iRegene Gets FDA IND Nod to Begin Parkinson's Clinical Trial

WUHAN, China, June 24, 2024 – On June 20, the U.S. Food and Drug Administration (FDA) approved the Investigational New Drug (IND) application for NouvNeu001, a cell therapy product developed by iRegene Therapeutics for the treatment of Parkinson's Disease. This approval marks a historic moment as NouvNeu001 is the world's first chemically induced allogeneic cell therapy to enter clinical trials for Parkinson’s disease in the United States. The "Combined Phase I/II Clinical Study" of NouvNeu001 had already received approval from China’s National Medical Products Administration (NMPA) in August 2023, demonstrating promising safety and efficacy in Phase I trials. The FDA’s approval on June 20, 2024, allows NouvNeu001 to be used in treating mid-to-late stage Parkinson's disease in the U.S.

In March 2024, iRegene received an exemption from the FDA, acknowledging the company’s innovative platform and production systems. Dr. Cai Meng, CMO of iRegene, explained that this exemption, followed by the IND approval, highlights iRegene's expertise in developing their innovative chemically induced platform. This milestone paves the way for iRegene's further international operations.

iRegene is one of the pioneering biotech companies globally to utilize "AI + chemical induction" for the specific functional modification of cells. This platform offers significant benefits, including increased efficiency, safety, cost-effectiveness, and scalability for large-scale industrial production. Utilizing this platform, iRegene has developed a comprehensive pipeline of universal induced pluripotent stem cell (iPSC)-derived products aimed at treating difficult diseases such as neurodegenerative disorders, including Parkinson's disease and blindness. In early 2024, clinical trials for NouvNeu001 began at Beijing Hospital and Zhongnan Hospital of Wuhan University in China. These trials are systematically evaluating the safety, tolerability, and efficacy of stereotactic bilateral intracerebral injections of NouvNeu001 for mid-to-late stage Parkinson's disease. The project is currently in its acceleration stage, with preliminary results showing significant improvements in motor function and good safety profiles with no adverse effects related to the cell product.

Following the successful progress of NouvNeu001’s clinical trials and the initial positive efficacy and safety data, iRegene plans to start patient recruitment for the clinical trial of its early-onset Parkinson's disease product, NouvNeu003, which was approved by China NMPA in December 2023.

In addition to neurodegenerative diseases, iRegene is exploring treatments for other challenging diseases. In March 2024, the FDA granted Orphan Drug Designation (ODD) to NouvSight001, an ophthalmic product targeting retinal diseases. This product is a first-of-its-kind iPSC-derived allogeneic cell therapy, developed using iRegene's unique "AI + chemical induction" platform.

Dr. Wei Jun, CEO of iRegene, emphasized the company's commitment to maintaining high standards in developing innovative products while ensuring steady progress in research and development. The project advancement plan is meticulously scheduled to the week, establishing a solid foundation for timely clinical entry of key product pipelines. Dr. Wei Jun stated, "Through diligent R&D efforts, iRegene aims to leverage its innovative technologies to bring breakthroughs for many currently incurable diseases, offering hope to patients and families affected by these conditions. The dual approval of IND applications for NouvNeu001 by the U.S. FDA and China NMPA boosts the confidence of iRegene's global team in building a world-class innovative platform."

NouvNeu001, iRegene’s proprietary product, is a chemically induced human dopaminergic neuron precursor derived from iPSC. It is the world’s first iPSC-derived chemically induced allogeneic cell therapy product for clinical use. Approved by China’s NMPA on August 2, 2023, for Phase I/II clinical trials, it aims to evaluate its safety, tolerability, and efficacy in treating mid-to-late stage Parkinson's disease. The trial began in January 2024 at Beijing Hospital, with more information available at clinicaltrials.gov (NCT#06167681). The FDA's approval on June 20, 2024, allows the continuation of this groundbreaking research in the U.S.

iRegene Therapeutics is a pioneering biotech company focused on accurately reprogramming cell fate and functions through chemicals. With its "AI + chemical induction" platform, the company has developed extensive pipelines of universal iPSC-derived cell products. iRegene is committed to advancing universal cell therapy products and introducing novel cell replacement therapies for difficult neurodegenerative diseases, including Parkinson's disease and retinal diseases.