255 people, 15 hospitals, one hard disease: an ALS drug candidate in China fills its pivotal trial
Amyotrophic lateral sclerosis — ALS, the disease that progressively takes away movement — has very few treatment options. Shanghai company Zhimeng Biopharma has now completed enrolling 255 patients across 15 research centres in China into the pivotal Phase 2/3 trial of CB03-154, its next-generation drug designed to calm over-excited nerve cells by opening specific potassium channels — tiny gates that control electrical signals in neurons. The trial, led nationally by Professor Fan Dongsheng of Peking University Third Hospital, will track patients' function over 39 weeks. The drug already holds US orphan-drug designation, a status meant to speed development for rare diseases, and is also in trials for epilepsy. Enrolment is a milestone, not a result: whether the drug helps will only be known when the data are read out, expected in 2027.
SDG 3 · Good health and well-being — Mechanism: tech innovation