Sarepta Therapeutics Inc (NASDAQ:SRPT), a developer of innovative RNA-based therapeutics, announced that the Annals of Neurology published online positive efficacy and safety results from a Phase IIb long-term open-label extension study of eteplirsen in patients with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. The study found that at three years of treatment, patients experienced a slower rate of disease progression when compared to untreated matched historical controls and the investigational drug continued to be well-tolerated.

This analysis used historical data from the Italian Telethon Network and the Leuven Neuromuscular Reference Group for comparative analysis of 6MWT performance at baseline and Months 12, 24, and 36. Patients were matched to the eteplirsen group based on age, corticosteroid use and genotype.

At 36 months, eteplirsen-treated patients demonstrated a statistically significant difference of 151 meters in six minute walk test (6MWT), compared to the external cohort. The eteplirsen-treated patients experienced a lower incidence of loss of ambulation (16.7%) compared to natural history control patients (46.2%).

“As a clinician with extensive experience treating patients with Duchenne muscular dystrophy, it is apparent based on these data, that the boys treated with eteplirsen in this study are showing more signs of stability than would normally be expected at this stage of the disease. Remaining ambulatory is critical to greater independence over a longer period of time and a 151 meter difference indicates a marked slowing of disease progression,” said Jerry Mendell, M.D. director of the Center for Gene Therapy and Muscular Dystrophy at Nationwide Children’s Hospital, Columbus, Ohio., and lead author of the publication. “The safety and tolerability demonstrated by eteplirsen is also important, as exon skipping therapies will require lifelong dosing to be effective. For over three years, it continues to be one of the safest drugs I have used in my career.”

“Over the last 10 years, there has been an effort to harmonize standards of care, which has resulted in published guidelines that have allowed our center to generate and publish more reliable natural history data for Duchenne muscular dystrophy,” said Eugenio Mercuri, Professor of Pediatric Neurology, Università Cattolica del Sacro Cuore, Rome, Italy. “The observations of the eteplirsen-treated boys in comparison to this matched natural history group shows an encouraging difference from what one would normally expect from the natural history of the disease.”

During the follow-up period of the Phase IIb study, the most common adverse events (AE) at 36 months included flushing, erythema, and mild temperature elevation. No pulmonary embolisms, hospitalizations, injection site reactions or thrombocytopenia have been observed.

“We are pleased to have these results, which indicate that eteplirsen-treated patients have a slower disease progression at three years of treatment, published in such a prestigious peer-reviewed journal,” said Edward Kaye, M.D., Sarepta’s interim chief executive officer and chief medical officer. “The use of natural history studies will become even more important for the development of drugs for rare exons, where there is currently no suitable regulatory pathway due to limited patient populations.” (Original Source)

Shares of Sarepta Therapeutics closed yesterday at $25.94. SRPT has a 1-year high of $41.97 and a 1-year low of $11.33. The stock’s 50-day moving average is $29.32 and its 200-day moving average is $29.72.

On the ratings front, Sarepta has been the subject of a number of recent research reports. In a report issued on November 6, Roth Capital analyst Debjit Chattopadhyay reiterated a Buy rating on SRPT, with a price target of $50, which implies an upside of 92.8% from current levels. Separately, on the same day, Needham’s Chad Messer reiterated a Buy rating on the stock and has a price target of $50.

According to, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Debjit Chattopadhyay and Chad Messer have a total average return of 8.9% and -4.0% respectively. Chattopadhyay has a success rate of 49.5% and is ranked #290 out of 3847 analysts, while Messer has a success rate of 36.3% and is ranked #3445.

Overall, one research analyst has assigned a Hold rating and 9 research analysts have given a Buy rating to the stock. When considering if perhaps the stock is under or overvalued, the average price target is $49.20 which is 89.7% above where the stock closed yesterday.

Sarepta Therapeutics Inc is a biopharmaceutical company focused on the discovery and development of unique RNA-targeted therapeutics for the treatment of rare, infectious and other diseases.