Aviragen, Vaxart merge to develop oral vaccines for influenza, norovirus and RSV
Tuesday, October 31, 2017 12:00 AM

For some antigens like norovirus and RSV, there are no approved vaccines. For others, like the seasonal flu, immunization rates are often far from ideal. Now, a new company, the product of a merger between Aviragen Therapeutics and Vaxart, aims to develop oral vaccines against these diseases.

After the transaction, which executives believe will close early next year, the combined company will take Vaxart’s name. Vaxart CEO Walter Latour will lead the combined company and shares will trade on Nasdaq under the proposed ticker “VXRT.”

The new Vaxart will have about $30 million in cash, which it will use to advance several key candidates in the company’s pipeline, Wouter Latour, M.D., CEO of Vaxart, said during a conference call on Monday about the transaction. Recent positive clinical data have derisked Vaxart’s platform, Aviragen CEO Joseph Patti, Ph.D., said on the call, leading the Nasdaq-listed company to pursue the merger after an extensive review of about 60 strategic options.

 
Shire Receives Orphan Drug Designation for Gene Therapy Candidate SHP654 (BAX 888) for the Treatment
Wednesday, October 25, 2017 12:00 AM

Lexington, MA – October 25, 2017 – Shire plc (LSE: SHP, NASDAQ: SHPG), the global biotechnology leader in rare diseases, today announced the U.S. Food and Drug Administration (FDA) awarded Orphan Drug Designation to Shire’s gene therapy candidate SHP654 (also designated as BAX 888), an investigational factor VIII (FVIII) gene therapy for the treatment of hemophilia A. The regulatory agency also granted Shire investigational new drug (IND) status for SHP654.

Orphan Drug Designation is intended to advance drug development for rare diseases.1 The FDA provides Orphan Drug Designation to drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S.1 Hemophilia A is the most common type of hemophilia, a bleeding disorder that causes longer-than-normal bleeding due to a lack of clotting factor VIII (FVIII) activity in the blood.2 It is estimated that approximately 14,000 people in the U.S. suffer from hemophilia A.3

“This important Orphan Drug Designation highlights Shire’s commitment to patients with rare diseases; and for hemophilia patients specifically our aim is to help them achieve zero bleeds,” says Paul Monahan, M.D., Senior Medical Director, Gene Therapy, Shire. “We know that hemophilia care is not one-size-fits-all and that every patient is unique, which is why we continue to focus on optimizing personal outcomes for hemophilia patients by developing innovations to transform care.”

Gene therapy is the delivery of a functional copy of the defective gene.4 For hemophilia, this is the gene for factor VIII or factor IX so the body can produce its own factor instead of relying on exogenous factor administration.5 The goal of hemophilia gene therapy is to provide a constant level of factor expression over several years and eliminate the peaks and troughs associated with factor replacement therapy.6 Shire is studying SHP654 as a potential treatment for hemophilia A patients through the delivery of a long-term, constant level of factor VIII expression.7

Shire also received FDA clearance for the IND application it submitted earlier this year to initiate a global multi-center study with SHP654 to evaluate the safety and optimal dose needed to boost factor VIII activity levels and affect hemophilic bleeding. The company anticipates that the SHP654 Phase 1/2 study will begin by the end of 2017.

About SHP654 (BAX 888)
Shire is developing SHP654, which includes technology acquired from Chatham Therapeutics, LLC, a spin-out of Asklepios Biopharmaceutical, IncSHP654 is an investigational factor VIII (FVIII) gene therapy intended to treat hemophilia A using a adeno-associated virus serotype 8 (AAV8) vector to deliver a codon-optimized, B-domain deleted FVIII (BDD-FVIII) specifically to a patient’s liver, where FVIII would then be produced and used to manage bleeds.7,8

About Hemophilia A
Hemophilia A, the most common type of hemophilia, is a rare bleeding disorder that causes longer-than-normal bleeding due to lack of clotting factor VIII activity in the blood.2 The severity of hemophilia A is determined by the amount of factor in the blood, with more severity associated with lower amounts of factor activity.9 More than half of patients with hemophilia A have the severe form of the condition.9 Hemophilia A primarily affects males, with an incidence of one in 5,000 male births.9,10

