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Amolyt Pharma Reports Positive Group I Results in a Phase 2A Trial Evaluation of AZP-3601 for Treatment of Hypoparathyroidism

LYON, France and Cambridge, MA, June 1, 2022 (GLOBE NEWSWIRE) — Amolyt Pharma, a company that specializes in developing therapeutic peptides targeting rare endocrine diseases, today announced positive results from its first set of clinical trials for proof. For the concept of AZP-3601 for the treatment of hypoparathyroidism. The data demonstrate that AZP-3601 was well tolerated and that its daily administration allowed patients to discontinue standard therapy while maintaining an average calcium level within target values. Detailed data from the first cohort will be presented later this year at a medical conference, while full data is expected in the fall of 2022.

This group is the first of the ongoing Phase 2a experiment, and consists of two consecutive groups. In this first group, 12 patients with hypoparathyroidism received a fixed dose of AZP-3601 for four weeks while oral calcium and active vitamin D supplementation were withdrawn gradually, followed by a 2-month extension period during which individual titration was allowed.

Main results:

  • The AZP-3601 was well tolerated without any safety issues. No serious or serious adverse events have been reported.
  • Daily administration of AZP-3601 allowed patients with hypoparathyroidism to discontinue standard therapy (oral calcium and active vitamin D supplementation) while maintaining mean serum calcium within target values.
  • In addition, consistent with its mode of action, AZP-3601 caused a reduction and normalization of 24-hour urinary calcium excretion in all patients in the group, as well as a slight, physiological increase in bone biomarkers.

In addition to its efficacy on serum calcium, the data obtained in the trial on these early patients with hypoparathyroidism show that AZP-3601 rapidly normalized 24-hour urinary calcium excretion in all patients and especially in those with Elevated primary urine calcium level,” said Soraya Alas, MD, PhD, senior vice president of clinical development and regulatory affairs at Amolyt Pharma. These findings are important because patients with hypoparathyroidism have a risk of developing kidney stones and chronic kidney disease about 5 times higher than normal. The bone biomarker data from this study is very encouraging, as it indicates the resumption of physiological bone turnover without excessive bone resorption. This element is necessary if we consider that 17% of hypoparathyroid patients suffer from osteoporosis or osteoporosis and that 53% of women in the pre- and post-menopausal period are at risk of developing osteoporosis. We await with interest the results of the placebo group for a better characterization of the dose-response relationship to treatment and to finalize the design of our pivotal trial. »

Thierry Aprebat, founder and CEO of Amolyt Pharma, concluded: “We are very satisfied with the results of this important cohort study. AZP-3601 does not appear to achieve the primary therapeutic goal of controlling serum calcium and inhibiting active calcium and vitamin D supplementation. Not only does its effect on urinary calcium excretion and bone biomarkers support the hypothesis of a long-term protective effect on kidney and bone health.This is exactly the target product profile we are aiming for: a product based on an innovative mechanism of action, which has a more selective effect on re-absorption. kidneys for calcium without a negative effect on bone metabolism. »

About hypoparathyroidism
Hypoparathyroidism is characterized by a deficiency of parathyroid hormone (PTH) that causes hypocalcemia and elevated phosphorous levels in the blood. About 80,000 people in the United States and 110,000 in Europe have hypoparathyroidism, 80% of whom are women. Despite available treatments, patients have persistent and severe symptoms. They often develop complications and comorbidities that affect their quality of life, and therefore represent population segments with specific clinical needs. Clinical manifestations are varied and may involve many tissues and organs, particularly the kidneys and bones. 17% of patients with hypoparathyroidism have osteopenia or osteoporosis and 53% of peri- or postmenopausal women at risk of developing osteoporosis. It is also estimated that 26% of hypoparathyroid patients have chronic renal disease or renal failure, supporting the therapeutic need to reduce urinary calcium excretion..

About AZP-3601
AZP-3601 is an investigational peptide therapy that selectively acts on a specific modulation of the parathyroid hormone (PTH) receptor to induce a long-term effect on calcium metabolism and thus control the symptoms of hypoparathyroidism. Its action can also limit urinary calcium excretion by restoring calcium absorption by the kidneys, with the goal of preventing chronic kidney disease. In addition, AZP-3601’s unique mode of action and short half-life should preserve bone integrity, which is a significant advantage given that the majority of patients with hypoparathyroidism are perimenopause, often at risk for osteoporosis. orthopedic;

Domolet Pharma’s Suggestion
Amolyt Pharma, a clinical-stage biotechnology company, leverages its team’s knowledge and expertise in therapeutic peptides to develop treatments to improve the lives of patients with rare endocrine diseases. Its development pipeline includes AZP3601, a potential treatment for hypoparathyroidism, and AZP-3813, a potential treatment for acromegaly. Amolyt Pharma intends to continue building its product portfolio by drawing on its global endocrinology network, and supported by a syndicate of international investors. To learn more, visit https://amolytpharma.com/ or follow us on Twitter @AmolytPharma.

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White McClelland
LifeSci . Communications
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