Navigating the challenge of covering breakthrough therapies at MedCity INVEST Precision Medicine

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Our virtual MedCity INVEST Precision Medicine conference, December 9-11, will highlight aspects of precision medicine from the biopharma companies developing cell and gene therapies to diagnostics and the data sharing initiatives to support precision and personalized medicine.

One vexing part of any conversation on precision medicine and breakthrough therapies that have been developed and are continuing to work their way through clinical trials for cancer to rare disease is how to address the practical challenge of their high price tags. Although there are several models to cover the cost of these therapies such as Cigna’s Embarc Benefit Protection program, what happens when someone changes insurers when they change their job? What is fair for patients and what’s fair for companies? When do these conversations even start?

The panel, Reimbursement Models for Cell and Gene Therapies, will highlight some of the pros and cons of different models that are coming to market. Panelists include Laura Okpala, Director, Reimbursement Policy, Gilead Sciences, and Mark Trusheim, Strategic Director, NEWDIGS initiative at the MIT Center for Biomedical Innovation.

Here’s a preview of some of the sessions. Click here to see the agenda.

Interoperability Progress Report

How far have we progressed with sharing patient medical records? Patient data is key in unlocking riddles of medical science but interoperability is necessary to facilitate this. What companies are making an impact on a regional and national scale? What milestones are on the horizon? What obstacles continue to vex further advancement? How can we make medical records more accessible to patients?

Moderator: Elise Reuter, Senior Reporter, MedCity News
Speakers:
Kevin Chaney, Senior Program Manager, Office of the National Coordinator for Health IT
Ida Sim, M.D., Ph.D., Professor of Medicine, University of California, San Francisco
Niko Skievaski, Co-Founder and President, Redox

What It Takes To Build A Successful, Regional BioInnovation Hub
Philadelphia is one of many cities seeking to support the continued growth of cell and gene therapy and connected health industries. What do cities need to do to address education, training and other needs to support these sectors? This session will be held as part of Venture Cafe Philadelphia.

(sponsored by IBX)

Lisa Dalton, Chief People Officer, Spark Therapeutics
Audrey Greenberg, Executive Managing Director, The Discovery Labs
Tiffany Wilson, President & CEO, University City Science Center

Moderator: Michelle Histand, Director of Innovation, Independence Blue Cross

Register now and be part of the conversation at INVEST Precision Medicine.

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TFF Pharmaceuticals, Inc. and Augmenta Bioworks, Inc. Enter Into a Worldwide Joint Development Agreement for COVID-19 Monoclonal Antibody Therapies

Companies to collaborate in first-of-its-kind uses of Thin Film Freezing technology applied to monoclonal antibodies

TFF Pharmaceuticals, Inc. (NASDAQ: TFFP), a clinical-stage biopharmaceutical company, and Augmenta Bioworks, Inc., a biotechnology company enabling breakthroughs in medicine through immune profiling, today jointly announce that both companies have entered into a worldwide Joint Development and Collaboration Agreement to develop novel commercial products incorporating Augmenta’s human-derived monoclonal antibodies (mAbs) for potential COVID-19 therapeutics.

Under the terms of the Agreement, both companies will collaborate in a Joint Development Project to develop one or more commercial therapeutics based on, derived from, and/or incorporating Augmenta’s human monoclonal antibodies to potentially treat patients with COVID-19. These products will be developed utilizing TFF Pharmaceuticals’ Thin-Film Freezing technology to manufacture dry powder formulations of these specific mAbs for inhalation delivery directly to the lungs of patients. The Agreement also includes the development of formulations suitable for parenteral administration, where the Thin Film Freezing dry powder formulations can be reconstituted, potentially mitigating the impacts of cold-chain storage and handling. TFF Pharmaceuticals will also have the option to develop two additional Augmenta mAbs for indications other than COVID-19.

Augmenta Bioworks and TFF Pharmaceuticals will allocate patent license rights to their respective technologies to allow each company to jointly commercialize the products developed under the Joint Development Project. The companies have agreed to a 50-50 split of all costs and expenses to further the Joint Development Project and both companies have agreed to the same 50-50 split of all revenues, cash payments and/or future cash payments related to the sale and/or license of the products resulting from the Joint Development Project to a third party.