References

  1. U.S. Food and Drug Administration. Developing Products for Rare Diseases & Conditions FDA website. https://www.fda.gov/forindustry/developingproductsforrarediseasesconditions/default.htmAccessed October 17, 2017
  2. World Federation of Hemophilia. “What is hemophilia?” World Federation of Hemophilia website. http://www.wfh.org/en/page.aspx?pid=646. Accessed October 17, 2017.
  3. World Federation of Hemophilia. Report on the Annual Global Survey 2015. World Federation of Hemophilia website. http://www1.wfh.org/publication/files/pdf-1669.pdf. Accessed October 17, 2017.
  4. U.S. Food and Drug Administration. What is Gene Therapy? FDA website https://www.fda.gov/BiologicsBloodVaccines/CellularGeneTherapyProducts/ucm573960.htm Accessed October 17, 2017.
  5. National Hemophilia Foundation. Future Therapies. National Hemophilia Foundation website. https://www.hemophilia.org/Bleeding-Disorders/Future-Therapies Accessed October 17, 2017 
  6. Herzog R. “Hemophilia Gene Therapy: Caught Between a Cure and an Immune Response.” Molecular Therapy. 2015;23(9):1411-1412. 
  7. Falkner et al. “Development of SHP654 a highly efficient AAV8-based BDD-FVIII gene therapy vector for treatment of hemophilia A.” Available at: http://onlinelibrary.wiley.com/doi/10.1111/rth2.2017.1.issue-S1/issuetoc
  8. Hoellriegl et al. “Dose response and long-term expression of a human FVIII gene therapy construct in hemophilia A mice.” Available at: http://onlinelibrary.wiley.com/doi/10.1111/rth2.2017.1.issue-S1/issuetoc
  9. National Hemophilia Foundation. “Hemophilia A.” National Hemophilia Foundation website. https://www.hemophilia.org/Bleeding-Disorders/Types-of-Bleeding-Disorders/Hemophilia-A. Accessed October 17, 2017.
  10. Centers for Disease Control and Prevention. “Hemophilia.” Centers for Disease Control and Prevention website. http://www.cdc.gov/ncbddd/hemophilia/facts.html. Accessed October 17, 2017.

For further information please contact:

Investor Relations    
Ian Karp [email protected] +1 781 482 9018
Robert Coates [email protected] +44 1256 894874
Media    
Gwen Fisher [email protected] +1 781 482 9649
Molly Poarch [email protected] +1 312 965 3413

NOTES TO EDITORS

About Shire
Shire is the leading global biotechnology company focused on serving people with rare diseases. We strive to develop best-in-class products, many of which are available in more than 100 countries, across core therapeutic areas including Hematology, Immunology, Neuroscience, Ophthalmics, Lysosomal Storage Disorders, Gastrointestinal / Internal Medicine / Endocrine and Hereditary Angioedema; and a growing franchise in Oncology.

Our employees come to work every day with a shared mission: to develop and deliver breakthrough therapies for the hundreds of millions of people in the world affected by rare diseases and other high-need conditions, and who lack effective therapies to live their lives to the fullest.

www.shire.com

Forward-Looking Statements

Statements included herein that are not historical facts, including without limitation statements concerning future strategy, plans, objectives, expectations and intentions, the anticipated timing of clinical trials and approvals for, and the commercial potential of, inline or pipeline products, are forward-looking statements. Such forward-looking statements involve a number of risks and uncertainties and are subject to change at any time. In the event such risks or uncertainties materialize, Shire’s results could be materially adversely affected. The risks and uncertainties include, but are not limited to, the following:

