FDA Clears HELP Therapeutics' IND for iPSC-Derived Heart Failure Cell Therapy

NANJING, China, Oct. 22, 2024 /PRNewswire/ -- HELP Therapeutics Co. Ltd, a clinical-stage cell therapy company, has received clearance from the U.S. Food and Drug Administration (FDA) for its Investigational New Drug (IND) application. This approval is for the administration of "Allogeneic Human iPSC-derived cardiomyocytes (HiCM-188)" via intramyocardial injection during coronary artery bypass graft surgery.

Heart failure (HF) is a significant public health concern affecting over 7 million individuals in the United States and about 65 million globally. With an increasing prevalence among the elderly, heart failure is a leading cause of death and hospitalization in older adults. HiCM-188, a prominent program developed by HELP Therapeutics, comprises purified allogeneic cardiomyocytes derived from human induced pluripotent stem (iPS) cells. These cells are reprogrammed under proprietary conditions that are serum-free, genetic vector-free, and integration-free.

HiCM-188 is the first investigational iPSC-derived cell therapy to enter clinical trials in the United States for treating end-stage heart failure. The primary goal of the Phase I study is to assess the safety and tolerability of HiCM-188 one year after transplantation. The study will evaluate various dosages and is expected to enroll participants across multiple sites in the United States. Professor Emerson C. Perin, Medical Director at The Texas Heart Institute, one of the trial centers, remarked on the significance of this milestone, expressing optimism that it could advance the field of cell therapy. He is looking forward to collaborating with HELP Therapeutics on this clinical program.

Professor Junbo Ge, a member of the Chinese Academy of Sciences and Chief of Cardiology at Zhongshan Hospital, Fudan University, is also encouraged by the FDA's approval. He highlighted that this innovative drug, developed independently by HELP Therapeutics, is the first IND approved by the FDA using iPSC technology for cardiovascular indications. He sees this as a significant achievement for China's cardiovascular drug development and a new chapter in cell therapy innovations.

Professor Philippe Menasché from the University of Paris Descartes emphasized the importance of cardiomyocytes derived from pluripotent stem cells in heart regeneration. He noted that the FDA's approval marks a major milestone in the field of cardiac regeneration, paving the way for large-scale efficacy trials. These trials have the potential to provide strong evidence for the use of cardiac-committed cells to repair failing hearts and expand the clinical applications of this innovative therapy.

Jiaxian Wang, MD, PhD, Founder and CEO of HELP Therapeutics, commented on the increasing prominence of heart failure due to the aging population. He pointed out the shortage of heart transplant donors and the high costs associated with artificial heart technologies, which leave many heart failure patients without adequate treatment options. Wang expressed confidence that their product, the Regenerative Cardiac Cell Injection (iPS-CMs: HiCM188), offers a promising alternative by focusing on precise myocardial repair rather than whole heart transplantation. This approach involves transplanting fresh myocardial cells into damaged areas, presenting a revolutionary treatment for heart failure patients. The company is committed to making innovative drugs affordable and accessible to a broad patient population.

Founded in 2016, HELP Therapeutics is a global clinical-stage biopharmaceutical company focused on developing iPSC-derived cell therapeutics for degenerative diseases. The company aims to establish a State National iPS Cell Bank and drive the development of off-the-shelf cell therapy products for cardiac failure, other degenerative diseases, and oncology. HELP Therapeutics is dedicated to providing superior development and manufacturing technologies to deliver safe and effective cell therapy products to patients in need.