Sarepta Therapeutics, Inc. 's SRPT shares shot up 22.3% after the company announced additional long-term efficacy and safety data from a phase IIb program on its Duchenne muscular dystrophy (DMD) candidate, eteplirsen.
Results showed that patients when treated with eteplirsen demonstrated a statistically significant 151 meter difference in the 6-minute walk test at three years compared to the control arm. Patients on eteplirsen witnessed loss in ambulation at a lower rate than the control arm over three years with pulmonary function remaining relatively stable through four years. Additionally, data from the fourth biopsy established that exon skipping occurred in 100% of patients after 180 weeks of treatment with an increase in dystrophin production in patients who received eteplirsen. On the safety front, eteplirsen was found to be well tolerated with a consistent safety profile.
We note that eteplirsen is the company's lead pipeline candidate and is currently under the priority review in the U.S. with a response expected by Feb 26, 2016. The candidate enjoys Fast Track and orphan status in the U.S. In Aug 2015, eteplirsen also received a rare pediatric disease designation from the FDA for DMD.
Eteplirsen's approval would be a huge boost for the company - it would be able to address about 13% of the total DMD population. However, we note that several companies including BioMarin Pharmaceutical Inc. BMRN are working on bringing DMD treatments to the market. In fact, BioMarin is a step ahead of Sarepta and could gain FDA approval for its DMD candidate, drisapersen, by Dec 27, 2015.
But before that, both eteplirsen and drisapersen will most likely be reviewed by the FDA's Peripheral and Central Nervous System Drugs Advisory Committee in late November. BioMarin would have a head start if approval comes on schedule. The company also received rare pediatric disease status from the FDA for drisapersen.
Sarepta currently carries a Zacks Rank #3 (Hold). A couple of better-ranked stocks in the health care sector are Regeneron Pharmaceuticals, Inc. REGN and Endo International plc ENDP , each carrying a Zacks Rank #1 (Strong Buy).