Sarepta Therapeutics (NASDAQ: SRPT) reported completion of its ESSENCE confirmatory study for AMONDYS 45 and VYONDYS 53, treatments for Duchenne muscular dystrophy. The company posted third-quarter net product revenues of $370.0 million.
The ESSENCE study evaluated the efficacy and safety of the two therapies in 225 patients aged 6-13 years with Duchenne mutations amenable to exon 45 or 53 skipping. While the study did not achieve statistical significance on its primary endpoint of 4-step ascend velocity at 96 weeks, Sarepta reported positive trends favoring treatment over placebo.
The company noted the study was impacted by the COVID-19 pandemic. When excluding data from participants whose treatment period overlapped with the pandemic, the analysis showed a 30% reduction in disease progression over two years compared to placebo.
Sarepta plans to schedule a meeting with the FDA to discuss converting from accelerated to traditional approval based on the study results and real-world evidence. The company has treated over 1,800 patients worldwide with its PMO therapies.
For the third quarter, total revenues reached $399.4 million, down from $467.2 million in the prior year period. The decrease primarily reflected lower ELEVIDYS volumes following the company's decision to suspend shipments to non-ambulatory patients in the U.S. in June 2025.
The company reported a GAAP net loss of $179.9 million for the quarter, compared to net income of $33.6 million in the prior year. Non-GAAP net loss was $12.9 million versus non-GAAP net income of $69.9 million in the prior year quarter.
Sarepta completed refinancing of a portion of its 2027 convertible notes and implemented cost restructuring initiatives during the quarter. The company expects to conclude ongoing FDA discussions regarding ELEVIDYS labeling in the near term.
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