Dive Brief:
- BioMarin Pharmaceutical is boosting its pipeline of experimental drugs for skeletal and muscle diseases, announcing Tuesday the acquisition of privately held Alesta Therapeutics and its therapy for a rare bone-wasting disease.
- Per deal terms, BioMarin will pay $275 million up front and potentially as much as $215 million more based on the achievement of certain development and regulatory milestones. Before the completion of the deal, which is expected by the end of September, Alesta will spin out a separate company with all of its employees to develop another unnamed drug.
- Alesta’s lead candidate, called ALE1, is in a Phase 1/2 trial for a genetic disease called hypophosphatasia, which affects bone mineralization and can result in fractures, tooth loss and muscle weakness. ALE1 “has the potential to reach our largest addressable patient population,” said BioMarin CEO Alexander Hardy in a statement.
Dive Insight:
The acquisition is BioMarin’s third in the past two years, following the $4.8 billlion takeout of Amicus Therapeutics and the more modest $270 million purchase of Inozyme Pharma. With Amicus, BioMarin gained a slate of marketed drugs for rare metabolic diseases, whereas Inozyme offered drug for an uncommon musculoskeletal condition that has since seen a setback.
The company is in the market for more. “We plan to continue to seek these kinds of opportunities as we focus on clinical-stage innovation to drive durable growth for BioMarin,” Hardy said.
The Alesta deal will be funded with company cash on hand, BioMarin said, and its shareholders will take a per-share earnings hit because of the cost of the transaction. It plans to provide updated guidance after the transaction closes. In its second quarter earnings, BioMarin estimated full-year per-share earnings of between $4.90 and $5.10.
ALE1 will join a portfolio of muscle and bone disease drugs that includes Voxzogo, a treatment that BioMarin is positioning for various types of dwarfism; a Voxzogo follow-on called BMN 333; and BMN 351, an oligonucleotide therapy for Duchenne muscular dystrophy. Alesta’s experimental drug acts on a “novel target” that lowers levels of inorganic pyrophosphate, a metabolite at the center of hypophosphatasia.
The Phase 1/2 trial that is underway tests ALE1’s safety and biological activity in healthy volunteers and patients.
Netherlands-based Alesta closed a 65 million euro Series A round in early 2025 led by Frazier Life Sciences and Droia Ventures, with Novartis’ venture arm one of the other participants. As for the remaining pipeline that will be developed by the spinout company, Alesta hasn’t disclosed much beyond stating it is a “second therapeutic candidate for another large indication with a major unmet need” at the J.P. Morgan Healthcare Conference in January 2026.
