4DMT Announces FDA Clearance of IND Application for 4D-175 Genetic Medicine for the Treatment of Geographic Atrophy

• 4D-175 comprises the proprietary low-dose intravitreal R100 AAV vector and a codon-optimized transgene encoding a highly functional shortened form of human complement factor H (sCFH)
  
• Complement factor H (CFH) variants with reduced function are a well-validated genetic risk factor for geographic atrophy (GA), with approximately 75% of age-related macular degeneration (AMD) patients carrying a high-risk variant of CFH
• Over 150 patients have been treated with the R100 vector, including those with wet AMD and diabetic macular edema, further validating the modularity of our Therapeutic Vector Evolution platform
• Enrollment for Phase 1 GAZE clinical trial is expected to begin in H2 2024

EMERYVILLE, Calif., June 24, 2024 (GLOBE NEWSWIRE) -- 4D Molecular Therapeutics (Nasdaq: FDMT, 4DMT or the Company), a leading clinical-stage genetic medicines company focused on unlocking the full potential of genetic medicines to treat large market diseases, today announced U.S. Food and Drug Administration (FDA) clearance of the Investigational New Drug Application (IND) for 4D-175, an R100 vector-based intravitreal genetic medicine, for the treatment of patients with GA.

The Phase 1 GAZE clinical trial will assess 4D-175 in patients with GA secondary to AMD. The study design consists of an open-label, sequential cohort Dose Exploration stage, in which patients will receive a single intravitreal injection of 4D-175 at one of three dose levels. Clinical trial objectives include safety and tolerability, definition of the Phase 2 trial dose level(s), transgene expression and biological activity. The IND clearance enables the initiation of GAZE clinical study sites, and 4DMT expects to begin enrollment in H2 2024.

“GA is a leading cause of irreversible vision loss for over 5 million people globally and while current bolus complement inhibitor treatments reduce the rate of growth in GA lesions, they require burdensome monthly or bimonthly intravitreal injections and do not demonstrate functional vision benefit,” said David Kirn, M.D., Co-founder and Chief Executive Officer of 4DMT. “4D-175 has the potential for durable clinical benefit with a single intravitreal injection, greatly reducing the current treatment burden for patients, which may lead to better long-term vision outcomes. In Phase 1, we aim to explore safety and transgene expression levels to select doses for Phase 2. We look forward to beginning enrollment in the Phase 1 GAZE clinical trial in the second half of 2024.”