Endocrine Abstracts

Background: Prader-Willi syndrome (PWS) is a rare disease characterized by hyperphagia and abnormal food-related behaviors that contribute to severe morbidity and early mortality and to a significant burden on patients and caregivers. There is no approved treatment for hyperphagia in PWS. Patients with PWS have increased circulating levels of the orexigenic hormone acylated ghrelin (AG) with a relative deficit of unacylated ghrelin (UAG). Livoletide (AZP-531) is a first-in-cla...

Prader-Willi syndrome (PWS) is a rare complex endocrine disease characterized by hyperphagia and abnormal food-related behaviors that contribute to severe morbidity and early mortality and to a significant burden on patients and caregivers. There are no approved treatments for hyperphagia in PWS. Patients with PWS have increased circulating levels of the orexigenic hormone acylated ghrelin (AG) with a relative deficit of unacylated ghrelin (UAG). These abnormalities in AG and ...

Hypoparathyroidism is a rare disease characterized by a deficiency in parathyroid hormone (PTH) that results in hypocalcemia and hyperphosphatemia. Current treatment approaches, including high dose oral calcium and active vitamin D, as well as recombinant human PTH (1–84), do not provide adequate or consistent control of either serum calcium or clinical symptoms over a full 24-hour period. AZP-3601 is a novel 36 amino-acid PTH analog that has been designed to potently bi...

Maintaining normal serum and urinary calcium in patients with chronic hypoparathyroidism (cHP) remains a therapeutic challenge. Parathyroid hormone (PTH) replacement therapy is not ideal due to its short half-life and transient activation of the PTH 1 receptor (PTH1R). Eneboparatide is a hybrid analog of PTH and PTH-related peptide specifically designed to induce prolonged activation of the PTH1R. Despite a short half-life, eneboparatide is able to normalize serum and urinary ...

Eneboparatide (AZP-3601) is a novel, synthetic, 36-amino-acid peptide agonist of the parathyroid hormone type 1 receptor (PTHR1), with potent selectivity for the R0 conformation. This results in prolonged calcemic responses, while having a short circulating half-life. Eneboparatide is being developed for the treatment of chronic hypoparathyroidism (cHP). Studies in hypoparathyroid animal models and, most recently, in hypoparathyroid patients, have demonstrated that eneboparati...

ObjectivesThere is a paucity of real-world studies analyzing comorbidities, lab testing and treatment patterns of patients with chronic hypoparathyroidism (cHP). This study describes a large cohort of cHP patients identified using a diagnosis-based approach from a US claims database.MethodsThis retrospective study was conducted using a large (130 million individuals) claims database (HealthVerity Closed Payer...

ObjectivesThere is a paucity of real-world studies on the clinical burden and practice patterns associated with chronic hypoparathyroidism (cHP). This study assesses the comorbidities, treatment and lab testing patterns in cHP patients identified using surgery-based criteria.MethodsThis was a retrospective study conducted using a large (130 million individuals) US claims database (HealthVerity Closed Payer Cl...

Background: Acromegaly is a rare disease typically caused by a benign growth hormone (GH)-secreting pituitary adenoma that stimulates over-production of insulin-like growth factor-1 (IGF1) from the liver. Treatment with somatostatin analog (SSA) monotherapy does not provide optimal control of circulating IGF-1 levels in the majority of patients. AZP-3813, a 16-amino acid, bicyclic GH receptor antagonist (GHRA) binds to the GH receptor and prevents endogenous GH from stimulatin...

Background: Conventional therapy for chronic hypoparathyroidism (cHP) is often unable to maintain stable normal serum calcium (Ca) levels for a full 24 h, to control symptoms, to prevent the detrimental long-term effects on the kidney and to preserve normal bone architecture. Eneboparatide (AZP-3601) is a novel 36-amino-acid peptide specifically designed to activate the R0 conformation of the PTH receptor 1, that results in a prolonged calcemic response and a sustai...

Background: Conventional therapy with oral calcium (Ca) and active vitamin D (vitD) supplementation for chronic hypoparathyroidism (cHP) can induce or aggravate hypercalciuria and may lead to detrimental long-term renal complications. Eneboparatide (AZP-3601) is a novel 36-amino-acid peptide with a short half-life designed to activate the R0 conformation of the PTH 1 receptor which produces a prolonged calcemic response. This phase 2a study examined the effects of e...