“This important agreement represents the culmination of many months of work by our scientific team, as we work towards the development of a never-before-achieved formulation of monoclonal antibodies into a dry powder therapeutic,” said Glenn Mattes, CEO, of TFF Pharmaceuticals, Inc. “It is a testament to the remarkable flexibility and capability of our Thin Film Freezing platform and we are eager to develop these potentially breakthrough mAb therapies internally, along with our other programs in Invasive Pulmonary Aspergillosis, solid organ transplant anti-rejection, and botanicals.”

“Confirmed discovery of novel anti-SARS-Cov-2 antibodies in 8 days was an achievement made possible by years of technology development, and a clear indication of the power and potential of our platform,” said Christopher Emig, Ph.D., CEO and Co-Founder of Augmenta Bioworks, Inc. “We are excited to enter this partnership to bring our COVID-19 treatment into clinical development, and are looking forward to the world’s first effective, affordable and scalable antibody therapeutic to mitigate the devastating effects of this disease.”

“We believe the interest in monoclonal-antibody therapeutics for the treatment of COVID-19 is extremely high, with the promise that they will harness the immune system’s natural response to viral invaders,” said Robert O. Williams III, Ph.D., Division Head of the University of Texas at Austin’s Division of Molecular Pharmaceutics and Drug Delivery and inventor of TFF Pharmaceuticals’ Thin Film Freezing technology.

“The challenge

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Europe Anti-Viral Therapies Market Forecast to 2027

The Europe anti-viral therapies market is expected to reach US$ 21,122. 66 million by 2027 from US$ 11,401. 67 million in 2019; it is estimated to grow at a CAGR of 8. 1% during 2020–2027. The market is growth is primarily attributed to the increasing R&D expenditures in pharmaceutical companies and rising government support for research activities and clinical trials in Europe.

New York, Oct. 28, 2020 (GLOBE NEWSWIRE) — Reportlinker.com announces the release of the report “Europe Anti-Viral Therapies Market Forecast to 2027 – COVID-19 Impact and Regional Analysis By Type, Mechanism of Action, and Application, and Country” – https://www.reportlinker.com/p05978841/?utm_source=GNW
Additionally, strong pipeline of anti-viral drugs, and growing emphasis on launching anti-viral agents are likely to fuel the growth of the Europe anti-viral therapies market during the forecast period.

High cost of drug development is a key factor restraining the growth of the market.
Antiviral therapy is one of the most exciting branches of virology. These therapies are based on several strategies—direct-acting antivirals target viral proteins, enzymes, or nucleic acids; passive antibodies neutralize circulating viruses; and several other antivirals target cellular proteins or processes essential for viral replication.

Research and development (R&D) is an essential part of any business.Pharmaceutical companies focus on R&D to introduce new drugs with enhanced medical and commercial potential.

These companies invest majorly in R&D activities with an aim to deliver high-quality and innovative products in the market. As per the Association of the British Pharmaceutical Industry Pharmaceutical R&D expenditures grew by >11% in the UK in 2001, reaching an estimated US$ 5.23 billion. Johnson & Johnson, and Pfizer are among the top 10 leading R&D investing companies. The UK’s relative stability compared to the rest of Europe as a base for investment to an increase in R&D activity there by the three largest pharmaceutical investors. AstraZeneca, GlaxoSmithKline, and Pfizer collectively signify over 70% of total R&D spending in the UK, while other manufacturers with a significant presence in the country include Merck, Sharp & Dohme, Organon, Eli Lilly, and Novartis. Additionally, the pharma companies in the Germany are highly engaged in R&D activities.

Europe has witnessed exponential growth in the count of COVID-19 cases.Spain, Italy, Germany, France, and the UK are among the most affected European countries.

Growing research activities to develop treatment against corona virus is likely to favor the growth of the market in this region.In June 2020, the scientists from the University of Oxford tested existing drugs as therapies against this novel virus.

Further, the European Commission in July authorized the anti-viral drug Remdesivir for its use in the treatment of COVID-19 patients, making it the first approved drug in the European Union for the treatment.

In 2019, the branded drugs segment accounted for a larger share of the Europe antiviral therapies market.The branded antiviral drugs are more trusted than the generic drugs.

Further, robust research and development activities, increasing number of drug discoveries, and rising number of patents for drugs are the factors supporting the growth of this segment.

