Sarepta CEO Gives CMO Role to Former Amgen-Regeneron Alum

April 4, 2017
By Mark Terry, BioSpace.com Breaking News Staff

Cambridge, Mass.-based Sarepta Therapeutics announced that Catherine Stehman-Breen was appointed as the company’s chief medical officer. She is replacing Ed Kaye, who is currently the company’s chief executive officer.

Kay served as chief medical officer since 2011. He became interim chief executive officer in April 2015 when Chris Garabedian abruptly resigned. In September 2016, Kaye was named permanent chief executive officer after the company received U.S. Food and Drug Administration (FDA) approval for Exondys 51, the first-ever treatment for Duchenne muscular dystrophy.

Prior to joining Sarepta, Stehman-Breen was vice president, clinical development and regulatory affairs at Regeneron Pharmaceuticals , since 2015. Before that, from 2003 to 2015, she held senior leadership positions at Amgen , including vice president, global development. She led the company’s neuroscience, nephrology and bone therapeutic divisions.

“I deeply admire Sarepta’s profound commitment to improving the lives of boys with Duchenne muscular dystrophy and the exciting and innovative PMO and PPMO platform that is being harnessed to achieve this goal,” Stehman-Breen said in a statement. “I am excited to join the company at a time when it is rapidly building and look forward to working closely with the internal team and external collaborators as we seek to develop and commercialize novel therapies that address this significant unmet medical need.”

Stehman-Breen received her MD from the University of Chicago. She underwent her residency and fellowship training at the University of Washington. At the same time, she received a Master of Science in Epidemiology. From 1997 to 2003, she was faculty in the Division of Nephrology at the University of Washington, where she managed the Clinical Research Training Program and developed the Epidemiology and Clinical Trials Research Program.

The last 18 months has been dramatic for Sarepta, as it tried to get Exondys 51 (eteplirsen) approved by the FDA. It faced more than its fair share of delays and controversies, both in the public and within the FDA. Because there is no other treatment, families and DMD advocates, as well as Congress, pushed the FDA to approve. A number of scientists, including several prominent agency staffers, had significant concerns over the drug and whether Sarepta had proven its efficacy.

The primary question was the size of the clinical trials—12 boys, and two of them were basically excluded from the data analysis because the company argued they were too old for the drug to be effective anyway—and the lack of placebo controls. Nonetheless, on September 19, the FDA approved the drug, although the company will need to continue to conduct a two-year, randomized controlled trial to verify the drug’s benefits. More data is needed to prove that the drug actually improves motor functions. If that trial fails, FDA approval could be withdrawn.

The drug is now on the market with a $300,000 per year price tag, although that figure is based on the weight of the patient. The recommended dose by Humana, for example, is 30 mg/kg of body weight once weekly.

For the most part, insurers have agreed to pay for the drug. One exception is Anthem, which argued that Exondys 51 was “investigational and not medically necessary.”

Humana agreed to pay, but there are caveats. It will pay for six months of the drug if it’s covered by initial approval. Humana will cover the next six months if the patient continues to be ambulatory.

Sarepta is expanding its development operations for DMD to include gene therapy treatments, as well as therapeutics that target RNA.

The Boston Business Journal notes that Stehman-Breen doesn’t seem to have any experience with Duchenne drugs. However, Kaye said in a statement, “We are thrilled to have Dr. Stehman-Breen join Sarepta and our mission to develop treatments for boys with Duchenne muscular dystrophy. Her extensive experience in global development, clinical operations and research across multiple therapeutic areas, at leading biopharmaceutical companies, positions her well to lead our medical teams and rapidly advance our RNA-targeted platforms and gene therapy programs.”