Encaleret & ADH1 Overview - Encaleret is an investigational oral calcilytic drug being developed as a potential treatment for Autosomal Dominant Hypocalcemia Type 1 (ADH1)[35, 38] - ADH1 is caused by activating variants in the CASR gene, leading to dysregulation of calcium homeostasis, decreased blood calcium, and increased urinary calcium[16, 17] - Diagnosis of ADH1 requires genetic testing, but is often delayed with a median age of 25 years[23, 24] - Conventional therapy for ADH1, including calcium and activated vitamin D, does not correct the underlying pathophysiology and may worsen long-term complications[17, 27] Clinical Development & Results - Phase 2 study of encaleret in 13 ADH1 patients showed rapid and sustained normalization of serum calcium, urine calcium, and PTH levels over 42 months[49, 56, 57] - In the Phase 2 study, 69% of participants responded to encaleret, achieving both blood and urine calcium in the target range at Week 24[59] - Encaleret was generally well-tolerated over 42 months of outpatient administration in the Phase 2 study, with most Treatment-Emergent Adverse Events (TEAEs) being mild[54] - The CALIBRATE Phase 3 study of encaleret in ADH1 is ongoing, with topline results expected in Fall 2025[62, 63] Market Opportunity - The estimated prevalent population of ADH1 in the US is approximately 12,000 individuals, with 73% being symptomatic[72] - ADH1 is emerging as the leading isolated cause of nonsurgical hypoparathyroidism, accounting for approximately 20% of all cases[76, 78] - The company projects peak year sales of encaleret to be over $1 billion, drawing a comparison to the XLH market[73]

Anticipated Upcoming Milestones Announced positive top-line results from the registration-directed Part 2 of the SUMMIT clinical trial in NonAdvanced Systemic Mastocytosis (NonAdvSM) patients. The SUMMIT trial, which was designed to assess the clinical benefit of bezuclastinib versus placebo, achieved its primary endpoint with a highly statistically significant difference in the mean change in Total Symptom Score (TSS) at 24 weeks (p=0.0002). TSS was assessed by the Mastocytosis Symptom Severity Daily Diary ...

Three Registration-Directed Top-line Data Readouts Remain on Track in 2025: SUMMIT in NonAdvanced SM expected in July, APEX in Advanced SM expected in second half of the-year and PEAK in GIST expected by end of year Ended 1Q 2025 with $245.7 million in cash, sufficient to fund operations into late 2026 WALTHAM, Mass. and BOULDER, Colo., May 06, 2025 (GLOBE NEWSWIRE) -- Cogent Biosciences, Inc. (Nasdaq: COGT), a biotechnology company focused on developing precision therapies for genetically defined diseases, ...