A

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Taysha Gene Therapies Receives Rare Pediatric Disease Designation and Orphan Drug Designation for TSHA-104 to Treat SURF1-Associated Leigh Syndrome

Taysha anticipated to submit Investigational New Drug Application for TSHA-104 to FDA in 2021

Rare pediatric disease and orphan drug designations now obtained in multiple pipeline programs, including TSHA-101 for GM2 gangliosidosis, TSHA-102 for Rett syndrome and TSHA-118 for CLN1

Taysha Gene Therapies Inc. (Nasdaq: TSHA), a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system in both rare and large patient populations, today announced that it has received rare pediatric disease designation and orphan drug designation from the U.S. Food and Drug Administration (FDA) for TSHA-104, an AAV9-based gene therapy in development for SURF1-associated Leigh syndrome. Taysha anticipates it will submit an Investigational New Drug (IND) application to the FDA for TSHA-104 in 2021.

“We have now obtained rare pediatric disease and orphan drug designations in multiple gene therapy programs, which we believe will allow us to work more effectively with the FDA as we advance our broad portfolio,” said RA Session II, President, CEO and Founder of Taysha. “The receipt of these designations highlights the dedication that our team has to advancing our gene therapy pipeline as efficiently and rapidly as possible.”

Leigh syndrome is a severe neurological disorder that usually presents in the first year of life. It is characterized by progressive loss of mental and movement abilities that can result in death within two to three years. Approximately 10-15% of people with Leigh syndrome have a SURF1 mutation.

“Being diagnosed with a mutation in the SURF1 gene is a truly devastating event for families,” said Kasey Woleben, Founder of Cure SURF1 Foundation. “Taysha’s commitment to developing a gene therapy for SURF1 deficiency is greatly welcomed by the patient community and has the potential to save the lives of children afflicted with this progressive disorder.”

Taysha has secured rare pediatric disease designation and orphan drug designation for multiple of its programs, including GM2 gangliosidosis, CLN1, Rett syndrome and now SURF1. In addition to these designations, the company also has fast track status for the CLN1 program.

“SURF1 deficiency is a monogenic mitochondrial disorder and is the most common cause of cytochrome c oxidase deficient Leigh syndrome,” said Steven Gray, Ph.D., Chief Scientific Advisor of Taysha and Associate Professor in the Department of Pediatrics at UT Southwestern. “Obtaining these key designations highlights our commitment to developing a gene therapy for the treatment of SURF1 deficiency.”

The FDA defines a rare pediatric disease as a serious or life-threatening disease in which the disease manifestations primarily affect individuals aged from birth to 18 years. Pediatric diseases recognized as “rare” affect under 200,000 people in the U.S. The Rare Pediatric Disease Priority Review Voucher Program is intended to address the challenges that drug companies face when developing treatments for these unique patient populations. Under this program, companies are eligible to receive a priority review voucher following approval of a product with rare pediatric disease designation if the marketing application submitted for the product satisfies certain

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The US now seems to be pinning all of its hopes on COVID-19 therapies and vaccines

Almost eight months after the White House first announced it would move from containment to mitigation efforts to stop the spread of the COVID-19 epidemic, the Administration is now pinning its hopes on vaccines to inoculate the population and therapies to treat the disease.

Months after announcing it would be working with technology giants Apple and Google on a contact tracing app (and nearly two months after Google and Apple rolled out their exposure notification features) and initiating wide spread testing efforts nationwide with the largest national pharmacies (which never received the coordinated support it needed),  the Administration appears to be giving up on a national effort to stop the spread of the COVID-19 epidemic.

In an interview with CNN’s Jake Tapper White House Chief of Staff Mark Meadows said that the US is “not going to control the pandemic… We are gonna control the fact that we get vaccines, therapeutics and other mitigation.”

The admission is a final nail in the coffin for a federal response that could have involved a return to lockdowns to stop the spread of the virus, or national testing and contact tracing and other mitigation measures. Meadows statement comes as the US experiences a second peak in infection rates. There are now over 8.1 million cases and over 220,000 deaths since the first confirmed infection on US soil on January 20. 

Now, the focus is all on the vaccines, therapies and treatments being developed by large pharma companies and startups alike that are making their way through the approval processes of regulatory agencies around the world.

