4DMT Gains Alignment with FDA on Plan to Lift Clinical Hold on Phase 1/2 INGLAXA Clinical Trial for 4D-310 for Fabry Disease Cardiomyopathy

• Initiated single non-human primate (NHP) safety study evaluating intravenous 4D-310 combined with rituximab/sirolimus (R/S) immunosuppressive regimen
• Amended INGLAXA protocol to minimize risk of atypical hemolytic uremic syndrome (aHUS) associated with intravenous (IV) AAV dosing, including addition of R/S immunosuppressive regimen
• 4D-310 combines a novel, targeted next generation AAV vector (C102) and GLA transgene for a single low dose IV delivery to cardiomyocytes
• Interim clinical data from INGLAXA expected to be presented in Q1 2024

EMERYVILLE, Calif., Oct. 30, 2023 (GLOBE NEWSWIRE) -- 4D Molecular Therapeutics (Nasdaq: FDMT, 4DMT or the Company), a clinical-stage genetic medicines company with three novel, highly targeted next generation AAV vectors currently in the clinic, today announced alignment with the U.S. Food and Drug Administration (FDA) on a plan to lift the clinical hold on the Phase 1/2 INGLAXA clinical trial in the United States for 4D-310 for Fabry disease cardiomyopathy.

“We are delighted to have reached an agreement with the FDA on our proposed plan to address the clinical hold and continue trial development,” said David Kirn, M.D., Co-founder and Chief Executive Officer of 4DMT. “We have shared with the FDA the totality of the most up to date clinical activity and safety data already generated from INGLAXA trials, as of August 2023, and look forward to progressing this critical work on behalf of all patients with Fabry disease.”

In January 2023, the Company announced that it would pause enrollment in both the United States and Asia-Pacific trials after observing instances of atypical hemolytic uremic syndrome (aHUS). Consistent with the Company’s plans, the FDA subsequently notified the Company of a clinical hold in the U.S. The Asia-Pacific clinical trial program was not placed on clinical hold by any regulatory agency.

The Company’s plan includes a single non-clinical study, currently underway, that will evaluate the safety and biodistribution in NHPs of IV 4D-310 with the R/S immunosuppressive regimen compared to the prior prednisone regimen. 4DMT expects to submit the results to the FDA in Q2 2024. In addition, the INGLAXA clinical trial protocol has been amended to minimize the risk of aHUS following IV 4D-310 dosing, including requiring the R/S immunosuppressive regimen.