iRegene Therapeutics' NouvNeu001 Receives FDA Fast Track Designation and Expanded Indication Authorization

iRegene Therapeutics' iPSC-derived project NouvNeu001 received FDA's Special Exemption in March 2024, and further obtained support from FDA's FTD accelerated pathway.This significant milestone marks the official entry of NouvNeu001 into the international accelerated pathway, as well as the US FDA's strong recognition of iRegene's NouvNeu001 clinical data.iRegene's NouvNeu001 Demonstrates Statistically Significant Safety Data Over 15 Months in Phase I Clinical Trials. More importantly, the Phase I clinical data not only shows significantly superior results compared to similar cell therapy products but also surpasses existing clinical treatment methods with statistical significance. The MDS-UPDRS III scores reflect a faster improvement time window and more pronounced improvement magnitude.

In obtainingFollowing the FTD designation, 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 obtained 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 intended use, applying it to other compassionate use cases.More notably, FTD can be implemented in parallel with Breakthrough Therapy Designation (BTD), Priority Review, and Accelerated Approval., accelerate the launch of iRegene's NouvNeu001, and bring this drug with significant clinical implications to the market as soon as possible.

iRegeneDr. Cai Meng, CMO of iRegene, stated: "This milestone demonstrates that the clinical value of NouvNeu001 has gained recognition from regulatory authorities worldwide. Moreover, due to its breakthrough efficacy in treating Parkinson's disease, the FDA has not only provided tailored support for the regulation and market approval of NouvNeu001 but has also agreed to expand its use to other compassionate use cases, significantly broadening the clinical application and commercial potential 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, it obtained FDA special exemption in March 2024, and the FDA approved its IND in June 2024 to initiate an international multi-center Phase I clinical trial in the United States and other countries. On August 15, 2025, iRegene further received Fast Track Designation (FTD) granted by the FDA.This is 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 progenitor) received FTD designation in October 2023, BlueRock Therapeutics' OpCT-001 (iPSC-derived photoreceptor cells) received FTD designation in February 2025, and BlueRock Therapeutics' Bemdaneprocel (BRT-DA01), a cell therapy product derived from embryonic stem cells, 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 for moderate to severe Parkinson's disease.

• In December 2023, NouvNeu001 was officially launched in Phase I clinical trials 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 US FDA approved its IND to initiate an international multi-center 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 granted FDA Fast Track Designation (FTD).

Fast Track Designation（FTD）It is an accelerated development and review pathway by the U.S. Food and Drug Administration (FDA), designed to expedite the approval and market entry of new drugs that treat serious or life-threatening diseases and address unmet medical needs. Key benefits include more frequent regulatory interactions and rolling review. In 1997, Fast Track Designation (FTD) was formally established as part of the FDA Modernization Act (FDAMA). FTD stands alongside the FDA’s three other expedited programs (Breakthrough Therapy Designation BTD, Accelerated Approval AA, Priority Review PR) as one of the four acceleration tools for "serious conditions—unmet needs." In 2012, the FTD strategy was further revised and strengthened under the FDASIA.

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Hetang VenturesRestructured in 2014 under Tsinghua University's strategic initiative to promote the commercialization of scientific and technological achievements, Hotang Ventures is a professional investment management institution primarily focused on early-stage investments in science and technology innovation enterprises. Hotang Ventures concentrates on key areas such as life sciences, medical devices, information technology, and modern manufacturing, with a long-term focus on investing in innovative enterprises with core hard technologies. Additionally, it specializes in incubating investments based on scientific and technological achievements from universities and top-tier hospital clinical experts. Currently managing nearly 4 billion yuan in funds, Hotang Ventures has invested in over 100 projects, supporting scientists and entrepreneurs with significant research discoveries and technological innovations to achieve "commercial transformation from 0 to 1." It consistently adheres to selecting high-barrier "first-of-its-kind technologies" for early-stage investment. With outstanding performance, Hotang Ventures has received honors such as being ranked in the "Top 50 Early-Stage Investment Institutions in China" by The Financing and "Top 50 Best Early-Stage Venture Capital Institutions in China" by CVSource.