IPS HEART wins rare pediatric drug designation for stem cell therapies

Both GIVI-MPC and ISX9-CPC were awarded orphan drug status by FDA

Margarida Maia, PhD avatar

by Margarida Maia, PhD |

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IPS Heart has received rare pediatric drug designation for its GIVI-MPC and ISX9-CPC, two experimental single-course stem cell therapies for Duchenne muscular dystrophy (DMD).

The designation is given by the U.S. Food and Drug Administration (FDA) to spur treatments for any rare disease that can be serious or life-threatening for people from birth to age 18. With this incentive, developers may request a voucher when applying for regulatory approval, which can be used for a faster review of a subsequent application of another treatment or transferred to another company.

As part of the process, the company held a pre-investigational new drug (IND) application meeting with the agency, which occurs early in development to help decide on the best strategies to begin testing in humans.

“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,” Rauf Ashraf, CEO of IPS Heart, said in a company press release. “We are in dialogue with large pharmaceutical companies [and] investors to partner/invest with us to help rapidly advance both therapies to the clinic.”

DMD is a type of muscular dystrophy caused by mutations in a gene that codes for the protein dystrophin, which helps keep muscles strong and working properly.

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Because dystrophin production is disrupted in DMD, patients’ muscles weaken over time. This can cause difficulty with walking, climbing stairs, and other physical activities.

The symptoms of DMD usually appear around the age of 2 or 3. Over time, the muscles that control breathing and the heart can also become affected, which can be life-threatening.

There’s no cure for DMD, but medications and other therapies can help relieve symptoms and improve quality of life.

According to IPS Heart, none of the current treatments can generate new skeletal muscle, the type that helps the body move, or provide the amount of dystrophin needed to completely restore muscle function.

GIVI-MPC uses a small molecule called givinostat to make new, fully working skeletal muscle from human pluripotent stem cells, which are able to morph into many different cell types. Moreover, preclinical studies of DMD mouse and pig models showed it created human skeletal muscle with full-length dystrophin.

ISX9-CPC uses another small molecule called ISX-9 to reprogram human pluripotent stem cells into functional cardiac muscle, the type of muscle that makes up the heart.

It’s designed as a candidate for treating heart failure and Duchenne cardiomyopathy, two heart problems that can arise as complications of DMD.

GIVI-MPC was designated an orphan drug by the FDA in February. IPS Heart received the same designation for ISX9-CPC in 2022 and expects it to be the first of the two therapies for human testing.

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