FDA Fast Tracks Amolyt Pharma's Eneboparatide for Hypoparathyroidism

Amolyt Pharma, a global biotechnology firm specializing in therapeutic peptides for rare endocrine diseases, recently announced that its leading candidate, eneboparatide, has been granted fast track designation by the U.S. Food and Drug Administration (FDA). Eneboparatide is currently in Phase 3 development for treating hypoparathyroidism, a rare endocrine disorder characterized by insufficient parathyroid hormone (PTH) leading to abnormal calcium and phosphorus levels in the blood. The FDA’s Fast Track designation aims to speed up the development and review of drugs that address serious conditions and unmet medical needs, facilitating quicker access to new therapies for patients.

Thierry Abribat, Ph.D., founder and CEO of Amolyt Pharma, expressed that the FDA's fast track designation underscores the significant unmet needs of hypoparathyroidism patients. Abribat emphasized the company's commitment to working closely with the FDA to efficiently bring new treatment options to patients suffering from this rare and complex condition.

Current treatment options for hypoparathyroidism, primarily oral calcium and vitamin D supplementation, often fail to manage the disease's symptoms and complications effectively. Mark Sumeray, M.D., Chief Medical Officer at Amolyt Pharma, highlighted that many patients face risks such as declining kidney function and poor bone quality. In previous studies, eneboparatide has demonstrated the ability to normalize serum calcium levels, reduce urinary calcium excretion, and restore balanced bone turnover. Building on successful Phase 2 trial results, the company is actively conducting the Calypso Phase 3 study, with anticipated top-line data expected in 2025.

The Calypso study is a multicenter, randomized, placebo-controlled, double-blind trial designed to assess the efficacy and safety of eneboparatide in chronic hypoparathyroidism patients. About 165 patients on standard care will be randomized in a 2:1 ratio to receive either eneboparatide or a placebo. The primary efficacy endpoint is achieving normal albumin-adjusted serum calcium levels and independence from standard care after 24 weeks of treatment. Secondary endpoints include normalizing 24-hour urinary calcium in patients with hypercalciuria at baseline and evaluating patient-reported outcomes related to physical and cognitive function and quality of life. Additional exploratory endpoints will assess bone quantity and quality through DXA and high-resolution peripheral quantitative CT scanning. Following the initial 24-week period, all patients will receive eneboparatide in an open-label extension phase for another 28 weeks.

Hypoparathyroidism is a rare condition marked by a lack of PTH, leading to reduced calcium and increased phosphorus levels in the blood. The disease affects approximately 80,000 people in the U.S and 110,000 in the European Union, with about 80% being women. Despite existing treatments, patients often suffer from persistent symptoms and complications that significantly impact their quality of life. The disease predominantly affects the kidneys and bones, with over half of the patients being post-menopausal women who face increased risks of osteoporosis and fragility fractures. A significant portion also suffers from chronic kidney disease or failure, highlighting the necessity of reducing urinary calcium excretion as a crucial treatment objective.

Eneboparatide is an investigational therapeutic peptide designed to bind effectively to a specific PTH receptor conformation. This binding results in stable calcium levels in the blood, addressing hypoparathyroidism symptoms and reducing urinary calcium excretion, thereby potentially preventing kidney function decline and chronic kidney disease. The peptide's unique receptor profile and short half-life aim to preserve bone integrity, particularly beneficial for peri- and postmenopausal women at higher risk of osteoporosis.

Amolyt Pharma continues to leverage its expertise in rare endocrine diseases to develop life-changing treatments. Its portfolio includes eneboparatide as a potential hypoparathyroidism treatment and AZP-3813, a peptide growth hormone receptor antagonist for acromegaly. The company aims to expand its portfolio by utilizing its global endocrinology network and support from international investors.