4D Molecular provides update on Fabry Disease Cardiomyopathy

4D-310 for Fabry Disease Cardiomyopathy: Interim 4D-310 INGLAXA Phase 1/2 Clinical Trials Data in Patients with Fabry Disease: Two clinical trials, one in the U.S. and one in Taiwan and Australia are evaluating a single intravenous administration of 4D-310 in patients with classic or late-onset Fabry disease. Detailed safety data will be presented at the WORLDSymposium on February 25, 2023. Next Steps for 4D-310 Clinical Development: As of January 2023, no additional patients will be enrolled on the current Fabry disease clinical trials. The program will be evaluated in the second half of 2023 after 12-month clinical data are obtained on all six of the currently enrolled patients, including on-going safety and cardiac endpoints for a potential pivotal trial as recommended by the FDA: peak VO2, Quality-of-life and left ventricular function by global longitudinal strain. 4DMT does not plan to utilize the current corticosteroid regimen with 4D-310 in any future studies it decides to initiate. In parallel with patient followup, 4DMT will evaluate its preferred approach of utilizing the rituximab-sirolimus immune inhibition regimen with 4D-310; the rituximab and sirolimus combination is an established clinical regimen to prevent AAV-associated aHUS. 4DMT anticipates that any future clinical development of 4D-310 would be with rituximab-sirolimus under new clinical protocol(s) and an amended or new INDs.