BioMarin Pharmaceutical has announced that it is purchasing Alesta Therapeutics for the company’s clinical-stage asset, ALE1, to expand its rare diseases pipeline.
ALE1 is an orally-active, small molecule for the potential treatment of hypophosphastasia (HPP), a rare bone disease caused by mutations in the APLP gene. The therapy reduces excess inorganic pyrophosphate (PPi) by inhibiting a novel target that regulates PPi. The first of its kind, ALE1 is currently undergoing a phase 1/2a clinical trial.
Through the definitive agreement, BioMarin will pay $275 million upfront plus up to $215 million in additional payments upon reaching developmental and regulatory milestones related to ALE1. All non-ALE1 assets will spin out to a new entity, and Alesta employees will transfer to this new entity.