Ascletis Pharma Inc. announced the poster presentation of Phase II study final results of ASC40, a first-in-class fatty acid synthase (FASN) inhibitor for treatment of acne, at the 2024 American Academy of Dermatology (AAD) Annual Meeting in San Diego, the United States. Method: This phase 2 trial (NCT05104125) was a randomized, double-blind, placebo-controlled, multicenter study. 180 patients were 1:1:1:1 assigned to the ASC40 255075 mg or placebo QD for 12-week treatment and 2-week follow-up.

Efficacy and safety of ASC40 vs placebo were assessed. Results: At week 2, 4, 8 and 12, percentage and absolute change from baseline in total lesion, inflammatory and non-inflammatory lesion counts as well as treatment success and Investigator's Global Assessment (IGA) reduction = 2 were assessed. At all doses, above efficacy measures generally improved from week 2 to week 12.

50 mg QD demonstrated the best efficacy: placebo-adjusted proportion of patients with treatment success and IGA reduction = 2 were 14.3% and 16.2%, respectively. Placebo-adjusted median percentage (absolute) change from baseline in total lesion and inflammatory counts were -27.1% (-23.5) and -33.5% (-13), respectively (p = 0.008 (0.030) and 0.003 (0.003)). Safety: The incidence rates of study drug related AEs were comparable among 25 mg (grade 1 = 28.9%; grade 2 = 20.0%), 50mg (grade 1 = 36.4%; grade 2 = 11.4%), 75 mg (grade 1 = 44.4%; grade 2 = 17.8%) ASC40 and placebo (grade 1 = 35.6%; grade 2 = 13.3%).

The most common study drug related AE was dry eyes whose incidence rates were similar among 25 mg (grade 1 =17.8%; grade 2 = 6.6%), 50 mg (grade 1 = 22.7%; grade 2 = 2.3%), 75 mg (grade 1 = 15.5%; grade 2 =11.1%) ASC40 and placebo (grade 1 = 28.9%; grade 2 = 6.6%). There were no clinically significant findings in clinical laboratory, vital signs and electrocardiography. There were no ASC40 related grade 3 or 4 AEs and no ASC40 related serious AEs (SAEs).

Conclusion: Based on efficacy and safety data from this phase 2 trial, a phase 3 clinical trial of 50 mg QD ASC40 with 12-week treatment has been initiated.