5-HT1 receptor

AstraZeneca is bolstering its rare disease pipeline with a deal to buy Amolyt Pharma, a biotech company in late-stage development with a therapy designed to treat a rare hormone deficiency whose only FDA-approved treatment will soon cease production. According to deal terms announced Thursday, AstraZeneca is paying Amolyt shareholders $800 million up front. Another $250 million is tied to the achievement of a regulatory milestone that was not disclosed. Considering that the drug is in Phase 3 testing, that milestone could be a regulatory approval. Lyon, France-based Amolyt is developing a therapy for hypoparathyroidism, a disease in which the parathyroid glands do not produce enough parathyroid hormone. This hormone is important for regulating calcium and phosphate levels in the blood. Disease symptoms include tingling or burning sensations in the fingertips and toes, muscle aches or cramps, muscle twitching or spasms, and fatigue. Hypoparathyroidism disproportionately affects women. Calcium and vitamin D supplements can help, but at high doses, calcium supplements can lead to gastrointestinal problems such as constipation. When supplements alone aren’t enough to manage hypoparathyroidism, the only FDA-approved therapy is Natpara, an engineered version of parathyroid hormone sold by Takeda Pharmaceutical. Amolyt could bring patients a different way to manage hypoparathyroidism. The biotech’s drug candidate, eneboparatide, is a peptide designed to bind to PTH receptor 1, the receptor that native parathyroid hormone binds to. In Phase 2 results reported in 2022, Amolyt said its drug led to normal calcium levels in the blood as well the potential to eliminate the need for daily calcium and vitamin D supplements. Furthermore, the drug preserved bone mineral density, which is important for patients at higher risk of developing bone problems such as osteopenia or osteoporosis. The market need for new hypoparathyroidism treatments is becoming urgent. Takeda’s Natpara (Natpara outside of the U.S.) has had a rocky commercialization history since its FDA approval in 2015. In 2019, the FDA issued a recall due to potential contamination from tiny rubber particles from the cartridge housing the injectable product. Takeda’s efforts to resolve the problem to the FDA’s satisfaction have been unsuccessful. In late 2022, Takeda said it planned to discontinue manufacturing of Natpara/Natpar by the end of 2024. After that, the company said it would supply the remaining doses of the drug until inventory is depleted or the product is expired. The Amolyt acquisition brings AstraZeneca into a group of companies vying to fill the hypoparathyroidism treatment need that will be left when Takeda’s drug is no longer available. Extend Biosciences is in Phase 1 testing with a drug derived from parathyroid hormone. MBX Biosciences has reached Phase 2 testing with a peptide drug. Both drug candidates are injectable. Septerna is developing an oral drug for hypoparathyroidism. Last year, the South San Francisco-based startup raised $150 million in financing to advance the small molecule to Phase 1 testing. Amolyt last raised money in early 2023, a €130 million (about $138 million) Series C financing to support its pipeline. Besides the hypoparathyroidism program, that pipeline includes AZP-3813, a potential treatment for acromegaly, a disease in which the body produces too much growth hormone. Phase 1 testing began last summer. Amolyt has said it expects preliminary data in the first half of 2024. AstraZeneca expects to complete the Amolyt acquisition by the end of the third quarter of this year. When the deal closes, Amolyt will become part of Alexion, AstraZeneca Rare Disease, the pharmaceutical giant’s Boston-based rare disease division. AstraZeneca said the acquisition will enable it to expand its rare disease research into endocrinology. “Chronic hypoparathyroid patients face a significant need for an alternative to current supportive therapies, which do not address the underlying hormone deficiency,” Marc Dunoyer, CEO of Alexion, AstraZeneca Rare Disease, said in a prepared statement. “As leaders in rare disease, Alexion is uniquely positioned to drive the late-stage development and global commercialization of eneboparatide, which has the potential to lessen the often debilitating impact of low parathyroid hormone and avoid the risks of high-dose calcium supplementation.”

AstraZeneca is splashing out $800 million upfront to bolster its rare disease pipeline with the purchase of Amolyt Pharma and its Phase III asset for chronic hypoparathyroidism.Marc Dunoyer, chief executive of AstraZeneca’s rare-disease unit Alexion, noted that patients with chronic hypoparathyroidism “face a significant need for an alternative to current supportive therapies,” with Amolyt’s eneboparatide holding “the potential to lessen the often debilitating impact of low parathyroid hormone and avoid the risks of high-dose calcium supplementation.”Eneboparatide – also known as AZP-3601 – is a PTH receptor 1 agonist that in Phase II studies has shown the ability to achieve normalisation of serum calcium levels, as well as the potential to eliminate dependence on daily calcium and vitamin D supplementation. AstraZeneca noted that in adults with chronic hypoparathyroidism and hypercalciuria, eneboparatide normalised calcium in urine, while it also preserved bone mineral density.According to Amolyt’s website, its pipeline also includes AZP-3813 for acromegaly - with initial Phase I results expected later this year - and the preclinical candidate AZP-40XX for primary hyperparathyroidism and humoral hypercalcemia of malignancy.Along with the upfront payment, Amolyt shareholders are eligible to receive an additional $250 million payable upon achievement of a specified regulatory milestone. The acquisition is expected to close by the end of the third quarter.

