4DMT Presents Interim Data from 4D-310 INGLAXA Phase 1/2 Clinical Trials for Fabry Disease Cardiomyopathy at WORLDSymposium 2024

• 4D-310 demonstrated clinically meaningful cardiac endpoint improvements through 12-24 months in contractility (echocardiography), peak VO₂ (cardiopulmonary exercise testing) and/or cardiac quality of life (KCCQ) in all five evaluable patients
• Cardiac biopsies were obtained from a patient at week 6 and 24 and showed robust and durable delivery, transgene expression and clearance of Gb3 substrate in cardiomyocytes
• No new treatment-related adverse events > Grade 1 since last interim update and through 12-33 months; previously reported cases of atypical hemolytic uremic syndrome (aHUS) (n=3) fully resolved
• FDA submission of data from non-human primate (NHP) study evaluating 4D-310 with rituximab/sirolimus (R/S) immunosuppressive regimen in Q2 2024
• Efficient delivery, transgene expression and clinical activity now demonstrated in the clinic with three proprietary vectors generated from 4DMT’s Therapeutic Vector Evolution platform
  

EMERYVILLE, Calif., Feb. 12, 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 updated interim safety and efficacy data on six adults with Fabry disease cardiomyopathy treated with a single intravenous (IV) infusion of 4D-310 (1E13 vg/kg) with follow-up of 12-33 months overall, including 12-24 month follow-up data on cardiac contractility, exercise capacity, quality of life and cardiac biopsy data. The data was presented in a late-breaking session at WORLDSymposium 2024 in San Diego, California on Friday, February 9, 2024.

“We are pleased to see 4D-310 continue to consistently demonstrate clinical activity across multiple important cardiac endpoints including cardiac function, exercise capacity and quality of life,” said Robert Kim, M.D., Chief Medical Officer of 4DMT. “Current therapies do not adequately address Fabry-related cardiovascular manifestations, and cardiovascular disease is the most common cause of death in these patients. 4D-310 continues to be well tolerated, with the previously reported cases of aHUS having fully resolved and no new drug-related adverse events over Grade 1 reported.”