Sarepta Makes Headway With Phase 2 Program

So far, the data demonstrated that eteplirsen provided a statistically significant advantage of 151 meters in the ability of study participants to walk at three years, compared with external controls. Additionally, the fourth biopsy data has confirmed the mechanism of action of eteplirsen, which demonstrated exon skipping in all patients and dystrophin production in nearly all patients. Safety data remained consistent with prior results.

Eteplirsen is designed to target the underlying cause of DMD by enabling the production of a functional dystrophin protein in patients with mutations amenable to exon 51 skipping. Roughly 13% of people with DMD are estimated to have a mutation targeted by eteplirsen/exon 51 skipping.

Edward Kaye, M.D., Sarepta’s interim CEO and chief medical officer, said:

We are encouraged by the positive clinical outcomes, such as the statistically significant difference in the 6MWT in eteplirsen-treated patients compared to a control, especially since we see them accompanied by data that continues to demonstrate exon skipping and dystrophin production in most patients. We are committed to bringing eteplirsen and our other investigational exon skipping therapies to patients with DMD and will continue to work with all stakeholders to advance these programs as quickly as possible so we can better address the unmet need for treatments in the DMD community.

Shares of Sarepta closed Wednesday up 0.7%, at $32.11 in a 52-week trading range of $11.33 to $41.47. In early trading indications on Thursday, shares were up 18% at $37.90. The stock has a consensus analyst price target of $41.75.

ALSO READ: 2 Deutsche Bank Health Care Focus Stocks With Big Upside Potential