MBX Biosciences, Inc. experienced a significant surge in its stock value following the release of positive results from its Phase 2 clinical trial of once-weekly canvuparatide, a treatment for chronic hypoparathyroidism. The trial, named Avail, successfully met its primary endpoint at Week 12 with statistical significance, marking an important milestone for the company’s ongoing research.
The study involved 64 adult patients, all of whom completed the 12-week randomized trial phase. Impressively, 94% of participants chose to continue onto the open-label extension (OLE) of the study. In this phase, 79% of patients receiving canvuparatide achieved responder status at six months, indicating maintained normal serum calcium levels and independence from conventional therapies.
The canvuparatide treatment showed a marked improvement over placebo, with 63% of its patients meeting the primary composite endpoint compared to just 31% in the placebo group. This outcome underscores the potential efficacy of canvuparatide in managing hypoparathyroidism.
In terms of secondary and exploratory endpoints, the results were equally promising. Pharmacokinetic data supported the feasibility of a once-weekly dosing schedule. Additionally, markers of bone turnover and formation were elevated in the treatment group, consistent with enhanced bone remodeling. Among patients with initially high urine calcium levels, those treated with canvuparatide saw a 48% reduction by Week 12, compared to a 33% reduction in the placebo group.
The treatment was generally well-tolerated, with no discontinuations due to adverse effects, and most side effects were mild or moderate. There were no reports of mortality among participants.
Given these promising results, MBX Biosciences is preparing to move forward with a Phase 3 clinical trial for canvuparatide, set for 2026. The positive reaction from the market was immediate, with the company’s stock price soaring by 98.60% to $19.86 during premarket trading. This significant increase reflects investor confidence in the potential of canvuparatide to address unmet needs in the treatment of chronic hypoparathyroidism.