IPS HEART Receives U.S. FDA Rare Pediatric Drug Designation for ISX9-CPC Stem Cell Therapy for Treatment of Cardiomyopathy Associated with Danon disease

• Marks third Rare Pediatric Drug Designation granted by FDA for pipeline candidate

HOUSTON--(BUSINESS WIRE)--IPS HEART, a privately held cell therapy company advancing its stem cell platform to develop new skeletal muscle and cardiac muscle generation treatments for Duchenne muscular dystrophy (a rare disease) and heart failure, today announced that the U.S. Food and Drug Administration (FDA) awarded a third Rare Pediatric Drug Designation (RPDD) for its product candidate, ISX9-CPC, for treatment of cardiomyopathy associated with Danon disease. ISX9-CPC stem cell therapeutic candidate is designed to create new functional human heart muscle along with reducing cardiac fibrosis.

The FDA previously awarded RPDDs for ISX9-CPC and GIVI-MPC for the treatment of Duchenne muscular dystrophy. Separately, the FDA granted Orphan Drug Designation for its GIVI-MPC for the treatment of Duchenne muscular dystrophy.

“While the rare disease community is largely focused on gene therapy approaches, gene therapy, unfortunately, cannot create new heart muscle nor can it create any new skeletal muscle or deliver full length dystrophin,” said Rauf Ashraf, Chief Executive Officer of IPS HEART. “Large pharmaceutical companies are starting to show clinical success with IPS stem cell therapy as Vertex recently reported two diabetic patients drastically cut their A1c levels while freeing them from their insulin shots for a full year. We are hopeful similar advances will be seen in devastating rare diseases.”

About Danon disease

Danon disease is a rare fatal disease leading to sudden death or the need for cardiac transplantation by the second or third decade of life.

About Duchenne muscular dystrophy

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

About IPS HEART

IPS HEART is a privately held cell therapy company advancing its two stem cell platforms to develop new skeletal muscle and cardiac muscle treatments. ISX9-CPC is IPS HEART’s first patented drug candidate that uses the small molecule ISX-9 to reprogram iPSCs into new, functional heart muscle for the treatment of heart failure, Duchenne Cardiomyopathy and Danon Cardiomyopathy. 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.