companys current expectations regarding its plans for future equity financings; the timing for the Company or its partners to initiate the planned clinical trials for its lead assets, PDS0101 and PDS0102; the Companys interpretation of the results of its Phase 1 trial for PDS0101 and whether such results are sufficient to support additional trials or the future success of such trials;the successful implementation of the Companys research and development programs and collaborations; the acceptance by the market of the Companys product candidates, if approved;the timing of and the Companys ability to obtain and maintainU.S. Food and Drug Administrationor other regulatory authority approval of, or other action with respect to, the Companys product candidates;and other factors, including legislative, regulatory, political and economic developmentsnot within the Companys control. The foregoing review of important factors that could cause actual events to differ from expectations should not be construed as exhaustive and should be read in conjunction with statements that are included herein and elsewhere, including the risk factors included in the Companys annual and periodic reports filed with the SEC. The forward-looking statements are made only as of the date of this press release and, except as required by applicable law, the Company undertakes no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, whether as a result of new information, future events or otherwise.

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PDS Biotechnology Accepted for Oral Presentation at the 34th Annual Society for Immunotherapy of Cancer Annual Meeting - Yahoo Finance