4D Molecular Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted the RMAT designation for the investigational genetic medicine candidate 4D-150 for intravitreal treatment of wet age-related macular degeneration (wet AMD). The FDA granted RMAT designation based on the potential of 4D-150 to address the unmet medical need within this population. RMAT designation is part of the 21st Century Cures Act.

The program was created to expedite the development and review of regenerative medicine therapies intended to treat, modify, reverse or cure a serious condition. Receiving RMAT designation offers sponsor companies all the benefits of the fast track and breakthrough therapy designation programs, allowing for early, close and frequent interactions with the FDA with the goal of expediting drug development. The designation follows interim Phase 1 PRISM clinical data for 4D-150 that demonstrated an encouraging safety, tolerability and clinical activity profile.

4D-150 has the potential to address an unmet medical need to safely maintain long-term visual acuity outcomes while avoiding the need for repeated intravitreal injections. 4DMT is currently working with the FDA and EMA on preliminary Phase 3 clinical trial plans and expects to provide an update in February 2024 along with 4D-150 interim randomized Phase 2 PRISM trial data. In July 2023, 4DMT announced that a dose response was demonstrated in favor of the highest tested dose of 3E10 vg/eye, including a promising reduction in supplemental anti-VEGF injections (4 of 4 evaluable patients injection-free) and a clinically meaningful reduction in mean central subfield thickness (CST) at 36 weeks in a patient population with high anti-VEGF need.

As of July 2023, intravitreal 4D-150 remained well tolerated at all doses in all patients, with no Grade =1 inflammatory cells, hypotony, dose-limiting toxicities or treatment-related serious adverse events (SAE) during follow-up through 36 weeks in all 15 patients. Additionally, 4DMT announced that the 3E10 vg/eye dose cohort showed a durable reduction in supplemental anti-VEGF injections beyond 36 weeks, with 3 of 4 evaluable participants remaining injection-free beyond one year and one patient remaining injection-free during a maximum follow-up of 80 weeks with no change in safety profile observed.