AstraZeneca Expands Rare Disease Pipeline with Amolyt Pharma Acquisition

AstraZeneca has announced it is set to acquire Amolyt Pharma, a French clinical-stage biotechnology company focused on developing novel treatments for rare endocrine diseases, for a total consideration of up to $1.05 billion. The purchase will expand AstraZeneca’s rare disease pipeline beyond complement inhibition, building on the company’s success in bone metabolism and opportunity in rare endocrinology.

The proposed acquisition stands to fortify the Rare Disease late-stage pipeline of Alexion and AstraZeneca, while also expanding its bone metabolism franchise through the inclusion of eneboparatide (AZP-3601), a Phase III investigational therapeutic peptide. Eneboparatide offers a novel mechanism of action tailored to address critical therapeutic objectives for hypoparathyroidism.

Hypoparathyroidism, characterized by insufficient production of parathyroid hormone (PTH), leads to substantial disruptions in calcium and phosphate regulation, often resulting in debilitating symptoms and complications, such as chronic kidney disease. This rare disease affects a considerable number of individuals globally, with estimates indicating its impact on over 115,000 people in the United States and 107,000 individuals in the European Union, predominantly women.

Marc Dunoyer, Chief Executive Officer, Alexion, AstraZeneca Rare Disease, said:

“Chronic hypoparathyroid patients face a significant need for an alternative to current supportive therapies, which do not address the underlying hormone deficiency. As leaders in rare disease, Alexion is uniquely positioned to drive the late-stage development and global commercialisation 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. We believe this programme, together with Amolyt’s talented team, expertise and earlier pipeline, will enable our expansion into rare endocrinology.”

Thierry Abribat, Chief Executive Officer, Amolyt Pharma, said:

“We enthusiastically welcome the proposed acquisition of Amolyt by AstraZeneca, an organisation that shares our dedication to delivering life-changing treatments to people living with rare diseases. This agreement offers the opportunity to meaningfully advance our pipeline therapies. Strong Phase II data suggest eneboparatide has the potential to improve outcomes for patients and to shift the treatment paradigm for hypoparathyroidism, and we look forward to seeing the continued advancement of the Phase III trial.”

Eneboparatide, functioning as a PTH receptor 1 (PTHR1) agonist, offers a novel mechanism of action aimed at addressing the complex therapeutic requirements of hypoparathyroidism. Phase II data showed that eneboparatide achieved normalisation of serum calcium levels as well as the potential to eliminate dependence on daily calcium and vitamin D supplementation. In adults with chronic hypoparathyroidism and hypercalciuria, results have shown that eneboparatide normalised calcium in the urine. In addition, for patients with hypoparathyroidism, eneboparatide preserved bone mineral density, a key potential benefit in patients with an increased risk of osteopenia or osteoporosis.

Pending the fulfillment of standard closing conditions outlined in the acquisition agreement, including regulatory approvals, the transaction is anticipated to conclude by the conclusion of the third quarter of 2024.

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Image Credit: AstraZeneca

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