By: IPP Bureau
Last updated : October 23, 2023 9:16 am
CB03 is a candidate drug for the treatment of ALS and other central nerve system (CNS) diseases
Shanghai Zhimeng Biopharma (Zhimeng) announced that one of its innovative small-molecule KCNQ2/3 selective opener compounds (CB03), developed for the treatment of amyotrophic lateral sclerosis (ALS), received orphan drug designation (ODD) from US FDA. CB03 is also being developed for other central nervous diseases, such as refractory epilepsy, major depressive disorders (MDD), etc.
CB03 is a candidate drug for the treatment of ALS and other central nerve system (CNS) diseases independently developed by Zhimeng. A phase 1 study for the evaluation of the safety, tolerability, and pharmacokinetics of CB03 in healthy subjects is currently ongoing in Australia and US. The phase 1 study is expected to complete the dosing phase (both single dose and multiple dose) before the end of 2023.
"The orphan drug designation by US FDA for CB03 is an important milestone in its global clinical development for ALS and other central nervous system diseases," said Dr. Huanming Chen, President and CEO of Zhimeng.
"Since ALS is a very serious disease without adequate treatment options, we hope our dedicated efforts will allow us to bring a safer and more effective medicine to ALS patients worldwide as the first indication for CB03."