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Shares of Sarepta Therapeutics (SRPT) closed 35% higher on Wednesday despite Wall Street raising concerns on the company’s early clinical results for two of its programs in neuromuscular diseases.
Leerink analyst Joseph Schwartz said the initial data released by the company earlier on Wednesday in genetic diseases myotonic dystrophy type 1 (DM1) and facioscapulohumeral muscular dystrophy type 1 (FSHD1) are "a bit of a mixed bag." While it seems like they can achieve "impressive" muscle concentration, the DM1 data are "very limited and safety remains an open debate," said the analyst, who has a ‘Market Perform’ rating on Sarepta shares.
Oppenheimer analyst Kostas Biliouris, meanwhile, said Sarepta's data in the programs "appear promising." However, the early nature of data and use of different sample sizes across measurements may bias the results, preventing conclusion, the analyst told investors in a research note. The firm awaits additional data in the second half of 2026 to better assess Sarepta's programs. The firm has an ‘Outperform’ rating on Sarepta with a $37 price target.
Sarepta said earlier in the day that early results from the studies of its investigational siRNA treatment SRP-1001 in FSHD1 and DM1 demonstrated dose- dependent muscle exposure, early biomarker effects, and favorable tolerability, which the company expects will lead to better clinical efficacy.
Both DM1 and FSDH1 are genetic disorders caused by overexpression of mutant proteins or toxic mRNA and characterized by progressive muscle weakness. The experimental treatment is designed to reduce, or knock down, the production of DUX4 protein in skeletal muscle.
On Stocktwits, retail sentiment around SRPT stock jumped from ‘bullish’ to ‘extremely bullish’ territory over the past 24 hours, while message volume rose from ‘low’ to ‘extremely high’ levels.
SRPT stock has dropped 68% over the past 12 months.
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