LYON, France and CAMBRIDGE, Mass., Sept. 27, 2023 (GLOBE NEWSWIRE) -- Amolyt Pharma, a global company specialized in developing therapeutic peptides for rare endocrine and related diseases, today announced that it will present data on eneboparatide, the company’s investigational lead product candidate for the treatment of hypoparathyroidism, at the 39th Congress of the French Society of Endocrinology, being held October 4-7, 2023, in Marseille, France. Jean-Philippe Bertocchio, M.D., Ph.D., Clinical Nephrologist at Pitié-Salpêtrière Hospital, Paris, and Michel Ovize, M.D., Ph.D., Senior Medical Director of Amolyt, will discuss the effects of eneboparatide on the kidneys of patients with hypoparathyroidism. The presentation will also include data on eneboparatide’s effect on the bone and the results of the company’s Phase 2a clinical trial in more detail. An interactive Q&A with doctors Bertocchio and Ovize, moderated by Thomas Cuny, M.D., Ph.D., will conclude the session. Presentation details: Title: The kidney of the patient with hypoparathyroidism: Effect of eneboparatide, a new agonist of the PTH receptor 1Date: Thursday, October 5, 2023 Time: 7:45 – 8:30 a.m. CET Presenters: Jean-Philippe Bertocchio, M.D., Ph.D., Michel Ovize, M.D., Ph.D. A replay of the presentation will be available to all registrants following conclusion of the event. Additional details can be found on the French Society of Endocrinology website. About HypoparathyroidismHypoparathyroidism is a rare condition defined by a deficiency of parathyroid hormone (PTH) that results in decreased calcium and elevated phosphorus levels in the blood. Approximately 80% of the estimated 80,000 people in the U.S. and 110,000 in the European Union with hypoparathyroidism are women. Despite available treatments, patients experience persistent, life-altering symptoms and often develop complications and comorbidities that diminish quality of life and create segments of the patient population with specific clinical needs. Clinical manifestations of hypoparathyroidism impact many tissues and organ systems, in particular, the kidneys and bone. More than half of all patients are post-menopausal women who are at an increased risk for developing osteoporosis. In a 515 hypoparathyroidism patient chart review, 17% were diagnosed with osteopenia or osteoporosis, and in the eneboparatide Phase 2a trial, 43% of patients had osteopenia. Approximately 26% of patients with hypoparathyroidism have chronic kidney disease or failure, highlighting the importance of reducing urinary calcium excretion as a key treatment goal. About EneboparatideEneboparatide is an investigational therapeutic peptide designed to target a specific conformation of the parathyroid hormone (PTH) receptor to produce sustained and stable levels of calcium in the blood and thereby manage the symptoms of hypoparathyroidism, and to limit urine calcium excretion by restoring calcium reabsorption by the kidney, with the goal of consequently preventing progressive decline in kidney function and the development of chronic kidney disease. In addition to its unique receptor profile, eneboparatide is also designed to have a short half-life to potentially preserve bone integrity, an important potential benefit, since the majority of patients are peri- and postmenopausal women with an increased risk of developing osteoporosis. About Amolyt Pharma Amolyt Pharma, a clinical stage biotechnology company, is building on its team’s established expertise to deliver life-changing treatments to patients suffering from rare endocrine and related diseases. Its development portfolio includes eneboparatide (AZP-3601), a long-acting PTH1 receptor agonist as a potential treatment for hypoparathyroidism, and AZP-3813, a peptide growth hormone receptor antagonist for the potential treatment of acromegaly. Amolyt Pharma aims to further expand and develop its portfolio by leveraging its global network in the field of endocrinology and with support from a strong syndicate of international investors. To learn more, visit https://amolytpharma.com/ or follow us on Twitter and LinkedIn. Media Inquiries: Jordyn TemperatoLifeSci Communications jtemperato@lifescicomms.com Investors Inquiries:Ashley RobinsonLifeSci Advisors, LLCarr@lifesciadvisors.com+1.617.430.7577