RARE Daily

FDA Grants Rare Pediatric Drug Designations for both of IPS HEART’s Stem Cell Drugs for Duchenne

April 14, 2023

Rare Daily Staff

The U.S. Food and Drug Administration granted Rare Pediatric Drug designation to IPS Heart for GIVI-MPC, a first-in-class stem cell therapeutic to create new skeletal muscle with 100 percent 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.

The FDA also recently granted an orphan drug designation 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 percent full length human dystrophin. GIVI-MPC has succeeded in creating new human skeletal muscle with 100 percent full length human dystrophin in both mdx mice and dystrophic pigs.

Duchenne muscular dystrophy (DMD) is a rare disease caused by mutations in the gene that encodes for dystrophin, a protein critical for the normal function of muscle cells. These mutations, the majority of which are deletions, result in the lack of dystrophin protein and progressive loss of muscle function. DMD occurs primarily in males and affects an estimated 12,000 to 15,000 individuals in the U.S. and 25,000 in Europe. Loss of strength and function typically first appears in pre-school age boys and worsens as they age. As the disease progresses, the severity of damage to skeletal and cardiac muscle often results in patients experiencing total loss of ambulation by their early teenage years and includes worsening cardiac and respiratory symptoms and loss of upper body function by the later teens. There is no cure for DMD and currently approved therapies provide limited benefit.

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 percent full-length human dystrophin for the treatment of DMD. 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.

ISX9-CPC, a patented therapeutic for heart failure and Duchenne cardiomyopathy, has created significant new human heart muscle with more than 50 percent Ejection Fraction improvement and 70 percent scar tissue reduction 90 days after heart attack. With GMP manufacturing in place from 2020, ISX9-CPC will be IPS HEART’s first drug to start 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,” said Rauf Ashraf, CEO of IPS HEART. “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 Rare Pediatric Disease designation program is offered by the FDA to encourage the development of new drugs for rare pediatric diseases (diseases affecting children 18 years of age and younger and fewer than 200,000 people in the United States). Under the RPD program, a sponsor who receives approval for a drug or biologic for a rare pediatric disease for which the RPD designation has been granted, may qualify for a priority review voucher (PRV) at the time of market approval. Holders of a PRV can redeem the voucher to obtain priority review, which shortens review from 10-months to 6-months, for any subsequent marketing application, or they can sell or transfer it to other developers. Recently, Bluebird Bio sold two PRVs, one for $95 million and one for $102 million.

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