Objective: This Phase 1 study aims to evaluate the safety, tolerability, preliminary efficacy, and optimal dose of NouvNeu001 for treating mid to late-stage Parkinson’s disease (PD) (NCT#06167681).

Background: PD is characterized by degenerative loss of dopaminergic neurons in the substantia nigra. There remains no therapeutic approach can reverse the disease progression in PD. NouvNeu001 is induced pluripotent stem cell (iPSC)-derived and chemically induced human dopaminergic neuron precursor for PD treatment. The transplantation of NouvNeu001 in the putamen is expected to restore dopamine pathways in the brain and revert dopaminergic function.

Method: In this multi-center, open label study, mid to late-stage patients (50-75 yrs) were randomly assigned to the low dose (5×10⁶, N=5) or the high dose (1.5×10⁷, N=5) group. The safety, tolerability, and preliminary efficacy of transplantation of NouvNeu001 in the putamen were evaluated.

Results: 10 patients received the transplantation of NouvNeu001 in the putamen and completed 9-month and 3-month follow-ups in the low dose group and high dose group, respectively. The results showed good safety and tolerability with no reported AEs related to the cell product and no tumor formation detected by MRI in either group. The motor symptom improvement was maintained at 9-month follow-up in the low dose group, showing a reduction trend in UPDRS Part III score “ON” state (change from baseline 25.10±4.91 to 9-month 16.20±5.81) and “OFF” state (change from baseline 58.60±7.26 to 9-month 40.00±11.55), and Hoehn & Yahr stage rating scale “ON” state (change from baseline 2.25±0.22 to 9-month 1.40±0.25) and “OFF” state (change from baseline 3.05±0.25 to 9-month 2.30±0.38). In addition, PET imaging from first patient revealed a steady increase in CFT-DAT post-transplantation during the 12-month observation, indicating the long-term survival of transplanted NouvNeu001 and restoration of DA neuron function.

Conclusion: The Phase 1 results of NouvNeu001 demonstrated the good safety, tolerability, and preliminary efficacy of NouvNeu001, providing supportive evidence for further development of PD treatment. A multi-center Phase II trial of NouvNeu001 is planned to start in 2025H1.

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MDS Abstracts - https://www.mdsabstracts.org/abstract/safety-tolerability-and-preliminary-efficacy-of-nouvneu001-for-the-treatment-of-advanced-parkinsons-disease-a-multi-center-open-label-phase-1-study/