HOUSTON--(BUSINESS WIRE)--IPS HEART has been awarded Orphan Drug Designation (ODD) by the FDA for GIVI-MPCs based on its unique ability to create new muscle with full length dystrophin in Becker Muscular Dystrophy(BMD). IPS HEART has now shown GIVI-MPCs create new human muscle with full length human dystrophin in dystrophic pigs, young and extremely aged Duchenne Muscular Dystrophy (DMD) mice and in a sarcopenia mouse model. The FDA previously awarded GIVI-MPCs Orphan Drug Designation for DMD, highlighting the unique platform potential of GIVI-MPCs to reverse devastating diseases. While today the market is focused almost exclusively on gene therapy delivering partial length synthetic dystrophin, after muscle loss no gene therapy can create new muscle or even deliver full length dystrophin, in contrast to the unique potential of pluripotent stem cell therapy.
IPS HEART has also been awarded Rare Pediatric Drug Designation for Duchenne cardiomyopathy as ISX9-CPCs have been shown to make new functional cardiac muscle. Given the death of both cardiac muscle and skeletal muscle in muscular dystrophy patients and the successful completion of our second pre-IND meeting with the FDA last year (attended by 4 FDA branch chiefs), which also included approving our proposed/submitted Phase I/II clinical trial design, we are committed to rapidly advancing both therapeutics to the clinic.
“With our successful FDA pre-IND meeting and our ongoing developmental efforts on both drugs, we believe that we will be the first company with bona fide disease modifying therapies to advance both drugs into human clinical trials, unlike all current drugs that largely only provide symptomatic relief by delivering novel biomarkers. We are also pursuing ongoing partnerships with various large pharmaceutical companies while attending the J.P. Morgan Healthcare Conference to partner with us to help rapidly advance both therapies to the clinic as we appreciate the many dozens of DMD patients reaching out to us directly requesting to participate in our future clinical trial,” said Rauf Ashraf, CEO of IPS HEART.
About IPS HEART/GIVI-MPCs
GIVI-MPCs form new skeletal muscle with 100% full-length human dystrophin for DMD and BMD. IPS HEART has also developed ISX-9-CPCs, a human iPSC-derived cardiac therapy for treatment of Duchenne cardiomyopathy and heart failure that develops new heart muscle.
About Becker & Duchenne Muscular Dystrophy
BMD is a muscular disease and DMD is a fatal neuromuscular disease.
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