SHANGHAI, Oct. 31, 2026 /PRNewswire/ -- Shanghai Zhimeng Biopharma, Inc. ( "Zhimeng Biopharma") announced today that its independently developed next-generation potassium channel opener, CB03-154, intended for the treatment of amyotrophic lateral sclerosis (ALS), has successfully completed patient enrollment in its pivotal Phase 2/3 clinical trial, in total enrolled 255 patients. This significant milestone marks the entry of the critical stage in the clinical development of CB03-154 for ALS in China, laying a solid foundation for the upcoming analysis of topline data and potential new drug application submissions.

This pivotal clinical trial has been conducted in China, with Professor Fan Dongsheng from the 3^rd Affiliated Hospital of Peking University serving as the national leading principal investigator (Leading PI), and involving a total of 15 clinical research centers in Mainland China. The study aims to evaluate the efficacy and safety of CB03-154 in patients with ALS, with the primary efficacy endpoint being the change from baseline in the ALSFRS‑R score at Week 39. Previously, CB03-154 received approval from the China CDE in July 2025 to initiate Phase 2/3 clinical trials for ALS, and in October 2023 it was granted Orphan Drug Designation (ODD) by the U.S. FDA.

As a rare disease, ALS poses numerous unique challenges in drug development. Notably, based on the current clinical progress, if the Phase 2 topline results are positive in the third quarter of 2027, Zhimeng Biopharma plans to submit a marketing application to the China CDE for a conditional approval. This strategy is expected to enable ALS patients to gain access to innovative therapies sooner, reflecting the company's patient‑centered R&D philosophy in the rare‑disease space.

CB03-154 is a next-generation, selective potassium‑channel opener of the voltage‑gated KCNQ2/3, independently discovered and developed by Zhimeng Biopharma, and possesses potential best-in-class properties. By modulating neuronal excitability and restoring the balance between excitation and inhibition, this compound has demonstrated neuroprotective effects on motor neurons in preclinical studies. The completion of enrollment in this pivotal clinical trial marks the entry of a critical phase of validating the efficacy and safety of CB03-154 in patients with ALS.

In terms of clinical development, CB03-154 has been steadily advancing across multiple CNS indications. As of October 2026, in addition to the ALS indication, the drug has already entered the Phase 2 clinical trial for Focal Epilepsy in China and Australia. Furthermore, in June 2026, it received approval from the China CDE to initiate a Phase 2 trial for Major Depressive Disorder (MDD). The concurrent advancement of these three clinical studies fully underscores CB03-154's broad development potential in the field of central nervous system disorders.

Dr. Huanming Chen, the Founder and CEO of Zhimeng Biopharma, stated: "The completion of patient enrollment for the pivotal Phase 2/3 clinical trial of CB03-154 marks a strategically significant milestone for Zhimeng Biopharma in the field of ALS treatment. I would like to extend my sincere appreciation to Professor Fan Dongsheng and the investigators at all clinical research centers across China for their dedicated efforts, as well as to all ALS patients and their families who participated in the trial for their selfless commitment. Moving forward, we will spare no effort to advance clinical development and regulatory submissions, striving to bring a truly effective new treatment option to ALS patients in China and around the world as soon as possible."

About Zhimeng

Founded in 2018, Shanghai Zhimeng Biopharma, Inc, is a clinical stage biopharmaceutical company dedicated to the research and development of original small‑molecule drugs. The company focuses on liver diseases and central nervous system disorders, with a strong pipeline targeting treatment options for refractory epilepsy, neuropathic pain, amyotrophic lateral sclerosis (ALS), major depressive disorder (MDD), and other neurological conditions. In addition to its CNS portfolio, Zhimeng Biopharma has also made significant progress in the field of functional cure for chronic hepatitis B; its leading product, ZM‑H1505R (capsid inhibitor), has entered pivotal Phase 3 trial in China.

Disclaimer

This press release contains forward-looking statements. While Zhimeng considers the projections to be based on reasonable assumptions, these forward-looking statements may be called into question by a number of hazards and uncertainties, so that actual results may differ materially from those anticipated in such forward-looking statements.

For more information, please visit: www.corebiopharma.com