4D Molecular Therapeutics (FDMT) 2024 Cantor Fitzgerald Global Healthcare Conference summary

Key clinical and development updates

• 4D-150 demonstrated robust and durable clinical activity with strong safety across all wet AMD populations studied, including severe, broad, and recently diagnosed patients.

• Injection-free rates improved from 44% in severe, 70% in broad, to 87% in recently diagnosed patients at 52 weeks, with treatment burden reductions up to 98%.

• Safety profile remains excellent, with no significant inflammation or adverse events, and is comparable to approved anti-VEGF agents; IOI rates are favorable.

• Data cutoffs for clinical activity and safety were September 3, 2024, and August 23, 2024, respectively.

• Product candidates are in preclinical and clinical stages and not yet FDA approved.

Phase 3 study design and leadership

• 4FRONT Phase 3 program will start in Q1 next year, targeting treatment-naive wet AMD, with primary endpoint of BCVA non-inferiority and secondary endpoint of treatment burden reduction.

• Study design includes anti-VEGF responsive patients, 3 monthly loading doses, and comparator arm with Q8W dosing; 500 patients will be randomized, with supplemental aflibercept allowed in the 4D-150 arm.

• Comparator for phase III is 2 mg Eylea, as required by FDA, despite market shifts to higher doses and new agents.

• Senior ophthalmology leadership team has over 100 years of experience and multiple product approvals and launches.

• Key inclusion criteria include treatment-naïve wet AMD and BCVA of 25-78 letters.

Market and regulatory strategy

• Study design aligns with FDA and EMA expectations, leveraging prior data and expert input to maximize regulatory and commercial success.

• No need for an SPA with FDA due to strong alignment; EMA engagement ongoing with PRIME designation and agreement on study structure.

• Commercially, phase II data suggest a compelling value proposition versus current therapies, with potential for broad market appeal and pricing flexibility.

• U.S. commercialization is feasible independently, with a manageable specialist audience; ex-U.S. partnerships considered only if value accretive.

• Forward-looking statements reflect current views as of the presentation date and are subject to change.