iRegene Therapeutics Announces FDA Fast Track Designation for NouvNeu001, the World’s First Allogeneic iPSC-Derived Cell Therapy for Parkinson’s Disease, with Expanded Use Authorization

On August 15, 2025, iRegene announced that its self-developed NouvNeu001 injection had been granted Fast Track Designation (FTD) by the FDA and approved for an expanded scope of application. This marks the first-ever FTD qualification for a universal iPSC-derived Parkinson's cell therapy product globally. This breakthrough not only signifies iRegene's leading position in the field of cell therapy worldwide but also reflects the company’s steadfast commitment and efficient execution in delivering innovative solutions for Parkinson's patients.

In addition to iRegene Therapeutics' iPSC-derived project NouvNeu001 receiving FDA Special Exemption in March 2024, it has further obtained FTD (Fast Track Designation) support from the FDA. This significant milestone marks NouvNeu001's official entry into the international accelerated pathway, as well as the FDA’s strong recognition of iRegene's NouvNeu001 clinical data. In Phase I clinical trials, NouvNeu001 demonstrated statistically significant safety data over a 15-month dosing period. More importantly, the Phase I clinical data not only showed superiority over similar cell therapy products but also surpassed existing clinical treatment methods with statistically significant results. The MDS-UPDRS III scores highlighted a faster improvement time window and a more pronounced improvement magnitude.

After obtaining the FTD qualification, the FDA will engage in more frequent interactions with iRegene. The FDA will provide targeted one-on-one guidance to iRegene on aspects such as clinical trial design and data requirements. Additionally, iRegene has also gained the qualification for rolling communication with the FDA without time restrictions. More importantly, the granting of this FTD also allows iRegene's NouvNeu001 to expand its scope of application to other compassionate use cases. More notably, FTD can be combined withBreakthrough Therapy Designation (BTD), Priority Review, Accelerated ApprovalIn parallel, accelerate the pace of iRegene's NouvNeu001 to market and bring this drug with significant clinical implications to patients as soon as possible.

Dr. Cai Meng, CMO of iRegene, stated: This milestone event demonstrates that the clinical value of NouvNeu001 has been recognized by regulatory authorities worldwide. Moreover, due to its breakthrough effectiveness in treating Parkinson's disease, the FDA has not only provided customized support for the regulation and market approval of NouvNeu001 but also agreed to expand its use to other compassionate use scenarios, significantly broadening the clinical application value and commercial scope of NouvNeu001.

Globally, iRegene Therapeutics' iPSC-derived universal cell therapy project, NouvNeu001 (chemically induced dopaminergic neural progenitor cells), has received multiple designations from the FDA. First, in March 2024, it obtained special exemption from the FDA, and in June 2024, the FDA approved its IND to initiate an international multicenter Phase I clinical trial in the United States and other countries. On August 15, 2025, iRegene further received Fast Track Designation (FTD) from the FDA, making it the world's first iPSC-derived universal Parkinson’s cell therapy product to receive FDA Fast Track Designation (FTD). Prior to this, Aspen Neuroscience’s ANPD001 (autologous iPSC-derived dopaminergic neural progenitors) received FTD designation in October 2023, and BlueRock Therapeutics’ OpCT-001 (iPSC-derived photoreceptor cells) received FTD designation in February 2025. Bemdaneprocel (BRT-DA01), a cell therapy product derived from embryonic stem cells developed by BlueRock Therapeutics, received RMAT (Regenerative Medicine Advanced Therapy) designation in August 2023. This breakthrough further solidifies iRegene’s leading position in the global industry.

Milestone Timeline for iRegene Therapeutics' NouvNeu001 Project:

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

• In December 2023, NouvNeu001 officially launched its Phase I clinical trial at the National Center for Geriatrics of 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 related U.S. Pharmacopeia requirements.

• In June 2024, the US 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 patient enrollment for Phase I clinical trials in China.

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

• From April to June 2025, iRegene launched the overseas multi-center Phase I clinical trial of NouvNeu001 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, further received FDA Fast Track Designation (FTD).

Fast Track Designation (FTD) is a designation by the U.S. Food and Drug Administration (FDA).Accelerated Development and Review Pathway`, aiming to`Promote the treatment of serious or life-threatening diseases and meet unmet medical needsThe new drug can enter the approval and marketing process more quickly. The core conveniences includeMore Frequent Regulatory Interactions and Rolling Review。1997FTD was first formally established as part of the FDAMA (FDA Modernization Act). FTD, along with the FDA's other three expedited mechanisms (Breakthrough Therapy Designation (BTD), Accelerated Approval (AA), Priority Review (PR)) in parallel, is one of the four acceleration tools in the "Severe Disease-Unmet Need" scenario.In 2012, the FTD StrategyFurther revised and strengthened in the FDASIA.

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