iRegene Therapeutics Completes Phase I Enrollment of NouvNeu001, the World’s First Chemically Induced iPSC-Derived Cell Therapy for Mid-to-Late Stage Parkinson’s Disease

Recently, there is new news in the field of Parkinson's disease cell drug treatment.NouvNeu001 Injection, Fully Independently Developed by iRegene for the Treatment of Mid-to-Late Stage Parkinson's Disease, Completes Dosing of the Last Subject in the High-Dose Group Phase I Clinical Study at Beijing Hospital.

It is reported that NouvNeu001, fully independently developed by iRegene, is a type of chemically induced functional human dopaminergic neuronal precursor cell, and alsoThe World's First Universal Cell Therapy Product Based on Chemical Induction to Enter Clinical Stage, which has preliminarily demonstrated the potential to treat mid-to-late stage Parkinson's disease in the current Phase I clinical study.

"At present, the overall safety of subjects in the low-dose group and high-dose group is good, both showing an improving trend in motor function as transplantation time increases," said Dr. Wei Jun, founder and CEO of iRegene, in an exclusive interview with PharmaDJ. This also preliminarily confirms the therapeutic effect of chemically induced functional human-derived dopamine precursor cell products on mid-to-late stage Parkinson's disease.

Public information shows that iRegene was founded in 2017 as an international innovative biopharmaceutical company focused on the field of regenerative medicine. Through its unique technology platform, the company concentrates on small-molecule chemical transcriptional regulation of iPSC and has established the world’s first one-stop cell drug development platform based on "artificial intelligence (AI) + chemical induction." It has multiple FIC pipeline products covering neurodegenerative diseases and ophthalmology. In October this year, the company completed a Series B round of financing exceeding 100 million yuan.

On August 2, 2023, the National Medical Products Administration (NMPA) officially approved the I-II phase IND application for iRegene's Parkinson's disease drug NouvNeu001. This makes it the world's first chemically-induced, universal-type cell therapy product to enter the clinical stage in the field of Parkinson's treatment.

"Currently, in clinical practice, both the chemical drug treatment regimen represented by levodopa and the physical treatment regimen represented by DBS are compensatory therapies. They cannot address the issue of missing functional neurons in patients' brains and have significant side effects such as the end-of-dose effect," said Dr. Wei Jun in an exclusive interview. In response to this, iRegene began actively exploring the possibility of cell drug therapy for treating Parkinson's disease at its inception.

According to Dr. Wei Jun, unlike conventional drug or surgical treatments, NouvNeu001 focuses on clinical needs, utilizing compounds to achieve precise cellular function modification and high-purity neuronal subtype differentiation. This enables the cells to connect with the body's original neurons, enhancing cellular secretion functions and strengthening the improvement of the original lesion by transplanted cells to achieve comprehensive therapeutic effects. "It can be said that NouvNeu001 is an innovative universal cell therapy product that was initiated with clinical needs in mind and developed following the 'end-to-beginning' logic."

The interim results of this Phase I clinical study have also preliminarily confirmed the therapeutic effect of this product on mid-to-late stage Parkinson's disease.

Dr. Wei Jun introduced that the overall safety of the low-dose group of NouvNeu001 was good, with no cell drug-related adverse reactions. Moreover, some motor functions of the subjects showed stable improvement six months after transplantation, with reduced dyskinesia and extended "on" periods, among other positive effects. The dopamine transporter activity in the transplant area was significantly enhanced, confirming the healthy survival of NouvNeu001, a universal cellular therapeutic product, within the patients' bodies post-transplantation. Meanwhile, sleep and quality of life data also indicated an improving trend in non-motor symptoms among the subjects.

In addition, after completing the dosing of the low-dose group and a detailed safety evaluation by the expert panel, the Phase I clinical trial of NouvNeu001 also initiated the enrollment of subjects in the high-dose group. Similarly, an improvement trend in motor function with increased transplantation time was observed, and the overall safety of the subjects remained favorable.

While achieving phased progress in China, in June this year, the U.S. Food and Drug Administration (FDA) also officially approved the IND application of NouvNeu001, formally realizing the "dual filing and dual approval" of IND in both China and the United States.

According to official information from iRegene, the Phase II clinical trial of NouvNeu001 in China is also expected to begin enrollment in the first quarter of 2025.

In addition to NouvNeu001, iRegene's other FIC cell drug pipelines are also advancing steadily.

In December last year, the National Medical Products Administration (NMPA) officially approved iRegene's IND application for NouvNeu003, a pipeline under research targeting early-onset Parkinson's disease, marking the second IND approval for iRegene that year.

This pipeline targets Parkinson's patients with an onset age of less than 50, and together with NouvNeu001, establishes a strategic layout that fully covers the entire life cycle and treatment cycle for two major patient populations: "early onset" and "elderly onset," encompassing both genetic and non-genetic types.

In Dr. Wei Jun's view, there is a strong synergy between the NouvNeu003 and NouvNeu001 pipelines. Moreover, since early-onset Parkinson's disease is rarer, NouvNeu003 has the potential to become an "orphan drug."

In addition, iRegene's ophthalmic product NouvSight001 for "retinitis pigmentosa series of indications" has been granted orphan drug designation by the U.S. FDA; the world's first chemically induced cell-derived extracellular vesicle product NouvSoma001 for "neuromyelitis optica spectrum disorder" also held a clinical trial initiation meeting in September this year.

In March this year, iRegene was granted a Special Exemption by the FDA. Dr. Wei Jun introduced that iRegene has become the world's only cell therapy company to receive special exemption qualifications from the FDA. "This not only represents the FDA's recognition of the company’s pioneering platform and the innovative production and quality systems built on this platform, but also marks the company as an internationally leading team in the field of Parkinson's disease cell therapy, ranking first globally in terms of recognition by regulatory agencies, as well as the number, quality, and progress of clinical trials."

It is precisely based on the latest progress of the aforementioned FIC self-developed pipeline that, in 2024 when financing in the pharmaceutical sector has generally cooled down, iRegene once again secured a large-scale financing round exceeding 100 million yuan.

Public information shows that in October this year, iRegene officially completed a Series B financing round of over 100 million yuan. This round was jointly led by Ceyuan Capital and Guosheng Capital, with TAIHO Pharmaceutical and Wuhan Optics Valley Industrial Investment following up. The funds will be used to accelerate the clinical advancement in both China and the U.S. for NouvNeu001, NouvNeu003, and the ophthalmic treatment product NouvSight001, which has been granted "Orphan Drug" designation by the U.S. FDA.

Dr. Fang Yang, Executive Director of CEC Capital, who served as the financial advisor for iRegene's Series B financing round, pointed out, "iRegene's solid R&D and clinical advancement have established its leading position in product development, industrial implementation, and various other aspects."

Dr. Wangqian Luo, a partner at Huafang Capital who participated in iRegene's earlier financing rounds, also once remarked that iRegene’s style is "steadfast yet sharp."

"They often bring surprises to us investors. They won't talk about things they are not sure about yet, and what they say they can always achieve as scheduled," said Luo Wangqian.

Notably, the latest information reveals that iRegene's B+ round of financing is also nearing its final stage and will be closed shortly.

"iRegene has always adhered to the principle of addressing genuine clinical needs by starting from the patients' demands, using original innovation to develop entirely new therapies that can truly reverse disease progression," said Dr. Wei Jun. In the future, the company will also fully advance its subsequent clinical pipelines and global trials, with the aim of benefiting more patients worldwide as soon as possible.