4D Molecular Therapeutics (FDMT) Morgan Stanley 23rd Annual Global Healthcare Conference summary

Platform and pipeline overview

• Utilizes directed evolution to create customized gene therapy vectors for targeted tissue delivery, aiming for improved safety and lower costs.

• Lead candidate 4D-150 targets wet AMD and diabetic eye disease, currently in two phase III trials with strong enrollment and commercial potential.

• 4D-150 expresses aflibercept for multi-year durability and includes a VEGF-C siRNA component to potentially enhance efficacy.

• Cystic fibrosis program (4D-710) uses an aerosolized vector for lung delivery, showing high gene expression and early clinical activity, advancing into phase II.

• Cash balance of $417 million supports operations and clinical milestones through 2028.

Clinical data and safety profile

• Over 100 patients treated with 4D-150, with 80 at the phase III dose; more than 97% experienced no inflammation or toxicity.

• Significant reduction in injection burden: up to 94% reduction in newly diagnosed patients, with 80% injection-free at one year.

• Consistent dose response and robust efficacy across diverse patient populations, including severe and newly diagnosed cases.

• Safety profile has gained strong support from practitioners, enabling front-line use in large trials.

• DME safety data (zero toxicity/inflammation) further boosts confidence among investigators.

Market needs and practice integration

• Extended durability and maintaining vision gains are top unmet needs for wet AMD and DME patients.

• Retina specialists value predictable workflow; 4D-150's storage and administration align with current anti-VEGF practices, minimizing disruption.

• Steroid regimen for prophylaxis is well tolerated, accessible, and easily integrated into patient routines.

• Growing AMD prevalence and limited specialist supply highlight the need for innovations that reduce treatment burden.