MBX Biosciences, Inc. (NASDAQ: MBX) has announced positive results from the phase 2 AVAIL™ trial of once-weekly canvuparatide, a potential treatment for chronic hypoparathyroidism (HP). The trial demonstrated a 63% responder rate at 12 weeks, with 79% of patients achieving responder status at 6 months in the open-label extension (OLE) phase. Notably, all patients completed the 12-week trial, with 94% choosing to enroll in the OLE.
In the 12-week randomized portion of the trial, 63% of canvuparatide-treated patients met the primary composite endpoint, compared to 31% in the placebo group. Furthermore, in the OLE, 79% of patients achieved responder status at 6 months, indicating promising long-term efficacy.
Canvuparatide was generally well-tolerated, with no treatment-related serious adverse events or discontinuations during the 12-week trial. The company is now preparing to initiate a phase 3 trial in 2026.
Additional findings from the trial include consistent pharmacokinetic (PK) results supporting a once-weekly dosing schedule, as well as positive effects on bone and kidney biomarkers. Specifically, bone turnover and formation markers increased over 12 weeks compared to placebo, and in patients with elevated urine calcium at screening that normalized at week 12, mean urine calcium was reduced by 48% in patients treated with once-weekly canvuparatide compared with 33% on placebo.
Patty Keating, Executive Director of the Hypoparathyroidism Association, expressed excitement about the trial results, noting that a once-weekly treatment option could significantly improve the quality of life for HP patients.
The company intends to present additional data from the phase 2 trial and OLE at an upcoming major medical meeting. MBX Biosciences will also host a conference call and webcast to discuss the results from the AVAIL™ phase 2 trial.
Canvuparatide, a parathyroid hormone peptide prodrug, is designed to provide convenient, once-weekly administration and continuous, infusion-like PTH exposure with lower daily peak-to-trough ratios than observed with daily PTH dosing regimens. It has received orphan drug designation from the U.S. Food and Drug Administration for the treatment of HP.
MBX Biosciences is a clinical-stage biopharmaceutical company focused on the discovery and development of novel precision peptide therapies for the treatment of endocrine and metabolic disorders. The company's pipeline includes candidates for HP, post-bariatric hypoglycemia, and obesity. The market has reacted to these announcements by moving the company's shares 100.8% to a price of $20.08. For the full picture, make sure to review MBX Biosciences's 8-K report.
