Cogent Biosciences (NASDAQ:COGT) Chief Executive Officer Andrew Robbins said the company is preparing for potential commercial launches of its lead drug candidate, bezuclastinib, later this year, citing regulatory progress in gastrointestinal stromal tumors, or GIST, and systemic mastocytosis.
Speaking at the Jefferies Global Healthcare Conference in New York with Jefferies Senior Biotech Analyst Faisal Khurshid, Robbins described Cogent as focused on developing small-molecule treatments for rare diseases caused by mutations, both in oncology and non-oncology settings. He said bezuclastinib is under FDA review for GIST and non-advanced systemic mastocytosis, with the company hoping the drug will soon be under review for advanced systemic mastocytosis as well.
GIST Data Presented at ASCO
Robbins highlighted results from Cogent’s Phase 3 PEAK study, which evaluated bezuclastinib in combination with sunitinib in second-line GIST patients. He said the combination produced a median progression-free survival of 16.5 months, compared with 9.2 months for sunitinib alone. The result was statistically significant, with a hazard ratio of 0.5, representing a 50% reduction in the risk of progression or death.
Robbins said the study was notable because second-line GIST has not seen a new approved drug in 20 years, and because the trial was the first in GIST to show statistical significance against an active comparator.
At the American Society of Clinical Oncology meeting, Cogent also presented subgroup data showing what Robbins described as consistent benefit across mutation-defined patient groups. He said the company also presented a PFS-2 analysis, a measure that tracks outcomes through the next line of therapy, which showed a hazard ratio of 0.57 in favor of starting patients on the bezuclastinib-sunitinib combination.
Robbins said overall survival data remain “very immature,” which he described as favorable for patients. He also said the mean expected duration of treatment in the study reached 21.4 months, compared with what he characterized as a mean expected duration for sunitinib of less than 10 months.
Regulatory Timeline and FDA Review
Cogent has a Nov. 30 PDUFA action date for bezuclastinib in GIST. Robbins said the drug has Breakthrough Therapy Designation and is being reviewed under the FDA’s Real-Time Oncology Review program, which allowed the company to begin providing portions of its application before the full filing was completed at the end of March.
Robbins said those designations helped support priority review, shortening the review timeline compared with a standard review that would have had an end-of-March action date. Asked whether approval could come ahead of the PDUFA date, Robbins said it is difficult to rely on historical FDA timing, but added that Cogent would be ready if the agency moved sooner.
Robbins said Cogent’s interactions with FDA review teams have remained consistent despite broader leadership changes at the agency. He said the GIST application is being reviewed by the Division of Oncology Products, while the mastocytosis applications are reviewed by the Division of Non-Malignant Hematology.
Labeling and Safety Considerations
Asked about potential labeling related to liver enzyme elevations, Robbins said bezuclastinib has shown asymptomatic and reversible AST and ALT elevations across studies. He said the company has rarely seen bilirubin elevations and has not observed clinical liver-related complications from those events.
Robbins said any potential label language could include monitoring of transaminases, particularly early in treatment, noting that higher-grade transaminase events typically occurred within the first five cycles. He said such monitoring would be consistent with current physician workflows in mastocytosis, where bloodwork is already used to track disease markers and drug effects.
Systemic Mastocytosis Positioning
Robbins also outlined Cogent’s view of bezuclastinib in non-advanced systemic mastocytosis compared with avapritinib, marketed as AYVAKIT. He said both drugs are potent against the D816V KIT mutation, but argued that bezuclastinib’s lack of central nervous system penetration and greater KIT selectivity could support higher intended dosing and differentiated tolerability.
Robbins said Cogent has seen rapid and sustained symptom improvement in its SUMMIT trial, along with objective disease response measures. He said leading pathologists have observed complete elimination of mast cell aggregates in the bone marrow in a high percentage of patients, which he said has generated excitement in the systemic mastocytosis community.
Discussing the market opportunity, Robbins said Sanofi’s avapritinib sales have continued to rise following its acquisition of Blueprint Medicines, though disclosure has become “more opaque.” He said Sanofi’s first-quarter figures implied avapritinib could be tracking toward roughly $1 billion in annual sales as a rare disease oral therapy.
Commercial Plans and Pipeline
Robbins said Cogent plans to use a single sales force for bezuclastinib across indications, rather than separate teams for oncology and mastocytosis. He said the physicians treating the diseases are largely distinct, but often practice in overlapping settings, supporting a focused commercial effort of “several dozen” sales representatives.
He added that Cogent has already identified the individuals who would make up the sales force.
Robbins also discussed earlier-stage pipeline programs, saying the company’s pan-KRAS inhibitor and JAK2 V617F inhibitor are the programs investors most often ask about. He said both are expected to have INDs filed this year. He also pointed to Cogent’s CNS-penetrant ErbB2 and alpha-selective PI3K programs as clinical-stage assets with potential.
About Cogent Biosciences (NASDAQ:COGT)
Cogent Biosciences is a clinical-stage biopharmaceutical company focused on the discovery and development of small-molecule therapies that modulate the tumor microenvironment. The company’s research centers on targeting colony-stimulating factor 1 receptor (CSF1R), a key regulator of tumor-associated macrophages that can promote tumor growth and immune evasion. By selectively inhibiting CSF1R, Cogent Biosciences aims to restore immune surveillance and enhance the efficacy of existing cancer treatments.
The company’s lead asset is an orally bioavailable CSF1R inhibitor that has advanced into early-stage clinical trials for various solid tumors.
