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# Zhimeng Bio Gets Orphan Status for ALS Drug
- URL: https://www.fdaweb.com/zhimeng-bio-gets-orphan-status-for-als-drug/
- Published: 2023-10-24T12:00:00.000Z
- Updated: 2026-09-14T14:14:08.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5155637

FDA has granted Shanghai-based Zhimeng Biopharma an orphan drug designation for its small-molecule KCNQ2/3 selective opener compound (CB03) for treating amyotrophic lateral sclerosis (ALS, also known as Lou Gehrig’s disease). The company says a Phase 1 study evaluating the safety, tolerability, and pharmacokinetics of CB03 in healthy subjects is currently ongoing in Australia and the U.S. It is expected to complete the dosing phase (both single dose and multiple dose) before the end of the year.

Zhimeng says studies have shown that hyperexcitability of neurons is one of the important underlying causes associated with neurodegenerative and neuropsychic central nervous system (CNS) diseases. “As the most widely distributed and the most diverse group of ion channels, potassium (K+) channels are mainly involved in the modulation of neuronal excitability and the frequency and amplitude of action potential discharges,” it says. KCNQ2/3 potassium ion channel opener CB03 thus has the potential to become a safe and effective treatment option for ALS and other CNS diseases, it adds.