Objective: This ongoing, phase 1/2, multi-center, open label study aims to evaluate the safety, tolerability, and efficacy of NouvNeu001 (NCT#06167681) for treating mid to late-stage Parkinson’s disease (PD).

Background: PD is pathologically characterized by the progressive nigral dopaminergic neuron degeneration, and currently no disease-modifying therapy can reverse this neurodegenerative process. To replace the damaged dopaminergic neurons in PD patients, NouvNeu001 has been developed as a chemically induced pluripotent stem cell (iPSC)-derived human dopaminergic neuron precursor.

Method: This multi-center, open label study aims to enroll PD patients aged between 50-75 years. All patients will receive the transplantation of NouvNeu001, enabling evaluation of safety, efficacy, and tolerability for PD treatment.

Results: Phase 1 study revealed that NouvNeu001 was well tolerated, and no AEs or SAEs related to cell product were observed. Furthermore, in the low dose group (5×10⁶ cells, N=5, except N=4 at 15 months due to one participant unable to make the visit) at 15 months post transplantation, the mean of UPDRS Part III score decreased by 14.50±4.51 points from baseline (25.10±4.91 points) in the “ON” state, and decreased by 24.75±10.92 points from baseline (58.60±7.26 points) in “OFF” state; the average value of H-Y stage decreased by 1.06±0.33 from baseline (2.25±0.22) in the “ON” state, and decreased by 1.06±0.39 from baseline (3.05±0.25) in “OFF” state. In the high dose group (1.5×10⁷ cells, N=3) at 9 months post transplantation, the mean of UPDRS Part III score decreased by 9.67±5.85 points from baseline (29.33±1.36 points) in the “ON” state, and decreased by 23.33±2.46 points from baseline (65.00±5.22 points) in “OFF” state; the average value of H-Y stage decreased by 0.50±0.14 from baseline (2.50±0.25) in the “ON” state, and decreased by 0.83±0.17 from baseline (3.33±0.33) in “OFF” state. Currently, the longest follow-up for dosed patients has exceeded two years, with favorable safety and efficacy profiles. NouvNeu001 has successfully completed phase 1 and proceeded to phase 2.

Conclusion: Phase 1 study demonstrated a significant improvement of motor symptom, providing supportive evidence for therapeutic potential of NouvNeu001 for treating PD. The phase 2 clinical trial of NouvNeu001 aims to finish enrollment no later than 2026H1.

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MDS Abstracts - https://www.mdsabstracts.org/abstract/an-ongoing-phase-1-2-multi-center-open-label-study-of-nouvneu001-for-the-treatment-of-advanced-parkinsons-disease/