PresentationWelcome, everyone, to the 44th Annual JPMorgan Healthcare Conference. My name is Tessa Romero, and I'm one of the senior biotech analysts here at JPMorgan. Our next presenting company is Edgewise Therapeutics. And presenting on behalf of the company, we have President and CEO, Kevin Koch. Kevin, over to you.Kevin KochPresident, CEO & Director Okay. Thanks, Tessa. Always great to thanks for joining today. Let me get organized here. These are the forward-looking statements. Edgewise Therapeutics i ...

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]

Core Insights - Edgewise Therapeutics, Inc. is a leading biopharmaceutical company focused on developing novel therapeutics for muscle diseases and serious cardiac conditions [3] Group 1: Company Overview - Edgewise Therapeutics specializes in therapeutics for muscular dystrophies and cardiac conditions, leveraging deep expertise in muscle physiology [3] - The company is developing Sevasemten, a first-in-class fast skeletal myosin inhibitor, which is in late-stage clinical trials for Becker and Duchenne muscular dystrophies [3] - EDG-7500 is a novel cardiac sarcomere modulator for hypertrophic cardiomyopathy, currently in Phase 2 clinical development [3] - EDG-15400 is another cardiac sarcomere modulator aimed at treating heart failure, which is in Phase 1 clinical development [3] - The mission of Edgewise is to change the lives of patients and families affected by serious muscle diseases [3] Group 2: Event Details - Kevin Koch, Ph.D., President and CEO of Edgewise Therapeutics, will present at the 44th Annual J.P. Morgan Healthcare Conference on January 13, 2026, at 8:15 am PT [1][2] - The presentation will be available via a live webcast, with a link accessible on the Edgewise Events & Presentations page [2]

Core Insights - Edgewise Therapeutics, Inc. has appointed Christopher Martin, a biotechnology veteran, to its Board of Directors, enhancing its leadership team as it prepares for significant product launches [1][2][3] Company Overview - Edgewise Therapeutics is focused on developing novel therapeutics for muscular dystrophies and serious cardiac conditions, with a strong emphasis on muscle physiology [4] - The company is advancing its lead product, Sevasemten, which is in late-stage clinical trials for Becker and Duchenne muscular dystrophies, and is also developing EDG-7500 and EDG-15400 for cardiac conditions [4] Leadership Background - Christopher Martin previously served as Chief Commercial Officer for Verona Pharma, which was acquired by Merck & Co. for approximately $10 billion in October 2025, and has extensive experience in building commercial organizations and launch strategies [3] - His past roles include Executive Director of Marketing at SK Life Science and Head of Marketing at Cempra, where he led the development and launch strategies for multiple products [3]

Core Insights - Edgewise Therapeutics, Inc. is a leading biopharmaceutical company focused on developing novel therapeutics for muscle diseases and serious cardiac conditions [2][3]. Group 1: Investor Conferences - Management will participate in multiple investor conferences, including the Guggenheim 2nd Annual Healthcare Innovation Conference on November 11, 2025, at 8:30 am ET, the Piper Sandler 37th Annual Healthcare Conference on December 2, 2025, at 2 pm ET, and the 8th Annual Evercore Healthcare Conference on December 3, 2025, at 3:25 pm ET [1]. Group 2: Product Development - Sevasemten is an orally administered first-in-class fast skeletal myosin inhibitor in late-stage clinical trials for Becker and Duchenne muscular dystrophies [2]. - EDG-7500 is a novel cardiac sarcomere modulator for treating hypertrophic cardiomyopathy and other diseases of diastolic dysfunction, currently in Phase 2 clinical development [2]. - EDG-15400 is another cardiac sarcomere modulator for heart failure, currently in Phase 1 clinical development [2]. Group 3: Company Mission - The company is dedicated to changing the lives of patients and families affected by serious muscle diseases [2].

Summary of Arrowhead Pharmaceuticals (ARWR) 2025 Conference Call Company Overview - Arrowhead Pharmaceuticals is focused on developing RNAi-based therapeutics with a strong pipeline aimed at addressing severe diseases, particularly in the areas of hypertriglyceridemia and alpha-1 antitrypsin deficiency [2][3] Key Developments - The company has solidified its balance sheet and has capital projected to last until 2028, which is crucial for upcoming drug launches [3][4] - Planned drug launches include: - **Plazasiran** for familial chylomicronemia syndrome (FCS) expected in late 2025 - **Fazisiran** for alpha-1 antitrypsin deficiency (AAT) in 2027-2028 - **Olpassiran** with Amgen also anticipated around the same timeframe [4] Drug Pipeline Insights - **Plazasiran**: - First drug from Arrowhead's pipeline expected to receive FDA approval with a PDUFA date set for November 18, 2025 - Demonstrated significant triglyceride reduction in clinical trials, with 100% of patients showing lowered triglycerides and an average reduction of around 80% from baseline [6][7] - Safety profile appears favorable, with no anticipated advisory committee meeting [6][7] - **Shasta Five Trial**: - A dedicated trial to assess the acute pancreatitis benefit of plazasiran, targeting patients with triglycerides above 1000 mg/dL and a history of pancreatitis [24][25] - This trial is not required for regulatory approval but is seen as beneficial for payer acceptance [25] Competitive Landscape - The market for severe hypertriglyceridemia is viewed as underserved, with Arrowhead positioned to be among the first to provide effective treatments [16][17] - The company acknowledges competition but believes that having multiple players will enhance market education and acceptance [19] Safety and Efficacy Considerations - Concerns regarding HbA1c levels were addressed, with the company viewing any increases as a biological signal rather than a safety issue [22][23] - The company is focused on ensuring that the drug's efficacy translates into meaningful clinical benefits for patients [22][23] Obesity Pipeline - Arrowhead is pursuing dual targets (inhibin E and ALK7) for obesity treatment, with the rationale that both pathways could induce lipolysis and fat burning [28][30] - The company plans to evaluate which target performs better in human trials before advancing one into later-stage development [35] Alpha-1 Antitrypsin Program - The collaboration with Takeda is seen as transformative, with expectations for full enrollment in the pivotal trial by the end of the year [74][75] - The drug is anticipated to significantly impact the treatment landscape for alpha-1 antitrypsin deficiency, with a profit-sharing model in place [78] Business Development Strategy - Arrowhead aims to pursue additional business development deals to extend its runway, with a focus on maintaining a balance between new partnerships and internal drug development [79][80] - The company is open to exploring discovery components in future deals to enhance its pipeline [80] Conclusion - Arrowhead Pharmaceuticals is strategically positioned with a robust pipeline and a clear focus on addressing significant unmet medical needs in hypertriglyceridemia and alpha-1 antitrypsin deficiency, while also exploring opportunities in obesity treatment and maintaining a proactive approach to business development [2][3][4][79]