Bezuclastimib appears to significantly alleviate symptoms in patients with nonadvanced systemic mastocytosis (SM), Cogent Biosciences Inc. announced recently.
Bezuclastinib’s safety and efficacy are currently being tested in SUMMIT, the registration-directed, randomized, double-blind, placebo-controlled, global, multicenter clinical trial. The study enrolled more than 170 patients with nonadvanced SMNonadvanced SM In these subtypes of SM, mast cells accumulate in the body but do not usually cause severe organ damage. Nonadvanced SM includes the subtypes indolent SM and smoldering SM..
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“The SUMMIT data reported today are encouraging to thousands of NonAdvSM patients around the world who are waiting for a novel treatment that can rapidly and meaningfully improve a wide variety of symptoms that impact their daily lives,” said Andrew Robbins, Cogent’s president and CEO, in a statement.
What is SM?
Systemic mastocytosisMastocytosis Rare disease caused by the buildup of mast cells. Cutaneous mastocytosis primarily affects the skin and is more common in children, while systemic mastocytosis affects internal organs and is more common in adults. (SM) is a rare hematological disease characterized by mast cells that are overactive and accumulate in different parts of the body such as the bone marrow, liver, spleen, gastrointestinal tract and lymph nodes.
Preliminary results after 24 weeks showed that patients receiving 100mg of bezuclastinib had better results in the Mastocytosis Symptom Severity Daily Diary (MS2D2) and the Mastocytosis Quality-of-Life (MC-QoL) scales than patients receiving placebo, the company said. Patients taking bezuclastinib reported a 56% improvement in the total symptoms score (TSS), and 31% of patients discontinued best supportive care medications.
Read more about SM therapies
Laboratory workups revealed that patients in the bezuclastinib group had a significant decrease in serum tryptaseTryptase A protein enzyme that is primarily produced by mast cells and stored in small pockets within the cells, known as granules. High levels of tryptase are a key indicator of SM. levels, with close to 90% of patients experiencing a 50% decrease.
The majority of adverse effects associated with bezuclastinib were mild or moderate, with hair discoloration and transitory liver enzyme elevations being the most frequent, the company said.
Bezuclastinib is an experimental drug that acts as a selective tyrosine kinase inhibitorTyrosine kinase inhibitor Targeted therapies designed to block the KIT D816V mutation that drives most cases of SM. Includes avapritinib, midostaurin and imatinib.. The drug is currently being tested in clinical trials for its potential therapeutic uses in SM and gastrointestinal stromal tumors (GIST).
By selectively inhibiting the KIT D816V mutation, bezuclastinib can prevent mast cellMast cell A type of white blood cell produced in the bone marrow. They help defend against infections and play a key role in allergic reactions. In SM, mast cells become overactive and build up throughout the body. proliferation and survival, decreasing the symptom burden in patients with SM, the company said.
