#China #Shanghai #ALS #Zhimeng Biopharma #CB03-154

Zhimeng Biopharma Achieves Key Enrollment Milestone in ALS Trial

Shanghai Zhimeng Biopharma, Inc. has proudly announced the completion of patient enrollment for its promising investigational drug, CB03-154, in a critical Phase 2/3 clinical trial aimed at treating Amyotrophic Lateral Sclerosis (ALS). The successful enrollment of 255 patients marks a significant step forward in the drug's development process, setting the stage for crucial analyses and potential new drug applications.

Professor Fan Dongsheng from the 3rd Affiliated Hospital of Peking University takes on the role of the principal investigator in this noteworthy trial, which encompasses 15 clinical research centers across Mainland China. The primary objective of this trial is to evaluate both the efficacy and safety of CB03-154, with the primary efficacy endpoint focused on changes from baseline in the ALS Functional Rating Scale-Revised (ALSFRS-R) score over a 39-week period.

Zhimeng Biopharma has previously received regulatory approval from the China Center for Drug Evaluation (CDE) back in July 2025 to commence this phase of clinical trials. Moreover, in a significant recognition of its potential, CB03-154 was granted Orphan Drug Designation by the U.S. FDA in October 2023. Given the unique challenges associated with developing drugs for ALS, a rare disease, Zhimeng Biopharma aims to expedite the approval process. Should the topline results of this Phase 2 evaluation be promising, the company plans to submit a marketing application to the CDE for conditional approval, thereby allowing ALS patients quicker access to groundbreaking therapies.

CB03-154 stands out as a next-generation selective potassium-channel opener specifically targeting KCNQ2/3 channels. This compound promises potential best-in-class efficacy, as it has demonstrated neuroprotective effects on motor neurons in preclinical studies, primarily by functionally modulating neuronal excitability. The completion of patient enrollment signifies the transition to an essential phase of validating CB03-154's safety and effectiveness for ALS patients.

In addition to ALS, Zhimeng Biopharma is actively progressing CB03-154 in other Central Nervous System (CNS) indications. By October 2026, the drug had also initiated Phase 2 trials for Focal Epilepsy in both China and Australia. Furthermore, in June 2026, Zhimeng Biopharma obtained approval from the CDE to launch a Phase 2 trial targeting Major Depressive Disorder (MDD). The simultaneous advancement of these trials accentuates the broader developmental prospects for CB03-154 in addressing various CNS disorders.

Dr. Huanming Chen, the esteemed Founder and CEO of Zhimeng Biopharma, expressed profound gratitude to Professor Fan Dongsheng and all collaborating researchers for their dedicated efforts throughout the trial. He also acknowledged the courage and commitment of ALS patients and their families who contributed by participating in the study. Looking ahead, Dr. Chen reaffirmed Zhimeng's commitment to propelling the clinical development process and regulatory submissions, aiming to deliver effective treatment options to ALS patients both in China and globally.

Founded in 2018, Zhimeng Biopharma focuses on innovating original small-molecule drugs addressing liver diseases and CNS disorders. The company boasts a robust portfolio aimed at treating refractory epilepsy, neuropathic pain, ALS, MDD, and other neurological conditions. In addition, Zhimeng is making notable strides in pursuing a functional cure for chronic hepatitis B, with its leading product currently undergoing pivotal Phase 3 trials in China.

As Zhimeng Biopharma navigates the complexities of drug development in the rare disease space, the completion of patient enrollment for CB03-154 underscores significant progress toward increasing patient access to potentially life-changing therapies, reaffirming the company's patient-centered philosophy in research and development.