BioMarin (BMRN) Makes a $275 Million Bet on an Oral Treatment for Rare Bone Disease

BioMarin (BMRN) is adding another rare-disease program to its pipeline through the planned acquisition of Alesta Therapeutics.

The numbers:

• $275M upfront

• Up to $215M in additional development and regulatory milestones

• Up to $490M in total potential consideration

• 1 primary asset: ALE1

• >9,000 people diagnosed with HPP in the U.S.

Alesta's lead program, ALE1, is an investigational orally active small molecule being developed for hypophosphatasia (HPP)—a rare genetic bone disease caused by mutations in the ALPL gene. HPP can impair bone and tooth mineralization, resulting in frequent bone breaks and early tooth loss. In adults, the disease can also cause clinically significant muscle weakness, fatigue and pain.

ALE1 is designed to inhibit a novel target that regulates PPi levels. By lowering excess PPi, the goal is to restore healthier bone and mineral metabolism.

Early Indications:

ALE1 is currently in an ongoing Phase 1/2a clinical trial evaluating safety, tolerability and pharmacokinetics/pharmacodynamics in healthy volunteers and adults with HPP.

Before closing, Alesta will spin out all non-ALE1 assets into a separate company. Alesta's employees will transfer to that spinout, meaning no Alesta employees are expected to join BioMarin as part of the transaction.

BioMarin Reporting:

Just 12 days before announcing the transaction, BioMarin reported Q2 revenue: $990M, +20% YoY.

The company also raised its 2026 guidance for total revenue, VOXZOGO revenue and non-GAAP diluted EPS.