BioMarin Pharmaceutical has agreed to buy Alesta Therapeutics for $275 million upfront, positioning the biopharma to challenge AstraZeneca for a rare bone disease market.
Alesta is developing a small molecule to treat hypophosphatasia (HPP), a rare genetic bone disease. The pediatric HPP market is already served by AstraZeneca’s enzyme replacement therapy (ERT) Strensiq, and the Big Pharma has tipped its next-generation asset to generate blockbuster sales. Yet the limitations of Strensiq and ERT more broadly leave a potential opportunity open to competitors.
BioMarin has identified Alesta’s lead asset, ALE1, as a molecule capable of seizing this opportunity. ALE1 is an oral drug candidate against a target that regulates levels of inorganic pyrophosphate (PPi). Lowering excess PPi could restore bone and mineral metabolism in people with HPP.
Alesta began a phase 1/2 trial of the drug candidate in September. The study is assessing ALE1 in healthy volunteers and adults with HPP.
The focus on adults positions ALE1 to address a currently underserved part of the HPP market. Strensiq is only FDA-approved in infantile- and juvenile-onset HPP, and a phase 3 trial of AstraZeneca’s next-generation ERT in people 12 years and older missed its primary endpoint.
Unlocking the adult HPP market is one of several ways that BioMarin could turn ALE1 into a commercial success. BioMarin could also challenge AstraZeneca directly, tapping into the potential convenience and cost advantages of small molecules over ERTs to win market share. Patients take Strensiq subcutaneously three or six times a week, while AstraZeneca’s next-generation ERT is administered every two weeks.
BioMarin is paying the upfront fee and up to $215 million in milestones for ALE1. Alesta will spin out all non-ALE1 assets to a new entity staffed by its current employees. No Alesta employees are transferring to BioMarin.
The deal strengthens BioMarin’s pipeline following the failure of an earlier attempt to expand through acquisition. BioMarin recently ended development of an ERT for ENPP1 deficiency that it acquired last year through the $270 million takeover of Inozyme. The termination followed the failure of a phase 3 trial.
ALE1 will slot into the skeletal condition unit that BioMarin created when it revised its strategy in 2024. The unit features Voxzogo, an approved treatment for a cause of dwarfism, and clinical candidates for a clutch of skeletal diseases.