On Aug. 18, 2026, BioMarin Pharmaceutical announced a definitive agreement to acquire Alesta Therapeutics to gain Alesta’s lead clinical-stage asset, ALE1. 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 this quarter, subject to customary closing conditions.

ALE1 is an orally active, small molecule for the potential treatment of hypophosphatasia (HPP), a rare genetic bone disease caused by mutations in the ALPL gene. ALE1 has the potential to be the first oral therapy for HPP and is currently being evaluated in an ongoing Phase 1/2a clinical trial assessing safety, tolerability and pharmacokinetics/pharmacodynamics in healthy volunteers and adults with HPP.

HPP is a serious condition that can affect bone and tooth mineralization, resulting in easy or frequent bone breaks, early tooth loss, and, in adults, clinically significant muscle weakness, fatigue and pain. If approved, ALE1 is expected to be the first oral therapy approach that targets the central disease metabolite, PPi (inorganic pyrophosphate), with the potential to impact both skeletal and broader manifestations of HPP through systemic correction of disease biology. The program will become part of BioMarin’s Skeletal Conditions Business Unit following close.

Under the terms of 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. BioMarin intends to fund the transaction with cash on hand. BioMarin expects to provide updated full-year 2026 guidance reflecting the acquisition of Alesta following the closing of the transaction. Excluding the upfront consideration, the transaction is expected to have a modestly dilutive impact on 2026 financial results.

ALE1 is designed to inhibit a novel target that regulates levels of inorganic pyrophosphate (PPi), the metabolite at the center of HPP pathology. By lowering excess PPi, ALE1 aims to restore healthier bone and mineral metabolism across the full spectrum of HPP. More than 9,000 people have been diagnosed with HPP in the U.S., however, the disease is often underdiagnosed due to a broad spectrum of symptoms that can mimic more common conditions.