Cogent Biosciences (COGT) Bezuclastinib showed "rapid and deep" clinical benefit and met primary and secondary endpoints in patients with advanced systemic mastocytosis during a clinical trial, the company said Friday.
The illness is a rare blood and bone marrow disease that can lead to severe organ damage.
Among the 81 patients in the study, 65% and 81% showed objective response, depending on the various measurement criteria, the company said.
The study also met a secondary endpoint of response per pure pathological response with an 81% objective response rate, the company said.
The drug also had a "powerful effect" on the overproduced mast cells which characterize the disease, with around 90% of patients achieving a more than 50% reduction, the company said.
The drug also showed a favorable safety and tolerability profile, the company said.
The company plans to submit the data as part of a new drug application to the US Food and Drug Administration this month, it said.
Separately, Cogent's treatment showed promise in preclinical trials as a treatment for the JAK2 V617F mutation, a rare blood condition, the company said.
"This highly selective, potent inhibitor of JAK2 V617F has the potential to address the underlying mutational driver of disease while mitigating off-target hematological effects," said Andrew Robbins, Cogent's president and chief executive officer.
The company is on track to summit an investigational new drug application for the treatment this year.
The mutation is the most common in BCR-ABL-negative myeloproliferative neoplasms, stem cell disorders which occur in patients with polycythemia vera, primary myelofibrosis, or essential thrombocythemia, all rare blood diseases.
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