Summary of Edgewise Therapeutics FY Conference Call Company Overview - **Company**: Edgewise Therapeutics (NasdaqGS:EWTX) - **Event**: 2025 Healthcare Innovations Conference - **Date**: November 11, 2025 Key Points Industry and Product Focus - Edgewise Therapeutics is focused on developing treatments for hypertrophic cardiomyopathy (HCM) and heart failure with preserved ejection fraction (HFpEF) [1][2] PART-D Study Insights - The PART-D data is expected to be released by the end of the year, with ongoing discussions about the efficacy data from the 25 mg dose [7][9] - Initial data from the PART-BC study showed no correlation between ejection fraction changes and drug concentration, indicating a differentiated profile compared to current treatments [2][13] - The company has faced enrollment challenges but has implemented measures to ensure a representative patient group [4][6] Efficacy and Safety Observations - Significant improvements were noted in patient feel and function, as measured by New York Heart Association (NYHA) changes and Kansas City Cardiomyopathy Questionnaire (KCCQ) scores [2][21] - The compound was generally well tolerated, with a few cases of atrial fibrillation observed, which is common in HCM patients [2][3] - The company aims to eliminate the need for multiple echocardiograms in dosing, which is a barrier for many cardiology practices [14][15][20] Competitive Landscape - Edgewise's drug aims to provide a safer alternative to current treatments that require titration and monitoring of ejection fraction [14][15] - The company believes that their drug can be a first-line treatment for patients with borderline ejection fraction, potentially replacing current therapies that have safety concerns [16][17] Future Studies and Registration Plans - Plans for future studies include potentially head-to-head trials against beta blockers and similar studies to previous successful trials [27][30] - The primary endpoints for future studies will likely include KCCQ and NT-proBNP levels, with a focus on achieving significant clinical benefits [33][37] HFpEF Program Development - The company plans to initiate a phase 2A study in HFpEF, focusing on NT-proBNP as a primary measure of efficacy [36][38] - The anticipated timeline for this study is in 2026, with a focus on patient tolerability and dose range [37][38] Sarasantan and DMD Strategy - Discussions are ongoing regarding the registration study for Sarasantan in Duchenne Muscular Dystrophy (DMD), with a focus on patient backgrounds and competitive landscape [39][40] - The company is considering using SV95 as a primary endpoint for DMD studies, pending discussions with regulatory agencies [42][43] Financial Position - Edgewise Therapeutics has $560 million in cash, which is expected to fund operations through 2028, supporting their commercialization plans [47][51] Conclusion - Edgewise Therapeutics is positioned to make significant advancements in the treatment of HCM and HFpEF, with promising data and a strong financial foundation to support ongoing and future studies [52][56]

Precision BioSciences (DTIL) Conference Call Summary Company Overview - **Company**: Precision BioSciences - **Focus**: Gene editing therapies, particularly for Duchenne muscular dystrophy (DMD) and chronic hepatitis B (HBV) Key Points from the Conference Call Industry and Market Context - **Duchenne Muscular Dystrophy (DMD)**: A genetic disorder leading to progressive muscle degeneration, with approximately 300,000 to 400,000 patients globally and 15,000 new cases in the U.S. annually [9] - **Current Treatment Landscape**: Existing therapies, including microdystrophins and exon skipping, have not achieved significant long-term muscle functional improvement [7][43] Core Program Updates - **PBGene DMD**: - A clinical-stage candidate aimed at providing durable muscle functional improvement for DMD patients, focusing on correcting mutations in the dystrophin gene [5][6] - The program is designed to address the highest unmet needs in DMD, with a unique mechanism that allows for the production of a near full-length dystrophin protein [20][31] - Preclinical data shows significant improvements in muscle force output in treated mice, maintaining improvements up to nine months post-treatment [22][23] Safety and Efficacy - **Safety Profile**: The use of lower doses of AAV (adeno-associated virus) is expected to enhance safety, reducing the risk associated with high doses [35][49] - **Durability of Treatment**: The ability to edit satellite cells is crucial for long-term muscle function improvement, with evidence of increased dystrophin protein expression over time [25][28][99] Regulatory Pathway - **FDA Interactions**: Precision BioSciences has had positive discussions with the FDA regarding their clinical trial design and biomarker linkage to functional improvement [59][102] - **Clinical Trial Timeline**: Targeting to file a CTA or IND by late 2025, with clinical trials expected to commence in 2026 [61][66] Financial and Operational Updates - **Resource Allocation**: The company is prioritizing its resources towards the HBV and DMD programs, pausing the PBGene 3243 mitochondrial DNA elimination program for fiscal reasons [62][64] - **Cash Runway**: The company has sufficient cash runway into the second half of 2026 to meet Phase 1 clinical requirements [68] Additional Insights - **Patient Perspective**: The potential for improved durability and better dystrophin expression is seen as a significant advancement over current therapies, which have struggled to demonstrate clinical efficacy [46][53] - **Commercial Considerations**: Screening for neutralizing antibodies will be critical, as many patients may have previously received AAV therapies, impacting eligibility for new treatments [80][83] Conclusion - Precision BioSciences is positioned to make significant advancements in the treatment of DMD and HBV, with promising preclinical data and a clear regulatory pathway. The focus on safety, efficacy, and patient needs underscores the company's commitment to addressing high unmet medical needs in these areas.