4D Molecular Therapeutics Inc. (NASDAQ: FDMT) has announced updated preliminary clinical findings from the current Phase 1/2 clinical study of 4D-310 in patients with Fabry Diseases at the 18th WoRLDSyposim.
4D-310 targets tissues via cross-correction from continued blood AGA activity
4D-310’s dual mechanism of action allows it to heal target tissues via cross-correction from continued high blood AGA action, as well as immediate transduction and AGA production in target tissues such as the kidney, heart, and blood vessels.
Senior Vice President & Therapeutic Area Head, Lysosomal Storage Diseases and Cardiology, Raphael Schiffmann, said, “The evidence of AGA clinical activity and tolerability of 4D-310, as well as the initial encouraging effects on cardiac endpoints, strengthen our belief that 4D-310 represents a promising therapeutic approach for a broad range of patients with Fabry disease.”
Principal Investigator on the 4D-310 Phase 1/2 clinical trial and Chief of Genetic and Genomic Medicine at the University of Pittsburgh School of Medicine Jerry Vockley said, “These updated data highlight encouraging trends toward stability of high levels of blood AGA activity following discontinuation of ERT. In addition, the initial effects on cardiac endpoints suggest the design of 4D-310 has potential for benefit in the heart. ”
4D-310 is well tolerated with the promise of treating Fabry disease
These clinical findings imply that 4D-310 is well tolerated across time and can potentially treat a wide variety of Fabry disease patients. D-310 has maintained its tolerable safety profile. As of the data cutoff, no cardiac risk had been documented. Multiple blood biomarkers, electrocardiograms, and echocardiograms were used to determine cardiac safety.
4D-310 was developed for a novel dual mode of action following a single IV injection and used the tailored and modified C102 vector to provide a complete copy of the GLA gene. The product is intended to provide both sustained high blood levels of AGA for broad tissue cross-correction and a complementing high local synthesis of AGA precisely within severely damaged organs such as the heart, blood arteries, and kidneys. The product approach has the ability to meet large unmet medical requirements in Fabry disease patients.
