Cowen analyst Ritu Baral‘s stance on eteplirsen, Sarepta Therapeutics Inc (NASDAQ:SRPT) pipeline drug for Duchenne Muscular Dystrophy, DMD, is unchanged. This comes after Santhera announced that the FDA considered Raxone’s, a competitor’s drug for DMD, data package insufficient for NDA submission.
The FDA required data from Santhera’s “Ph3 SIDEROS trial of Raxone in DMD patients either with or without concomitant glucocorticoid therapy for its NDA filing.” They initially intended to use this data as a “confirmatory study to expand the indication.”
The FDA’s strict stance on the data set they receive shows that the Agency will not be lenient with DMD indication and require a full dataset from appropriate trials. The analyst believes the FDA’s hesitation is due to “the respiratory endpoint of the pivotal DELOS trail (change in peak expiratory flow, PEF) which has not been previously validated as an endpoint in DMD.” Furthermore, this shows that a placebo-controlled, stat sig trial does not guarantee approval in DMD and rather the Agency is more focused on “demonstrable efficacy in validated, clinically meaningful measures.”
Even with the FDA’s strict stance, the analyst does not believe it will impact the FDA’s decision on Sarepta’s eteplirsen. She feels that the result depends on the PROMOVI dystrophin biomarker data with results expected back in early September. Baral anticipates that “dystrophin production at or above the levels demonstrated in the Ph2 ‘2021/202 study in the PROMOVI data would be supportive of Subpart H accelerated approval by FDA.” If this is the case, then the rest of the PROMOVI would be a confirmatory study.
Although the analyst expects to see positive results, she remains cautious because 13 patients of “even rigorous positive dystrophin data may be too little to support an acceptable NDA.” She also expects the next relevant data regarding eteplirsen t o be released when new data is submitted to the FDA.
Baral maintained her Market Perform rating for Sarepta. According to TipRanks, the analyst has a yearly average return of 4.6% and a 42% success rate. The analyst has a 20% average loss when recommending Sarepta, and is ranked #649 out of 4,064 analysts.
TipRanks shows that out of the 14 analysts who rated Sarepta in the last 3 months, 36% gave a Buy rating, 36% gave a Hold rating and 28% gave a Sell rating. The average 12-month price target for the stock is $22.27, marking a 6.30% upside from current levels.