MBX Biosciences Inc. (NASDAQ:MBX) on Thursday reported full results from its 12-week Avail Phase 2 trial and new one-year data from an ongoing open-label extension study of once-weekly canvuparatide in adults with chronic hypoparathyroidism.
Hypoparathyroidism is a rare condition in which the parathyroid glands in the neck produce too little parathyroid hormone (PTH), leading to low blood calcium (hypocalcemia) and high phosphorus levels.
The data highlighted sustained disease control and continued progress toward a Phase 3 program.
The company said the latest findings support the potential of canvuparatide as a once-weekly treatment designed to restore physiologic parathyroid hormone (PTH) activity while reducing treatment burden for patients.
Phase 2 Trial Met Primary Endpoint
As previously reported, 63% of patients treated with canvuparatide achieved the study's primary composite endpoint at 12 weeks, compared with 31% of placebo-treated patients.
The endpoint required patients to maintain normal albumin-adjusted serum calcium levels while remaining independent from conventional therapy, including active vitamin D and more than 600 milligrams per day of calcium supplements.
At the one-year mark, 57% of evaluable patients achieved responder status without any contribution from rescue therapy during the final week of treatment.
Long-Term Data Show Sustained Physiologic Effects
According to the company, pharmacokinetic data continued to support once-weekly dosing.
Canvuparatide demonstrated consistent drug concentrations throughout the week, with minimal fluctuations and a peak-to-trough ratio of approximately 1.3.
Researchers also reported evidence of sustained physiologic PTH replacement through one year. Mean serum calcium levels remained within the normal range, while 24-hour urine calcium levels declined from baseline and stayed within normal limits.
The company added that the estimated glomerular filtration rate, a measure of kidney function, improved by Week 12 and remained elevated through one year of treatment.
Bone remodeling markers, including CTx and P1NP, showed patterns consistent with expected bone turnover associated with PTH replacement therapy. Changes in bone mineral density scores were also consistent with restoration of physiologic bone remodeling.
Safety Profile Remained Consistent
The company reported that canvuparatide was generally well tolerated through one year, with no new safety signals identified.
MBX Biosciences said it remains on track to initiate a Phase 3 trial of canvuparatide in the third quarter of 2026.
MBX Stock Price Activity: MBX Biosciences shares were down 4.86% at $35.44 at the time of publication on Friday, according to Benzinga Pro data.
Image created using artificial intelligence via Midjourney.
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