Ruijian Pharmaceuticals' NouvNeu001 Becomes First iPSC Therapy Globally to Receive FDA's Dual Designation of Fast Track and RMAT for Parkinson’s Disease

December 22, 2025, iRegene（Chengdu）iRegene's Self-Developed NouvNeu001 Injection Receives FDA's Regenerative Medicine Advanced Therapy Designation（Regenerative Medicine Advanced Therapy, RMAT）Recognition. NouvNeu001 becomes the world's first iPSC chemically induced universal cell therapy product approved for RMAT in the field of neurological diseases. The full name of RMAT by the FDA is Regenerative Medicine Advanced Therapy.(Advanced Regenerative Medicine Therapies), which was established in 2016 under the 21st Century Cures Act as an accelerated approval pathway specifically for regenerative medicine therapies. It is oriented towards significant clinical value, facilitating their rapid market entry to address unmet medical needs.

NouvNeu001 was granted RMAT designation shortly after receiving FTD from the FDA in August 2025.Becoming the world's first iPSC product to date to receive both FDA FTD and RMAT designations reflects the FDA’s high recognition of the clinical data for NouvNeu001. The data integrity, clinical trial rigor, as well as the superior safety and efficacy data demonstrated by iRegene's NouvNeu001 in Phase I clinical trials for Parkinson's disease have been highly affirmed by the FDA. The RMAT designation underscores the significant therapeutic value of this product in the international Parkinson's disease market. The FDA will further engage in close communication with iRegene to provide customized guidance for subsequent clinical development, including discussions on trial design, selection of clinical endpoints, and expedited approval pathways, thereby shortening the time from clinical trials to market. The achievement of RMAT status also marks iRegene's innovative strength in the iPSC field and the clinical value of its pipeline being highly recognized by international authoritative regulatory agencies, fully demonstrating iRegene's team breakthrough from "0 to 1" and their continued leadership in the iPSC field on an international scale.

As the second most common neurodegenerative disease globally, the current therapeutic approaches for Parkinson's disease focus on symptomatic relief or "complication management," rather than offering "disease-modifying" treatments. The development of NouvNeu001 directly addresses this fundamental treatment gap, aiming to fundamentally repair the loss of dopaminergic neurons through cell replacement therapy, reverse the progression of Parkinson's disease, and provide patients with potential clinical benefits that could alter the course of the disease.

iRegene's proprietary "AI + chemical induction" technology can achieve directed differentiation of high-purity specific dopaminergic neuron subtypes and simultaneously complete various functional modifications, including cellular biochemistry, during the induction process to meet genuine clinical needs. After transplantation, the cells repair the circuit deficiencies caused by Parkinson’s disease in patients by filling the void of functional cells at the lesion site, restoring the patients' dopamine self-secretion function. This presents a potential for reversing disease progression that traditional symptomatic treatments cannot achieve, representing a true First-In-Class technological approach.

iRegene's team adhered to the principle of prioritizing patient benefits from the project's inception. Compared with global peers, iRegene adopts the world's first single-needle-path stereotactic drug delivery method, achieving maximum safety in the drug delivery process and enabling long-term survival and integration of transplanted cells. Its efficacy data also demonstrate international leadership; subjects showed motor function scores 12 months after a single dose.（MDS-UPDRS III）An average improvement of 30.6 points in the OFF state(Improvement Amplitude 52.82%)，ON status improved by an average of 13.6 points(Improvement Amplitude 54.67%), significantly superior to existing therapies. These statistically significant clinical results demonstrate the tremendous therapeutic value of NouvNeu001 in the international Parkinson's disease market.

Since its inception, iRegene has established a normalized and forward-looking collaborative working mechanism with regulatory agencies, promoting the continuous transformation of key R&D milestones into regulatory approvals. With solid non-clinical data and a comprehensive and robust CMC system, NouvNeu001 was the first to receive FDA special exemption in March 2024 and was approved for IND to conduct Phase I clinical trials overseas in June of the same year. Subsequently, based on positive clinical progress, the project received Fast Track designation in August 2025.（FTD）In December of the same year, it once again obtained advanced regenerative medicine therapy.（RMAT）Recognition. This consistent series of regulatory breakthroughs not only highlights the FDA's full affirmation of the therapeutic potential of NouvNeu001 and iRegene's innovative CMC and development strategy, but will also further assist the iRegene team in providing comprehensive support in clinical trial design, advancing registration-based Phase III trials with the FDA, and optimizing the market entry process.

Dr. Meng Cai, Chief Medical Officer of iRegene, stated: "Receiving the RMAT designation is an important regulatory milestone in the development of NouvNeu001, fully demonstrating its clinical value and innovation. This not only signifies the FDA's recognition of the product's therapeutic potential for serious unmet medical needs but also provides us with a structured pathway for in-depth and efficient collaboration with regulatory authorities. We will make full use of this qualification to accelerate global multi-center clinical trials and strive to bring this revolutionary therapy to patients worldwide as soon as possible."

As of now, the development of NouvNeu001 continues to lead:

● On August 3, 2023, NouvNeu001 received approval from China's NMPA to conduct Phase I/II combined clinical trials in China for moderate to severe Parkinson's disease.

● In December 2023, NouvNeu001 officially launched its Phase I clinical trial at the National Center for Geriatric Medicine, Beijing Hospital, Ministry of Health;

● In March 2024, the U.S. FDA granted NouvNeu001 special exemption status, recognizing iRegene's breakthrough innovation platform and comprehensive quality system, and exempting NouvNeu001 from relevant requirements of the U.S. Pharmacopeia.

● In June 2024, the U.S. FDA approved its IND to initiate an international multicenter Phase I clinical trial in the United States and other countries;

● In November 2024, NouvNeu001 completed the enrollment for Phase I clinical trial in China;

● In April 2025, NouvNeu001 officially launched the Phase II clinical trial in China;

● From April to June 2025, NouvNeu001 will initiate multi-center Phase I clinical trials overseas in multiple countries;

● In July 2025, the first group of patients in the Phase II clinical trial of NouvNeu001 completed dosing;

● On August 15, 2025, NouvNeu001 was granted Fast Track Designation by the FDA.（FTD） ；

● On December 22, 2025, NouvNeu001 further received FDA Regenerative Medicine Advanced Therapy designation. (RMAT) Recognition.

RMAT（Regenerative Medicine Advanced Therapy）It is the U.S. FDA in the "21st Century Cures Act"（21st Century Cures Act）A specialized review pathway established under the framework aims to accelerate the development and evaluation of regenerative medicine products used for treating, delaying, reversing, or curing serious or life-threatening diseases. Products granted RMAT designation can enjoy benefits similar to those of breakthrough therapies.（BTD）Similar accelerated support measures, including: earlier and more frequent communication with the FDA; rolling review（Rolling Review）Qualification; Possible Accelerated Approval（Accelerated Approval）And Priority Review（Priority Review）。   

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