AstraZeneca is bolstering its uncommon illness pipeline with a deal to purchase Amolyt Pharma, a biotech firm in late-stage growth with a remedy designed to deal with a uncommon hormone deficiency whose solely FDA-approved therapy will quickly stop manufacturing.
In line with deal phrases introduced Thursday, AstraZeneca is paying Amolyt shareholders $800 million up entrance. One other $250 million is tied to the achievement of a regulatory milestone that was not disclosed. Contemplating that the drug is in Section 3 testing, that milestone may very well be a regulatory approval.
Lyon, France-based Amolyt is growing a remedy for hypoparathyroidism, a illness during which the parathyroid glands don’t produce sufficient parathyroid hormone. This hormone is necessary for regulating calcium and phosphate ranges within the blood. Illness signs embody tingling or burning sensations within the fingertips and toes, muscle aches or cramps, muscle twitching or spasms, and fatigue.
Hypoparathyroidism disproportionately impacts ladies. Calcium and vitamin D dietary supplements may also help, however at excessive doses, calcium dietary supplements can result in gastrointestinal issues equivalent to constipation. When dietary supplements alone aren’t sufficient to handle hypoparathyroidism, the one FDA-approved remedy is Natpara, an engineered model of parathyroid hormone offered by Takeda Pharmaceutical.
Amolyt may deliver sufferers a special solution to handle 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 Section 2 outcomes reported in 2022, Amolyt mentioned its drug led to regular calcium ranges within the blood in addition to the potential to eradicate the necessity for each day calcium and vitamin D dietary supplements. Moreover, the drug preserved bone mineral density, which is necessary for sufferers at larger threat of growing bone issues equivalent to osteopenia or osteoporosis. Amolyt has mentioned it expects preliminary Section 3 information will grow to be accessible by the top of 2024.
The market want for brand new hypoparathyroidism therapies is turning into pressing. Takeda’s Natpara (Natpar exterior of the U.S.) has had a rocky commercialization historical past since its FDA approval in 2015. In 2019, the FDA issued a recall as a consequence of potential contamination from tiny rubber particles from the cartridge housing the injectable product. Takeda’s efforts to resolve the issue to the FDA’s satisfaction have been unsuccessful. In late 2022, Takeda mentioned it deliberate to discontinue manufacturing of Natpara/Natpar by the top of 2024. After that, the corporate mentioned it will provide the remaining doses of the drug till stock is depleted or the product is expired.
The Amolyt acquisition brings AstraZeneca into a gaggle of corporations vying to fill the hypoparathyroidism therapy want that might be left when Takeda’s drug is now not accessible. An FDA choice is anticipated in Could for Ascendis Pharma’s peptide drug Transcon PTH. Prolong Biosciences is in Section 1 testing with a drug derived from parathyroid hormone. MBX Biosciences has reached Section 2 testing with a peptide drug. These drug candidates are injectable. Septerna is growing an oral drug for hypoparathyroidism. Final yr, the South San Francisco-based startup raised $150 million in financing to advance the small molecule to Section 1 testing.
Amolyt final raised cash in early 2023, a €130 million (about $138 million) Collection C financing to assist its pipeline. In addition to the hypoparathyroidism program, that pipeline contains AZP-3813, a possible therapy for acromegaly, a illness during which the physique produces an excessive amount of progress hormone. Section 1 testing started final summer time. Amolyt has mentioned it expects preliminary information within the first half of 2024.
AstraZeneca expects to finish the Amolyt acquisition by the top of the third quarter of this yr. When the deal closes, Amolyt will grow to be a part of Alexion, AstraZeneca Uncommon Illness, the pharmaceutical big’s Boston-based uncommon illness division. AstraZeneca mentioned the acquisition will allow it to increase its uncommon illness analysis into endocrinology.
“Persistent hypoparathyroid sufferers face a major want for a substitute for present supportive therapies, which don’t deal with the underlying hormone deficiency,” Marc Dunoyer, CEO of Alexion, AstraZeneca Uncommon Illness, mentioned in a ready assertion. “As leaders in uncommon illness, Alexion is uniquely positioned to drive the late-stage growth and world commercialization of eneboparatide, which has the potential to minimize the usually debilitating influence of low parathyroid hormone and keep away from the dangers of high-dose calcium supplementation.”
Picture: Christopher Furlong, Getty Photos
