BioMarin agreed to acquire Alesta Therapeutics in a transaction valued at $275 million upfront, plus up to $215 million in development and regulatory milestones, for Alesta’s lead candidate ALE1. ALE1 is an orally active small molecule intended to treat hypophosphatasia (HPP), a rare genetic bone disease caused by ALPL mutations. BioMarin characterized ALE1 as a strategic fit to add a potential first-in-class oral option to an HPP landscape currently dominated by injectable approaches. The program is in an ongoing Phase I/IIa study (NCT07179640), evaluating safety, tolerability, and pharmacokinetics/pharmacodynamics in adults and healthy volunteers. The deal includes a spinout of non-ALE1 assets to a new entity prior to closing, with former Alesta employees transferring to the spinout rather than BioMarin, according to the companies’ structure for the transaction.