Summary of 4D Molecular Therapeutics Conference Call Company Overview - **Company**: 4D Molecular Therapeutics (FDMT) - **Focus**: Gene therapy portfolio, specifically targeting neovascular diseases and cystic fibrosis Key Programs 1. **4,150 for Neovascular Diseases** - Targets wet AMD and diabetic eye disease - Currently in two Phase III trials (FORFORNTA I and FORFORNTA II) - Strong enthusiasm and enrollment observed - Recent survey indicated 50% of physicians view gene therapy as the most exciting novel approach for wet AMD - Expected completion of enrollment for FORFORNTA I in Q1 2026, with a 52-week endpoint anticipated in 2027 [3][4] 2. **4,710 for Cystic Fibrosis** - An aerosol-delivered vector for cystic fibrosis patients, particularly those not amenable to modulators - Currently in a Phase I dose-finding study - Initial doses were found to be overdosed; adjustments made to lower doses for better physiological expression - Expected readouts on lung function and quality of life in Q4 2025 [4][38] Clinical Trial Insights - **4,150 Program** - Strong dose response observed in Phase I and II trials, with treatment burden reduction between 83% to 94% - Safety profile is robust, with less than 3% transient inflammation reported [6][7] - Changes from Phase II to Phase III include refined patient population and minor tweaks to reinjection criteria to enhance safety and efficacy [12][14] - **4,710 Program** - High expression levels observed initially, but adjustments made to avoid overdosing - Focus on long-term data from patients at various dose levels, with follow-up ranging from 3 to 18 months [39][44] - Emphasis on multiple endpoints including FEV1, Lung Clearance Index, and quality of life metrics [46][50] Market Position and Strategy - The company is well-positioned with cash reserves extending into 2028, allowing for continued development of its gene therapy programs [3] - The focus is on large market opportunities rather than rare diseases, reflecting a strategic shift in the pharmaceutical landscape [62] Regulatory Considerations - The company aims to align with FDA and EMA guidelines, particularly regarding trial designs and patient populations [20][23] - Emphasis on demonstrating significant reductions in treatment burden to meet regulatory expectations [36][37] Future Directions - Potential for collaborations in gene therapy, but with a focus on maintaining the integrity of their own product development [62] - Plans for post-approval studies to assess real-world efficacy and safety of their therapies [26][27] Conclusion 4D Molecular Therapeutics is advancing its gene therapy programs with promising data and a strategic focus on large market opportunities. The company is committed to refining its clinical trials and ensuring alignment with regulatory expectations to maximize the potential of its innovative therapies.