#NouvNeu001 #FDA Designation #iRegene Therapeutics

iRegene's NouvNeu001: A Groundbreaking Leap in Parkinson's Disease Treatment

In a significant milestone for the field of biotechnology, iRegene Therapeutics Co., Ltd. has announced that its leading product, NouvNeu001, has been awarded Regenerative Medicine Advanced Therapy (RMAT) designation by the U.S. Food and Drug Administration (FDA). This impressive achievement marks NouvNeu001 as the world's first allogeneic cell therapy derived from induced pluripotent stem cells (iPSC) to secure both RMAT and FDA Fast Track Designation (FTD), the latter having been granted back in August 2025. Such recognitions reflect the promising clinical data and the innovative chemical induction platform utilized to develop the therapy, which aims to modify the course of Parkinson's disease (PD).

Addressing a Serious Medical Need

Parkinson's disease is recognized as the second most common neurodegenerative disorder globally, with existing treatments primarily aimed at alleviating symptoms rather than addressing the root causes of neuronal degeneration. Traditional therapies have not significantly altered disease progression, leaving a substantial gap in patient care. iRegene's NouvNeu001 is designed to close this gap, as it utilizes allogeneic dopaminergic progenitor cells derived from iPSCs to replace dopaminergic neurons that have been lost due to the disease. This approach not only aims to restore disrupted neural circuits but also to enable patients to regain their own dopamine production.

Dr. Meng Cai, the Chief Medical Officer of iRegene Therapeutics, stated, "The RMAT designation is a pivotal regulatory milestone for NouvNeu001 and a compelling endorsement of its clinical value. It underscores the FDA's recognition of the urgent need for effective therapies that can potentially modify the course of Parkinson’s disease." He emphasized the importance of this designation for facilitating collaboration with the FDA, which he believes will accelerate the global clinical development of the therapy, making it available for patients as soon as possible.

Clinical Data: Concrete Evidence of Efficacy

The initial Phase I clinical trial data for NouvNeu001 provided robust evidence of its clinical efficacy, particularly in terms of motor functionality—an essential aspect of daily living for those affected by Parkinson's disease. The trial results demonstrated significant improvements in the Movement Disorder Society-Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) Part III motor scores, which is considered the gold standard for evaluating motor impairment in PD patients.

In the low-dose cohort, participants saw an improvement of 30.6 points (a rise of 52.82% from baseline) in OFF states and 12.9 points (54.67% improvement) in ON states after 12 months. Meanwhile, the high-dose cohort experienced increases of 23.3 points (OFF) and 9.67 points (ON) at the nine-month mark. Notably, improvements persisted through 15 months post-treatment, suggesting the long-lasting benefits of the injected cells—an indication that NouvNeu001 could offer hope where traditional therapies fail.

Future Prospects and Company Vision

Looking ahead, iRegene's aspirations extend beyond the confines of a single product. The company is dedicated to harnessing its innovative AI and chemical induction technology to develop a pipeline of iPSC-derived therapies aimed at various currently