  • Shire’s products may not be a commercial success;
  • increased pricing pressures and limits on patient access as a result of governmental regulations and market developments may affect Shire’s future revenues, financial condition and results of operations;
  • Shire conducts its own manufacturing operations for certain of its products and is reliant on third party contract manufacturers to manufacture other products and to provide goods and services. Some of Shire’s products or ingredients are only available from a single approved source for manufacture. Any disruption to the supply chain for any of Shire’s products may result in Shire being unable to continue marketing or developing a product or may result in Shire being unable to do so on a commercially viable basis for some period of time;
  • the manufacture of Shire’s products is subject to extensive oversight by various regulatory agencies. Regulatory approvals or interventions associated with changes to manufacturing sites, ingredients or manufacturing processes could lead to, among other things, significant delays, an increase in operating costs, lost product sales, an interruption of research activities or the delay of new product launches;
  • certain of Shire’s therapies involve lengthy and complex processes, which may prevent Shire from timely responding to market forces and effectively managing its production capacity;
  • Shire has a portfolio of products in various stages of research and development. The successful development of these products is highly uncertain and requires significant expenditures and time, and there is no guarantee that these products will receive regulatory approval;
  • the actions of certain customers could affect Shire’s ability to sell or market products profitably. Fluctuations in buying or distribution patterns by such customers can adversely affect Shire’s revenues, financial conditions or results of operations;
  • Shire’s products and product candidates face substantial competition in the product markets in which it operates, including competition from generics;
  • adverse outcomes in legal matters, tax audits and other disputes, including Shire’s ability to enforce and defend patents and other intellectual property rights required for its business, could have a material adverse effect on the Company’s revenues, financial condition or results of operations;
  • inability to successfully compete for highly qualified personnel from other companies and organizations;
  • failure to achieve the strategic objectives, including expected operating efficiencies, cost savings, revenue enhancements, synergies or other benefits at the time anticipated or at all with respect to Shire’s acquisitions, including NPS Pharmaceuticals Inc., Dyax Corp. or Baxalta Incorporated may adversely affect Shire’s financial condition and results of operations;
  • Shire’s growth strategy depends in part upon its ability to expand its product portfolio through external collaborations, which, if unsuccessful, may adversely affect the development and sale of its products;
  • a slowdown of global economic growth, or economic instability of countries in which Shire does business, as well as changes in foreign currency exchange rates and interest rates, that adversely impact the availability and cost of credit and customer purchasing and payment patterns, including the collectability of customer accounts receivable;
  • failure of a marketed product to work effectively or if such a product is the cause of adverse side effects could result in damage to Shire’s reputation, the withdrawal of the product and legal action against Shire;
  • investigations or enforcement action by regulatory authorities or law enforcement agencies relating to Shire’s activities in the highly regulated markets in which it operates may result in significant legal costs and the payment of substantial compensation or fines;
  • Shire is dependent on information technology and its systems and infrastructure face certain risks, including from service disruptions, the loss of sensitive or confidential information, cyber-attacks and other security breaches or data leakages that could have a material adverse effect on Shire’s revenues, financial condition or results of operations;
  • Shire incurred substantial additional indebtedness to finance the Baxalta acquisition, which has increased its borrowing costs may decrease its business flexibility; and

a further list and description of risks, uncertainties and other matters can be found in Shire’s most recent Annual Report on Form 10-K and in Shire’s subsequent Quarterly Reports on Form 10-Q, in each case including those risks outlined in “ITEM 1A: Risk Factors”, and in Shire’s subsequent reports on Form 8-K and other Securities and Exchange Commission filings, all of which are available on Shire’s website.

All forward-looking statements attributable to us or any person acting on our behalf are expressly qualified in their entirety by this cautionary statement. Readers are cautioned not to place undue reliance on these forward-looking statements that speak only as of the date hereof. Except to the extent otherwise required by applicable law, we do not undertake any obligation to update or revise forward-looking statements, whether as a result of new information, future events or otherwise.

 
Biden Cancer Initiative President & FasterCures Executive Director to Keynote Georgia Bio Innovation Summit October 24
Friday, October 20, 2017 12:00 AM

Atlanta, GA (October 20, 2017) – The Georgia Bio Innovation Summit will welcome patient advocate and visionary strategist Greg Simon, who leads the Biden Cancer Initiative, and Kim McCleary, Acting Executive Director at FasterCures, a center of the Milken Institute, as the plenary speakers for the October 24, 2017 event at Cobb Galleria. 

Immediately following her remarks, Ms. McCleary will moderate a panel discussion on “Engaging for Success: Patient Centricity Comes of Age” with: Robert K. Coughlin, President & CEO of the Massachusetts Biotechnology Council and a cystic fibrosis patient advocate, and Jason Spangler MD, MPH, FACPM, Executive Director for Value, Quality, & Medical Policy at Amgen.

The Summit is the largest gathering of life science leaders in the state. According to a 2016 TEConomy report, the bioscience industry in Georgia is sizable and growing, reaching nearly 30,000 jobs across 2,047 business establishments. Georgia’s bioscience and biomedical research complex is large with nearly $967 million in bioscience academic R&D. 

As many as 500 CEOs, senior executives, investors, university scientists, physicians, and public policy experts are expected to attend the day-long Summit. In addition to the keynote speakers, the Summit will feature more than 50 experts in panel discussions highlighting the full range of bioscience innovation in Georgia. The day-long program will cover the span of life science sectors including biotech/pharma, medical devices, diagnostics, academic research, agricultural biotechnology, and healthcare IT.  Details are available on the Summit website at www.georgiabiosummit.org.    

