Shanghai-based biotech Abbisko Therapeutics Co., Ltd (HKG: 2256) has announced the dosing of the first patient in a Phase II clinical study for its Category 1 drug ABSK021 (pimicotinib) in tendon sheath giant cell tumor (TGCT) in the United States.
Phase III Study Design and Objectives
The upcoming randomized, double-blind, placebo-controlled Phase III study is expected to enroll 100 subjects from China, the US, and Europe. The study aims to assess the efficacy and safety of ABSK021 in TGCT patients. Enrolled patients will receive 50mg QD of ABSK021 or matched placebo treatment under double-blind conditions, with a 28-day cycle. The primary endpoint is the objective response rate (ORR) at 25 weeks, evaluated by the Blind Independent Review Committee (BIRC). The study had previously completed the administration of its first patient in China in April of this year.
Pimicotinib’s Mechanism of Action and Previous Study Results
ABSK021 is an orally administrated, highly potent, and selective small-molecule inhibitor of CSF-1R. Research indicates that blocking the CSF1/CSF-1R signaling pathway can regulate and alter macrophage function, playing a role in various macrophage-related diseases. In the Phase Ib study, the drug demonstrated a 77.4% objective response rate (ORR) in inoperable tenosynovial giant cell tumor (TGCT), with a good safety and PK/PD profile.
Regulatory Designations and Global Recognition
Breakthrough therapy designations (BTDs) for ABSK021 have been obtained from the Center for Drug Evaluation (CDE), the US Food and Drug Administration (FDA), and European Medicine Agency (EMA) in July 2022, January 2023, and June 2023, respectively, highlighting the global recognition of its potential impact on TGCT treatment.-Fineline Info & Tech
