Summary of Sarepta Therapeutics FY Conference Call Company Overview - **Company**: Sarepta Therapeutics (NasdaqGS:SRPT) - **Industry**: Biotechnology, specifically focused on therapies for Duchenne muscular dystrophy (DMD) Key Financial Highlights - **Total Net Product Revenue for FY 2025**: $1.86 billion, with Q4 revenue of $370 million, exceeding expectations [2][3] - **Elevidys Revenue**: $899 million for the year, a 9% increase from the previous year [2] - **PMOs Revenue**: $966 million, flat compared to the prior year despite some cannibalization from Elevidys [2] - **Cash Position**: Ended the year with $954 million in cash and cash equivalents, an increase of $89 million from the previous quarter [3] Product Performance and Opportunities - **Elevidys**: - Currently treating over 1,100 boys, with 80% of the addressable ambulatory population still untreated, indicating a significant market opportunity [4] - Aims to communicate the efficacy and disease-slowing nature of Elevidys more effectively in 2026 [5][6] - Statistical significance achieved in secondary endpoints in trials, with disease progression slowed by over 100% in some measures [8][9] - **PMOs (Exondys 51, Vyondys 53, Amondys 45)**: - Over 2,000 boys treated, with a stellar safety profile and over 90% compliance rates [16] - Proven to increase survival by nearly 5.5 years and delay loss of ambulation by 3-4 years [17] - Plans to meet with the FDA to discuss transitioning from accelerated to traditional approval [18] Pipeline Developments - **Next-Generation siRNA Pipeline**: - Focused on therapies for DMD and other conditions, with significant U.S. prevalence numbers indicating a large market opportunity [19] - Collaboration with Arrowhead's TRIM platform for superior tissue-targeting and safety [20][21] - **CNS Programs**: - Utilizing a transferrin receptor approach to cross the blood-brain barrier, with promising preclinical data for Huntington's disease [22][23][25][27] Strategic Initiatives - **Safety and Efficacy Communication**: - Plans to enhance communication regarding the efficacy of Elevidys while maintaining focus on safety [34] - Enhanced monitoring and proactive measures to improve safety profiles [36][38] - **Newborn Screening Initiative**: - Duchenne newborn screening added to the Federal Registry, which is expected to facilitate early intervention and improve patient outcomes [40][41][42] Financial Outlook - **Profitability**: - Anticipated non-GAAP profit of approximately $400 million for the year, marking the second consecutive year of profitability [28] - Expected to remain cash flow positive throughout the decade, with a strong cash balance and a $600 million revolver available for strategic opportunities [29][30] Challenges and Considerations - **2025 Challenges**: - The company faced significant challenges in 2025, including safety concerns that impacted communication about Elevidys [33][34] - Ongoing discussions with the FDA regarding the treatment of non-ambulatory patients and the potential use of sirolimus to enhance safety [47][48] Conclusion - Sarepta Therapeutics is positioned strongly in the biotechnology sector with a robust financial outlook and significant opportunities in both existing therapies and its pipeline. The company is focused on improving communication regarding the efficacy of its products while addressing safety concerns and expanding its market reach through strategic initiatives.

HOPE-3 Trial Overview - The HOPE-3 Phase 3 trial met both the primary endpoint (PUL v20) and the key secondary cardiac endpoint (LVEF), achieving statistical significance with p=003 and p=004 respectively[27] - Statistical significance was achieved in all type 1 error controlled secondary endpoints[27] - The trial involved 106 patients randomized 1:1 to Deramiocel or placebo[13, 14] - The study population consisted of late-ambulatory patients with DMD and 10MWR > 10 seconds[15] Safety Profile - In the Placebo group, 827% (43/52) experienced any Treatment Emergent Adverse Events (TEAEs), while in the Deramiocel group, 943% (50/53) experienced any TEAEs[18] - TEAEs related to the Investigational Product (IP) or administration procedure were reported in 365% (19/52) of the placebo group and 830% (44/53) of the Deramiocel group[18] - Serious TEAEs occurred in 96% (5/52) of the placebo group and 19% (1/53) of the Deramiocel group[18] Deramiocel Potential - Deramiocel is a potential first-in-class therapy designed to treat DMD skeletal and cardiomyopathy[27] - The company plans to submit a response to the Complete Response Letter incorporating HOPE-3 data, following prior alignment with the FDA[27]

Core Points - Entrada Therapeutics announced the recipients of its third annual DREAMS Grant Program, awarding $50,000 each to Jett Foundation (U.S.) and Parent Project aps (Italy) to support the Duchenne muscular dystrophy community [1][2] - The grants aim to fund projects that enhance equity, accessibility, and inclusion for underrepresented or underserved individuals living with Duchenne [1][2] Group 1: Grant Program Details - The DREAMS Grant Program is designed to support organizations working to identify and reach members of the Duchenne community globally [1][2] - The 2025 grants were announced ahead of World Duchenne Awareness Day on September 7 [1] - Jett Foundation will use the funds to support its Camp Promise program, which focuses on independent living and self-advocacy for individuals aged 18 and older [5] - Parent Project aps will utilize the grant to provide home-based care and teleconsultation services in underserved regions of Southern Italy [5] Group 2: Organizational Commitment - Entrada Therapeutics emphasizes its commitment to the Duchenne community beyond therapeutic development, engaging with patients and families to understand their needs [2] - The expansion of the grant program to include organizations from the U.S., EU, and U.K. reflects this commitment [2] - The independent grant review committee, composed of neuromuscular leaders and advocates, played a crucial role in selecting the grant recipients [4] Group 3: Organizational Background - Jett Foundation is based in Massachusetts and focuses on enriching the lives of individuals affected by Duchenne through family support services and educational initiatives [5] - Parent Project aps, founded by parents, aims to promote scientific research and improve access to care for individuals with Duchenne and Becker muscular dystrophy [5] - Entrada Therapeutics is a clinical-stage biopharmaceutical company developing a new class of medicines targeting intracellular delivery for neuromuscular diseases [6]