CARMEL, Ind., and BURLINGTON, Mass. — MBX Biosciences, Inc. (Nasdaq: MBX), a clinical-stage biopharmaceutical company focused on the discovery and development of novel precision peptide therapies for the treatment of endocrine and metabolic disorders, today announced that the first patient has been dosed in its pivotal Phase 3 oPTimize clinical trial evaluating once-weekly canvuparatide for the treatment of chronic hypoparathyroidism (HP).
“The initiation of our pivotal Phase 3 program marks an important milestone for MBX as we advance once-weekly canvuparatide toward potential registration,” said Steve Hoerter, Chairman and Chief Executive Officer of MBX Biosciences. “The promising efficacy observed in the Phase 2 Avail™ trial and the sustained clinical effect demonstrated through one year of treatment reinforce our confidence in canvuparatide’s potential to become a new standard of care in the treatment of HP. We look forward to advancing the Phase 3 oPTimize trial and bringing once-weekly canvuparatide one step closer to patients living with chronic HP.”
Phase 3 oPTimize Trial Design
The Phase 3 oPTimize trial (NCT07699471) is a global, multicenter, randomized, double-blind, placebo-controlled study evaluating the efficacy and safety of once-weekly canvuparatide in adults with chronic hypoparathyroidism, followed by a 78-week open-label extension. Approximately 160 patients will be enrolled and randomized 3:1 to receive canvuparatide or placebo during the 26-week double-blind treatment period. Canvuparatide will be administered once weekly using the same device intended for commercial use. More information on the Phase 3 oPTimize trial is available at https://www.optimize-study.com/.
The 26-week double-blind treatment period consists of:
- A four-week fixed-dose period at 600 micrograms or placebo once weekly
- An 18-week individualized dose-titration period (up to 1,600 micrograms once weekly)
- A four-week dose-maintenance period through Week 26 without increases in study drug, active vitamin D, or calcium supplementation.
The primary endpoint will evaluate the proportion of patients achieving a composite responder endpoint at Week 26, defined as meeting all the following criteria:
- Maintenance of normal albumin-adjusted serum calcium levels
- Independence from active vitamin D
- Reduction of calcium supplementation to ≤600 milligrams per day
- No increases in study drug dose during the four weeks preceding the Week 26 assessment
Key secondary endpoints include normalization of urinary calcium excretion while maintaining normal serum calcium in patients with elevated baseline urinary calcium excretion, and improvement of physical functioning and hypocalcemia-related symptoms. The trial will also evaluate additional measures of bone mineral metabolism, kidney health, bone biomarkers, patient-reported outcomes and long-term safety.
The Phase 3 oPTimize trial builds on positive results from the Phase 2 Avail™ trial and ongoing open-label extension study, which demonstrated results consistent with restoration of systemic PTH activity, including normalization of serum calcium, reduction of urinary calcium excretion, restoration of bone metabolism and increase of eGFR. Together, these findings support once-weekly canvuparatide’s potential to become a best-in-class PTH replacement therapy for people living with chronic hypoparathyroidism.
About Hypoparathyroidism
Hypoparathyroidism is a rare endocrine disorder characterized by deficient or absent PTH production, resulting in hypocalcemia and disruptions in mineral homeostasis. Current first line standard-of-care treatment consists of calcium supplements and active vitamin D, which do not replace the missing hormone or restore physiologic homeostasis and may be associated with significant treatment burden and long-term complications. Chronic HP affects over 250,000 patients in the US and EU along, with an annual incidence estimated at over 7,000 patients per year.
About Once-Weekly Canvuparatide
Canvuparatide is an investigational long-acting parathyroid hormone peptide prodrug being developed as a once-weekly PTH replacement therapy for adults with hypoparathyroidism. Canvuparatide is designed to provide continuous, infusion-like exposure to active PTH through a convenient weekly subcutaneous injection. Canvuparatide has received Orphan Drug Designation from the U.S. Food and Drug Administration and Orphan Designation from the European Medicines Agency for the treatment of hypoparathyroidism.
About MBX Biosciences
MBX Biosciences is a biopharmaceutical company focused on the discovery, development and commercialization of novel precision peptide therapies based on its proprietary PEP™ platform, for the treatment of endocrine and metabolic disorders. The Company is advancing a pipeline of novel candidates for endocrine and metabolic disorders with clinically validated targets, established endpoints for regulatory approval, significant unmet medical needs and large potential market opportunities. The Company’s pipeline includes canvuparatide (MBX 2109) for the treatment of chronic hypoparathyroidism in Phase 3; and an obesity portfolio that includes MBX 4291 in Phase 1, and MBX 5765 and MBX 6180 in preclinical development, as well as additional discovery candidates. The Company is based in Carmel, Indiana and Burlington, Massachusetts. To learn more, please visit the company website at www.mbxbio.com and follow it on LinkedIn.
About MBX’s Proprietary Precision Endocrine Peptide (PEP™) Platform
MBX was founded by global leaders with a transformative approach to peptide drug design and development. Leveraging this expertise, the Company designed its proprietary Precision Endocrine Peptide™ (PEP™) platform to overcome the key limitations of unmodified and modified peptide therapies and to improve clinical outcomes and simplify disease management for patients. PEPs are selectively engineered to have optimized pharmaceutical properties, including extended time-action profiles and consistent drug concentrations with low peak-to-trough concentration ratios, consistent exposure to target tissues, and less frequent dosing.
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