Shanghai-based biotech Abbisko Therapeutics Co., Ltd (HKG: 2256) has announced that it has received approval from the European Medicines Agency (EMA) to conduct a Phase III clinical study for its Category 1 innovative drug ABSK021 (pimicotinib), which is being assessed as a treatment for tendon sheath giant cell tumor (TGCT).
Design and Structure of the Phase III Study
The Phase III study is a randomized, double-blind, placebo-controlled, multi-center trial designed in two parts to evaluate the efficacy and safety of ABSK021 in TGCT. In Part 1, subjects will be randomly assigned to receive 50mg QD of ABSK021 treatment or placebo under double-blind conditions for a 28-day treatment cycle. Participants who complete Part 1 will be eligible to enter Part 2, an open-label treatment phase where all participants receive 50mg QD ABSK021 treatment for 24 weeks or choose to withdraw from the study. The primary endpoint of the study is the 25-week objective response rate (ORR), and approximately 100 subjects are aimed to be enrolled. Enrollment has commenced in the US and China, with the data to support New Drug Application (NDA) filings in Chinese, US, and European markets.
Pimicotinib’s Profile and Potential Impact
Pimicotinib is an orally administrated, highly potent, and selective small-molecule inhibitor of CSF-1R. It has received breakthrough therapy designation (BTD) and priority review awards in China, the US, and Europe. Tendon sheath giant cell tumor (TGCT) is a locally invasive tumor characterized by swelling, pain, stiffness, and limited mobility of the affected joints, significantly impacting patients’ quality of life. Currently, no targeted therapy exists in China for TGCT, although Daiichi-Sankyo’s pexidartinib has been approved for use outside of the country for this indication.-Fineline Info & Tech
