India, Aug. 18 -- BioMarin Pharmaceutical Inc. (BMRN) has entered into a definitive agreement to acquire Alesta Therapeutics, securing rights to its lead clinical-stage candidate ALE1.
ALE1 is an orally active, small molecule being developed for hypophosphatasia (HPP), a rare genetic bone disease caused by mutations in the ALPL gene. The therapy is currently in a Phase 1/2a trial evaluating safety, tolerability and pharmacokinetics/pharmacodynamics in healthy volunteers and adults with HPP.
If approved, ALE1 could become the first oral treatment for HPP, targeting inorganic pyrophosphate (PPi) to address skeletal and systemic manifestations of the disease.
Under the terms of the deal, BioMarin will pay $275 million upfront, with addition...