Sarepta Appoints Interim CEO to Accelerate DMD Drug Process

Sarepta Therapeutics has appointed Edward Kaye, MD, as its new interim Chief Executive Officer (CEO) to lead the  effort to develop their lead product candidate, eteplirsen, for the treatment of Duchenne muscular dystrophy (DMD).

Serving as CMO since June, 2011, Kaye was in charge of the medical and clinical operations undertaken by Sarepta. He will now gather manage both positions and until the company finds a permanent CEO, given the resignation of the former president, CEO and Board member, Christopher Garabedian. The company is currently dedicated to the regulatory and clinical process regarding eteplirsen, as well as on developing other “exon” treatments for DMD, as announced in a press release.

“We believe this change will facilitate the company’s clinical and regulatory discussions and relationships with the goal of meeting its stated timelines for bringing a potentially disease-modifying treatment to patients with DMD as soon as possible,” stated the interim Chairman of Sarepta’s Board, John Hodgman.

“Dr. Kaye has a proven track record of leading teams that have brought some of the most successful rare-disease drugs to market, including Myozyme, Lumizyme and Fabrazyme,” continued Hodgman. “Further, he has excellent relationships with the clinical, regulatory and patient advocacy communities so critical to making this treatment a reality for this underserved patient population.”

Kaye is currently a Neurological Consultant at the Children’s Hospital of Boston and sits on the editorial boards of several journals, as well as on the Medical or Scientific Advisory Boards of a series of organizations, including Spinal Muscular Atrophy Foundation, CureCMD and CureDuchenne. Prior to joining Sarepta, he served as Group Vice President of Clinical Development at Genzyme Corp, where he was responsible for company’s clinical development programs for rare diseases.

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Eteplirsen, the lead drug candidate being studied by Sarepta, is meant to impact the underlying causes of DMD and facilitate the production of functional dystrophin protein, which is responsible for providing a key structure in muscle fiber function by skipping the mutation that affects the exon 51 of the dystrophin gene. Therefore, the experimental therapy is expected to restore the capacity of the gene to make a functional, albeit short form of the protein from mRNA and slow the degenerative process of muscular dystrophy.

However, last November the U.S. Food and Drug Administration (FDA) refused approval for the new DMD drug treatment, asking the company for more research. The FDA noticed discrepancies in the methodologies used by the company to study eteplirsen, and decided to request new trials, a decision that is forcing Sarepta to delay the process until at least mid-2015.

“We remain on track in collecting and analyzing the data requested by the FDA necessary for us to submit the eteplirsen New Drug Application in the middle of this year as planned,” explained Kaye. “I’m pleased to say that I have the full support of the entire executive team and the Board to optimally focus and prepare for this important milestone.

“I look forward to further strengthening our relationship with the FDA and other regulatory agencies that share our goal of doing what’s in the best interest of DMD patients and their families. I could not be more excited to take full charge of our DMD clinical and regulatory efforts, as well as to lead the advancement of our broader pipeline of treatments for rare diseases,” he added.

Kaye will now be mainly focused on fulfilling the FDA requirements for eteplirsen, as the company had already presented research findings confirming the dosages of the drug in Duchenne patients with a baseline 6-minute walk test score. Sarepta is now conducting new studies to determine Eteplirsen’s safety and efficacy when administered for one year to 160 DMD patients enrolled in several centers across the United States.