MBX Biosciences announced that its once-weekly investigational drug, canvuparatide, achieved the prespecified primary composite endpoint in its Phase 2 Avail trial in patients with chronic hypoparathyroidism, according to the company.
In the 12-week randomized period, 63 % of canvuparatide-treated patients met the primary endpoint — maintaining normal serum calcium while remaining independent of conventional therapy — with no reliance on rescue therapy. In the open-label extension, 79 % of patients were responders at 6 months. All 64 enrolled patients completed the 12-week trial, and 94 % elected to continue into the extension phase.
The company reported favorable signals in secondary and exploratory measures, including increases in bone turnover markers and reductions in urinary calcium in patients with elevated levels at baseline. Pharmacokinetic data were consistent with a once-weekly dosing regimen. Safety findings were generally benign: no treatment-related serious adverse events or discontinuations were reported, and most adverse events were mild to moderate; injection site reactions occurred in 19 % of treated patients versus 13 % in placebo.
With these results, MBX is preparing to launch a Phase 3 trial in 2026. The company claims the data provide a foundation for canvuparatide to become a potential best-in-class therapy for hypoparathyroidism and to simplify treatment burdens for patients.
