AZP-3601: Hypoparathyroidism​

AZP-3601 is parathyroid hormone receptor 1 (PTHR1) agonist with a novel mechanism of action rationally designed to meet the therapeutic goals of hypoparathyroidism. It targets a specific conformation of the PTH1 receptor to achieve three key therapeutic goals: 1) safely produce sustained and stable levels of calcium in the blood to effectively address symptoms while removing oral calcium and active Vitamin D supplements, 2) normalize urine calcium excretion to prevent kidney disease, and 3) restore bone turnover to a more physiologic state while preventing excessive bone resorption over formation to help preserve bone integrity.

Bone graph

AZP-3601 has demonstrated an optimal pharmacological profile1,2 and has successfully completed pre-investigational new drug activities. A Phase 1 clinical trial in healthy volunteers demonstrated that AZP-3601 induced a rapid, dose-dependent increase in serum calcium levels that was sustained and stable over the treatment period. In addition, no increase in urinary calcium excretion was observed, and bone biomarkers were unchanged.

Data from a Phase 2a trial showed that AZP-3601 was well-tolerated, and daily administration enabled 93% of patients to discontinue standard of care therapy (calcium and vitamin D supplementation) while mean serum calcium was maintained within the target range. Mean urinary calcium excretion was rapidly normalized and bone biomarkers increased in line with a resumption of a normalized physiological level of bone turnover. Consistent with the findings in bone biomarkers, bone mineral density (BMD) was stable, including in patients with osteopenia.

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