NewsHK
The rare disease drug market has long run on injectable therapies, a delivery format patients tolerate but rarely prefer.
Against that backdrop, BioMarin Pharmaceutical announced Tuesday it has entered a definitive agreement to acquire Alesta Therapeutics for $275 million upfront, with up to $215 million in additional payments tied to development and regulatory milestones.
The target asset is ALE1, an orally active small molecule designed to treat hypophosphatasia, or HPP.
HPP is a rare genetic disorder caused by mutations in the ALPL gene, impairing bone and tooth mineralization and, in adults, contributing to muscle weakness, fatigue, and pain.
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