Ascletis Pharma-B (01672) announced positive topline results from a Phase III open-label study (NCT06248008) of denifostat (ASC40), a first-in-class, once-daily oral small-molecule fatty acid synthase (FASN) inhibitor, in patients with moderate-to-severe acne vulgaris.
This recently completed second Phase III study was an open-label, multi-center trial conducted in China, designed to evaluate the long-term safety of denifostat (ASC40) in 240 patients with moderate-to-severe acne vulgaris.
All 240 patients received once-daily denifostat (ASC40) treatment for 40 weeks, having previously undergone 12 weeks of treatment with either denifostat (ASC40) or a placebo.
The primary endpoints included the incidence of treatment-emergent adverse events (TEAEs), the incidence of serious adverse events (SAEs), and the incidence of treatment discontinuation due to adverse events (AEs).
Denifostat (ASC40) demonstrated a favorable safety and tolerability profile.
The majority of treatment-emergent adverse events were mild (Grade 1) or moderate (Grade 2) in severity.
There were no Grade 3 or 4 adverse events related to denifostat (ASC40), and no serious adverse events associated with the treatment were reported.
No deaths were reported.
The company had previously announced on June 4, 2025, that a randomized, double-blind, placebo-controlled Phase III clinical study (NCT06192264) of denifostat (ASC40) in 480 patients with moderate-to-severe acne vulgaris had met all primary, key secondary, and secondary endpoints.
The mechanism of action for denifostat (ASC40) in treating acne involves the direct inhibition of sebum production by suppressing de novo lipogenesis (DNL) in human sebocytes; it also combats inflammation by reducing cytokine secretion and Th17 cell differentiation.
Excessive sebum production is a primary driver of acne, and the unique mechanism of denifostat (ASC40) directly addresses this overproduction, setting it apart from most other acne treatments that do not target the root cause of the condition.
Ascletis has obtained the exclusive rights for denifostat (ASC40) in Greater China from Sagimet Biosciences Inc. (NASDAQ: SGMT).