Yuhan Corporation presented population PK/PD modeling results for YH35995 at the American Conference on Pharmacometrics (ACoP 2026) on October 12, 2026.
YH35995 is a novel oral glucosylceramide synthase (GCS) inhibitor with high blood-brain barrier penetration, being developed for Gaucher disease type 3. It has received orphan drug designation from both the U.S. FDA and the European Medicines Agency (EMA). A multiple-ascending-dose study evaluating Q4W dosing is ongoing.
Based on Phase 1 data from 50 healthy participants (NCT06517914), PK/PD modeling supported the potential for once-monthly (Q4W) oral dosing of YH35995. At steady-state, the model predicted approximately 40%, 70% and 80% reductions in plasma glucosylceramide (GL-1) at doses of 10, 30, and 60 mg, respectively. These findings, together with YH35995’s prolonged elimination half-life, support the feasibility of Q4W dosing at exposures projected to remain within established safety margins.
These findings support the potential of YH35995 as a convenient once-monthly oral therapy for Gaucher disease type 3, pending clinical confirmation.
Source: ACoP 2026, Poster M-096, “Population PK/PD modeling of YH35995, a novel glucosylceramide synthase inhibitor for the treatment of Gaucher disease type 3, to inform multiple doses in Phase 1 study” (10/12/2026)
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