Balancing the Risk-Reward in Rare Disease Drug Development

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Monday, February 10, 2:00pm - 2:55pm  |  Jade

There were certainly positive developments in the rare diseases space last year – as we saw with the success of recent IPOs and new deals. These possibilities of large returns often outweigh the inherent risk involved, and while the path to approval is not always as straightforward as it seems, success is there to be had. Challenges also exist throughout development however, from validating endpoints and reimbursement costs, to the difficulties of trial recruitment. How do you navigate this dynamic environment? CEOs of companies paving the way will be on-hand to discuss the advantages and challenges involved in bringing rare disease treatments to market, and industry experts will dissect some of the myths that have revolved around the space.

Moderator

  • Liisa A. Bayko, Managing Director, Senior Analyst, Biotechnology, JMP Securities

Panelists

  • Seng H. Cheng, PhD, Head of Research & Early Development, Rare Diseases Division, Genzyme
  • Timothy R. Coté, MD, MPH, Principal & CEO, Coté Orphan Consulting
  • Rajesh Ranganathan, PhD, Director, Translational Research, NIH/NINDS
  • Hans GCP Schikan, PharmD, Chief Executive Officer, Prosensa

Who's Who

Liisa A. Bayko
Managing Director, Senior Analyst, Biotechnology, JMP Securities

Liisa Bayko is a Managing Director and senior research analyst covering the biotechnology industry. Prior to joining JMP, Liisa was a Director and senior biotechnology analyst at Next Generation Equity Research. She previously served as an equity research associate at Prudential Equity Group and worked as a research analyst at Baker Brothers Investments, a biotechnology-focused asset management firm. She began her career at Pharmacia Corporation as a senior analyst in the strategy development group. In 2011,

Liisa ranked first among all stock pickers in the Pharmaceuticals category and fourth in the Biotechnology category in the annual “Best on the Street” analyst survey conducted by The Wall Street Journal.

In 2010, she ranked second in the Biotechnology category. Liisa holds an MBA from the Kellogg School of Management at Northwestern University, an MS in medical biophysics from the University of Toronto and a BS with honors from Queen’s University in Kingston, Canada.

Seng H. Cheng, PhD
Head of Research & Early Development, Rare Diseases Division, Genzyme

Seng Cheng is Head of Research and Early Development of the Rare Diseases Division at Genzyme, a Sanofi Company. Dr. Cheng received his BSc, and PhD degrees in Biochemistry from the University of London, U.K. He trained as a postdoctoral fellow at the National Institute for Medical Research in London, U.K., in the field of tumor biology. He was a Staff Scientist at Integrated Genetics Inc., and later joined Genzyme Corporation to work on several discovery projects including the structure and function of the cystic fibrosis transmembrane conductance regulator. As Group Vice President of Genetic Diseases Science at Genzyme, he also managed the development of novel gene delivery vector systems as well as translational research of genetic diseases, a number of which transitioned to clinical testing. Areas of focus included inherited metabolic, muscle, lung and neurodegenerative diseases. He has published over 240 research articles and reviews, and is a named inventor on 45 issued patents in the area of biotechnology. In his current position, he is responsible for directing the translational research and early clinical development activities in rare genetic diseases.

Dr. Tim Coté

Timothy R. Coté, MD, MPH
Principal & CEO, Coté Orphan Consulting

Timothy R. Coté MD MPH is one of the leading regulatory experts in orphan drug development dedicated to assisting biotech and pharmaceutical companies through regulatory hurdles in the US and EU and specializing in helping clients obtain orphan status designation for their therapeutics. He has worked in the field for over two decades and has acquired extensive specialized knowledge of rare diseases and orphan drugs. He is a Professor of Regulatory Practice at the Keck Graduate Institute, where he also co-directs the KGI Center for Rare Disease Therapies and co-edits the Journal of Rare Diseases and Orphan Drugs.

Prior to establishing Coté Orphan Consulting, Dr. Coté served as Chief Medical Officer for the National Organization for Rare Disorders (NORD) and as the Director of the FDA’s Office of Orphan Product Development. In these positions, he advanced NORD relationships with industry, Congress, FDA and member groups and oversaw a staff of 45 and a budget of $17M in implementation of the US Orphan Drug act. Dr. Coté was instrumental in implementing the Orphan Drug Act and personally signed over 800 orphan drug designations during his time at the FDA. Before joining the FDA, Dr. Coté held positions at CDC, NIH and USDA.

Rajesh Ranganathan, PhD
Director, Translational Research, NIH/NINDS

Dr. Rajesh Ranganathan is currently the Director of the Office of Translational Research (OTR) at National Institutes for Neurological Disorders and Stroke (NINDS) at the National Institutes of Health (NIH) and has been in this role since Jan. 2012. The OTR currently has five funding programs that aim to provide a breadth of opportunities for academic investigators and small businesses to engage in the spectrum of work, beginning with assay development through to initial test of clinical candidate in human trials. The emerging priorities of the office are to ensure that therapeutic development is partnered with the development of mechanistic biomarkers to facilitate deeper understanding of the mechanism of action of the therapeutic candidate. Additionally, there will be emphasis going forward on engaging other stakeholders, such as pharma, biotech, venture capital, and patient organizations to ensure that projects in the office's portfolio have been appropriately de-risked to ensure downstream investments, which will accelerate getting the much-needed therapies to patients suffering from neurological disorders.

Previously, Dr. Ranganathan served in the NIH Office of the Director beginning in Nov. 2010 as the Senior Advisor to the NIH Director for Translational Medicine, where he led efforts to assess the translational medicine pipeline across the NIH and helped formulate an NIH-wide strategy which culminated in the formation of the new NIH National Center for Advancing Translational Sciences (NCATS). Prior to that, spent 7+ years at the Novartis Institutes for BioMedical Research Inc. where he was a Director in the scientific strategy and portfolio management group and also founded and led a Global Office of Scientific Education for the Institutes. Dr. Ranganathan received his Bachelor’s Degrees in Biology and Chemistry from Amherst College, performed his doctoral training with Dr. H. Robert Horvitz at MIT in C. elegans neurobiology, and his postdoctoral training with Dr. Linda Buck at Harvard Medical School and the Fred. Hutchinson Cancer Research Center in the area of mammalian sensory regulation.

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. 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, Non-executive Director of Sobi (Swedish Orphan Biovitrum) and Member of the Core Team of the Dutch Top Sector Life Sciences & Health. He is also past Chairman of Nefarma, the Dutch Association of Research Based Pharmaceutical Industry. He has a PharmD from Utrecht University.