Ruijian Medicine Secures Second IND Approval of the Year for NouvNeu003, a First-in-Class Cell Therapy Targeting Early-Onset Parkinson’s Disease

On December 11, the National Medical Products Administration (NMPA) officially approved the IND application for iRegene's pipeline NouvNeu003. This pipeline targets early-onset Parkinson's disease and is aimed at Parkinson's patients under the age of 50.This is also the second IND approved for iRegene this year, following the approval in August for the "combined Phase I-II clinical trial" of the company's new Parkinson's drug NouvNeu001.

In terms of indications, NouvNeu003 and NouvNeu001 respectively target the two major patient populations of Parkinson's disease: "one elderly and one early-stage," while encompassing both genetic and non-genetic types. This achieves iRegene's strategic layout for complete coverage of the entire life cycle and full treatment cycle in the Parkinson's field.

When it comes to Parkinson's disease, the keywords "tremors" and "elderly" always come to mind. However, Parkinson's disease can also occur in young people. According to statistics, the youngest age of onset for Parkinson’s is just 7 years old, and onset before the age of 50 is referred to as Early-Onset Parkinson's Disease (EOPD).

In 2018, the National Health Commission included early-onset Parkinson's disease in the first national edition of the rare disease directory. According to statistics, there are currently more than 3 million Parkinson's patients in China, of which about 300,000 suffer from early-onset Parkinson's disease. However, with the advent of an aging society and increasing social pressures, it is estimated that by 2030, the number of Parkinson's patients in China will surge to around 5 million, with early-onset Parkinson's patients expected to account for 15-20% of the total patient population.

As the world's earliest high-tech pharmaceutical company focused on researching efficient chemical small molecule-induced functional cell regeneration, iRegene has hoped to reverse the progression of neurodegenerative diseases such as Parkinson's through cell fate regulation and transformation since its establishment.

In August this year, NouvNeu001, a cell therapy product independently developed by iRegene for the Parkinson's disease field, received IND approval from the National Medical Products Administration to conduct "combined Phase I-II clinical trials." The product achieves high-purity neuronal subtype reconstruction and functional optimization through compound modulation, forms connections with the body's original neurons, and enhances cellular secretion functions. This further strengthens the transplanted cells' improvement of the original lesion, achieving comprehensive therapeutic effects. This is also the world’s first chemically induced, off-the-shelf cell therapy product to enter the clinical stage in the field of Parkinson's disease treatment.

NouvNeu003, which has received approval for a new drug clinical trial application, targets early-onset Parkinson's disease, specifically for Parkinson's patients under the age of 50.

Dr. Luo Wangqian, partner of Huafang Capital, the earliest investor in iRegene, pointed out that NouvNeu003 has significant social value. Since early Parkinson's patients are usually under 50 years old, in their prime working age and mostly the main breadwinners of the family, the impact on the entire family is enormous once they become ill. The clinical demand for new drugs in this field is very strong.

There is also a strong synergy between the two pipelines, NouvNeu003 and NouvNeu001, with the potential for collaborative reference in terms of pathogenesis and clinical treatment options. "Since early-stage Parkinson's disease is even rarer, NouvNeu003 could potentially become an 'orphan drug.' This may accelerate its clinical progress and market approval, benefiting patients sooner. In this process, NouvNeu003 can also provide experiential guidance for the clinical work of NouvNeu001, helping it reach the market faster," said Dr. Luo Wangqian.

iRegene demonstrates robust operational capabilities and strong execution by advancing two innovative pipelines to pass IND within a year.

Liu Xi, Investment Director of Fortune Venture Capital, who led the Series A financing of iRegene, recalled that in June 2018, when they first contacted iRegene, the company clearly proposed to develop Parkinson's treatment products derived from iPSC. "Looking back, it’s been almost six years; the core R&D direction and key pipelines have never changed. This kind of persistence is quite commendable in the bustling biopharmaceutical R&D environment of the past few years. Although the progress of pipeline development has seen phased adjustments or delays due to the pandemic and unexpected industry incidents leading to tightened reviews, it has ultimately landed steadily within expectations. This not only reflects iRegene's pioneering spirit but also demonstrates the professionalism and resilience of the team."

"Extremely well-planned." Dr. Luo Wangqian also shared a similar assessment of iRegene. "(iRegene) has a style that is both steady and sharp, often surprising us investors. They won't talk about things they are not confident in yet, but when they do, they always deliver as promised."

The confidence of iRegene in its commitment to action and results actually stems from the company's clear strategy, innovative technology platform, and a young team that embodies "passion, professionalism, and execution capabilities."

In the view of Dr. Wei Jun, co-founder and CEO of iRegene, the steady development of Biotech must first clarify its direction, avoid chasing hotspots, and plan clearly. She revealed that before the initiation of the Parkinson's disease pipeline, iRegene had already conducted extensive research and market surveys on drugs, devices, and even preclinical data within the field to identify the actual problems the product needed to solve from the user’s perspective. "We believe that addressing 'real clinical issues' should be the top priority. Only by leveraging the team’s best creativity on the correct clinical needs and using breakthrough technology platforms to efficiently resolve real clinical problems can we create products with market value. In this process, efficient management capabilities are also required to ensure the practical implementation of the technology."

iRegene's steady style also makes it adopt a more rigorous attitude towards innovative drug research and development.

Previously, iRegene had extensive discussions with the Center for Drug Evaluation (CDE) of the National Medical Products Administration to advance regulatory details in the iPSC field. Meanwhile, the company independently developed a platform based on "AI + compound induction" for the targeted modification of cell fine functions. Through precise differential analysis, the platform determines the induction protocols for derivative cells, achieving efficient transformation of induced pluripotent stem cells and ensuring drug feasibility.

Dr. Wei Jun stated that innovative therapies like iPSC require a balance between data rigor and R&D progress during implementation, which is a comprehensive management art. The project advancement plans previously formulated by the company were precisely timed down to the "week," ultimately enabling two internationally pioneering original products to enter clinical trials as scheduled.

The development of new drugs does not always go smoothly. In 2020, when the company's Parkinson's disease pipeline was preparing to enter the pilot stage, it encountered the outbreak of the COVID-19 pandemic, which delayed the trial plan by half a year. To make up for the loss of time, the company adjusted details such as the supply chain and service providers in subsequent plans, eventually catching up with the schedule. "The professionalism, creativity, execution, and resilience demonstrated by the entire team in the face of unexpected difficulties far exceeded the expectations of the founding team."

"At present, the industry has entered a period of adjustment and calm. In fact, everyone suddenly realizes that it's easy to create a beautiful PPT, but the ability to efficiently execute and 'deliver your promise' is the most precious core competitiveness of a startup company," Liu Xi lamented. The efficient teamwork and execution capabilities of iRegene, the high efficiency in the use of funds, as well as the unique core competitive advantages of the company’s chemical small molecule induction technology platform in the CMC process, have all played a crucial role in the company fulfilling its promises to investors.

"Against the backdrop of industry downturn, looking back and forward, these points will become clearer." Liu Xi said.