Ruijian Medicine's iPSC-Derived Cell Therapy NouvNeu001 Receives FDA IND Approval for Parkinson’s Disease

▎WuXi AppTec Content Team Report

News on June 24,iRegene AnnouncesThe U.S. FDA officially approved the IND application for its Parkinson's pipeline NouvNeu001 on June 20 local time.NouvNeu001 is a universal cell therapy product based on chemical induction.The phase 1/2 clinical study of this drug has been approved by China's NMPA in August 2023, and positive safety and efficacy data were obtained in the phase 1 clinical trial. Notably, in March 2024, the FDA granted iRegene’s pioneering product special Exemption.

In this regard, Dr. Cai Meng, Chief Medical Officer (CMO) of iRegene, stated that after obtaining the FDA special exemption, the further FDA approval of NouvNeu001 IND demonstrates the FDA's high recognition of iRegene's pioneering chemical induction platform and its innovative products.

Public information shows that iRegene is one of the earlier companies globally to apply "AI + chemical induction" to the specific functional modification of cells. This platform offers advantages of higher efficiency, safety, lower cost, and better suitability for large-scale industrial production in terms of cell function modification. Based on this platform,iRegene has established a rich universal typeInduced Pluripotent Stem Cells (iPSC)Product Pipeline: Developing Universal Cell Therapy Products for "Incurable" Diseases Such as Neurodegenerative Disorders Represented by Parkinson's Disease and Blindness, bringing the possibility of a cure.

NouvNeu001 is a universal cell therapy product based on chemical induction., which achieves high-purity neuronal subtype reconstruction and functional optimization through compound regulation, forms connections with the original neurons in the body, and enhances cellular secretion functions, further strengthening the improvement of the original lesion by the transplanted cells to achieve comprehensive therapeutic effects.

At the beginning of 2024, NouvNeu001 has 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, researchers will systematically evaluate the safety, tolerability, and efficacy of stereotactic bilateral intracerebral injection of NouvNeu001 in treating mid-to-late stage Parkinson's disease. Currently, this clinical project has entered an accelerated phase. Based on the results of multiple cases independently completed across multiple centers, treatment with NouvNeu001 not only achieved rapid and significant improvement in MDS-UPDRS scores but also greatly enhanced patients' quality of life, with good safety and no related adverse reactions. The NouvNeu001 project is about to enter the dose expansion phase.

In the field of neurodegenerative diseases, iRegene has also developed another research product, NouvNeu003. In December 2023,NouvNeu003 has been approved by NMPA for clinical trials, targeting indications primarily for early-onset Parkinson's patients with an age of onset under 50.According to the news release from iRegene, the company will also initiate patient recruitment for the NouvNeu003 clinical trial in the near future.

Beyond neurodegenerative diseases, iRegene continues to explore other "incurable" disease areas. In March this year, the company's breakthrough ophthalmology product NouvSight001 received orphan drug designation from the U.S. FDA. This product is the first developed by iRegene.iPSC-Derived Universal Cell Therapy Products for Ophthalmology, intended for development to treat "a series of indications related to retinitis pigmentosa".

Dr. Wei Jun, CEO of iRegene, stated: "In the field of iPSC innovative therapies, iRegene has maintained a high level of rigor while balancing the pace of research and development. The project advancement plans previously set by the company were precise to the ‘week’, which laid an essential foundation for continuously pushing core pipelines into clinical trials on schedule. On the basis of cautious and steady R&D, iRegene hopes to leverage its innovative technologies to bring breakthroughs to a large number of currently incurable diseases, creating blessings for patients and families suffering from related conditions. The achievement of 'dual filing and dual approval' in China and the U.S. for NouvNeu001 has further strengthened the confidence of iRegene's international team in building a world-class innovation platform."

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