BioMarin Pharmaceutical, a San Rafael, California-based bio/pharmaceutical company focused on rare diseases, has agreed to acquire Alesta Therapeutics, a Leiden, the Netherlands-based clinical-stage bio/pharmaceutical company developing small-molecule drugs for rare diseases, in a deal worth up to $490 million ($275 million upfront and $215 million in milestone payments).  

Alesta’s lead asset is ALE1, an orally active small molecule for the potential treatment of hypophosphatasia (HPP), a rare genetic bone disease caused by mutations in the ALPL gene. The ALPL gene provides instructions for making tissue-nonspecific alkaline phosphatase, an enzyme that plays an important role in the growth and development of bones and teeth. ALE1 has the potential to be the first oral therapy for HPP and is currently being evaluated in an ongoing Phase I/IIa clinical trial. 

Under the agreement, BioMarin will acquire Alesta, and Alesta shareholders will receive an upfront payment of $275 million plus up to $215 million in subsequent payments upon achievement of certain development and regulatory milestones. Additionally, immediately prior to the close of BioMarin’s acquisition of Alesta, Alesta will spin out all non-ALE1 assets to a new entity and former Alesta employees will transfer to the spinout entity. As a result, no Alesta employees will become employees of BioMarin in connection with the transaction. 

Alesta plans to spin out all non-ALE1 assets prior to the close of the transaction, which has been approved by the Boards of Directors of both companies and is expected to be completed in the third quarter of 2026, subject to customary closing conditions. 

Source: BioMarin Pharmaceutical