Orphan Drugs: Making Rare Diseases Rarer

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Tuesday, February 12th, 9:00am - 9:55am

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 with hopes of achieving the ever elusive blockbuster drug. Some companies have even taken a step beyond and focused primarily on ultra rare disease, potentially providing an even more compelling opportunity. This panel will provide an in-depth analysis of the thriving market opportunity that is rare disease by featuring industry, clinical and regulatory experts specializing in this exciting field.

Moderator:

  • Ritu S. Baral, Principal, Sr. Analyst – Biotechnology Equity Research, Canaccord Genuity

Panelists:

  • Marc Beer, Chief Executive Officer, Aegerion Pharmaceuticals
  • John F. Crowley, Chairman & CEO, Amicus Therapeutics
  • Pamela Gavin, Senior Vice President, National Organization for Rare Disorders (NORD)
  • Hans GCP Schikan, PharmD, Chief Executive Officer, Prosensa
  • Alvin V. Shih, MD, Chief Operating Officer, Pfizer Rare Disease Research Unit

 

 

Ritu S. Baral
Principal, Sr. Analyst – Biotechnology Equity Research, Canaccord Genuity

Ritu Baral first joined Canaccord as Senior Research Associate in Biotechnology in August 2007. She has over seven years of experience in biotechnology finance, including over 3 years in biotechnology equity research. Previously, Ritu was biotechnology equity research associate at JMP Securities and before that was a Senior Associate at the Trout Group, a biotechnology focused investor relations agency. Prior to this, Ritu was a research associate at Columbia University's Department of Medicine where she participated in neuroendocrine research focused on appetite and metabolism regulation, and completed graduate coursework in Immunology. Ritu graduated with a B.A. in Biological Sciences from Barnard College.

Marc Beer
Chief Executive Officer, Aegerion Pharmaceuticals

Marc Beer is the Chief Executive Officer of Aegerion Pharmaceuticals, Inc. (NASDAQ: AEGR). Aegerion is a biopharmaceutical company dedicated to the development and commercialization of innovative, life-altering therapies for patients with debilitating often fatal, rare diseases. Marc took the company public in October 2010. The Company’s first marketed product, JUXTAPID™ (lomitapide) was approved by the U.S. Food and Drug Administration in December 2012. The company also has a Marketing Authorization Application for lomitapide under review in the EU. Marc brings over 20 years of development and commercialization experience in biotechnology, pharmaceuticals and diagnostics. In April 2000, Marc became the founding CEO of ViaCell (VIAC), a biotechnology company specializing in the collection, preservation, and development of umbilical cord blood stem cells, and over a period of seven years led the company from its inception to a rapidly growing and robust commercial organization. Under Marc's leadership, the company grew to more than 300 employees, went public in 2005 and was ultimately acquired in 2007 by PerkinElmer (PKI). Prior to founding ViaCell, Marc held various positions within Genzyme (GENZ), most recently as Vice President of Global Marketing where he was responsible for the commercial launch of several products addressing orphan disease populations on a global scale. Prior to Genzyme, he held a variety of sales and marketing roles in the pharmaceutical and diagnostic divisions of Abbott Laboratories (ABT). He also was a former member of the Mass Life Science Board of the Commonwealth of Massachusetts. Marc serves on the Biotechnology Industry Organization (BIO) Emerging Companies Section Governing Board as well as on the board of directors of Good Start Genetics, Inc. where he is Chairman; ERYtech Pharma; Strategic Consultant to OvaScience; and a member of the Graduate Studies and Research Advisory Council of Notre Dame University. Marc holds a B.S. from Miami University (Ohio).

John F. Crowley
Chairman & CEO, Amicus Therapeutics

John F. Crowley is the Chairman and CEO of Amicus Therapeutics, Inc. John's involvement with biotechnology stems from the 1998 diagnosis of two of his children with Pompe disease-a severe and often fatal neuromuscular disorder. In his drive to find a cure for them, he left his position at Bristol-Myers Squibb and became an entrepreneur as the co-founder, president and CEO of Novazyme Pharmaceuticals, a biotech start-up conducting research on a new experimental treatment for Pompe disease (which he credits as ultimately saving his children's lives.) In 2001, Novazyme merged into Genzyme Corporation and John continued to play a lead role in the development of a drug for Pompe disease as Senior Vice President, Genzyme Therapeutics.

John and his family have been profiled on the front page of The Wall Street Journal and are the subjects of a book by Pulitzer prize-winning journalist Geeta Anand, "The Cure: How a Father Raised $100 Million-And Bucked the Medical Establishment-In a Quest to Save His Children." The major motion picture, Extraordinary Measures, starring Brendan Fraser and Harrison Ford, is inspired by the Crowley family journey. John is the author of a personal memoir: Chasing Miracles: The Crowley Family Journey of Strength, Hope and Joy.

John is also a commissioned officer in the U.S. Navy Reserve, assigned to the United States Special Operations Command and is a veteran of the global war on terrorism, with service in Afghanistan. He graduated with a B.S. in Foreign Service from Georgetown University, and earned a J.D. from the University of Notre Dame Law School and an M.B.A. from Harvard. The Crowley family was the recipient of the 2011 Family Exemplar Award from the University of Notre Dame. John is a Henry Crown Fellow at the Aspen Institute. He lives in Princeton, NJ with his wife, Aileen and their three children, John, Megan and Patrick. John serves on the executive committee of the National Board of Directors of the Make A Wish Foundation of America.

 

Pamela Gavin
Senior Vice President, National Organization for Rare Disorders (NORD)

Hans GCP Schikan, PharmD
Chief Executive Officer, 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.

Alvin V. Shih, MD
Chief Operating Officer, Pfizer Rare Disease Research Unit

Alvin joined the Pfizer Rare Disease Research Unit in 2010 and currently serves as the Chief Operating Officer for the Cambridge-based R&D group.

Prior to joining Pfizer, Alvin was a senior engagement manager at L.E.K. Consulting in Boston, where he led value-creating projects for a diverse set of biopharma clients, focusing on growth strategy development, M&A strategy, transactional due diligence, and new product planning.  Alvin was previously a member of the consulting staff at McKinsey & Company, where he participated in strategic projects for a variety of healthcare clients including academic medical centers and clinical research organizations.

Alvin is a board-certified internist and completed his training at the Massachusetts General Hospital.  He holds an MD from the University of Alabama and an MBA from the Kellogg School of Management at Northwestern University.