Shanghai-based Abbisko Therapeutics Co., Ltd (HKG: 2256) announced that its Category 1 drug ABSK021 has received breakthrough therapy designation (BTD) from the Center for Drug Evaluation (CDE) for treating inoperable giant cell tumor of tendon sheath. The drug, a CSF-1R inhibitor, is being developed for both tumor and non-tumor indications.
Drug Profile
ABSK021 is an orally administered, highly potent, and selective small-molecule inhibitor of CSF-1R. Preclinical studies demonstrated its efficacy in inhibiting CSF-1R signaling, which plays a key role in macrophage recruitment and tumor immunosuppression. The drug is currently in Phase Ib trials in the US and China for giant cell tumor of tendon sheath, triple-negative breast cancer, lung cancer, and pancreatic cancer.
Breakthrough Designation
The BTD status reflects the drug’s potential to address unmet medical needs. CSF-1R inhibition is a novel mechanism targeting tumor-associated macrophages (TAMs), aiming to reverse immunosuppression in the tumor microenvironment.
Strategic Partnership
In July 2021, Abbisko licensed ABSK021 to Sperogenix Therapeutics for development in rare nervous system diseases, with amyotrophic lateral sclerosis (ALS) as the initial indication.-Fineline Info & Tech
