Ruijian Medicine Achieves FDA Special Exemption and Dual IND Approval in the U.S. and China for Parkinson’s Cell Therapy NouvNeu001

On June 20th, US time, the US Food and Drug Administration (FDA) officially approved the IND application for iRegene's Parkinson's pipeline NouvNeu001.As the world's first chemically induced, universal cell therapy product in the Parkinson's field to enter the clinical stage, NouvNeu001’s "combined Phase I-II clinical" study was approved by the National Medical Products Administration (NMPA) in August 2023 and has achieved positive safety and efficacy data in Phase I clinical trials. On June 20, 2024, iRegene also achieved "dual IND submission and approval" in both China and the U.S. for its Parkinson’s pipeline.

It is worth mentioning that in March this year, iRegene was granted a Special Exemption by the FDA, representing the FDA's recognition of the company's groundbreaking platform and the innovative production and quality systems established on this platform.

In response, Dr. Cai Meng, CMO of iRegene, stated that after obtaining the FDA special waiver, the NouvNeu001 IND received further FDA approval, demonstrating the FDA's high recognition of iRegene’s pioneering chemical induction platform and innovative products. This also cleared policy barriers for the company’s subsequent internationalization pathway, allowing universal cell therapy products produced in China to be directly exported for clinical trials.

Public information shows that iRegene is one of the earliest biotech companies globally to apply "AI + chemical induction" to the specific functional modification of cells. This platform offers more efficient, safer, lower-cost, and scalable advantages for industrial production in terms of cell function modification. Based on this unique platform, iRegene has established a rich pipeline of universal iPSC products, developing universal cellular therapeutics for currently "incurable" diseases such as Parkinson's disease and other neurodegenerative disorders, as well as blindness, bringing the possibility of a cure. Due to the originality of iRegene's technology platform and the strong advantages demonstrated in product development, the FDA granted special exemption status to iRegene’s groundbreaking product in March 2024.

At the beginning of 2024, NouvNeu001 successfully initiated clinical trials at Beijing Hospital and Zhongnan Hospital of Wuhan University. By integrating the advantageous clinical resources of neurosurgery, neurology, and other departments, a systematic evaluation was conducted on the safety, tolerability, and efficacy of stereotactic bilateral intracerebral injection of NouvNeu001 for the treatment of mid-to-late stage Parkinson's disease. The clinical project is now entering an accelerated phase. Based on the results of multiple cases independently completed across multiple centers, treatment with NouvNeu001 not only led to a rapid and significant improvement in MDS-UPDRS scores but also greatly enhanced patients' quality of life. Additionally, the treatment demonstrated good safety with no related adverse reactions. The NouvNeu001 project is about to enter the dose expansion phase.

Following significant progress in the NouvNeu001 project, which has demonstrated statistically significant efficacy and safety data, iRegene will soon initiate patient recruitment for the NouvNeu003 clinical trial. In December 2023, the NouvNeu003 IND for iRegene's early-onset Parkinson's disease project received approval from the National Medical Products Administration (NMPA), marking the second IND obtained by iRegene.

Beyond neurodegenerative diseases, iRegene continues to explore in other "incurable" disease fields.In March this year, NouvSight001, a breakthrough ophthalmic product by iRegene, was granted Orphan Drug Designation (ODD) by the U.S. FDA. Targeting the treatment of "retinitis pigmentosa-related indications," this product is the first off-the-shelf ophthalmic cell therapy derived from induced pluripotent stem cells (iPSC) developed by the iRegene team based on the company’s unique “AI + chemical induction” platform.

According to Dr. Wei Jun, CEO of iRegene, in the innovative therapy field of iPSC, iRegene has maintained a high level of rigor while balancing the progress of research and development. The project advancement plans previously formulated by the company were precisely timed to the "week", which laid an essential foundation for the company to continuously push its core pipelines into clinical trials as scheduled. "On the basis of prudent and steady R&D, iRegene hopes to leverage its innovative technology to bring breakthroughs to a large number of diseases that currently cannot be cured, creating blessings for patients and families suffering from related diseases. This time, NouvNeu001 achieved...'Dual Filing and Dual Approval'", further enhancing iRegene's confidence in building a world-class innovation platform with its international team."