Ruijian Medicine Launches Phase II Clinical Trial for Parkinson’s Stem Cell Therapy and Secures Over RMB 200 Million in Series B+ Financing

Text｜Hu Xiangyun

Editor｜Hai Ruo Jing

36Kr learned that "iRegene," which is engaged in the development of cell therapy drugs, has recently completed a B+ round of financing of over 100 million yuan. This round of financing was led by Fengchuan Capital, with participation from Hotang Venture Capital, and Easy Capital served as the exclusive financial advisor. The funds raised will be used to accelerate the Sino-US clinical trials of the company's core product pipeline, the research and development of multiple innovative pipelines, and the establishment of clinical capabilities.

It is also reported that not long ago, iRegene had just completed its Series B financing. The two rounds of Series B financing have raised more than 200 million yuan in total.

iRegene was founded in 2017, focusing on the development of small-molecule chemical transcriptional regulation-induced pluripotent stem cell (iPSC) products, and is also a company that 36Kr has been tracking for a long time.EnterpriseRecently, amidst the successive completion of large-scale fundraising, iRegene's independently developed core product pipeline for Parkinson’s treatment, NouvNeu001 (a chemically induced functional human dopaminergic neuron precursor cell), has recently entered Phase II clinical trials. Taking this opportunity, we revisited Wei Jun, founder and CEO of iRegene, to discuss the company’s business progress and expectations for the development of the stem cell sector.

Parkinson's disease is a common degenerative disease of the central nervous system in middle-aged and elderly people, and its root cause lies in the massive apoptosis of dopaminergic neurons in the patient's brain. However, current clinical treatments for Parkinson's disease mainly involve medication and surgery, which can only "alleviate symptoms" but cannot prevent the ongoing neurodegeneration at the site of the lesion. Moreover, drug treatments may lead to reduced efficacy or adverse reactions as the duration of use increases, while surgical treatments carry risks such as high costs, limited application scope, significant trauma, and are also unable to halt the progression of the disease.

Scientists thus believe that if the lost cells could be "re-transplanted," it might be possible to "reverse" the condition of Parkinson's patients, and iPSC technology is the foundational platform for realizing this concept. Wei Jun introduced that the ideal therapeutic effect comes from the transplanted cells being able to efficiently perform their comprehensive functions. The cells transplanted into the brain need to "within an efficient time window, reconnect the upstream and downstream neural circuitry lost due to Parkinson’s disease, while also possessing the ability to secrete dopamine."

Under this approach, iRegene focused on analyzing the following two key issues in its previous Phase I clinical study:First, whether the lesions in the patient's brain caused by the degenerative death of nerve cells can be repaired through the transplantation of new functional nerve cells; second, whether the repair of such lesions will further lead to improvements in the patient's behavioral and other symptoms.。

Based on the results of 10 enrolled cases accumulated from NouvNeu001, six months after transplantation, patients' various indicators were assessed using the MDS Unified Parkinson's Disease Rating Scale (MDS-UPDRS). The motor functions showed a significant trend of clinical improvement. Non-motor symptoms, including anxiety and "on-time" duration, also reached the standard of clinically significant improvement.

At the same time, PET imaging analysis also confirmed the colonization and function of the transplanted cells in the brain. The results showed that with the increase of transplantation time, the activity of dopamine transporter in the transplantation area was significantly enhanced.

Especially in the low-dose group, the longest follow-up time for participants has exceeded 60 weeks, and the behavioral improvement effects in patients are still stably continuing. This may indicate that "universal cell transplantation for Parkinson's disease is highly feasible, and the transplanted cells can function effectively in patients over the long term."

"Unlike the clinical trials of similar products overseas, we included a broader range of Parkinson's disease subjects in this clinical trial, covering moderate to very severe cases. The aim was to understand which patients responded better to NouvNeu001 and guide its optimal clinical application. Among them, some subjects who were already in a very severe state at enrollment and required family assistance for mobility could stand on their own shortly after receiving the treatment. After six months of treatment, their walking ability had significantly improved, greatly enhancing their quality of life," said Dr. Wei Jun.

It is also reported that, in order to achieve full-course coverage for Parkinson's treatment, iRegene has developed NouvNeu003, a cell therapy product mainly targeting early-onset Parkinson’s patients under 50 years old. This product was approved by the National Medical Products Administration (NMPA) to enter Phase I clinical trials at the end of 2023 and has now completed patient enrollment.

Since 2024, iRegene has made certain progress in advancing overseas clinical research. In the same year, the IND application for NouvNeu001 was approved in the United States, and it received special exemptions granted by the FDA, with Phase I clinical enrollment expected to officially commence in the first half of this year; another product, NouvSight001, which is designed for ophthalmic diseases, received orphan drug designation from the FDA in March of the previous year for the treatment of a series of indications related to retinitis pigmentosa.

In addition, when discussing the pricing of the company's stem cell therapy products, Wei Jun mentioned that the future product pricing would comprehensively consider factors such as the affordability of China's medical insurance system, patients' payment ability, and enterprise costs, aiming to achieve a win-win situation for all parties involved while ensuring patients can afford it. Currently, the company's cost advantages in the production process have laid a foundation for achieving this goal.

Founders and Investors' Perspectives:

Dr. Wei Jun, founder and CEO of iRegene, stated: "We are highly honored to continuously receive strong support from new shareholders during the development of iRegene, and we are also very grateful for the contributions made by our existing shareholders. Special thanks go to CEC Capital and the close cooperation of the iRegene team. iRegene has always adhered to the principle of 'starting from the needs of patients and addressing real clinical needs,' using 'original innovation' to develop entirely new therapies that can truly reverse disease progression. This round of financing will further solidify the foundation for iRegene to accelerate its clinical progress, and the team will continue to work hard to bring new therapeutic experiences to a wide range of patients. Additionally, the completion of this financing not only lays the groundwork for NouvNeu001 to enter the confirmatory clinical stage but also provides support for iRegene to further expand into international markets."

Xiang Duan, founder of Fengchuan Capital, stated: "Fengchuan Capital is honored to participate in iRegene's current round of investment. The company has developed a next-generation chemically induced universal cell therapy based on a globally leading small-molecule chemical transcription regulation iPSC technology platform. Its pipeline focuses on unmet clinical needs, demonstrating impressive safety and efficacy potential with broad commercial prospects. Through solid technical research and clinical progress, iRegene has gradually grown into one of the top global enterprises in the iPSC field. We look forward to iRegene further accelerating the industrialization and commercialization of its various pipelines through this round of financing, bringing revolutionary changes to the global healthcare sector. Fengchuan Capital will also continue to collaborate with Chinese biotech companies to promote the rapid development of China-originated innovative drugs."

Guo Kai, Managing Director of Hetang Ventures, stated: The drug development strategy of transplanting dopamine precursor cells for the treatment of Parkinson's disease has a clear biological mechanism and has recently been supported by preliminary clinical efficacy data. It is expected to become the first therapy capable of significantly delaying disease progression, offering tremendous social and commercial value. iRegene's core product, NouvNeu001, is the world’s first clinical-stage iPSC-derived dopamine precursor cell therapy, demonstrating significant advantages in terms of clinical progress, clinical efficacy, and production costs. We have high hopes for the company’s product and will support the ongoing clinical trials in both China and the U.S., aiming to benefit Parkinson's patients as soon as possible.