AstraZeneca is strengthening its uncommon illness pipeline with an settlement to accumulate Amolyt Pharma, a biotech firm in late-stage growth of a remedy designed to deal with a uncommon hormone deficiency whose solely FDA-approved remedy will quickly be can be discontinued.

Beneath deal phrases introduced Thursday, AstraZeneca pays Amolyt shareholders $800 million upfront. One other $250 million is tied to reaching a regulatory milestone that has not been disclosed. Contemplating the drug is in Part 3 testing, that milestone may very well be a regulatory approval.

Lyon, France-based Amolyt is creating a remedy for hypoparathyroidism, a illness by which the parathyroid glands don’t produce sufficient parathyroid hormone. This hormone is necessary for regulating the calcium and phosphate ranges within the blood. Illness signs embrace tingling or burning sensations within the fingertips and toes, muscle ache or cramps, muscle twitching or spasms, and fatigue.

Hypoparathyroidism disproportionately impacts ladies. Calcium and vitamin D dietary supplements might help, however at excessive doses, calcium dietary supplements can result in gastrointestinal issues equivalent to constipation. When dietary supplements alone aren’t sufficient to regulate hypoparathyroidism, the one FDA-approved remedy is Natpara, a modified model of parathyroid hormone, bought by Takeda Pharmaceutical.

Amolyt may supply sufferers one other option to deal with hypoparathyroidism. The biotech's drug candidate, eneboparatide, is a peptide designed to bind to PTH receptor 1, the receptor to which the pure parathyroid hormone binds. In Part 2 outcomes reported in 2022, Amolyt mentioned the drug led to regular blood calcium ranges, in addition to the potential to eradicate the necessity for day by day calcium and vitamin D dietary supplements. As well as, the drug helped preserve bone mineral density, which is necessary for sufferers at greater danger of creating bone issues equivalent to osteopenia or osteoporosis. Amolyt has mentioned it expects preliminary Part 3 knowledge to be out there by the tip of 2024.

The market want for brand new remedies for hypoparathyroidism is turning into pressing. Takeda's Natpara (Natpar exterior the US) has had a troubled commercialization historical past since its FDA approval in 2015. In 2019, the FDA issued a recall because of doable contamination by small rubber particles from the cartridge containing the injectable product. Takeda's makes an attempt to resolve the difficulty to the FDA's satisfaction have been unsuccessful. In late 2022, Takeda mentioned it deliberate to stop manufacturing of Natpara/Natpar by the tip of 2024. After that, the corporate mentioned it will provide the remaining doses of the drug till provides run out or the product expires.

The acquisition of Amolyt places AstraZeneca in a bunch of corporations vying to fill the remedy want for hypoparathyroidism that continues to be if Takeda's drug is not out there. An FDA choice on Ascendis Pharma's peptide drug Transcon PTH is predicted in Might. Lengthen Biosciences is in part 1 testing of a drug derived from parathyroid hormone. MBX Biosciences has reached Part 2 testing of a peptide drug. These drug candidates are injectable. Septerna is creating an oral drug for hypoparathyroidism. Final yr, the South San Francisco-based startup raised $150 million in funding to advance the small molecule to part 1 testing.

Amolyt final raised cash in early 2023, a Collection C financing of €130 million (about $138 million) to help its pipeline. Along with the hypoparathyroidism program, that pipeline contains AZP-3813, a possible remedy for acromegaly, a illness by which the physique produces an excessive amount of development hormone. Part 1 testing started final summer time. Amolyt has mentioned it expects preliminary knowledge within the first half of 2024.

AstraZeneca expects to finish the acquisition of Amolyt by the tip of the third quarter of this yr. When the deal closes, Amolyt will turn out to be a part of Alexion, AstraZeneca Uncommon Illness, the pharmaceutical large's Boston-based uncommon illness division. AstraZeneca mentioned the acquisition will enable the corporate to increase its uncommon illness analysis into endocrinology.

“Sufferers with continual hypoparathyroidism face a big want for an alternative choice to present supportive therapies, which don’t tackle the underlying hormone deficiency,” Marc Dunoyer, CEO of Alexion, AstraZeneca Uncommon Illness, mentioned in a ready assertion. “As a frontrunner in uncommon illnesses, Alexion is uniquely positioned to drive the superior growth and international commercialization of eneboparatide, which has the potential to cut back the customarily debilitating influence of low parathyroid hormone and the dangers of excessive doses keep away from calcium supplementation.”

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