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Sarepta (SRPT) Down on Unsatisfactory Data for DMD Candidate

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Sarepta Therapeutics, Inc. (SRPT - Free Report) announced top-line results from  part 1 of  Study 102, evaluating its micro-dystrophin gene therapy candidate, SRP-9001, for treating patients with Duchenne muscular dystrophy (“DMD”), a progressive muscular weakness primarily affecting boys.

The double-blind, placebo-controlled Study 102 is evaluating the safety, efficacy and tolerability of a single dose of SRP-9001 in 41 patients with DMD. The study met the primary biological endpoint of micro-dystrophin protein expression after 12 weeks of treatment with SRP-9001 participants versus placebo. Moreover, treatment with SRP-9001 led to an increase in NSAA total score, a 17-item test and the primary functional endpoint, as compared to placebo at 48 weeks. The NSAA test measures gross motor function in boys with DMD.

However, the study did not achieve the statistical significance for this primary functional endpoint.

Shares of Sarepta plunged 50% in after-hours trading on Thursday following the announcement of the news. However, the stock has rallied 28.4% in the past year compared with the industry’s increase of 4.4%.

price chart for SRPT

Per the press release, patients treated with SRP-9001 demonstrated a statistically significant increase in NSAA total score of 1.7 points compared to baseline, while patients in the placebo arm witnessed an increase of 0.9 points on the NSAA total score as compared to baseline. However, this was not statistically significant.

Also, 85% of participants who were treated with SRP-9001 experienced at least one treatment-related adverse side effect as compared to 43% in the placebo arm.

Notably, no new safety signals were observed in part 1 of Study 102, which underlines the favorable safety and tolerability profile of SRP-9001.

We note that Swiss pharma giant Roche (RHHBY - Free Report) has signed a collaboration deal with Sarepta related to commercialization of SRP-9001 in ex-U.S. markets. In the first nine months of 2020, Sarepta recorded $61.7 million in collaboration revenues from Roche related to its licensing agreement for commercialization of SRP-9001 in ex-U.S. markets.

Though the DMD market holds promise, competition remains stiff in this space as other companies like Solid Biosciences (SLDB - Free Report) and pharma bigwig Pfizer (PFE - Free Report) are also developing gene therapies for treating DMD. Successful development of gene therapies for DMD will increase competition for Sarepta in the long run.

Zacks Rank

Sarepta currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

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