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CAMBRIDGE - Sarepta Therapeutics, Inc. (NASDAQ:SRPT) announced Monday progress in its Phase 1/2 multiple ascending dose clinical study for SRP-1003, an investigational small interfering RNA therapeutic targeting type 1 myotonic dystrophy (DM1). The company’s shares, which have declined over 84% in the past year according to InvestingPro data, are trading at $17.69.
The company has completed the first two dosing cohorts (1.5 mg/kg and 3 mg/kg) and fully enrolled the third cohort (4.5 mg/kg), which is currently ongoing. Following a positive safety review, the study will continue with higher doses, with patients now receiving treatment in the fourth cohort (6 mg/kg).
Sarepta plans to begin dosing the final cohort (12 mg/kg) in early 2026, according to the press release statement.
The advancement triggers a $200 million milestone payment to Arrowhead Pharmaceuticals within 60 days, as the study has reached its pre-specified patient enrollment target.
SRP-1003, formerly known as ARO-DM1, is part of Sarepta’s siRNA platform focused on developing treatments for neurodegenerative and pulmonary diseases. The company’s siRNA pipeline includes investigational therapies for multiple conditions including facioscapulohumeral muscular dystrophy, spinocerebellar ataxia type 2, idiopathic pulmonary fibrosis, and Huntington’s disease.
Sarepta Therapeutics specializes in precision genetic medicine for rare diseases and holds leadership positions in Duchenne muscular dystrophy treatments. The company has an exclusive collaboration with Arrowhead Pharmaceuticals to develop therapies for skeletal muscle diseases and plans to pursue additional targets in muscle or central nervous system disorders.
In other recent news, Sarepta Therapeutics has seen several notable developments. The company announced that the U.S. Food and Drug Administration approved new labeling for its Elevidys gene therapy, which now includes a Boxed Warning for acute liver injury risks. This update follows reports of fatal liver failure in non-ambulatory patients, though the FDA maintained approval for use in ambulatory patients aged four and older. Despite safety concerns, Mizuho upgraded Sarepta’s stock to Outperform, citing strong demand for Elevidys and favorable insurance coverage. The firm raised its price target from $19.00 to $26.00, reflecting confidence in the therapy’s adoption.
Conversely, Baird lowered its price target for Sarepta to $15.00 due to the failure of the ESSENCE trial, which was a significant study in dystrophin replacement for Duchenne muscular dystrophy. Sarepta also updated the prescribing information for Elevidys, adding a boxed warning for liver injury and removing the non-ambulatory indication. Mizuho reiterated its Outperform rating despite investor concerns over new heart-safety risk warnings related to Elevidys. These recent developments highlight the complex landscape Sarepta navigates as it addresses both regulatory challenges and market opportunities.
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