Sarepta (SRPT) announced yesterday 96 week data from its 202 Study, which is a Phase IIb open-label extension study of eteplirsen in patients with Duchenne muscular dystrophy (DMD). Sarepta will be presenting the 96-week data at the World Muscle Society meeting next month.
At Week 48, eteplirsen met its primary endpoint of increased novel dystrophin as assessed by muscle biopsy and is now in the long-term extension phase. Patients will continue to be followed for safety and clinical outcomes. The company plans to meet with the FDA soon to discuss the path forward to an NDA filing for eteplirsen, which is likely by January of 2014. However, it's possible an NDA filing could come earlier, based on the 96 week data.
I have personally spoken with a person who has a child who is affected by DMD who has shown marked improvement with the disease while taking eteplirsen.
Prosensa (RNA), which was a potential competitor of Sarepta, received bad news last week as its DMD oligonucleotide therapeutic drisapersen failed in a Phase IIIm trial named DEMAND-III. The result of this failure saw Prosensa's stock plummet 70%. Based on Prosensa's failure, and the likeliness of Sarepta's success, Sarepta's stock has rallied to as high as $50 the other day. We feel it could rally even further if an NDA is filed sooner than expected, and we think there is a decent chance of this happening.