October 29, 2020, Shanghai—Abbisko Therapeutics Co., Ltd. (“Abbisko” hereafter) today announces that it has obtained the regulatory approval of ABSK021, a CSF-1R small molecule antagonist, by National Medical Products Administration (NMPA) of Chin上a, and will initiate a phase I clinical study in subjects with advanced solid tumors. This is the sixth IND approval obtained by Abbisko around the world.

Colony-stimulating factor 1 receptor (CSF-1R), is a type of transmembrane receptor tyrosine kinase thatplays critical roles in the regulation of macrophage proliferation and differentiation. Emerging evidences have revealed that CSF-1R signaling pathway is activated in many type of cancers and tumor-associated macrophages (TAM), which can lead to changes in the microenvironment within the tumor and thus produce immunosuppressive effects. Inhibition of CSF-1R may hold a potential synergistic effect in combination with a variety of anti-cancer therapies such as tumor immune checkpoint inhibitors. In Addition, CSF-1R antagonist may also be effective for treating diseases associated with dysfunctional macrophages such as tenosynovial giant cell tumor (TGCT), amyotrophic lateral sclerosis (ALS), Alzheimer’s disease. In past years, clinical studies on Pexidartinib, a non-selective CSF-1R antagonist, has proved that disruption of CSF-1R signaling pathway is essential and effective for TGCT treatment. In 2019, it obtained the market authorization approval by US FDA.

ABSK021 is independently discovered and developed by Abbisko who solely holds its global intellectual properties. This innovative drug candidate is an orally available small molecule with highly potent, selective and other advanced physicochemical properties. In August 2019, ABSK021 was firstly approved by US FDA and it is currently under dose-escalation phase I study in multiple US sites. Preliminary clinical data to date have revealed that patients are well tolerated by ascending doses of ABSK021 and its pharmacokinetic properties and effective inhibition of targets with potential ‘Best-in-class’ features are validated in humans.