The vaccines in phase three clinical trials

There are currently 12 vaccines in large scale, late-stage clinical trials around the world, including ones from American companies Novavax, Johnson & Johnson, Moderna Therapeutics, and Pfizer who are recruiting tens of thousands of people in the US and UK to volunteer for testing.

In China, the state run pharmaceutical company Sinopharm has filed its application to China’s regulatory commission for the approval of a vaccine and hundreds of thousands of civilians have already been vaccinated under emergency use approvals from the Chinese government, according to a report in the New Yorker. Meanwhile the privately held Chinese pharmaceutical company, Sinovac, is moving forward with phase three trials for its own vaccine in Brazil, Bangladesh and Indonesia. Another

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Ultragenyx and Solid Biosciences Announce Strategic Collaboration to Develop and Commercialize New Gene Therapies for Duchenne Muscular Dystrophy

Collaboration combines Solid’s differentiated microdystrophin construct and Ultragenyx’s HeLa PCL manufacturing platform for use with AAV8 and variants

Solid receives $40 million upfront via equity investment at a premium; up to $255 million in milestones plus royalty payments

Solid retains exclusive rights to all other uses of its microdystrophins, including its existing SGT-001 program

NOVATO, Calif. and CAMBRIDGE, Mass., Oct. 23, 2020 (GLOBE NEWSWIRE) — Ultragenyx Pharmaceutical Inc. (Nasdaq: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare diseases, and Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company focused on advancing meaningful therapies for Duchenne muscular dystrophy (Duchenne), today announced a strategic collaboration and license agreement to focus on the development and commercialization of new gene therapies for Duchenne. The parties will collaborate to develop products that combine Solid’s differentiated microdystrophin construct, Ultragenyx’s HeLa producer cell line (PCL) manufacturing platform, and AAV8 variants. The collaboration also brings together Solid’s expertise in muscle biology and Ultragenyx’s expertise in bringing novel therapies to patients with rare diseases.

Under the terms of the collaboration, Solid granted Ultragenyx an exclusive license for any pharmaceutical product that expresses Solid’s proprietary microdystrophin construct from AAV8 and variants thereof in clade E for use in the treatment of Duchenne and other diseases resulting from lack of functional dystrophin, including Becker muscular dystrophy. Ultragenyx has made a $40 million investment in Solid and has agreed to pay up to $255 million in cumulative milestone payments per product upon achievement of specified milestone events, and tiered royalties on worldwide net sales at low double digit to mid-teens percentages. Upon achievement of proof-of-concept, Solid has the right to opt-in to co-fund collaboration programs in return for participation in a profit share or increased royalty payments.

“We believe that Solid’s microdystrophin is best-in-class with its unique neuronal nitric oxide synthase binding domain,” said Emil D. Kakkis, MD, PhD, Chief Executive Officer and President of Ultragenyx. “By using an AAV8 variant validated in prior human and other studies combined with our scalable, efficient HeLa producer cell line platform, we believe we can leverage our mutual strengths to develop a high-quality AAV-based treatment alternative for Duchenne.”

“Ultragenyx has a demonstrated track record of success in developing and commercializing innovative therapies for rare diseases,” said Ilan Ganot, Co-Founder, President and Chief Executive Officer at Solid Biosciences. “We believe it is the partner of choice for exploring new gene therapy opportunities for patients with Duchenne.”

Solid’s proprietary microdystrophin construct has exhibited functional benefit in preclinical models. In preclinical studies, animals expressing a microdystrophin capable of restoring neuronal nitric oxide synthase (nNOS) resisted fatigue better than those expressing a microdystrophin that does not. Patients dosed with Solid’s proprietary microdystrophin construct at the 2E14 vg/kg dose in Solid’s ongoing IGNITE DMD clinical trial have also preliminarily demonstrated nNOS activity and function, further validating these preclinical results. Solid expects to dose the next patient in the IGNITE DMD clinical

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Aerogen Collaborates on Development of More Than 15 Potential COVID-19 Inhaled Therapies

  • Synairgen’s SNG001 among the many potential treatments and vaccines to be delivered via Aerogen’s aerosol drug delivery system

  • Adds to the thousands of COVID-19 patients already safely and effectively treated with Aerogen’s Solo aerosol drug delivery system in the intensive care unit 1

