Stem Cell Therapy Candidates for DMD Receive Rare Pediatric Drug Designation

Two new stem cell therapy candidates for the treatment of Duchenne muscular dystrophy (DMD) have received Rare Pediatric Drug designation from the US Food and Drug Administration (FDA), according to a press release from IPS HEART, the developers of the drug candidates.

“Given our successful pre-[Investigational New Drug (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,” Rauf Ashraf, the chief executive officer of IPS HEART, said. “We are in dialogue with large pharmaceutical companies and investors to partner/ invest with us to help rapidly advance both therapies to the clinic.” 

The 2 investigational therapies are called GIVI-MPC and ISX9-CPC. They are first-in-class stem cell therapeutics designed to create new skeletal muscles (GIVI-MPC) or new functional cardiac muscles (ISX9-CPC).

Read more about experimental therapies for DMD

GIVI-MPC has demonstrated the ability to create new human skeletal muscle from full-length human dystrophin in mouse and pig models of DMD, and it has been granted Orphan Drug designation by the FDA.

ISX9-CPC is a patented therapy for the treatment of heart failure and DMD cardiomyopathy. It has been shown to lead to the formation of new human heart muscles, improve ejection fraction by more than 50%, and reduce scar tissue formation by 70% ninety days following a heart attack.

DMD is a rare genetic disease caused by a mutation in the DMD gene, which codes for the essential muscle protein dystrophin. In the absence of dystrophin due to the mutation, both skeletal and cardiac muscles get damaged over time and are replaced by scar tissue.

The currently available treatments for DMD have limited therapeutic effects.

Reference

IPS HEART receives FDA Rare Pediatric Drug designations for both of its stem cell drugs for Duchenne muscular dystrophy. News release. IPS HEART; April 13, 2023.