BioMarin Pharmaceutical has outlaid $490m to acquire rare disease drug developer Alesta Therapeutics, though it plans to keep only one asset from the biotech’s pipeline.
BioMarin has agreed to buy Alesta to gain access to ALE1, the company’s lead clinical-stage asset being developed for rare genetic bone disease hypophosphatasia (HPP). As per the deal’s terms, BioMarin will pay $275m upfront and up to $215m in development and regulatory milestones.
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Alesta plans to spin out all non-ALE1 assets prior to the close of the transaction, which is expected this quarter. The biotech has neither disclosed the rest of its pipeline nor R&D unrelated to HPP. Meanwhile, BioMarin will add ALE1 to its Skeletal Conditions Business Unit following the deal’s close.
HPP is a rare, inherited metabolic disorder caused by mutations in the ALPL gene. It is charactered by low levels of the alkaline phosphatase (ALP) enzyme causing a buildup of inorganic pyrophosphate, disrupting bone and tooth mineralisation. This means that patients have soft bones, leading to frequent fractures and tooth problems.
ALE1, an oral administered small molecule drug, works by lowering levels of inorganic pyrophosphate. It is being evaluated in an ongoing Phase I/IIa clinical trial (NCT07179640), with an enrolment goal of 120 healthy adults and adults with HPP.
The only approved medication for HPP is AstraZeneca’s blockbuster Strensiq (asfotase alfa), an enzyme replacement therapy administered via an injection. If approved, ALE1 would be the first therapy that is both pill-based and that targets inorganic pyrophosphate.
In a research note, William Blair analysts said: “We view ALE1 as a logical addition to BioMarin’s skeletal disease franchise and believe the asset could create meaningful long-term value for the company, particularly given that AstraZeneca’s Strensiq generated about $1.7bn of revenue in 2025 as the only approved therapy for HPP.”
The analysts added that ALE1’s combination of oral administration and differentiated biology could put it in an advantageous position if approved. However, they caveated that Phase III development needs to occur, meaning the deal is a longer-term pipeline investment.
The Alesta deal marks the second pipeline reinforcement for BioMarin so far this year. In April, the biopharma completed its $4.8bn acquisition of Amicus Therapeutics. The transaction gave BioMarin access to Fabry disease and Pompe disease assets.
Commenting on the Alesta deal, BioMarin’s CEO Alexander Hardy called ALE1 a “strong strategic fit”.
“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,” Hardy commented.
