BioMarin Pharmaceutical has agreed to acquire Alesta Therapeutics in a deal aimed at adding a potential new treatment for hypophosphatasia (HPP), a rare genetic bone disease, to its pipeline.
The transaction will give BioMarin access to Alesta’s lead clinical-stage drug, ALE1. The companies said the deal has been approved by both boards and is expected to close in the third quarter of 2026, subject to customary closing conditions.
ALE1 is an oral small-molecule drug being developed as a potential treatment for HPP. The therapy is currently being tested in a Phase 1/2a clinical trial in healthy volunteers and adults with HPP. The study is evaluating its safety, tolerability, pharmacokinetics and pharmacodynamic effects.
HPP is caused by mutations in the ALPL gene and can affect the mineralization of bones and teeth. Patients may experience frequent bone fractures, early loss of teeth, muscle weakness, fatigue and pain. The disease can affect people of different ages and can have a major impact on daily life.
If successful, ALE1 could become the first oral treatment for HPP. The drug is designed to target PPi, or inorganic pyrophosphate, a key disease-related molecule. By addressing PPi levels throughout the body, BioMarin believes ALE1 could potentially improve both skeletal and other symptoms linked to HPP.
Following the acquisition, ALE1 will become part of BioMarin’s Skeletal Conditions business unit.
Under the terms of the agreement, BioMarin will pay Alesta shareholders $275 million upfront. An additional $215 million could be paid if certain development and regulatory milestones are achieved.
Before the acquisition closes, Alesta will separate its non-ALE1 assets into a new company. Alesta employees will move to that new entity, meaning no Alesta employees will join BioMarin as part of the transaction.
BioMarin plans to fund the acquisition using cash on hand. The company said it expects the deal to have a modest impact on its 2026 financial results, excluding the upfront payment. BioMarin also plans to update its full-year 2026 financial guidance after the transaction closes.
Alexander Hardy, President and CEO of BioMarin, said ALE1 fits the company’s focus on rare diseases and could provide an oral alternative to injectable treatments currently available for people with HPP.
Ilan Ganot, CEO of Alesta Therapeutics, said BioMarin’s experience in rare disease development and its global reach made it a strong partner for advancing ALE1.
The acquisition gives BioMarin another clinical-stage rare disease program as the company continues to expand its pipeline. The next key milestone for ALE1 will be the results from its ongoing Phase 1/2a clinical trial, which will provide further information on the drug’s safety and potential activity in HPP.