This year’s conference will offer the opportunity to hear about developments in the bioscience start-up community. The Company Investor Presentations features several Georgia-based startups describing their technologies, products and recent developments.  To get a glimpse of emerging technologies from Georgia’s universities and companies, stop by the Innovation Stageduring the exhibition breaks.  The always popular Life Science Leader Chats provide the opportunity to learn from seasoned executives about the skills every entrepreneur needs. 

The Summit serves as the only life sciences exposition in Georgia with an Exhibit Hall featuring the latest research, technology and services at companies, universities and other organizations.  The Summit also features a Scientific Poster Contest where representatives from Georgia-based academia, research institutes and industry alike will showcase their research. 

The event will close with a Casino Night Gala and Silent Auction where attendees can network and raise funds for the Georgia BioEd Institute, which provides K-12 STEM programming in bioscience and medtech throughout the state. Casino Night tickets are available onsite.

Georgia Bio (www.gabio.org) is the state’s private, non-profit life sciences association. Members include pharmaceutical, biotechnology and medical device companies, medical centers, universities and research institutes, government groups and other business organizations involved in the development of life sciences related products and services.

 Editor’s Note: Registration is complimentary for credentialed members of the news media. To request media registration, please contact Maria Thacker at [email protected] | 404-920-2042. 

 
The future of drug pricing: Value over volume
Thursday, October 19, 2017 12:00 AM

Doctors and hospitals are increasingly being paid not for the quantity of care they provide, but for the outcome or quality of care patients receive. The emerging trend in health care is about rewarding value, rather than volume. This is the future, where there is less focus on the number of tests or treatments a patient receives and more focus on whether a patient’s health is improving. 

While value-based pricing is showing great promise in other areas of health care, expanding this approach to prescription drugs is restricted by outdated federal rules. Yet this hasn’t stopped critics from labelling this innovative approach a failure before it has been given a chance to succeed. 

With a new wave of breakthrough cures and therapies expected in the coming years, ensuring prescription drugs are accessible and affordable will remain an important concern for drugmakers, policymakers, and the broader public. As the drug pricing debate continues, there are some key facts to keep in mind about a value-based model that highlight why it’s important for patients and the future of biomedical innovation.

 

Outcomes-based pricing is already happening.

This approach may be limited in the area of prescription drugs, but it isn’t new. Current value-based agreements cover patients with rheumatoid arthritis, hepatitis C, diabetes, congestive heart failure, and even a form of cancer in children and young adults. 

Roughly 20 agreements have been announced in recent months, and each is developed with differing details. For example, some result in larger rebates or lower prices if a drug does not lead to its intended results. Other arrangements let patients “try before you buy” and only require payment if the therapy works. Still others promote “pay for performance” in which a drug’s cost is tied to its effectiveness. 

These efforts are answering important questions and providing insights that will help expand this approach to more patients.

Future treatments will be more accessible and affordable. 

While nearly 90 percent of all drugs on the market are low-cost generic medicines, roughly 5 percent of patients take so-called “specialty” drugs to treat serious or life-threatening diseases. These drugs represent one-third of all drug spending, and this trend is expected to continue with the discovery of new treatments for rare diseases and other highly personalized medicines. 

Because drugmakers are accountable for patient outcomes, a value-based approach encourages insurers to ease coverage restrictions on more costly, innovative medicines. This occurs when an insurer places a drug on a lower cost-sharing tier, which reduces a patient’s out-of-pocket costs and encourages better medication adherence. What does this mean for patients? It means they have affordable access to the treatments they need and greater opportunity to enjoy healthier lives.

This will bend the cost curve in health care. 

Biomedical innovation not only saves and improves the lives of patients, it also reduces other forms of health care spending. For example, every $1 spent on medicine for congestive heart failure for adherent patients saves an estimated $8 in other health services. 

Further, the creation of the Medicare Part D prescription drug program reduced annual hospitalization costs by roughly $1.5 billion. It has been estimated that if researchers could develop a treatment to delay the onset of Alzheimer’s by five years, it would generate $367 billion in annual health care savings by 2050. 

The facts show that prescription medicines — when taken as prescribed — reduce the need for more costly health services (like hospital stays and doctor visits). By ensuring future cures are accessible and patients adhere to their medications, value-based pricing will lower the trajectory of health care spending.

