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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. 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

Systemic Mastocytosis occurs when mast cells inappropriately accumulate in various internal organs in the body. About 90% of people with Systemic Mastocytosis have Nonadvanced Systemic Mastocytosis (NonAdvSM), a life-long illness with chronic symptoms including headaches, urticaria pigmentosa, skin lesions, skin redness and warmth (flushing), abdominal pain, bloating, vomiting, diarrhea, and gastroesophageal reflux (GERD), that significantly impact the patient’s quality of life. Many patients are also at high risk for severe, life-threatening anaphylactic reactions to various triggers such as insect bites. Advanced Systemic Mastocytosis (AdvSM) is a rare, very aggressive form of Systemic Mastocytosis. Patients with AdvSM may suffer from a multitude of debilitating symptoms such as anemia, thrombocytopenia, ascites, bone fractures, gastrointestinal abnormalities, and enlargement of the liver, spleen, and lymph nodes, which ultimately lead to organ failure and early death. The median life expectancy for AdvSM is less than 3.5 years.

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 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

Gastrointestinal Stromal Tumors (GIST) are categorized by uncontrolled cell growth in the tissues of the gastrointestinal (GI) tract. At diagnosis, about 80% of GIST patients’ tumors are the result of primary KIT mutations. Approved therapies currently inhibit a subset of these mutations, but most patients develop resistance due to additional secondary KIT mutations, including mutations in Exon 17. Bezuclastinib is designed to be a potent and selective inhibitor of KIT Exon 17 mutations, and by combining bezuclastinib with drugs that inhibit additional KIT mutations, Cogent is addressing a clear unmet medical need for patients with GIST.

The safety profile of bezuclastinib has been clinically evaluated in two completed trials both as a single agent and as part of a combination therapy. In a Phase 1/2 trial testing the combination of bezuclastinib with sunitinib in 18 patients with advanced GIST, median progression free survival (PFS) reached 11 months. Most of these patients were heavily treated previously, having received sunitinib or other kinase inhibitors prior to treatment with bezuclastinib.

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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