This week's biotech landscape features key FDA approvals, clinical holds and go-aheads, trial discontinuations, and clinical trial data readouts across key therapeutic areas such as Endometrial cancer, Colorectal cancer, Weight Management, Acne, and Duchenne muscular dystrophy. Let us unpack the key developments and milestones in the biotech space this week.FDA Approvals & RejectionsIntellia Gets FDA Green Light to Resume MAGNITUDE-2 Phase 3 TrialIntellia Therapeutics, Inc. (NTLA) said the U.S. FDA has lif ...

Key Takeaways SRPT's phase III EMBARK study showed statistically significant gains across major functional endpoints.Sarepta Therapeutics said ~52 Elevidys patients kept NSAA scores above baseline three years post-infusion.SRPT reported disease progression slowed 73% by TTR and 70% by 10MWR versus external controls.Sarepta Therapeutics (SRPT) announced positive three-year top-line data from Part 1 of the phase III EMBARK study, evaluating Elevidys in ambulatory individuals with Duchenne muscular dystrophy ( ...

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Core Insights - The presentation focuses on the 3-year top line results from the EMBARK trial, which is a pivotal Phase III study for ELEVIDYS, highlighting the significance of the year 3 results [2] Group 1 - The results from the EMBARK trial are described as remarkably important, indicating a positive trend in long-term outcomes [2]

U.S. stocks were higher, with the Dow Jones gaining over 100 points on Monday.Shares of Sarepta Therapeutics Inc (NASDAQ:SRPT) rose sharply during Monday's session after the company released topline three-year functional results from Part 1-treated patients in the EMBARK (Study SRP-9001-301) Phase 3 study of Elevidys (delandistrogene moxeparvovec-rokl) in ambulatory individuals with Duchenne muscular dystrophy.Three years after treatment, patients who received Elevidys in Part 1 of EMBARK demonstrated stati ...

Summary of Sarepta Therapeutics EMBARK Trial Conference Call Company Overview - **Company**: Sarepta Therapeutics (NasdaqGS:SRPT) - **Focus**: Gene therapy for Duchenne muscular dystrophy (DMD) through the product ELEVIDYS Key Industry Insights - **Trial Name**: EMBARK, a Phase 3 pivotal trial - **Significance**: First long-term data measuring the disease-modifying impact of gene therapy over three years in a large, controlled clinical trial [2][3] Core Findings - **Three-Year Results**: - Patients treated with ELEVIDYS showed a **70% or greater reduction in disease progression** compared to an external control group, as measured by time to rise and 10-meter walk/run [4][5] - The North Star Ambulatory Assessment (NSAA) scores indicated that treated patients remained above baseline three years post-treatment [4][14] - A **4.39-point difference** in NSAA scores at year three compared to external controls, with a statistically significant p-value of **0.0002** [14] - Time to arise increased by **6 seconds** in the control group, while treated patients showed a significant slowing of disease progression [16] - The 10-meter walk/run assessment showed a **2.7 seconds** improvement in treated patients, with a p-value of **0.0039**, indicating a **70% slowing of disease progression** [17] Long-Term Implications - **Disease Trajectory**: The results demonstrate a sustained and growing separation from the expected decline in DMD, emphasizing the importance of early treatment to avoid irreparable muscle damage [3][5] - **Safety Profile**: No new safety signals were observed in year three, consistent with previous data from over 1,200 exposures in clinical trials [17] Additional Insights - **Patient Demographics**: Over **1,200 patients** have been treated with ELEVIDYS, ranging from ages two to adults with advanced disease [7] - **External Control Methodology**: The external control group was rigorously matched to the treated group, ensuring comparability in the analysis [12][29] - **Future Directions**: Continued long-term follow-up and data publication are planned to further inform treatment protocols and patient care [18] Educational and Communication Strategies - **Focus on Education**: The company plans to enhance communication with healthcare providers and the community regarding the efficacy and safety of ELEVIDYS, especially in light of the new three-year data [28][80] - **Sales Force Expansion**: Plans to double the sales force to improve outreach and education efforts [28] Conclusion - The three-year data from the EMBARK trial provides compelling evidence of the efficacy of ELEVIDYS in modifying the disease trajectory of DMD, highlighting the importance of early intervention and ongoing education for healthcare providers and patients [18][80]

Sarepta reports positive, statistically significant results in a study evaluating the efficacy of its blockbuster gene therapy. ...

SRP-9001-301 PART-1 3-Year Data Topline Results Doug Ingram Chief Executive Officer Louise R. Rodino-Klapac, Ph.D. President, R&D and Technical Operations James Richardson, MA (Oxon), BMBCh, MBA, MRCP (Lon) Executive Vice President, Chief Medical Officer January 26, 2026 ©SAREPTA THERAPEUTICS, INC. 2026. ALL RIGHTS RESERVED. 1 DILLON Living with Duchenne muscular dystrophy This presentation contains "forward-looking statements." Any statements that are not statements of historical fact may be deemed to be f ...

Sarepta Therapeutics' gene therapy for a progressive musclewasting disorder showed sustained benefit in patients' ability to control and coordinate movement, according to threeyear data from a late... ...

Biotech shares saw strong moves in Friday's after-hours session, with Sarepta leading on upcoming trial data and several peers advancing on recent clinical and regulatory updates.Sarepta Therapeutics Inc. (SRPT) surged 9.65% to $23.17, adding $2.04 in extended trading. The move comes ahead of a highly anticipated webcast and conference call scheduled for Monday, January 26, 2026, at 8:30 a.m. Eastern Time. Sarepta will present three-year topline functional results from Part 1 of EMBARK (Study 9001-301), it ...