BioMarin Pharmaceutical agreed to acquire Alesta Therapeutics for up to $490 million, gaining ALE1, a potential first oral therapy for hypophosphatasia (HPP). The deal includes $275 million upfront and up to $215 million in milestones, with closing expected this quarter.
BioMarin Pharmaceutical agreed to acquire Alesta Therapeutics for up to $490 million, gaining ALE1, an oral hypophosphatasia (HPP) therapy candidate.
"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," Alexander Hardy, president and CEO of BioMarin, said.
BioMarin will pay $275 million upfront and up to $215 million in development and regulatory milestones. The transaction, approved by both boards, is expected to close this quarter. Alesta will spin out all non-ALE1 assets into a separate company before closing, with Alesta employees transferring to the new entity.
HPP is a rare inherited metabolic disorder caused by mutations in the ALPL gene, leading to low alkaline phosphatase enzyme levels and a buildup of inorganic pyrophosphate that disrupts bone and tooth mineralization. Patients experience soft bones, frequent fractures, and tooth problems. More than 9,000 people in the US have been diagnosed with HPP, though the condition is believed to be underdiagnosed.
The only approved HPP therapy is AstraZeneca's Strensiq (asfotase alfa), an injectable enzyme replacement that generated about $1.7 billion in revenue in 2025. ALE1, an orally administered small molecule, works by lowering inorganic pyrophosphate levels. It is being evaluated in a Phase I/IIa trial (NCT07179640) with an enrollment goal of 120 healthy adults and adults with HPP. If approved, ALE1 would be the first pill-based therapy for the condition.
William Blair analysts said ALE1's combination of oral administration and differentiated biology could put it in an advantageous position if approved, though they noted Phase III development still needs to occur, making the deal a longer-term pipeline investment.
Ilan Ganot, CEO of Alesta, said BioMarin's global reach and expertise in rare disease drug development factored into the board's decision to approve the deal.
The acquisition marks BioMarin's second major pipeline move this year. In April, the biopharma completed its $4.8 billion acquisition of Amicus Therapeutics, gaining Fabry disease and Pompe disease assets. BioMarin currently has nine commercial therapies and will add ALE1 to its Skeletal Conditions Business Unit after the deal closes.
BioMarin shares rose 4.7 percent in premarket trading following the announcement. The company expects the acquisition, excluding the $275 million upfront payment, to have a modestly dilutive impact on 2026 financial results. Morgan Stanley served as BioMarin's financial advisor, with J.P. Morgan Securities advising Alesta.
The deal gives BioMarin a potential oral entrant in a rare disease market currently dominated by an injectable therapy, expanding its addressable patient population. Investors will watch for Phase II/III data from the ALE1 program as the next key milestone.
This article is for informational purposes only and does not constitute investment advice.
