BioMarin agreed to acquire Alesta Therapeutics’ lead program ALE1 in a deal valued at $275 million upfront with up to $490 million total, expanding BioMarin’s rare disease portfolio around hypophosphatasia (HPP). ALE1 is an orally active small molecule designed to address HPP by targeting inorganic pyrophosphate biology. ALE1 is in an ongoing Phase I/IIa trial (NCT07179640) evaluating safety, tolerability, and pharmacokinetics/pharmacodynamics in healthy volunteers and adults with HPP. BioMarin said the acquisition could create a large addressable patient opportunity and strengthen its early-stage clinical pipeline. The transaction received board approval from both companies, with Alesta planning to spin out all non-ALE1 assets into a new entity before closing. BioMarin will not directly absorb Alesta employees into the company as part of the restructuring, according to the deal structure.