FDA Approvals & Rejections - Intellia Therapeutics has received FDA approval to resume its MAGNITUDE-2 Phase 3 trial for nexiguran ziclumeran (nex-z) targeting hereditary transthyretin amyloidosis with polyneuropathy, increasing target enrollment from 50 to 60 patients [2][4] - Outset Medical's next-generation Tablo Hemodialysis System has been granted FDA 510(k) clearance, making it the first dialysis device to meet enhanced cybersecurity standards, with shipping expected to begin in Q2 2026 [6][7] - OKYO Pharma has received positive feedback from the FDA for its Phase 2b/3 trial design for Urcosimod, a candidate for neuropathic corneal pain, with plans to start the trial in the first half of 2026 [8][9] - REGENXBIO has faced clinical holds on its RGX-111 and RGX-121 gene therapy programs due to a case of CNS tumor in a child treated with RGX-111, although no similar findings were reported in other patients [10][11] - Almirall has received NMPA approval for Seysara in China for treating moderate-to-severe acne vulgaris, expanding its dermatology portfolio in the region [12][13] Clinical Trials - Breakthroughs - Aprea Therapeutics reported early clinical activity for APR-1051 in endometrial cancer, achieving a 50% reduction in target lesion size in a patient with PPP2R1A-mutated uterine serous carcinoma [19][21] - Fractyl Health's Revita demonstrated positive results in weight maintenance after GLP-1 drug discontinuation, showing a 4.5% weight regain compared to 7.5% in the sham group [22][24] - Ascletis Pharma announced positive Phase 3 results for Denifanstat in moderate-to-severe acne vulgaris, focusing on long-term safety in a trial with 240 patients [25][26] - GRI Bio reported new gene expression data from its Phase 2a study of GRI-0621 in idiopathic pulmonary fibrosis, showing significant improvements in lung injury and fibrosis progression [27][28] - Cardiff Oncology announced encouraging results from its Phase 2 trial of Onvansertib in RAS-mutated metastatic colorectal cancer, with a well-tolerated regimen and plans to advance to a registrational program [31][32] - Genentech's CT-388 Phase 2 trial for obesity showed a significant placebo-adjusted weight loss of 22.5% at 48 weeks, with a high percentage of participants achieving significant weight loss [34][36] - Sarepta Therapeutics reported positive three-year results from its EMBARK study for ELEVIDYS in Duchenne muscular dystrophy, showing significant slowing of disease progression in treated patients [38][41] Deals - YD Bio Limited has signed a letter of intent to acquire Safe Save Medical for approximately $26.87 million, aiming to enhance its capabilities in advanced cellular therapeutics [14][15][17]

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... ...

Core Insights - Biotech shares experienced significant movements in after-hours trading, with Sarepta Therapeutics leading due to anticipated trial data and other companies benefiting from recent clinical and regulatory updates [1] Company Summaries - Sarepta Therapeutics Inc. (SRPT) saw a surge of 9.65% to $23.17, driven by anticipation for a webcast on January 26, 2026, where three-year topline results from its Phase 3 trial of ELEVIDYS for Duchenne muscular dystrophy will be presented, potentially pivotal for its gene therapy program [2] - Fractyl Health, Inc. (GUTS) increased by 7.66% to $2.25, indicating speculative interest despite no new company news, suggesting momentum-driven trading [3] - Greenwich LifeSciences, Inc. (GLSI) advanced 2.08% to $26.00 following FDA approval of the first commercial lot of GP2 vials for the FLAMINGO-01 trial, marking a significant step in its commercialization efforts with the potential to prepare approximately 200,000 doses [4] - Anebulo Pharmaceuticals, Inc. (ANEB) rose 8.41% to $1.16, attracting buyers despite no new updates, reflecting investor interest [5] - OmniAb, Inc. (OABI) gained 4.59% to $2.05, similar to Anebulo, indicating broader investor interest in the biotech sector without fresh news [6] - Trevi Therapeutics, Inc. (TRVI) added 3.40% to $10.33 after publishing results from its Phase 2b CORAL trial in a peer-reviewed journal, enhancing credibility and investor enthusiasm [7] - AEON Biopharma, Inc. (AEON) edged up 1.80% to $1.13, confirming a meeting with the FDA and securing shareholder approval for financing transactions, simplifying its capital structure ahead of a second PIPE financing closing expected around January 27, 2026 [8]