4D Molecular Therapeutics (FDMT) Goldman Sachs 45th Annual Global Healthcare Conference summary

Platform and pipeline overview

• Directed evolution platform enables creation of customized gene therapy vectors for targeted tissue delivery, validated in retina, lung, and heart indications.

• Three clinical-stage vectors have shown high transduction and gene expression at lower doses, supporting broad pipeline expansion.

• Ophthalmology is the primary value driver, with lead programs in wet AMD, DME, DR, and geographic atrophy, all leveraging the same R100 vector.

• Upcoming catalysts include IND filings, phase 2 and 3 data presentations, and trial initiations over the next 9-12 months.

• Regulatory designations (PRIME in Europe, RMAT in the US) facilitate phase 3 planning and engagement with agencies.

Financial strategy and resource allocation

• Recent capital raise supports phase 3 studies in wet AMD; current cash balance is $589 million.

• Cystic fibrosis program benefits from partnership and funding with the Cystic Fibrosis Foundation.

• Ophthalmology receives the largest capital allocation, with ongoing studies in wet AMD and DME covered.

• Fabry disease program is positioned for partnership opportunities as clinical hold is lifted.

Ophthalmology clinical progress and differentiation

• Phase 2 PRISM study in severe wet AMD patients showed strong safety, no significant inflammation, and superior CST stabilization compared to aflibercept.

• 90% reduction in annualized injections, with 84% of patients needing no or one injection over six months.

• Durability data show stable CST and injection-free status up to two years in some patients.

• Differentiation includes intravitreal delivery, low inflammation, and potential for long-term efficacy.

• Phase 3 initiation planned for Q1 2025, pending team expansion and regulatory alignment.