financetom
Business
financetom
/
Business
/
Sarepta slumps as gene therapy setback adds to drug pipeline woes
News World Market Environment Technology Personal Finance Politics Retail Business Economy Cryptocurrency Forex Stocks Market Commodities
Sarepta slumps as gene therapy setback adds to drug pipeline woes
Nov 4, 2025 1:51 AM

(Reuters) -Shares of Sarepta sank 36% before the bell on Tuesday after a trial for two gene-targeted therapies for a muscle-wasting disease missed a key goal, deepening concerns about the company's treatment pipeline.

The latest setback comes after Sarepta's top-selling gene therapy, Elevidys, was briefly pulled from the market in July following the death of three patients from acute liver failure. The stock has lost about 80% of its value this year.

The latest trial enrolled 225 patients aged 6 to 13 with Duchenne muscular dystrophy (DMD) and tested casimersen and golodirsen, which belong to a class of drugs called phosphorodiamidate morpholino oligomers, or PMOs, designed to help produce functional dystrophin protein.

"The failure of the ... study exacerbates Sarepta's woes in the DMD space as both gene therapy and, now the PMO franchise, are likely to face increasing regulatory, payer, physician scrutiny," analysts at Baird said.

Duchenne muscular dystrophy is a rare genetic disorder that mostly affects boys, leading to progressive weakening of muscles over time.

"The miss is not entirely surprising, but adds some uncertainty around base business," said Brian Abrahams, head of global healthcare research at RBC Capital Markets.

"Pipeline may have potential, but remains early to meaningfully move the needle."

While patients showed numerical gains in climbing four steps after 96 weeks, the difference was not statistically significant.

Sarepta said the COVID-19 pandemic disrupted trial participation and data collection, potentially affecting the results and that it plans to meet with the U.S. Food and Drug Administration to discuss converting the drugs' current accelerated approvals into full approvals.

The company on Monday posted third-quarter revenue of $399.4 million, beating analysts' average expectation of $338.7 million.

(Reporting by Joel Jose in Bengaluru; Editing by Saumyadeb Chakrabarty)

Comments
Welcome to financetom comments! Please keep conversations courteous and on-topic. To fosterproductive and respectful conversations, you may see comments from our Community Managers.
Sign up to post
Sort by
Show More Comments
Related Articles >
Yum Brands begins strategic review for struggling Pizza Hut chain
Yum Brands begins strategic review for struggling Pizza Hut chain
Nov 4, 2025
(Reuters) -Yum Brands ( YUM ) said on Tuesday it was exploring strategic options for its Pizza Hut chain as the unit struggles to keep pace in a highly competitive fast-food industry vying for sales from a stressed consumer. Pizza Hut's performance indicates the need to take additional action to help the brand realize its full value, which may be...
Marathon Petroleum's Q3 Adjusted Income Increases, Total Revenue Declines
Marathon Petroleum's Q3 Adjusted Income Increases, Total Revenue Declines
Nov 4, 2025
06:53 AM EST, 11/04/2025 (MT Newswires) -- Marathon Petroleum ( MPC ) reported Q3 adjusted income Tuesday of $3.01 per diluted share, up from $1.87 a year earlier. Analysts polled by FactSet expected $3.15. Total revenue and other income for the quarter ended Sept. 30 were $35.85 billion, compared with $35.37 billion a year earlier. Analysts polled by FactSet expected...
Pfizer beats Q3 estimates, hikes FY profit outlook
Pfizer beats Q3 estimates, hikes FY profit outlook
Nov 4, 2025
Overview * Pfizer ( PFE ) Q3 revenue of $16.65 bln beats analyst expectations despite a 6% yr/yr decline * Adjusted EPS for Q3 beats consensus, with strong operational performance * Company raises full-year 2025 adjusted EPS guidance, reflecting confidence in business Outlook * Pfizer ( PFE ) reaffirms full-year 2025 revenue guidance of $61.0 to $64.0 bln * Raises...
Harley-Davidson's third-quarter profit rises
Harley-Davidson's third-quarter profit rises
Nov 4, 2025
Nov 4 (Reuters) - Harley-Davidson ( HOG ) reported a higher third-quarter profit on Tuesday, helped by cost-saving measures and strong margins from its custom touring models. Its quarterly profit rose to $377 million, or $3.10 per share, from $119 million, or 91 cents per share, a year earlier. ...
Copyright 2023-2026 - www.financetom.com All Rights Reserved