Shanghai Zhimeng Biopharma, Inc., a China-based clinical-stage biopharmaceutical company, announced on Friday that it has received orphan drug designation (ODD) from US FDA for one of its innovative small-molecule KCNQ2/3 selective opener compounds (CB03), developed for the treatment of amyotrophic lateral sclerosis (ALS).

The product is a candidate drug for the treatment of ALS and other central nerve system (CNS) diseases, independently developed by the company. Presently, the company is conducting a phase one study for the assessment of the safety, tolerability, and pharmacokinetics of CB03 in healthy subjects in Australia and the US. The phase one study is expected to complete the dosing phase (both single dose and multiple dose) before the end of 2023.

Dr Huanming Chen, Zhimeng president and CEO, said, '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. 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.'