Abbisko Therapeutics discovered novel CSF-1R inhibitor Pimicotinib（ABSK021) for the treatment of tenosynovial giant cell tumour (TGCT) patients not responding to surgery.
Pimicotinib is the first new generation CSF-1R drug candidate in the world. It is a novel, oral, highly selective and highly potent small molecule inhibitor of CSF-1R. Several studies have shown that blocking the CSF-1R signalling pathway can effectively modulate and alter macrophage functions, and has the potential to treat many macrophage-dependant human diseases. Pimicotinib is currently being studied for the treatment of tenosynovial giant cell tumour (TGCT) and cGVHD , and is being evaluated for, other indications.
Pimicotinib showed significant antitumor efficacy in a phase Ib trial in patients with TGCT, resulting a preliminary ORR of 68.0 per cent and favourable safety profile.
TGCT, a pigmented villonodular synovitis, is a locally aggressive neoplasm affecting synovial joints, mucous sacs, and tendon membranes, resulting in swelling, pain, stiffness, and decreased activity of the affected joints which seriously affect the patient's quality of life.
US Food and Drug Administration (FDA) grants breakthrough device designation for its CSF-1R inhibitor Pimicotinib (ABSK021).