BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) has agreed to acquire Alesta Therapeutics in a deal worth up to $490 million, adding a clinical-stage oral therapy for hypophosphatasia to its rare disease pipeline and expanding its investment in skeletal conditions
BioMarin will pay Alesta shareholders $275 million upfront and up to an additional $215 million tied to development and regulatory milestones. The San Rafael, California-based biotechnology company plans to fund the acquisition with cash on hand
The transaction has been approved by both companies’ boards and is expected to close this quarter, subject to customary closing conditions. Before completion, Alesta will spin out all assets other than ALE1, its lead clinical program, into a separate company. Alesta employees will transfer to the new entity, meaning none will join BioMarin as part of the acquisition
The structure makes ALE1 the central strategic rationale for the transaction. The orally active small molecule is being developed for hypophosphatasia, or HPP, a rare genetic bone disease caused by mutations in the ALPL gene. It is currently being evaluated in a Phase 1/2a trial involving <a href="https://healthylife7.com/physician-kirk-bass-to-speak-on-healthy-living-at-oak-ridge-meeting/” title=”Physician Kirk Bass to speak on healthy living at Oak Ridge meeting”>healthy volunteers and adults with HPP
If successfully developed and approved, BioMarin expects ALE1 could become the first oral therapy for HPP. Existing treatment options include injectable therapies, giving an oral medicine the potential to provide an alternative approach for patients while expanding BioMarin’s presence in a rare disease market with significant unmet need
“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,” President and CEO Alexander Hardy said
ALE1 is designed to inhibit a target that regulates inorganic pyrophosphate, or PPi, a metabolite central to HPP pathology. By reducing excess PPi, the therapy is intended to address abnormal bone and mineral metabolism and potentially affect both skeletal and broader manifestations of the disease
HPP can interfere with bone and tooth mineralization and can result in frequent or easily occurring fractures and early tooth loss. Adults can also experience muscle weakness, fatigue and pain. More than 9,000 people have been diagnosed with HPP in the United States, although the condition can be difficult to identify because its symptoms can resemble more common disorders
Following completion of the acquisition, ALE1 will become part of BioMarin’s Skeletal Conditions Business Unit. The company views the program as an opportunity to move into a larger rare disease market while adding a clinical-stage asset with the potential to address what BioMarin describes as its largest addressable patient population
The deal also reflects BioMarin’s broader capital allocation strategy around clinical-stage innovation. Hardy said the company intends to continue pursuing assets that can address substantial unmet needs while contributing to durable growth
Alesta CEO Ilan Ganot said BioMarin’s global reach and experience developing rare disease therapies were important considerations in selecting an acquirer for ALE1. The transaction gives the program access to BioMarin’s clinical development and commercialization infrastructure while allowing Alesta’s remaining programs and employees to continue independently through the planned spinout
BioMarin currently has nine commercial therapies along with clinical and preclinical development programs focused on genetically defined diseases. Founded in 1997, the company has built its business around developing medicines for rare conditions where biological understanding can support targeted therapeutic approaches
BioMarin expects the acquisition, excluding the $275 million upfront consideration, to have a modestly dilutive impact on its 2026 financial results. The company plans to update its full-year guidance after the transaction closes
Morgan Stanley is serving as BioMarin’s exclusive financial advisor, with Jones Day providing legal counsel. J.P. Morgan Securities is Alesta’s exclusive financial advisor, while Goodwin Procter and NautaDutilh are serving as its legal counsel
The acquisition represents a targeted deployment of BioMarin’s balance sheet toward a single clinical-stage program rather than a broader corporate combination. If ALE1 progresses successfully, the deal would give BioMarin a potential oral entrant in HPP while strengthening a skeletal disease portfolio that the company sees as an important component of its next phase of rare disease growth
