Discovering New Precision Medicines

Our Science

Building on an exceptional track record of innovation, this team brings a wealth of cross-functional expertise to successful rational drug design. We focus on pioneering best-in-class therapeutics to improve upon existing drugs with clear liabilities, as well as to create new breakthroughs for diseases where others have been unable to find solutions.

Established and emerging science will guide our plans, with an emphasis on rare disease patient populations with well-characterized biology, allowing us to move rapidly and create meaningful impact for patients.

Our Bezuclastinib Approach
Our FGFR Approach
Fibroblast growth factor receptors (FGFRs) are a family of transmembrane receptors consisting of isoforms FGFR1-FGFR4
Binding of a ligand to the inactive FGFR monomer leads to receptor dimerization, phosphorylation, and downstream signaling
Receptor mutations, amplifications, and fusions can result in activation of FGFRs and are recognized as well-established oncogenic drivers in multiple indications
Inhibition of FGFR3 mutations in urothelial cancer and FGFR2 fusions in cholangiocarcinoma has led to improved clinical outcomes in defined patient populations

Inhibition Ligand Independent Signaling in FGFR 2/3 Altered Tumors is a Clinically Proven Approach

Posters and Publications
December 2022
Preliminary Safety and Efficacy from Apex, a Phase 2 Study of Bezuclastinib (CGT9486), a Novel, Highly Selective, Potent KIT D816V Tyrosine Kinase Inhibitor, in Adults with Advanced Systemic Mastocytosis (AdvSM)
December 2022
Summit: A 3-Part, Phase 2 Study of Bezuclastinib (CGT9486), an Oral, Selective, and Potent KIT D816V Inhibitor, in Adult Patients with NonAdvanced Systemic Mastocytosis (NonAdvSM)
November 2022
A Phase 3 Randomized, Open-label, Multicenter Clinical Study of Bezuclastinib (CGT9486) and Sunitinib Versus Sunitinib in Patients with Gastrointestinal Stromal Tumors
October 2022
In Vivo Preclinical Characterization of a Novel FGFR2 Selective Inhibitor with Potency Against FGFR Activating Mutations
October 2022
Preclinical Characterization of a Novel EGFR Sparing ErbB2 Inhibitor with Activity Against Oncogenic ErbB2 Mutations
June 2022
A Phase 2 Study of Bezuclastinib (CGT9486), A Novel, Highly Selective, Potent KIT D816V Inhibitor, in Adults with Advanced Systemic Mastocytosis (Apex): Methods, Baseline Data, and Early Insights
April 2022
Pre-clinical characterization of a novel series of FGFR2 selective inhibitors with potency against clinically relevant mutations
April 2022
Bezuclastinib is a differentiated KIT inhibitor that exhibits unique selectivity to KIT A-loop mutations, minimal brain penetration, and favorable pharmacokinetic properties in preclinical models.
October 2021
Preclinical data identifies bezuclastinib as a differentiated KIT inhibitor with unique selectivity to KIT D816V and minimal evidence of brain penetration
November 2020
CTOS Oral Presentation: The Potent and Selective KIT Inhibitor PLX9486 Dosed in Combination with Sunitinib Demonstrates Promising Progression Free Survival (PFS) in Patients with Advanced Gastrointestinal Stromal Tumor (GIST): Final Results of a Phase 1/2 Study
June 2018
ASCO: A phase I pharmacokinetic and pharmacodynamic study of PLX9486 alone and in combination with the KIT inhibitors pexidartinib (pexi) or sunitinib in patients with advanced solid tumors and gastrointestinal stromal tumor (GIST)
November 2017
CTOS: A Phase I Pharmacokinetic (PK) and Pharmacodynamic (PD) Study of PLX9486, A Novel KIT Inhibitor with Potent Activity Against Exon 17/18 Activation Loop Mutations in Patients with Gastrointestinal Stromal Tumor (GIST)
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