BioMarin Pharmaceutical acquired Alesta Therapeutics for $275 million upfront, gaining the right to develop ALE1, an oral drug candidate for hypophosphatasia, a rare genetic disorder that impairs bone mineralization.
The deal includes up to $215 million in milestone payments tied to clinical and regulatory progress, according to the company's filing. Hypophosphatasia has limited treatment options; existing therapies require enzyme replacement infusions or subcutaneous administration, making an oral formulation a potential clinical advance for patients who cannot tolerate or access injected regimens.
ALE1 is currently in Phase 1/2a clinical testing. BioMarin, which focuses on genetic and metabolic rare diseases, has a portfolio including Palforzia for peanut allergy and Voxelotor for sickle cell disease. The company uses acquisitions to expand its pipeline; the Alesta deal follows its 2024 purchase of Reata Pharmaceuticals for $1.15 billion to acquire Fabhalta, a therapy for immunoglobulin A nephropathy. Hypophosphatasia affects approximately 1 in 100,000 people globally, though many cases go undiagnosed.
BioMarin closed the transaction on August 17, 2026, and announced it August 18. The upfront payment to Alesta represents 58 percent of the total deal value, with the remainder contingent on advancing ALE1 through regulatory milestones. Neither company disclosed the timing for Phase 2b expansion or anticipated regulatory filings.
The deal transfers early-stage oral therapy programs to a company with established manufacturing and regulatory infrastructure for rare disease drugs. BioMarin's existing route to market for orphan indications reduces the commercial risk associated with bringing a first-in-class oral agent to patients accustomed to enzyme therapy. The acquisition adds one more program to BioMarin's pipeline of candidates in Phase 2 or earlier, a stage where clinical and regulatory risk remains material.
