Shanghai-based biotech Abbisko Therapeutics Co., Ltd (HKG: 2256) has announced receiving approval from the National Medical Products Administration (NMPA) to conduct a Phase II clinical study for its Category 1 drug ABSK021 (pimicotinib) in chronic graft versus host disease (cGVHD). This marks a significant step forward in the development of this innovative therapy.
ABSK021: Mechanism and Development
ABSK021 is an orally administered, highly potent, and selective small-molecule inhibitor of CSF-1R, discovered and developed by Abbisko. The drug is under development to treat both tumor and non-tumor indications. A Phase Ib study for pimicotinib in advanced tenosynovial giant cell tumor (TGCT) demonstrated a 68.0% objective response rate (ORR) along with a favorable safety and pharmacokinetic/pharmacodynamic (PK/PD) profile. Based on these results, the drug was awarded breakthrough therapy designation (BTD) for inoperable TGCT in July of last year and subsequently obtained approval for a pivotal Phase III clinical study in October. The drug is currently undergoing a Phase Ib multi-cohort expansion study concurrently in China and the US, following the completion of a Phase Ia dosage escalation study in the US.
Partnership and Market Landscape
In July 2021, Sperogenix Therapeutics entered into a licensing agreement with Abbisko to co-develop the drug for rare nervous system diseases, with amyotrophic lateral sclerosis (ALS) as the first indication. Currently, there is no highly selective CSF-1R inhibitor commercially available in China, positioning ABSK021 as a potential first-in-class therapy.
Future Outlook
With NMPA approval for the Phase II study in cGVHD, Abbisko is poised to further explore the therapeutic potential of ABSK021. This development underscores Abbisko’s commitment to addressing significant unmet needs in both oncology and rare diseases.-Fineline Info & Tech
