Shanghai – October 31, 2026 -- Shanghai Zhimeng Biopharma has completed enrollment of 255 patients in the pivotal Phase 2/3 trial of CB03-154 for amyotrophic lateral sclerosis (ALS), moving the potassium channel opener toward topline data and a possible new drug application in China.
Trial runs at 15 Chinese centers with a Week 39 primary endpoint
The study is led by Professor Fan Dongsheng of the 3rd Affiliated Hospital of Peking University and spans 15 clinical research centers in Mainland China. Its primary efficacy endpoint is the change from baseline in ALSFRS-R score at Week 39. China's Center for Drug Evaluation (CDE) cleared the Phase 2/3 study in July 2025. The U.S. FDA granted CB03-154 Orphan Drug Designation in October 2023.
Zhimeng targets conditional approval filing after Q3 2027 topline data
Zhimeng expects Phase 2 topline results in the third quarter of 2027. If they are positive, it plans to file a marketing application with the CDE seeking conditional approval. The company says this route could give ALS patients earlier access to the therapy.
CB03-154 targets KCNQ2/3 channels and shows motor neuron protection in preclinical work
CB03-154 is a selective opener of voltage-gated KCNQ2/3 potassium channels, discovered and developed in-house by Zhimeng. In preclinical studies, it showed neuroprotective effects on motor neurons by modulating neuronal excitability and restoring the balance between excitation and inhibition. The company describes the compound as having potential best-in-class properties.
Two further CNS programs run alongside the ALS study
As of October 2026, CB03-154 is in a Phase 2 trial for focal epilepsy in China and Australia. In June 2026, the CDE approved a Phase 2 trial in major depressive disorder (MDD). Together with ALS, that gives the compound three concurrent clinical studies.
Founder and CEO Dr. Huanming Chen called the enrollment completion a strategically significant milestone. He said the company will focus on clinical development and regulatory submissions for ALS patients in China and globally.