Shanghai Zhimeng Begins Phase 2/3 Trial of Novel Potassium Channel Opener for ALS

Shanghai Zhimeng Begins Phase 2/3 Trial of Novel Potassium Channel Opener for ALS

By, Admin 1 November 2025

Shanghai Zhimeng Biopharma Inc. has officially launched the Phase 2/3 clinical trial of its new-generation potassium channel opener CB03-154, designed for the treatment of amyotrophic lateral sclerosis (ALS). The first patient has received the first dose at the 2nd Affiliated Hospital of Hebei Medical University, marking a major milestone in Zhimeng’s CNS pipeline.

In July 2025, China’s Center for Drug Evaluation (CDE) approved the study design, and in October 2023, CB03-154 was granted orphan drug designation by the U.S. FDA.

About the Study

The Phase 2/3 adaptive clinical trial is a multicenter, randomized, double-blind, placebo-controlled study with an open-label extension.

  • Lead Investigator: Professor Fan Dongsheng, 3rd Affiliated Hospital of Peking University
  • Planned Enrollment: ~240 ALS patients across 15 centers in China
  • Eligibility: Patients within 24 months of first symptom onset
  • Primary Endpoint: Change from baseline in ALS Functional Rating Scale-Revised (ALSFRS-R) score at Week 39

Understanding ALS

Amyotrophic Lateral Sclerosis (ALS) is a progressive and fatal neurodegenerative disease that damages motor neurons in the brain and spinal cord.

  • Key symptoms: Muscle weakness, atrophy, and respiratory failure
  • Median survival: 2–3 years after symptom onset
  • Current treatments: Riluzole, Edaravone, and Tofersen (Qalsody) — all with limited efficacy
  • Unmet need: No existing therapy can stop or reverse disease progression

The Science Behind CB03-154

CB03-154 is a selective voltage-gated potassium channel (KCNQ2/3) opener developed entirely by Zhimeng.

  • Mechanism: Regulates neuronal excitability by targeting potassium ion channels
  • Preclinical data:
    - Reduced hyperexcitability in ALS patient-derived motor neurons
    - Improved survival and motor performance in SOD-1 transgenic mouse models
    - Delayed disease onset and slowed muscle atrophy progression

This compound represents an improvement over the earlier-generation opener ritigabine (Potiga/Ezogabine), which showed potential in ALS but was withdrawn from the market in 2017 due to pigmentation-related side effects.

What This Means for ALS Patients

The start of this pivotal trial signals new hope for patients suffering from one of the most devastating neurodegenerative diseases. If successful, CB03-154 could become the first-in-class potassium channel opener for ALS and address a critical unmet need in neurotherapeutics.

“The initiation of this Phase 2/3 study marks a significant step forward in our rare disease program,” said a Zhimeng spokesperson. “CB03-154 has the potential to bring meaningful clinical benefit to ALS patients who currently have very limited options.”

About Shanghai Zhimeng Biopharma

Founded in 2018, Zhimeng Biopharma is a Shanghai-based biotechnology company focused on innovative small-molecule drug discovery. Its R&D pipeline includes programs for:

  • Chronic hepatitis B (CHB) functional cure
  • Central nervous system (CNS) disorders such as epilepsy, neuropathic pain, ALS, major depressive disorder, and bipolar disorder

For more information, visit the company’s website.

Optimize Your trial insights with Clival Database.

Are you exhausted from the uncertainty of trial insights pricing? Clival Database ensures the clarity in the midst of the global scenario for clinical trials to you.

Clival Database is one of the best databases that offers an outstanding number of clinical trial data in terms of 50,000+ molecules and from primary regulatory markets as well as new entrants like Indian and Chinese markets.

With Clival, you get accurate positioning of historical sales data, patent database, company profiling, safety & efficacy, and prediction of launch of new innovative molecules helping you to align your research and driving down the cost.

To add value, we further break down our analytics for you so that improving your operational effectiveness; optimizing your clinical trials; and offering you accurate and high-quality data at lowest possible prices becomes possible.

Elevate your trial success rate with the cutting-edge insights from Clival database.

Check it out today and make more informed sourcing decisions! Learn More!