  • Aerogen technology is featured in guidelines and papers on the treatment of COVID-19 patients across the globe 2,3,4

Aerogen, the global leader in aerosol drug delivery, today announced details of a broad collaborative effort to support pharmaceutical companies from around the world as they work to develop COVID-19 vaccines and treatments. To date, more than ten million patients globally have been treated safely and effectively with Aerogen’s drug delivery technology 1,5–7. As the global scientific community rallies against the pandemic, there has been unprecedented interest in inhaled delivery of antiviral drugs, leading to Aerogen’s involvement in multiple COVID-19 drug development initiatives with leading pharmaceutical companies and prominent academic groups.

This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20201022006039/en/

Aerogen Ultra (Photo: Business Wire)

Aerogen’s closed-circuit nebulizer technology, which addresses key concerns around safety and improves patient outcomes5–8, is being used in hospitals across the globe to deliver aerosolized medication to critically-ill ventilated COVID-19 patients1. Aerogen was quick to anticipate the critical role that development of new inhaled drugs will ultimately play in the COVID-19 response, and in March of this year formed a COVID response unit to support projects researching potential treatments and vaccines. This response unit is now working with pharmaceutical companies worldwide to ensure safe delivery of inhaled therapies. Several of these collaborations are already in clinical trials, with others on track to enter studies on moderately and severely ill COVID-19 patients over the weeks and months ahead.

In one such collaboration, Aerogen has signed an agreement with Synairgen plc, a Southampton, UK-based biotechnology company, to provide the market-leading Aerogen® Solo/Ultra nebulizer system for delivery of SNG001 directly into the lungs of COVID-19 patients. SNG001 is an inhaled interferon beta that stimulates the innate immune system. Initial investigation of SNG001 as a potential COVID-19 treatment has been promising – hospitalized patients receiving SNG001 were at reduced risk of developing severe disease and more than twice as likely to recover to the ‘no limitation of activities’ level on the ordinal scale over the course of treatment9,10.

“Aerogen is a highly regarded global company known for providing safe and effective aerosol drug delivery,” said Richard Marsden, CEO of Synairgen. “Ensuring that SGN001 is paired with optimal delivery technology is a vital component of our work to bring this potential treatment to market at scale. Aerogen is our choice because of its proven reputation for drug delivery efficiency and reliability, suitability for use with a wide range of ventilatory support modalities, established high-volume manufacturing and prior regulatory approvals across the globe.”

“In the early days of the pandemic, hospitals were discouraged from using any type of aerosol for COVID-19 treatment – which

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NIH Tests Therapies to Help Cut Hospital Stays for COVID-19 Patients | Top News

(Reuters) – The U.S. National Institutes of Health (NIH) has started a late-stage trial to evaluate if immune-modulating therapies from three drugmakers can help reduce the need for ventilators for COVID-19 patients and shorten their hospital stay.

The NIH said on Friday it has selected three agents for the study – Johnson & Johnson

unit Janssen Research’s Remicade, Bristol Myers Squibb’s

Orencia and Abbvie Inc’s

experimental drug cenicriviroc.

The study will enroll up to 2,100 hospitalized adults with moderate to severe COVID-19 symptoms in the United States and Latin America.

Immune-modulating therapies are medications that alter the way the immune system works. Severe infections are believed to be triggered by an over-reaction of the immune system, known as a “cytokine storm”, and drugs that suppress certain elements of the immune system can play a role in arresting a rapid escalation of symptoms.

This can lead to acute respiratory distress syndrome and multiple organ failure, among other life-threatening complications.

The NIH said its clinical trial – ACTIV-1 Immune Modulators (IM) – will last six months, and the agency will study if the therapeutics can restore balance by modulating that immune response.

All patients will be given Gilead Sciences Inc’s

antiviral drug remdesivir – the current standard of care – and also be randomly assigned to receive a placebo or one of the immune modulators as an add-on treatment, the NIH said in a statement.

Remdesivir was one of the drugs used to treat U.S. President Donald Trump’s coronavirus infection, and has been shown in previous studies to have cut time to recovery, though the European Union is investigating it for possible kidney injury.

(Reporting by Vishwadha Chander in Bengaluru, Editing by Sherry Jacob-Phillips)

Copyright 2020 Thomson Reuters.

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