Treating Genetically Defined Diseases

Cogent’s lead drug candidate, bezuclastinib, is designed to target exon 17 mutations found within the KIT receptor tyrosine kinase, including KIT D816V. When KIT D816V remains in a perpetual ‘on’ state causing mast cells, a type of white blood cell, to accumulate in various internal organs including the bone marrow. The result is an orphan disease called Systemic Mastocytosis.

Exon 17 mutations have also been found in advanced Gastrointestinal Stromal Tumors (GIST), which have a strong dependence on oncogenic KIT signaling. Bezuclastinib is a highly selective and potent KIT inhibitor with the potential to provide a powerful new treatment option for patients with both of these diseases.

About Systemic Mastocytosis

Cogent is currently enrolling Apex, a Phase 2 clinical trial of bezuclastinib in patients with AdvSM. Apex is an open-label, global, multicenter trial that will evaluate the safety, efficacy, pharmacokinetic, and pharmacodynamic profiles of bezuclastinib.

Cogent is also currently enrolling Summit, a global, randomized, double-blind, placebo-controlled Phase 2 clinical trial of bezuclastinib in patients with NonAdvSM. Learn more about Cogent’s clinical trials at

About GIST
Cogent is currently enrolling Peak, a randomized Phase 3 clinical trial of bezuclastinib in combination with sunitinib compared to sunitinib alone in patients with locally advanced, unresectable or metastatic GIST who have received prior treatment with imatinib.
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