MBX Biosciences completed enrollment in its Phase 2 Avail™ trial of canvuparatide, a parathyroid hormone (PTH) prodrug for chronic hypoparathyroidism (HP). The trial enrolled 64 patients, surpassing its initial target of 48, and aims to evaluate the drug’s safety, pharmacokinetics, and efficacy. Top-line results are anticipated in Q3 2025.
This successful enrollment signifies progress towards a potential new treatment option for HP, a rare endocrine disorder affecting approximately 120,000 people in the United States alone. Current treatment relies on high doses of calcium and vitamin D supplements, which only manage symptoms rather than addressing the underlying PTH deficiency. Canvuparatide offers a potential shift in the treatment paradigm by directly targeting the hormone deficiency, potentially leading to improved symptom control, better patient compliance, and a higher quality of life.
The Phase 2 trial is designed to determine the proportion of patients able to reduce or eliminate current standard-of-care medications while maintaining stable calcium levels after 12 weeks of treatment with canvuparatide. Secondary endpoints include assessment of the drug’s safety, tolerability, pharmacokinetic and pharmacodynamic activity, and effects on patient-reported outcomes.
Positive results from this trial would validate canvuparatide’s potential as a long-acting, once-weekly treatment for HP. This could lead to a significant advancement in HP management, offering patients a more convenient and effective therapy. Furthermore, it would reinforce the value of MBX Biosciences’ PEP™ platform technology and potentially pave the way for the development of similar precision peptide therapies for other endocrine and metabolic disorders.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