Outdated federal policies are standing in the way. 

Over the years, policymakers have adopted several policies designed to protect patients and taxpayers, such as the Medicaid “Best Price” rule and restrictions on communications between drugmakers and payers. These policies may have been appropriate for the old payment model based on volume or consumption, but they also act as barriers to a value-based approach. 

Providing more legal and regulatory flexibility — while maintaining strong protections for patients and taxpayers — will expand the use of outcomes-based drug pricing, and more importantly, advance patient health by providing better access to the treatments they need.

It’s one part of a broader effort to address drug costs.

Often policymakers respond to drug costs with calls for heavy-handed government intervention that would stymie innovation and harm patients who rely on new cures and treatments. That’s why the Council for Affordable Health Coverage — a coalition including drugmakers, insurers, pharmacy benefit managers, patient groups and employers — introduced a set of consensus reforms to tackle affordability of prescription drugs. These solutions leverage the power of the free market to promote greater access to affordable medicines and will protect biomedical innovation that saves lives.

In 2016, Congress passed the 21st Century Cures ActThe law reflects years of bipartisan work to deliver new hope to patients by accelerating the discovery of new cures and treatments. To achieve the full promise of the law, policymakers will need to think creatively about how the country pays for prescription drugs. 

For the sake of patients and the future of biomedical innovation, value-based drug pricing will need to be at the center of this important effort. 

Jim Greenwood, a former six-term member of Congress from Bucks County (Pa.), is the CEO of the Biotechnology Innovation Organization (BIO), the world’s largest biotechnology trade association.

 
Intent Solutions™ Well Positioned to Address U.S. Opioid Crisis
Tuesday, October 17, 2017 12:00 AM
ATLANTA (Oct. 17, 2017) — Intent Solutions™, creator of the breakthrough medical device technology, TAD™, has rounded out its executive team in preparation for going commercial later this year, which puts the company in a promising position to address the ongoing national opioid crisis. The commercialization efforts follow the company’s successful $1.5 million seed round capital raise in 2016 and an additional $1.5M in 2017. 

“Even with great doctors and groundbreaking medications, patient results will be ineffective if they don’t take their medication as directed,” said Sam Zamarripa, Intent Solutions CEO. “We have a mission inside healthcare and research — we’re looking to alter the very definition of what it means to be adherent.”

In addition to helping address the national opioid crisis as reported by the Atlanta Journal Constitution, the medication dispenser helps facilitate clinical trials with its ability to record adherence data, which is sent to a cloud database for analysis, reducing errors and simplifying data reconciliation. 

Since his promotion to CEO from his previous post as president, Zamarripa has led the development of Intent Solutions’ market strategy, helped to attract serious investments, and hired experienced executives to complete its management team, giving the company an edge as it looks to go commercial. 

“The board’s mandate to me is to grow the company and successfully take it to market,” Zamarripa said. “With the addition of new key executive members, Intent Solutions is poised for commercial success in the areas of addiction, pain treatment, clinical trials, and beyond.”

Recent staff additions include:
Ward Broom, chief operating officer: Broom co-founded a healthcare business and has experience in taking a product from development to an exit. Broom has two degrees from the Georgia Institute of Technology and is currently completing his MBA with a focus in management of technology.
Paul Quiner, chief strategy officer and general counsel: Having spent his entire career in healthcare, both on the law and business development sides, Quiner most recently oversaw acquisitions for his former employer, McKesson, a Fortune 5 company. He brings a unique perspective on regulatory issues and overall strategy. His role with Intent Solutions will include helping to prioritize all the opportunities the company has in healthcare, putting together a solid strategy for success. 

With the new energy and expertise of its additional hires, Intent Solutions is pushing into the final frontier of medicine. Knowing the need for a solution in addition and clinical trials, the company also is studying other markets including oncology drugs, specialty pharma, chronic pain, and tracking medication for adolescents and the elderly.
For more information on Intent Solutions or the TAD device, visit www.IntentSolutions.com or contact [email protected]

About Intent Solutions:
Headquartered in Atlanta, Georgia, Intent Solutions is a technology, software, and data service company focused on developing solutions for clinical research, pain management, and specialized pharmacy. Its products markedly improve the monitoring and management of medication adherence and provide real time behavioral data to more accurately measure compliance and efficacy. For more information, visit: intentsolutions.com.
 
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