Shanghai, China – September 11, 2025 – Shanghai Zhimeng Biopharma, Inc. today announced that its investigational therapy CB03-154, a novel next-generation KCNQ2/3 potassium channel opener, has received regulatory approval from China’s Center for Drug Evaluation (CDE) to initiate a Phase 2 clinical trial in refractory epilepsy. This milestone underscores Zhimeng’s growing leadership in central nervous system (CNS) drug innovation and strengthens its development pipeline, which also includes therapies for amyotrophic lateral sclerosis (ALS) and chronic hepatitis B.

Science Significance

Epilepsy, a disorder of neuronal hyperexcitability, affects nearly 90 million people globally, including 9 million in China, with about 30% of patients experiencing refractory forms that resist standard monotherapy. The disease is tightly linked to dysfunctional ion channels, making potassium (K+) channels particularly attractive therapeutic targets.

CB03-154 demonstrates high selectivity for KCNQ2/3 potassium channels, offering enhanced stability, safety, and efficacy compared with first-generation openers. Preclinical and early clinical studies suggest it has favorable pharmacokinetic properties, in vitro and in vivo anti-epileptic activity, and fewer safety issues than earlier compounds. Beyond epilepsy, the compound has potential applications in ALS, major depressive disorder (MDD), and neuropathic pain.

Regulatory Significance

The approval by China’s National Medical Products Administration (NMPA) CDE allows Zhimeng to conduct a randomized, double-blind, placebo-controlled, multinational, multicenter Phase 2 trial. This trial will evaluate the efficacy, safety, and tolerability of CB03-154 as an adjunctive therapy in adult patients aged 18–70 with focal epilepsy.

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This comes only months after CDE granted clearance for Phase 2/3 ALS studies of CB03-154, highlighting regulatory momentum. In addition, the U.S. FDA previously awarded Orphan Drug Designation (ODD) to CB03-154 for ALS, potentially accelerating global development timelines and incentives for the program.

Business Significance

For Zhimeng, founded in 2018, this regulatory progress validates its dual focus on antiviral and CNS therapeutic innovation. The company has raised multiple rounds of financing to support a pipeline that spans:

  • CB03-154 for epilepsy, ALS, neuropathic pain, and MDD.
  • ZM-H1505R, an HBV core protein inhibitor now in Phase 3 pivotal studies for chronic hepatitis B.

Advancing CB03-154 into Phase 2 strengthens the company’s value proposition as a clinical-stage biopharma with differentiated assets in underserved therapeutic areas.

Patients’ Significance

Patients with refractory epilepsy face severe unmet needs: uncontrolled seizures, impaired quality of life, and higher risks of morbidity and mortality. With 30% of epilepsy patients resistant to existing therapies, the approval of CB03-154 to enter Phase 2 offers new hope for adjunctive treatment strategies.

As Dr. Huanming Chen, CEO of Zhimeng Biopharma, emphasized: “We are committed to deepening our work in areas of unmet clinical need, progressing clinical trials at full speed, and providing patients with safer and more effective treatment options.”

Policy Significance

China’s NMPA approval reflects the government’s commitment to advancing innovative therapies for CNS disorders, a growing public health challenge. With epilepsy prevalence high in Asia and healthcare systems under pressure to provide affordable care, successful development of CB03-154 could reduce long-term healthcare costs while improving patient outcomes.

Furthermore, the global regulatory recognition (China NMPA, U.S. FDA ODD) illustrates how international collaboration in rare disease and CNS drug development can accelerate innovation pathways.

Transaction Highlights

Zhimeng Biopharma’s CB03-154 is cleared to begin a Phase 2, double-blind, placebo-controlled multinational trial for refractory focal epilepsy in adults. The compound has shown strong preclinical safety and efficacy, with approvals also secured for Phase 2/3 ALS studies. It carries U.S. FDA Orphan Drug Designation for ALS, strengthening its global development prospects. Alongside its Phase 3-ready HBV core inhibitor ZM-H1505R, Zhimeng is building a robust dual therapeutic pipeline in CNS and antiviral diseases. This milestone marks a significant advancement for both the company and the millions of patients worldwide awaiting better treatment options.

Source: Shanghai Zhimeng Biopharma, Inc., Press Release