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

Platform and pipeline overview

• Utilizes a proprietary directed evolution AAV platform with over one billion capsid sequences for next-generation gene therapies.

• Active in four therapeutic areas: ophthalmology, pulmonology, cardiology, and CNS, with five clinical candidates targeting seven patient populations.

• Only therapy in ophthalmology with both FDA RMAT and EMA PRIME designations for wet AMD.

• In-house manufacturing and a strong cash position of $589 million, expected to fund operations through H1 2027.

• Pipeline includes major programs in wet AMD, DME, geographic atrophy, cystic fibrosis, alpha-1 antitrypsin, and Fabry disease.

Clinical data and program updates

• 4D-150 (wet AMD) showed 89% reduction in treatment burden, 63% injection-free, and strong safety in advanced patients over 24 weeks.

• Long-term retinal control and durability up to two years demonstrated, with confidence in extended efficacy.

• 4D-710 (cystic fibrosis) dosed 10 patients, showing improved FEV1 (+5%, +6%) in two of three with six months follow-up and dose-dependent transgene expression.

• First aerosolized gene therapy to show widespread CFTR protein in the lung, with no effect from preexisting vector immunity.

• Next 4D-710 data update expected mid-2025; Phase II to use 1E15 or lower dose based on similar efficacy and safety at reduced doses.

Investor feedback and development strategy

• Positive feedback on program pace, though some investors expected more mature data for CF.

• Early efficacy in CF program seen as promising, with stability or improvement in FEV1 compared to expected annual decline.

• Durability of effect to be further assessed with 12-month biopsies in ongoing and future patients.

• Redosing interval for lung gene therapy estimated at 1.5–3 years, based on preclinical data and pending human results.

• No change to Phase III or regulatory plans for CF; next phase will enroll up to nine patients to inform pivotal trial.