Objective: To access the efficacy and safety of NouvNeu001 in models of Parkinson’s Disease (PD) and a Phase 1 clinical trial (NCT#06167681).

Background: Parkinson’s disease (PD) is a neurodegenerative disease without the treatment reversing the progression of the disease. NouvNeu001 is a chemically induced human dopaminergic neuron precursor derived from iPSCs for the treatment of moderate to severe PD. Therefore, the present studies aim to evaluate the preclinical and clinical therapeutic efficacy and safety of NouvNeu001 in PD.

Method: In the efficacy study, 6-OHDA Wistar rats (n=80) and MPTP cynomolgus monkeys (n=15) were used to evaluate parameters including behavioral tests and MRI scans. In the safety study, immunodeficient NOG mice (n=180), naive SD rats (n=120), immunodeficient BALB/c nude mice (n=36) and naive cynomolgus monkeys (n=42) were evaluated. The Phase 1 study is an open-label, exploratory study that recruited patients (age: 50-75 yrs) with mid- to late-stage PD. Efficacy and safety were evaluated post transplantation of NouvNeu001.

Results: NouvNeu001 transplantation leads to significant improvement in motor function and restoration of dopamine levels in diseased animals. The mean PD motor recovery rate was approximately 50% in the rats and the mean PD score improvement rate was approximately 60% (range 48.43%-88.78%) in the cynomolgus monkeys. NouvNeu001 differentiated into mature and functional dopaminergic neurons (85.0%-99.2% GIRK2⁺; 99.0%-99.6%TH⁺), and reconstituted the host neural circuits after transplantation in vivo. NouvNeu001 survived well in monkeys at 36 weeks post cell transplantation. NouvNeu001 showed no tumor formation or side effects at doses of up to 1.5×10⁷/monkey. Furthermore, NouvNeu001 has completed dosing of its first patient and the results demonstrated good safety and tolerability, significant improvement of up to 40% in UPDRS Part III (ON/OFF medication) as compared to baseline, and decreased from Hoehn & Yahr stage 2.5 to 1.5 at four weeks post-transplant.

Conclusion: These preclinical and first-in-human study results indicate that NouvNeu001 has the potential to be a safe and effective therapy for the treatment of PD patients with minimal risks.

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MDS Abstracts - https://www.mdsabstracts.org/abstract/nouvneu001-a-phase-1-stage-chemically-induced-human-dopaminergic-progenitor-cell-therapy-for-the-treatment-of-mid-to-late-stage-parkinsons-disease/