SRPT Stock Crash: ESSENCE Trial Miss Explained | Sarepta Q3 2025 Earnings & ELEVIDYS FDA Update скачать в хорошем качестве

SRPT Stock Crash: ESSENCE Trial Miss Explained | Sarepta Q3 2025 Earnings & ELEVIDYS FDA Update

*Sarepta Therapeutics (SRPT) stock plummeted over 40%* in after-hours trading following the release of its Q3 2025 earnings on Monday, November 3, 2025. The primary driver of the massive drop was the announcement that the *ESSENCE confirmatory study* for its Duchenne Muscular Dystrophy (DMD) therapies, *Amondys 45 (casimersen)* and **Vyondys 53 (golodirsen)**, failed to meet its primary endpoint with statistical significance. ESSENCE Study Results: Did the PMO Drugs Fail? The ESSENCE trial, required for traditional FDA approval for the exon-skipping drugs, evaluated efficacy and safety in 225 Duchenne patients. *Primary Endpoint Missed:* The study did not achieve statistical significance on the primary endpoint, the 4-step ascend velocity (4SA) at 96 weeks (P=0.309). *COVID-19 Impact:* Sarepta cited the *COVID-19 pandemic* as impacting the nine-year study, noting that 43% of COVID-impacted participants had an average of eight consecutive missed doses. *Positive Post-Hoc Data:* An analysis excluding COVID-impacted data (n=168) demonstrated a *meaningful treatment effect**, showing a **30% reduction in disease progression* over 2 years on the 4SA (P=0.09). *Path Forward:* Based on the encouraging trends, the favorable safety profile, and the **significant multi-year real-world evidence (RWE)**—which shows PMO therapies delaying loss of ambulation and need for nighttime ventilation—Sarepta plans to schedule a meeting with the FDA to discuss a path toward traditional approval. Q3 2025 Financial Overview Sarepta’s financial report for the quarter ending September 30, 2025, showed mixed performance: *Total Revenue:* **$399.4 million**, surpassing analyst estimates of $338.71 million. *GAAP Net Loss:* The company reported a substantial *GAAP net loss of $179.9 million* (GAAP EPS Loss of $1.80), a significant deepening from Q3 2024’s net income. *Revenue Breakdown:* Net product revenues were **$370.0 million**, including $238.5 million from PMO therapies (Amondys 45, Vyondys 53, and Exondys 51) and **$131.5 million from the gene therapy ELEVIDYS**. *Revenue Decrease:* Total revenue decreased by 15% year-over-year. This drop was largely attributed to $49.5 million less in net product revenue of ELEVIDYS following the decision to *suspend shipments to non-ambulatory patients* in June 2025. *Financial Strength:* Sarepta enhanced its financial position by refinancing its 2027 Notes and initiating cost restructuring efforts, achieving *positive cash flow* in the quarter (excluding certain collaboration costs and restructuring charges). ELEVIDYS and Pipeline Updates *Regulatory Labeling:* Labeling discussions with the FDA for the gene therapy *ELEVIDYS* (delandistrogene moxeparvovec-rokl) are nearing conclusion. The expected outcome includes a *black box warning* and the *removal of the non-ambulatory indication* from the Prescribing Information. *Enhanced Regimen Study:* Sarepta has submitted an amendment to Study 103 (ENDEAVOR) to study an *enhanced immunosuppression regimen* (including prophylactic sirolimus) in a new cohort of up to 25 non-ambulatory patients, aiming to assess the risk/benefit profile for this population. *R&D Milestones:* Preliminary data readouts for the siRNA programs *SRP-1003 (DM1)* and *SRP-1001 (FSHD)* are expected in *Early 2026**. The clinical trial for **SRP-1005 (Huntington’s Disease)* is on track to initiate by year-end 2025. --- Keywords & Tags: Sarepta Therapeutics, SRPT, SRPT stock, SRPT crash, Q3 2025 Earnings, Duchenne Muscular Dystrophy, DMD, ESSENCE study, Amondys 45, casimersen, Vyondys 53, golodirsen, PMO therapies, ELEVIDYS, gene therapy, delandistrogene moxeparvovec-rokl, FDA approval, confirmatory trial, black box warning, biotech news, stock market crash, clinical trial failure, SRP-1003, SRP-1001.