TLDR
- Sarepta Therapeutics stock dropped 36% premarket Tuesday following disappointing results from a nine-year clinical trial for DMD treatments.
- The study of casimersen and golodirsen in 225 patients showed numerical gains but failed to achieve statistical significance.
- This marks another setback after Elevidys gene therapy was temporarily removed from market in July following three patient deaths.
- Company blames COVID-19 pandemic for disrupting trial participation and data collection during the study period.
- Despite clinical failure, Sarepta posted Q3 revenue of $399.4 million, beating Wall Street estimates by over $60 million.
Sarepta Therapeutics experienced a massive selloff Tuesday with shares dropping 36% before market open. The decline followed news that a critical Duchenne muscular dystrophy drug trial failed to meet its primary goal.
Sarepta Therapeutics, Inc., SRPT
This latest blow adds to a rough year for the biotech firm. The stock has lost roughly 80% of its value in 2025.
The nine-year study enrolled 225 boys between 6 and 13 years old. It tested two PMO drugs called casimersen and golodirsen, which help produce dystrophin protein needed for muscle function.
After 96 weeks, patients demonstrated improvement in climbing four steps. However, the improvement wasn’t statistically significant, which is the benchmark that matters for approval decisions.
The measured difference came to only 0.05 steps per second. That tiny margin failed to convince anyone the treatments deliver meaningful benefits.
Company Points to Pandemic Interference
Sarepta officials blamed COVID-19 for compromising the trial’s integrity. They stated the pandemic interfered with patient participation and made data collection challenging.
Excluding COVID-impacted patients, the company suggests the drugs slowed disease progression by roughly 30%. Sarepta also highlighted long-term data indicating a three-year delay before patients need wheelchairs.
But selective data analysis after trial completion doesn’t typically satisfy regulators. The predetermined endpoints weren’t achieved, regardless of external factors.
This failure comes on the heels of serious trouble for Sarepta’s top product. Elevidys gene therapy was briefly withdrawn from the market in July after three patients died from acute liver failure.
Analysts at Baird expressed concern about mounting challenges. They noted both the gene therapy and PMO drug programs will face increased examination from regulators, insurers, and physicians.
Brian Abrahams from RBC Capital Markets called the miss unsurprising. He said it creates questions about the core business moving forward.
Regulatory Path Forward Remains Uncertain
Despite the setback, Sarepta intends to pursue full approval for both drugs. The company will meet with FDA officials to discuss converting current accelerated approvals to standard approvals.
Sarepta leadership maintains they aren’t worried about losing marketing authorization. They emphasize the drugs have demonstrated a strong safety profile throughout testing.
J.P. Morgan analyst Anupam Rama offered cautious optimism. He suggested pandemic-related disruptions could reasonably explain why the trial missed its target.
Rama pointed out that data excluding affected patients showed positive trends. He believes there’s a case for full approval, though he admits the regulatory landscape is unpredictable right now.
The company delivered positive financial news alongside the clinical disappointment. Third-quarter revenue totaled $399.4 million, crushing analyst projections of $338.7 million.
The earnings report showed a loss of $0.13 per share, much better than the anticipated $0.70 loss. Strong sales numbers weren’t enough to offset concerns about the pipeline though.
Analysts currently rate Sarepta stock as a Hold. The breakdown includes six Buy ratings, 13 Hold ratings, and five Sell ratings from Wall Street firms.
The consensus price target sits at $24.40 per share. That figure represents approximately where shares traded before Tuesday’s premarket plunge began.
Get 3 Free Stock Ebooks
Discover top-performing stocks in AI, Crypto, and Technology with expert analysis.
- Top 10 AI Stocks - Leading AI companies
- Top 10 Crypto Stocks - Blockchain leaders
- Top 10 Tech Stocks - Tech giants
