Edgewise Therapeutics (NasdaqGS:EWTX) FY Conference Transcript

Edgewise Therapeutics Conference Call Summary Company Overview - Company: Edgewise Therapeutics (NasdaqGS:EWTX) - Focus: Biopharmaceutical company specializing in muscle diseases, particularly muscular dystrophy and cardiac conditions [2][4] Key Programs and Developments Muscular Dystrophy - Sevasamtan: Drug for muscular dystrophy, pivotal study enrollment completed in February 2025, data readout expected by the end of 2026 [2][4] - Duchenne Program: Positive data from studies showing decreased decline rates in patients previously treated with steroids and gene therapy [2][3] - Becker Program: Focus on a rare genetic disease with a unique mechanism of action targeting muscle damage, showing promising results in clinical trials [5][6][8] Cardiovascular Assets - EDG-7500: Cardiac sarcomere modulator with a strong safety profile, showing no changes in left ventricular ejection fraction (LVEF) [3][4][18] - EDG-15400: Second-generation molecule filed for IND, expected to enter HFpEF studies in the second half of 2026 [4][25] Financial Position - Funding: Successfully raised $200 million, with a total of $563 million in cash and no debt, providing a runway through 2028 [4][24] Market Potential - Becker Disease Market: Estimated at $5 billion, with 6,000 patients in the U.S. and 12,000 in major markets [14] - Hypertrophic Cardiomyopathy (HCM) Market: Potentially a $10 billion opportunity, targeting 165,000 symptomatic patients [23][24] Clinical Data and Efficacy - Becker Program: - 99% of patients in open-label extension remain on study, indicating durable response [8] - Statistically significant decreases in biomarkers of muscle damage [7][10] - NorthStar endpoint measures functional capacity, with a target of a 1-2 point change [8][11] - EDG-7500: - 89% of patients showed significant improvement in KCCQ scores [20] - No relationship between drug concentration and changes in ejection fraction, indicating a unique safety profile [22][23] Regulatory and Development Strategy - FDA Engagement: Plans to discuss trial designs for phase three studies in obstructive and non-obstructive HCM with the FDA [25][44] - Commercial Strategy: Building a commercial backbone for Sevasamtan while executing on HCM opportunities [49][50] Upcoming Milestones - Data Readouts: - Becker pivotal study results expected in December 2026 [25] - 12-week study data for EDG-7500 anticipated in the second quarter of 2026 [25] - NDA Submissions: Planned for 2027 following successful trial outcomes [24][25] Conclusion - Edgewise Therapeutics is positioned for significant growth with two differentiated assets in the muscular dystrophy and cardiovascular spaces, backed by strong financials and promising clinical data. The company is focused on executing its development and commercial strategies effectively to capitalize on the substantial market opportunities ahead [24][26]