IPS HEART

GIVI-MPC targets site of muscle injury via direct injection and systemic delivery and creates new functional skeletal muscle for treatment of both Sarcopenia & Duchenne Muscular Dystrophy in aged mice 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 the peer-reviewed publication of in vivo and in vitro study with its GIVI-MPC stem cell therapy candidate in this month's issue of Cells (volume 12, issue 14). The study, “Chemokine/ITGA4 Interaction Directs iPSC-Derived Myogenic Progenitor Migration to Injury Sites in Aging Muscle for Regeneration,” demonstrated GIVI-MPC stem cell therapy’s ability to successfully differentiate and create new human skeletal muscle in extremely aged 18-month aged mice for sarcopenia repair. Sarcopenia is characterized by muscle mass loss and function decline with age and poses a threat to the quality of lifelong health as well as the general well-being of the elderly population. Weaker muscles with low mass and function are likely more susceptible to muscle damage and injury. Studies suggest that muscle mass decreases by about 3 to 8 percent by decade after age 30. Thus far, there is no effective therapeutic approved for muscle loss during aging. “During aging, increased inflammation and oxidative damages in skeletal muscle tissues occur,” said Rauf Ashraf, Chief Executive Officer of IPS HEART. “Given that GIVI-MPCs possess anti-oxidative and anti-inflammatory properties, and exhibit superior migration characteristics, GIVI-MPCs administered in 18-month aged NSG mice resulted in significant improved muscle regeneration, with hindlimb grip strength doubling and an over 30% reduction in fibrosis. We are thrilled with these significant results and are even more pleased that GIVI-MPC has been featured in a second peer reviewed publication.” IPS HEART previously reported that GIVI-MPC developed into new human skeletal muscle, and reduced fibrosis & necrosis upon transplantation in dystrophic mice and pigs. In the current publication, successful delivery via IV/systemic delivery and direct injection were both achieved as well as new skeletal muscle generation with full length human dystrophin developed in 16-month aged dystrophic mice. This development further validates the idea that the Company’s therapeutic candidate may not only slow the rate of decline as gene therapy approaches aim for, but more importantly can potentially help reverse Duchenne Muscular Dystrophy in both young and older DMD patients. Heart disease continues to kill more people annually than all forms of cancer combined. IPS HEART’s first stem cell therapeutic (ISX9-CPC) helps longevity by creating new heart functional heart muscle and when paired with GIVI-MPC, new skeletal muscle can be created for sarcopenia thus providing tangible aging therapeutics for improved longevity. About IPS HEART IPS HEART is advancing its stem cell platform to develop new skeletal & cardiac muscle generation therapies.

IPS HEART’s GIVI-MPC and ISX9-CPC are indicated to treat patients with Duchenne muscular dystrophy. Credit: Dr. Edwin P. Ewing, Jr. / commons.wikimedia.org. The US Food and Drug Administration (FDA) has granted rare paediatric drug designations for IPS HEART’s stem cell therapeutics, GIVI-MPC and ISX9-CPC, to treat Duchenne muscular dystrophy (DMD) patients. GIVI-MPC is intended to create new skeletal muscle with 100% full-length dystrophin, while ISX9-CPC is designed to create new functional cardiac muscle for indicated patients. Using the small molecule Givinostat, GIVI-MPC reprogrammes human induced pluripotent stem cells into new human skeletal muscle tissue, and delivers 100% full-length human dystrophin to treat DMD. The drug candidate received an orphan drug designation (ODD) from the FDA in February 2023. ISX9-CPC is IPS HEART’s first patented drug candidate, and uses small molecule ISX-9 to reprogramme the iPSCs into new functional human heart muscle to treat heart failure and Duchenne cardiomyopathy. It created new human heart muscle with a greater than 50% improvement in ejection fraction and a 70% reduction in scar tissue 90 days after a heart attack. ISX9-CPC is expected to become the company’s first drug to be used clinically in humans. IPS HEART CEO Rauf Ashraf said: “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 and investors to partner/ invest with us to help rapidly advance both therapies to the clinic”.

HOUSTON--( BUSINESS WIRE )-- 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. About IPS HEART 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.