Raring to Go: The Race to Treat Ultra-Rare Diseases

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Wednesday, October 10th, 2:00pm-2:55pm, Twin Peaks N/S

The signing of FDASIA by President Obama in July reinforces the country’s commitment to fostering innovation for rare diseases.  With a record 460 medicines in late-stage clinical trials, biopharmaceutical companies have embraced rare disease medicine and are rapidly developing the pipeline.  In anticipation of an even more compelling opportunity, many companies are taking a step beyond and focusing on ultra rare diseases. This panel will provide an in-depth analysis of this thriving market opportunity by featuring companies specializing in the ultra rare disease market alongside clinical and policy/regulatory experts in the field.

Moderator:

  • Ellen Licking, Senior Writer & Analyst, Real Endpoints

Panelists:

  • E. Cartier Esham, PhD, Senior Director, Emerging Companies Health Affairs, BIO
  • Emil Kakkis, MD, PhD, Chief Executive Officer & President, Ultragenex Pharmaceutical
  • Nick Leschly, CEO, bluebird bio
  • Hans GCP Schikan, PharmD, CEO, Prosensa
     

Who's Who

E. Cartier Esham, PhD
Senior Director, Emerging Companies Health and Regulatory Affairs, Biotechnology Industry Organization (BIO)

Cartier Esham is responsible for working on health and regulatory issues important to BIO's small biotechnology companies, the majority of which do not yet have a product in the market place. Issues of particular importance to emerging companies include intellectual property, technology transfer, clinical trials, and the Small Business Innovation Research Program. The emerging companies section also works on a wide range of FDA regulatory issues and is a strong advocate for NIH's biomedical research programs.

Prior to joining BIO, Esham was a Vice President and Director of Research at Dutko Worldwide, a private consulting firm in Washington, D.C. There she worked on a variety of environmental, education, science, technology, and health care related issues both on the federal and state/local levels. Esham has a PhD in Microbiology from the University of Georgia, a Master's degree in Marine Biology from the University of North Carolina at Wilmington, and a Bachelor of Science Degree from the University of Kentucky. She has published papers in peer-reviewed science journals on water quality, marine microbial ecology, and bacterial cloning. 

Emil Kakkis, MD, PhD
Chief Executive Officer & President, Ultragenex Pharmaceutical

Dr. Kakkis is best known for his work over the last 18years to develop novel treatments for rare disorders. He began his work developing an enzyme replacement therapy (Aldurazyme®) for the rare disorder MPS I, with minimal funding and support. The struggle to get the therapy translated from a successful canine model to patients succeeded due to the critical financial support of the Ryan Foundation, a patient organization formed by Mark and Jeanne Dant for their son Ryan.

Aldurazyme development was later supported by BioMarin Pharmaceutical and eventually their partner Genzyme leading to FDA approval in 2003. During his tenure at BioMarin, Dr. Kakkis guided the development and approval of two more treatments for rare disorders, MPS VI and PKU, and has contributed to the initiation of 7 other treatment programs for rare disorders, three of which are now in clinical development.

After 11 years at BioMarin, Dr. Kakkis left industry to initiate an effort to improve the regulatory and clinical development process for rare diseases. In early 2009, Dr. Kakkis launched and funded the Kakkis EveryLife Foundation to accelerate biotech innovation for rare diseases. The Foundation initiated a campaign to improve the regulatory and clinical development process for rare diseases. In just over a year, 160 patient organizations and physician society partners have endorsed the Campaign.

Dr. Kakkis has founded Ultragenyx™ to return to development of drugs for rare diseases. For many rare diseases, reasonable science exists that needs to get translated to patients. He will build on his previous experiences and will assemble an experienced team to efficiently develop treatments for rare diseases.

Dr. Kakkis is board certified in both Pediatrics and Medical Genetics. He graduated from Pomona College, magna cum laude and received combined MD and PhD degrees from the UCLA Medical Scientist Program and received the Bogen prize for his research. He completed a Pediatrics residency and Medical Genetics Training Fellowship at Harbor- UCLA Medical Center. He became an assistant professor of Pediatrics at Harbor-UCLA Medical Center from 1993 to 1998 where he initiated the enzyme therapy program for MPS I. In 1998, he joined BioMarin where he remained for 11 years in various titles eventually as Chief Medical Officer, before leaving in 2009.

Nick Leschly
CEO, bluebird bio

Nick Leschly is the president and CEO of bluebird bio, assuming the role in March 2010 in conjunction with Third Rock Venture’s investment in the bluebird’s Series B round. Formerly a partner of Third Rock Ventures since its founding in 2007, Nick played an integral role in the overall formation, development and business strategy of several of Third Rock’s portfolio companies, including Agios Pharmaceuticals and Edimer Pharmaceuticals. Prior to joining Third Rock, he worked at Millennium Pharmaceuticals, leading several early-stage drug development programs and served as the product and alliance leader for VELCADE™. Nick also founded and served as CEO of MedXtend Corporation. He holds a BS in molecular biology from Princeton University and an MBA from Wharton Business School.

Ellen Licking
Senior Writer & Analyst, Real Endpoints

Ellen joined Real Endpoints, a health information service company focused on product reimbursement, at its inception in December 2011. She has covered the medical industry for more than 12 years, as a reporter at BusinessWeek, then at Windhover Information, and since it’s acquisition by Elsevier, as business bureau chief and executive editor for the pharma group, where she was responsible for the editorial content of both IN VIVO and Start-Up. Ellen holds a BA in Biochemistry from Swarthmore College, an M. Phil. in Biochemistry from Cambridge University, and an MS in Biochemistry from Stanford University.

Hans GCP Schikan, PharmD
CEO, Prosensa

Hans Schikan is CEO of Prosensa, an innovative Dutch biopharmaceutical company focusing on the discovery, development and commercialization of novel treatments for rare diseases like Duchenne muscular dystrophy, myotonic dystrophy and Huntington’s disease, using its RNA modulation platform. In 2009 Prosensa announced a key agreement with GlaxoSmithKline for part of its Duchenne compounds at a value of nearly USD 700 million. Before joining Prosensa, Hans worked at Genzyme for five years in various executive roles, including as Vice President for Global Marketing and Strategic Development of Genzyme’s portfolio of products for rare genetic diseases. Prior to Genzyme, he spent 17 years at Organon, both at corporate level and in country operations which included assignments in Asia and Europe. Next to his role at Prosensa, Hans is currently Executive Board Member of the Dutch Top Institute Pharma and Non-executive Director of Swedish Orphan Biovitrum. He is also past Chairman of Nefarma, the Dutch Association of Research Based Pharmaceutical Industry. He has a PharmD from Utrecht University. Hans has given numerous presentations on orphan drugs, rare diseases and innovation.