MBX Biosciences Advances Phase 1 Clinical Trial of Long-Acting Parathyroid Hormone Peptide Prodrug

MBX Biosciences, Inc. recently announced it has initiated the multiple ascending dose portion of its ongoing Phase 1 clinical trial of MBX 2109. MBX 2109, the company’s lead investigational drug, is a long-acting parathyroid hormone (PTH) peptide prodrug in development for the treatment of hypoparathyroidism.

“This is an exciting achievement for MBX as we continue to advance our first clinical program,” said Kent Hawryluk, President and Chief Executive Officer of MBX Biosciences. “We believe MBX 2109 has the potential to address a substantial need in hypoparathyroidism, and this trial brings us a step closer to evaluating it in patients.”

The randomized, double blind, placebo-controlled Phase 1 trial is designed to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single and multiple ascending doses of MBX 2109 in healthy adult subjects. The single ascending dose portion of the trial, which began at the end of 2021, was completed successfully. MBX 2109 was observed to be safe and well tolerated, as compared to placebo. For the multiple ascending dose phase of the trial, subjects are to receive either MBX 2109 or placebo once weekly subcutaneously for four weeks. The primary outcome measures will evaluate safety and tolerability. Secondary outcome measures will evaluate pharmacokinetics and pharmacodynamics. For additional information about the trial, please visit clinicaltrials.gov using the identifier NCT05158335.

Hypoparathyroidism (HP) is a rare endocrine disorder caused by a deficiency of parathyroid hormone that results in decreased calcium and increased phosphorus levels in the blood. HP affects approximately 200,000 individuals worldwide, most of whom develop the condition following damage to or removal of the parathyroid glands during thyroid surgery. It is a complicated disorder associated with a wide range of symptoms including paresthesias, muscle cramps, seizures, an impaired quality of life, and an increased risk of comorbidities including kidney stones and impaired renal function. The goal of treatment is to maintain blood calcium levels in the low-normal range while preventing symptoms of hypocalcemia. Current standard of care consists of high doses of calcium supplements and active vitamin D, which may contribute to the risk of renal disease and does not address the underlying pathophysiology which is to restore the missing hormone.

MBX 2109 is an investigational long-acting parathyroid hormone peptide prodrug in development as a PTH replacement therapy. It was designed with the company’s novel, proprietary Precision Endocrine Peptide (PEP) platform to provide sustained PTH activity with a convenient once-weekly dosing regimen. MBX aims to simplify an individual’s disease management, while relieving both the symptoms of the disorder and long-term complications.

MBX Biosciences, Inc. is a clinical stage biopharmaceutical company pioneering investigational Precision Endocrine Peptides (PEPs). PEPs are designed to overcome key limitations of native peptide therapeutics to deliver superior pharmacologic properties. MBX is advancing a pipeline of PEP candidates to address the unmet needs of people with endocrine disorders. The company’s lead product candidate, MBX 2109 for the treatment of hypoparathyroidism, is currently in a Phase 1 clinical trial. Members of MBX’s leadership team have collaborated over several decades on the discovery, development, and commercialization of highly successful endocrine therapeutics including Forteo and Humalog. The company is supported by leading life science investors including Frazier Life Sciences, New Enterprise Associates and OrbiMed. For more information, visit the MBX website.