Ascletis Pharma has announced positive topline results from its U.S. Phase I clinical trial of ASC50, a novel oral small molecule targeting interleukin-17 (IL-17). 

 

The randomized, double-blind, placebo-controlled single ascending dose (SAD) study evaluated safety, tolerability, pharmacokinetics, and target engagement in 46 healthy participants.

 

Participants received doses ranging from 10 mg to 600 mg of ASC50 or placebo. 

 

Key findings included -- Long half-life: ASC50’s elimination half-life ranged from 43 to 104 hours across doses, supporting once-daily or potentially once-weekly oral dosing; Strong target engagement: Plasma IL-17A levels remained elevated up to seven days at higher doses.

 

ASC50 showed a consistent, dose-proportional profile from 10 mg to 600 mg. All adverse events were mild and transient. No serious adverse events or discontinuations occurred, and no hepatic safety signals were detected.

 

Based on these results, ASC50 has advanced into a multiple ascending dose study in participants with mild to moderate plaque psoriasis.

 

“These data demonstrate a favorable safety profile as well as a dose-dependent and differentiated pharmacokinetic profile of ASC50,” said Jinzi Jason Wu, Founder, Chairman and CEO of Ascletis. 

 

“We are encouraged by these data as ASC50 is the first oral small molecule drug candidate in immunology developed from our Artificial Intelligence-assisted Structure-Based Drug Discovery (AISBDD) technology. These findings underscore ASC50's potential as a best-in-class oral small molecule IL-17 inhibitor.”