Amolyt Pharma Initiates Phase 3 Clinical Trial of Eneboparatide for Hypoparathyroidism Treatment: The Calypso Study.

2023-05-10 06:28:54

2023-05-10
Amolyt Pharma Announces Phase 3 Clinical Trial of Eneboparatide for the Treatment of Hypoparathyroidism After Positive End-of-Phase 2 Interactions with the FDA

The Calypso study is expected to be the largest Phase 3 study ever conducted in hypoparathyroidism and the first data should be available at the end of 2024
In addition to serum calcium control and discontinuation of standard therapy, the study will assess normalization of urinary calcium as a key secondary efficacy endpoint
The effect of eneboparatide on bone quantity and quality will also be assessed
The Calypso study builds on the results of the phase 2a study on the control of serum calcium, the discontinuation of standard treatment, the normalization of hypercalciuria, and the restoration of balanced bone turnover

LYON, France and CAMBRIDGE, Mass., May 02, 2023 (GLOBE NEWSWIRE) — Amolyt Pharma, a company specializing in the development of therapeutic peptides targeting rare endocrine diseases, today announced the initiation of the Phase 3 clinical study of eneboparatide, a long-acting agonist of the parathyroid hormone PTH1 receptor, in patients with hypoparathyroidism, following receiving the end of Phase 2 recommendations from the FDA (Food and Drug Administration).

“We appreciated the constructive comments from the FDA during the end of Phase 2 meeting, and have designed our Phase 3 study taking into account their recommendations. We believe the Calypso study will be the largest prospective randomized clinical trial ever conducted in hypoparathyroidism. This is a major milestone, for patients with hypoparathyroidism and for Amolyt Pharma,” said Thierry Abribat, Ph.D., Founder and CEO of Amolyt Pharma. “We look forward to further evaluating the clinical potential of eneboparatide for the management of key clinical needs in hypoparathyroidism. Building on the Series C funding of €130 million we recently announced, we will put all our resources into conducting this study, with our aim to report the first data by the end of 2024.”

Mark Sumeray, MD, Medical Director of Amolyt Pharma, added, “The current gold standard treatment, high doses of calcium and vitamin D supplements, often fails to normalize serum calcium and exacerbates hypercalciuria, leading to kidney dysfunction and progressive kidney failure due to calcium deposits and kidney stones. In this phase 3 study, we will evaluate the efficacy of eneboparatide in controlling serum calcium, stopping the standard treatment and improving symptoms. Normalization of urinary calcium excretion in hypercalciuric patients will be the first key secondary efficacy endpoint. We have also included exploratory criteria assessing bone quantity and quality, as many patients with hypoparathyroidism have, or are at risk of developing osteopenia or osteoporosis. »

Calypso is a multicenter, randomized, placebo-controlled, double-blind phase 3 study designed to evaluate the efficacy and safety of eneboparatide in patients with chronic hypoparathyroidism. Approximately 165 patients on standard treatment will be randomized in a 2:1 ratio between eneboparatide and placebo. The primary efficacy endpoint is the proportion of patients who maintain normal serum calcium without being dependent on the reference treatment following 24 weeks of treatment. Secondary efficacy endpoints include, on the one hand, normalization of urinary excretion over 24 hours in hypercalciuric patients, and on the other hand, the evaluation of symptoms associated with reported physical and cognitive functions. by the patient, as well as the impact on quality of life. Other exploratory endpoints measure bone quantity and quality by DXA densitometry and high-resolution quantitative peripheral computed tomography. After the initial 24-week placebo-controlled period, all patients will be treated with eneboparatide, as part of an open-label extension phase of an additional 28 weeks.

The Calypso study builds on the results of our phase 2a study, which demonstrated that, in most patients, eneboparatide maintained mean serum calcium within the target range by allowing discontinuation of oral supplementation in 93% of patients, induced a balanced increase in bone biomarkers consistent with the restoration of more physiological bone turnover, and rapidly normalized urinary calcium excretion in all but one patient.
The Calypso study will be conducted at more than 50 centers in the United States, Europe, Canada and the United Kingdom.

About hypoparathyroidism

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

About eneboparatide

Eneboparatide is an investigational peptide therapeutic that acts selectively on a specific conformation of the parathyroid hormone (PTH) receptor to induce a prolonged effect on calcium metabolism and thereby control the symptoms of hypoparathyroidism. Its action might also limit the urinary excretion of calcium by restoring calcium reabsorption by the kidney, with the aim of preventing chronic kidney disease. In addition, eneboparatide’s unique mode of action and short half-life should preserve bone integrity, a major benefit considering that the majority of patients with hypoparathyroidism are peri- or post- menopausal, often at risk of osteoporosis.

About Amolyt Pharma

Amolyt Pharma, a clinical-stage biotechnology company, relies on the know-how and experience of its team to develop treatments aimed at improving the lives of patients suffering from rare endocrine diseases. Its development pipeline includes eneboparatide (AZP-3601), a long-acting PTH1 receptor agonist as a potential treatment for hypoparathyroidism, and AZP-3813, a growth hormone receptor antagonist for the treatment potential for acromegaly. Amolyt Pharma intends to continue building its product portfolio by relying on its global network in the field of endocrinology, and with the support of its syndicate of international investors.
To find out more, visit https://amolytpharma.com/