MBX Biosciences (Nasdaq: MBX) is making waves in the biopharmaceutical world! The company announced overwhelmingly positive results from its Phase 2 Avail™ trial of canvuparatide, a once-weekly treatment for chronic hypoparathyroidism (HP). The data, released today, shows significant progress in achieving the primary endpoint and sustained improvement over a six-month open-label extension (OLE).
Canvuparatide: A Potential Game-Changer for HP Patients
Hypoparathyroidism is a rare endocrine disorder characterized by insufficient parathyroid hormone production, leading to low calcium levels in the blood. Current treatments often involve daily doses of vitamin D and calcium supplements. Canvuparatide offers a potentially more convenient and effective alternative.
According to the company's press release, 63% of patients treated with canvuparatide in the 12-week randomized trial met the primary composite endpoint. This significant result was achieved without any need for rescue therapy. Furthermore, in the OLE, 79% of patients achieved responder status at 6 months, demonstrating the treatment's long-term efficacy.
Key Highlights from the Phase 2 Trial:
- Statistically significant responder rate at 12 weeks.
- Zero contribution from rescue therapy (PRN).
- Sustained improvement in open-label extension (OLE).
- Positive findings in bone and kidney biomarkers.
- All patients completed the 12-week Avail™ trial.
- 94% entered the OLE.
- Generally well-tolerated with no treatment-related serious adverse events or discontinuations.
MBX Biosciences is now preparing to initiate a Phase 3 trial in 2026. This pivotal trial will be crucial in determining the future of canvuparatide and its potential to become a leading treatment option for hypoparathyroidism.
MBX Biosciences CEO Kent Hawryluk will be hosting a conference call at 8:00 am ET today to discuss the results in further detail. Investors and analysts are keenly watching MBX as it seeks to compete with established players like Ascendis and AstraZeneca in the hypoparathyroidism market.