BioMarin has entered into 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 mineralisation, 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.

Alexander Hardy, President and Chief Executive Officer (CEO), BioMarin, said, "ALE1 is a strong strategic fit for BioMarin, bringing a potential oral alternative to the injectable therapies available today for people living with HPP around the world while meaningfully strengthening our early-stage clinical pipeline. This is exactly the kind of opportunity to address a significant unmet need that lets us compete in larger rare disease markets–adding an asset that has the potential to reach our largest addressable patient population. We plan to continue to seek these kinds of opportunities as we focus on clinical-stage innovation to drive durable growth for BioMarin."

Under the terms of the agreement, BioMarin will acquire Alesta and Alesta shareholders will receive an upfront payment of USD 275 million plus up to USD 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. It 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.

Ilan Ganot, CEO, Alesta Therapeutics, said, "We chose to partner with BioMarin due to their deep commitment to people living with rare diseases. BioMarin's global reach, scale, and proven expertise in rare disease drug development make it an ideal partner to advance ALE1 and realise its potential as a promising treatment for patients with HPP worldwide. This acquisition is also a testament to the extraordinary work, scientific expertise, and drug development capabilities of the Alesta team."