4D Molecular Therapeutics (FDMT) Barclays 28th Annual Global Healthcare Conference summary

Company overview and platform

• Focuses on next-generation gene therapy using directed evolution to create tissue-specific vectors and capsids, enabling safer dosing and targeting larger markets beyond rare diseases.

• Lead program 4D-150 targets retinal diseases, specifically wet age-related macular degeneration (AMD) and diabetic macular edema (DME), with a phase 3 trial for DME planned to start later this year.

• Additional pipeline includes a cystic fibrosis program, supported by the CF Foundation, with a phase 2 update expected in the second half of the year.

Differentiation and innovation in retinal gene therapy

• 4D-150 delivers aflibercept continuously via a single intravitreal injection, aiming for multi-year durability and potentially lifelong disease control for some patients.

• Designed for seamless integration into busy retina clinics, avoiding the need for subretinal surgery and matching current anti-VEGF delivery methods.

• Uses a proprietary vector developed in non-human primates, enabling lower dosing and improved safety.

Clinical data and efficacy

• Phase 2 PRISM data in hard-to-treat wet AMD patients showed an 80% reduction in treatment burden over two years, with vision and CST maintained.

• Recently diagnosed patients in PRISM, similar to the phase 3 population, saw a 90% reduction in treatment burden and 73% were injection-free at 18 months.

• Safety profile aligns with current standards, with intraocular inflammation rates below 3%.