As it continues its sales push for the controversial Duchenne med Exondys 51 (eteplirsen), Sarepta has taken on a new chief medical officer to help it develop its next-gen DMD pipeline.
The biotech announced this morning that Catherine Stehman-Breen, M.D., M.S., has become its new CMO after her two-year stint as VP of clinical development and regulatory affairs at Regeneron, and coming after a 12-year tenure at Amgen, where she led the neuroscience, nephrology and bone therapeutic areas.
She will take the role away from Ed Kaye, M.D., the company’s CEO, who had also been holding the dual position of chief medical officer since 2011.
“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,” said Stehman-Breen. “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.”
“We are thrilled to have Dr. Stehman-Breen join Sarepta and our mission to develop treatments for boys with Duchenne muscular dystrophy,” added Kaye. “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.”
Its current FDA-approved DMD med, which got the nod last fall despite having limited data and a negative AdComm, can only treat certain patients, namely those with the mutation of the dystrophin gene amenable to exon 51 skipping, which affects about 13% of the population with DMD.
Its pipeline is now trying to treat more boys with the genetic condition that will usually prove fatal in early adulthood, and includes research deals with Nationwide Children’s Hospital to work on their microdystrophin gene therapy program, as well as another form of gene therapy.
An initial phase 1/2a trial for the microdystrophin gene therapy is slated to begin at the end of the year and will be done at Nationwide Children’s.
It has also penned an exclusive license agreement with Nationwide for their Galgt2 gene therapy program. This early-stage program aims to research a potential surrogate gene therapy approach to DMD, whereby the gene therapy looks to induce genes that make proteins that can perform a similar function as dystrophin.
The goal here will be to produce a muscle cell that can function normally even when dystrophin is absent.