HELP Therapeutics Co. Ltd has announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for HiCM-188, an allogeneic human iPSC-derived cardiomyocyte therapy. This clearance paves the way for the first clinical trial in the United States evaluating an iPSC-derived cell therapy for end-stage heart failure.
The Phase I study aims to assess the safety and tolerability of HiCM-188 cell transplantation one year post-transplant. The trial will involve intramyocardial injection of HiCM-188 during coronary artery bypass graft surgery and will evaluate various dose levels across multiple sites in the United States.
Addressing Unmet Needs in Heart Failure Treatment
Heart failure is a significant public health concern, affecting at least 7 million people in the U.S. and approximately 65 million globally. The condition is a leading cause of death and hospitalization, particularly among the elderly, and the increasing aging population is exacerbating the issue. Current treatment options are limited, with scarcity of heart transplant donors and high costs associated with artificial heart technologies leaving many patients with unmet clinical needs.
HiCM-188: A Novel Approach to Myocardial Repair
HiCM-188 consists of highly purified allogeneic cardiomyocytes reprogrammed from human induced pluripotent stem (iPS) cells. The proprietary process used to create HiCM-188 is serum-free, genetic vector-free, and integration-free. This "off-the-shelf" product offers a shift from traditional whole heart transplantation to a more precise myocardial repair approach, transplanting fresh myocardial cells into damaged areas of the heart.
Expert Perspectives
Professor Emerson C. Perin, Medical Director at The Texas Heart Institute, a planned trial center, expressed enthusiasm for the trial. "I believe this milestone for HELP Therapeutics has the potential to advance the field of cell therapy and represents a significant step in their development program. I am eager to collaborate with them on this clinical program."
Professor Junbo Ge, Academician of Chinese Academy of Sciences, and the Chief of Cardiology at Zhongshan Hospital, Fudan University, highlighted the significance of this approval for China's pharmaceutical innovation. "The innovative drug independently developed by HELP Therapeutics is the first IND approved by the U.S. Food and Drug Administration using iPSC technology for cardiovascular indications. This is an exciting and proud achievement, representing a significant milestone in China’s cardiovascular innovative drugs and opening a new chapter in China’s cell therapy innovations."
Professor Philippe Menasché from University of Paris Descartes added, "Cardiomyocytes derived from pluripotent stem cells represent an effective means of increasing the pool of contractile cells in failing hearts and thus to improve their function. The IND released to HELP Therapeutics represents a major milestone in the landscape of cardiac regeneration as it paves the way for the first large-scale efficacy trial whose outcomes have the potential to strengthen the rationale for remuscularizing the heart with cardiac-committed cells and thus expand the clinical indications of this innovative therapy."
HELP Therapeutics' Vision
Jiaxian Wang, MD, PhD, Founder and CEO of Help Therapeutics, emphasized the company's commitment to developing affordable innovative drugs. "Our off-the-shelf product - the Regenerative Cardiac Cell Injection (iPS-CMs: HiCM188) - has successfully obtained IND clinical trial approval in the U.S... Additionally, the company is committed to developing affordable innovative drugs, aiming to create cell therapy products that are accessible and beneficial a broad patient population."
