Cogent Biosciences Reports Deepening Clinical Benefit for Bezuclastinib in Systemic Mastocytosis

Cogent Biosciences announced additional clinical results from the pivotal SUMMIT trial with bezuclastinib in patients with NonAdvanced Systemic Mastocytosis (NonAdvSM) at the American Academy of Allergy Asthma & Immunology (AAAAI) Annual Meeting. As previously reported, bezuclastinib demonstrated clinically meaningful and highly statistically significant improvements across the primary and all key secondary endpoints.

Treatment with bezuclastinib resulted in rapid, durable, statistically significant symptomatic improvements which continued to deepen out to 48 weeks. Patients on bezuclastinib reported a -32.0 point mean change in TSS, representing a 56% relative improvement in TSS from baseline. 86% of patients achieved a clinically meaningful threshold of 30% improvement in symptoms. 99% of patients had at least 50% reduction in serum tryptase, with 83.3% of patients achieving normalization.

Patients treated with bezuclastinib reported clear improvements across all relevant organ systems as evidenced by relative improvement at 48 weeks. Dermatologic symptoms showed a mean change from baseline of -13.94, representing a 65.0% relative improvement. Neurocognitive symptoms improved by -12.77 points, a 53.6% relative improvement. Gastrointestinal symptoms decreased by -6.63 points, a 62.5% relative improvement. Pain decreased by -5.43 points, a 45.8% relative improvement, and fatigue decreased by -3.02 points, a 41.3% relative improvement.

Bezuclastinib demonstrates strong evidence of disease modification in NonAdvSM patients based on significant correlation shown between serum tryptase reduction and symptomatic improvement as measured by TSS, all symptom domains, and 10 of 11 individual symptoms. Clinically meaningful improvements in bone mineral density (BMD) at week 24 were observed for patients treated with bezuclastinib, regardless of baseline bone health severity. 50% of patients reported a dose reduction and/or discontinuation of best supportive care medicines during the treatment period at week 48.

Bezuclastinib demonstrated consistent biomarker and symptomatic improvement in patients with smoldering SM, a subgroup of NonAdvSM with high unmet need. As previously reported, bezuclastinib demonstrated a favorable safety and tolerability profile, supporting its potential for chronic use in patients with NonAdvSM.

The company is pursuing multiple regulatory avenues for bezuclastinib. A New Drug Application (NDA) for the treatment of non-advanced systemic mastocytosis (NonAdvSM) was submitted in December. An application for gastrointestinal stromal tumors (GIST) is progressing under the FDA's expedited Real-Time Oncology Review program. A third NDA submission for advanced systemic mastocytosis (AdvSM) is scheduled for the first half of the year. The completion of the PEAK submission process is anticipated in April.

Bezuclastinib received Breakthrough Therapy Designation from the FDA for NonAdvSM and GIST in January, and orphan drug designation from both the FDA and EMA for Mastocytosis and GIST. The company expects to launch bezuclastinib commercially in the United States in the second half of 2026, pending regulatory approval, for the treatment of NonAdvSM and second-line GIST patients.

In the PEAK study, the bezuclastinib combination therapy demonstrated a median progression-free survival (mPFS) of 16.5 months, significantly outpacing the 9.2-month mPFS observed with standard treatment. The SUMMIT trial for NonAdvSM and the APEX trial for AdvSM achieved all primary and key secondary endpoints.

Cogent reported a net loss of $328.9 million for the full year 2025, an increase from $255.9 million in 2024. The fourth-quarter loss alone was $102.5 million. This was primarily driven by escalating research and development expenses, which reached $75.6 million in the final quarter and $269.8 million for the full year, an increase of $37.1 million from 2024, primarily due to costs related to clinical trials and personnel costs. General and administrative costs rose year-over-year from $43.3 million to $63.6 million for the full year.

A robust cash position of $900.8 million as of year-end 2025 provides the financial backbone for Cogent's operations. This capital is projected to fund the company's activities into 2028. The company raised approximately $878.2 million through various financing activities, including public offerings of common stock, issuance of convertible senior notes, and sales under an at-the-market offering program. The company entered into a loan and security agreement for a $400 million credit facility, of which $50 million was initially drawn and later repaid.

On February 25, 2026, the Compensation Committee of Cogent's Board of Directors approved the grants of inducement equity awards to three new employees under the company's 2020 Inducement Plan. The employees received, in the aggregate, nonqualified options to purchase 24,000 shares of Cogent common stock and 21,000 restricted stock units (RSUs). Each option has a 10-year term, an exercise price equal to the closing price of Cogent's common stock on the grant date, and a 4-year vesting schedule with 25% vesting on the 1-year anniversary of the grant date and the remainder vesting in equal monthly installments over the subsequent 36 months. The RSUs vest annually in equal installments over 4 years from the grant date.