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New DMD Drug Yields 'Compelling' Data
Research Report

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A statistical reanalysis of trial results from Sarepta Therapeutics Inc. shows improved patient function, noted an Oppenheimer report.

Sarepta Therapeutics Inc. (SRPT:NASDAQ) released new data showing patients experienced improved function at one, two, and four years out from treatment with SRP-9000, the company's investigational gene transfer therapy for Duchenne muscular dystrophy, reported Oppenheimer analyst Hartaj Singh in a July 7 research note.

"We believe this update demonstrated Sarepta's ability to successfully navigate this late stage Duchenne muscular dystrophy project and would be buyers on any weakness," Singh commented.

The update, which the drug developer presented at the 17th International Congress on Neuromuscular Diseases this week, consisted of functional data from Study 101, Part 1 of Study 102 and Study 103 (ENDEAVOR).

Singh presented the highlights of the update in his research report. Sarepta's statistical reanalysis, conducted using a plan approved by the U.S. Food and Drug Administration, of data from Study 102, Part 2 involving 20 patients "increased regulatory comfort" concerning SRP-9001," he wrote.

For the reanalysis, Sarepta used prespecified external controls and a linear regression model. In addition, the company matched the propensity score weighting based on age and scores on three tests, the North Star Ambulatory Assessment (NSAA), the 10-meter walk/run, and the rising from floor.

Also noteworthy, according to Singh, one-year data from Study 103 (of 20 patients ages four to just under eight years using commercial grade material) showed a 3.8 point improvement in NSAA score versus external control.

Four-year data from Study 101 (of four ambulatory patients ages four to seven) demonstrated a 9.9 point improvement versus a propensity-weighted external control.

An integrated analysis of all 52 patients who received a target dose of SRP-9001 while participating in either Study 101, 102, or 103 showed a 3.1 point improvement in NSAA score versus the propensity-weighted external control at one year.

"We believe the totality of the data for SRP-9001 (80-plus patients dosed) presented increases our conviction around the EMBARK study (38% probability of success, our base case scenario for approval)," Singh wrote. This is a Phase 3 pivotal global trial evaluating the safety and efficacy of SRP-9001 in Duchenne muscular dystrophy.

Sarepta identified a significant adverse event it had not seen before, myocarditis, which resolved after a month. Therefore, SRP-9001's safety profile "remains consistent across treated patients," Singh wrote.

The company and the FDA are continuing their discussions concerning possible accelerated approval of SRP-9001.

"We stay bullish," wrote Singh.

Oppenheimer has an Outperform rating and a $125 per share price target on Sarepta, the current share price of which is around $80.68.

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Disclosures for Oppenheimer & Co. Inc., Sarepta Therapeutics Inc., July 7, 2022

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