BioMarin Pharmaceutical has agreed to acquire Alesta Therapeutics for $275 million upfront, plus up to $215 million in development and regulatory milestone payments, gaining rights to ALE1, an investigational oral therapy for hypophosphatasia.
The deal has been approved by the boards of both companies and is expected to close during the current quarter, subject to customary closing conditions. BioMarin will fund the transaction with cash on hand.
ALE1 is an orally active small molecule being developed for hypophosphatasia, or HPP, a rare inherited bone disease caused by mutations in the ALPL gene. The medicine is being evaluated in an ongoing Phase 1/2a trial measuring safety, tolerability and pharmacokinetic and pharmacodynamic effects in healthy volunteers and adults with HPP.
BioMarin said ALE1 has the potential to become the first oral treatment for HPP. The candidate is designed to target inorganic pyrophosphate, or PPi, a central disease metabolite. The company said systemic correction of PPi could potentially affect skeletal symptoms and broader manifestations of the disease.
HPP can impair bone and tooth mineralization, leading to frequent or easily occurring fractures, early tooth loss, muscle weakness, fatigue and pain. BioMarin said ALE1 could offer an oral alternative to injectable therapies currently available to people with the disease.
After closing, the program will become part of BioMarin’s Skeletal Conditions Business Unit. Chief Executive Officer Alexander Hardy said ALE1 strengthens BioMarin’s early-stage clinical pipeline and represents the type of opportunity the company is seeking as it aims to expand into larger rare-disease markets.
Prior to the transaction’s close, Alesta plans to spin out all assets other than ALE1 into a new entity. Alesta employees will transfer to that spinout, meaning no Alesta employees will join BioMarin under the deal’s terms.
Alesta Chief Executive Officer Ilan Ganot said BioMarin’s global reach and rare-disease development experience make it a suitable company to advance ALE1.
BioMarin said it expects to update its full-year 2026 guidance after the acquisition closes. Excluding the upfront payment, the deal is expected to have a modestly dilutive effect on the company’s 2026 financial results.
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