IPS HEART receives FDA Rare Pediatric Drug Designations for both of its Stem Cell Drugs for Duchenne Muscular Dystrophy

HOUSTON--()--IPS HEART has been granted rare pediatric drug designation by the FDA for GIVI-MPC, a first-in-class stem cell therapeutic to create new skeletal muscle with 100% full length dystrophin and for ISX9-CPC, a first-in-class stem cell therapeutic for creating new functional cardiac muscle for the treatment of patients with Duchenne Muscular Dystrophy(DMD).

The FDA has also recently granted an orphan drug designation (ODD) to GIVI-MPC. Current treatments for DMD have limited therapeutic potential as unfortunately none can create any new functional skeletal muscle or can deliver 100% full length human dystrophin. GIVI-MPC has succeeded in creating new human skeletal muscle with 100% full length human dystrophin in both mdx mice and dystrophic pigs.

ISX9-CPC, a patented therapeutic for heart failure & Duchenne Cardiomyopathy, has created significant new human heart muscle with over 50% Ejection Fraction improvement and 70% scar tissue reduction 90 days after heart attack. With GMP manufacturing in place since 2020, ISX9-CPC will be IPS HEART’s first drug to start clinically in humans.

IPS HEART, founded in 2014, around cardiac and rare muscle diseases is on track to be the first company to advance its first in class disease modifying therapy into human clinical trials. “Given our successful pre-IND meeting with the FDA and our ongoing developmental efforts on both drugs, we believe that we will be the first company with a disease modifying therapy to advance both drugs into human clinical trials whereby all current drugs largely only provide symptomatic relief. We are in dialogue with large pharmaceutical companies & investors to partner/ invest with us to help rapidly advance both therapies to the clinic,” said Rauf Ashraf, CEO of IPS HEART.

About Duchenne Muscular Dystrophy

Duchenne muscular dystrophy is a rare, fatal neuromuscular genetic disease with patients usually succumbing to the disease in their twenties.


GIVI-MPC is IPS HEART’s second drug candidate that uses the small molecule Givinostat to reprogram human iPSCs into new skeletal muscle tissue while also delivering 100% full-length human dystrophin for the treatment of Duchenne Muscular Dystrophy. ISX9-CPC is IPS HEART’s first patented drug candidate that uses the small molecule ISX-9 to reprogram iPSC’s into new, functional heart muscle for the treatment of heart failure and Duchenne Cardiomyopathy.