Shanghai Zhimeng Biopharma Inc, a Chinese clinical-stage biopharmaceutical company involved in drug development for liver and central nervous system (CNS) diseases, announced on Wednesday that its self-discovered next-generation KCNQ2/3 potassium channel opener, CB03-154, has been approved by the Center for Drug Evaluation (CDE) of China's National Medical Products Administration (NMPA) to initiate a Phase 2/3 clinical study for amyotrophic lateral sclerosis (ALS, also known as Lou Gehrig's disease).

Previously, CB03-154 tablets were granted Orphan Drug Designation (ODD) by the US Food and Drug Administration (FDA) for the treatment of ALS patients.

Zhimeng Biopharma says that CB03-154 is a next-generation KCNQ2/3 potassium channel opener with excellent ion channel selectivity, chemical and metabolic stability, anti-neuronal hyperexcitability activity, and favourable pharmacokinetic and safety profiles. It holds potential not only for ALS but also for other CNS disorders such as epilepsy and major depressive disorder. According to the company, preclinical studies have demonstrated that CB03-154 significantly reduces the hyperexcitability of ALS motor neurons, markedly slows the deterioration of muscle strength-related functional parameters, delays the disease onset, extends the life expectancies, and normalises the morphology of muscle and neuronal cells.

CB03-154 is being evaluated in Phase 1 clinical trials taking place in the US and Australia for healthy adults, and has also completed the bridging pharmacokinetic and safety study in Chinese healthy subjects.

In 2024, the Phase 1 clinical trial results and the pre-clinical efficacy data of ALS and epilepsy of CB03-154 were presented at the American Neurological Association (ANA) Annual Meeting and the American Epilepsy Society (AES) Annual Meeting. Due to its innovation and efficacy data, the abstract on ALS-related pre-clinical findings received the honour of best poster of ANA 2024 annual meeting, and Zhimeng Biopharma was invited to give an oral presentation at the Movement Disorder Special Interest Group symposium.