MBX Biosciences Announces Once-Weekly Canvuparatide Achieved Primary Endpoint in Phase 2 Trial with 63% Responder Rate at 12 Weeks; 79% Responder Rate at 6 Months in Open-Label Extension

Core Insights - MBX Biosciences announced that once-weekly canvuparatide achieved statistical significance in the primary endpoint at Week 12 of the Phase 2 Avail trial for chronic hypoparathyroidism, with 63% of treated patients meeting the endpoint compared to 31% in the placebo group [2][3][7] - The company is preparing to initiate a Phase 3 clinical trial in 2026 based on the positive results from the Avail trial [4][5] - Canvuparatide was well tolerated, with no serious adverse events reported during the 12-week trial [1][13] Phase 2 Avail Trial Results - In the 12-week trial, 63% of canvuparatide-treated patients achieved the primary composite endpoint, while 79% maintained responder status at 6 months in the open-label extension [3][7] - The primary endpoint involved maintaining serum calcium levels in the normal range (8.2–10.6 mg/dL) and independence from conventional therapy [3][7] - All 64 patients completed the 12-week study, and 94% chose to enter the open-label extension [2][11] Safety and Tolerability - Canvuparatide was generally well tolerated, with most treatment-emergent adverse events classified as mild or moderate [13] - Injection site reactions occurred in 19% of the treatment group compared to 13% in the placebo group, with no treatment-related serious adverse events reported [13] Future Development - The company plans to present additional data from the Phase 2 trial and open-label extension at an upcoming major medical meeting [9] - The results support the potential of canvuparatide as a best-in-class treatment for hypoparathyroidism, addressing significant unmet medical needs [5][15] About Hypoparathyroidism - Hypoparathyroidism is a rare endocrine disease affecting over 250,000 individuals in the U.S. and Europe, characterized by low calcium levels due to parathyroid hormone deficiency [12] - Current treatments primarily involve high doses of oral calcium and active vitamin D supplements, which do not address the underlying cause of the disease [12]