China-based Shanghai Zhimeng Biopharma, Inc. is set to carry out a Phase II/III clinical study for its next-generation KCNQ2/3 potassium channel opener CB03-154 in amyotrophic lateral sclerosis (ALS) following approval from the Center for Drug Evaluation (CDE) of China’s National Medical Products Administration (NMPA).
ALS: A Severe Neurodegenerative Disease
ALS is a severe neurodegenerative disease characterized by the disruption, damage, or destruction of nerve cells in the brain and spinal cord, ultimately leading to loss of muscle control. While the exact cause of ALS remains unclear, research indicates that it is closely linked to ion channel dysfunction, impaired synaptic transmission, and neuronal excitation-inhibition imbalance.
CB03-154: A Promising Therapeutic Approach
CB03-154 is a novel KCNQ2/3 potassium channel opener that features high ion channel selectivity, excellent chemical and metabolic stability, strong anti-neuronal hyperexcitability activity, and favorable pharmacokinetic and safety profiles. It has shown potential in treating various central nervous system (CNS) disorders, including ALS, epilepsy, major depressive disorder (MDD), and others. Preclinical studies have demonstrated that CB03-154 significantly reduces motor neuron hyperexcitability in ALS models, slows the deterioration of muscle strength-related functional parameters, delays disease onset, extends survival, and normalizes muscle and neuronal morphology.-Fineline Info & Tech
