Category: Parkinson’s Disease: Clinical Trials
Objective: To investigate the efficacy and safety of TJ0113 for patients with early-stage Parkinson’s disease.
Background: Mitochondrial dysfunction, particularly decreased mitophagy, may contribute to the pathogenesis of Parkinson’s disease. TJ0113 is a first-in-class small molecule mitophagy activator, which may be clinically translated to mitophagy-based therapy.
Method: This 12-week, randomized, double-blind, phase 2 trial was conducted in China. Participants with early-stage Parkinson’s disease received an oral placebo, 200-mg TJ0113, or 400-mg TJ0113 once daily (1:1:1 ratio). All participants continued medications for Parkinson’s disease during the study. The primary endpoint was changes in scores for part III of the Movement Disorder Society-sponsored revision of the Unified Parkinson’s Disease Rating Scale (MDS-UPDRS III) from baseline to week 12.
Results: Of the 150 participants, 51 received a placebo, 50 received 200 mg TJ0113, and 49 received 400 mg TJ0113. At week 12, the mean (± SE) changes in the MDS-UPDRS III score were −0.7 ± 0.7 points in the placebo group, −7.1 ± 0.7 points in the 200-mg group (95% confidence interval [CI], [−8.1, −4.6]; P < 0.001), and −7.4 ± 0.7 points in the 400 mg group (95% CI, [−8.5, −5.0]; P < 0.001) off medication. MDS-UPDRS III scores measured on medication also tended to improve. The incidences of adverse events were not significantly different between the TJ0113 and placebo groups (TJ0113, 54.1% vs. Placebo, 49.0%).
Conclusion: TJ0113 treatment over 12 weeks significantly improved motor function in patients with early-stage Parkinson’s disease compared to placebo, and the safety profile was comparable to placebo. These data support the further investigation of TJ0113 in a phase 3 trial designed to confirm its potential effects on early-stage Parkinson’s disease.
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To cite this abstract in AMA style:
W. Luo, ZD. Cen, XF. Cen, XH. Chen, S. Wu, Y. Tan, LQ. Mao, XL. Mao, B. Zhang, W. Lv, SZ. Liu, YP. Wang, D. Liu, HG. Xia. Phase 2 Trial for the Treatment of Early-stage Parkinson’s Disease with TJ0113, a Small Molecule Mitophagy Activator [abstract]. Mov Disord. 2026; 41 (suppl 1). https://www.mdsabstracts.org/abstract/phase-2-trial-for-the-treatment-of-early-stage-parkinsons-disease-with-tj0113-a-small-molecule-mitophagy-activator/. Accessed October 1, 2026.« Back to 2026 International Congress
MDS Abstracts - https://www.mdsabstracts.org/abstract/phase-2-trial-for-the-treatment-of-early-stage-parkinsons-disease-with-tj0113-a-small-molecule-mitophagy-activator/