AZP-3601 is an investigational therapeutic peptide designed to target a specific conformation of the parathyroid hormone (PTH) receptor in order to safely produce sustained levels of calcium in the blood and thereby manage the symptoms of hypoparathyroidism.
AZP-3601 is designed to be selectively active through this distinct conformation of the PTH receptor and to limit urine calcium excretion by restoring calcium reabsorption by the kidney, with the goal of consequently preventing chronic kidney disease. In addition, AZP-3601 is designed to have a unique receptor profile and short half-life, which would have the potential to preserve bone integrity.
1Proprietary quantitative Market Research, 2021
2Proprietary retrospective Natural History Study, 2020
3Hadker and al., Endocrine Practice, Volume 20 No. 7 July 2014
AZP-3601 has demonstrated an optimal pharmacological profile in several preclinical studies1,2 and has successfully completed pre-investigational new drug activities. A Phase 1 clinical trial in healthy volunteers has been completed and positive results were announced in October 2021 at the American Society for Bone and Mineral Research. These results demonstrated that repeat administration of AZP-3601 to healthy volunteers 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, as was expected based on the unique mechanism of action of AZP-3601 and its short pharmacokinetic half-life.
Based on these positive results, we have initiated our next trial in patients. This is an important step forward in our quest to bring patients with hypoparathyroidism a potential new treatment option. The results from this trial will be reported in the first half of 2022.
- Shimizu, et al. “Pharmacodynamic Actions of a Long-Acting PTH Analog (LA-PTH) in Thyroparathyroidectomized (TPTX) Rats and Normal Monkeys.” J Bone Miner Res. 2016 Jul;31(7):1405-12.
- Bi, et al. “Diphtheria Toxin- and GFP-Based Mouse Models of Acquired Hypoparathyroidism and Treatment With a Long-Acting Parathyroid Hormone Analog.” J Bone Miner Res. 2016 May;31(5):975-84.
AZP-3601 (red) bound to the PTH1 receptor. Adapted from Zhao et al. Science 364:138, 2019.
Image generated with the PyMOL Molecular Graphics System, Version 2.3.2 Schrödinger, LLC.