Major rare disease player, AstraZeneca, has acquired privately-held French biotech, Amolyt Pharma, for an amount of up to US$1.05 bn (A$1.6 bn), consisting of US$800 m upfront with an additional contingent payment of US$250 m payable upon achievement of a specified regulatory milestone.
Amolyt’s key asset is eneboparatide (AZP-3601), an investigational therapeutic peptide with a novel mechanism of action which is currently in Phase 3 development as a treatment for chronic hypoparathyroidism. The study is due to complete at the end of this year. Amolyt’s other asset, a potential treatment for acromegaly, commenced Phase 1 clinical development mid-last year.
Hypoparathyroidism patients suffer from a deficiency in parathyroid hormone production, resulting in significant dysregulation of calcium and phosphate which can lead to serious debilitating diseases, including chronic kidney disease. While there are available treatments, they don’t address the underlying hormone deficiency.
Hypoparathyroidism affects approximately 115,000 people in the United States.
By way of comparison, ADPKD is estimated to affect approximately 300,000 people in the United States, with 80-85% of cases caused by mutation in the PKD1 gene, which is the target of PYC-003. As ADPKD is a highly debilitating and potentially lethal disease of unmet need, there is the potential for accelerated FDA approval, using a surrogate endpoint, following Phase 2.
Amolyt has moved from a start-up with preclinical assets to a Phase 3 company in less than five years. It has had little trouble raising funds - its largest investor is Novo Holdings, the controlling shareholder of Novo Nordisk.
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