SAN RAFAEL, Calif., August 18, 2026

BioMarin Pharmaceutical Inc. has entered into a definitive agreement to acquire Alesta Therapeutics, gaining access to ALE1, an investigational oral small-molecule therapy being developed for hypophosphatasia (HPP), a rare genetic disorder affecting bone and tooth mineralization. The transaction is expected to strengthen BioMarin’s rare disease pipeline while adding a potential oral treatment approach for patients with significant unmet medical needs.

BioMarin Plans $275 Million Upfront Acquisition

Under the proposed transaction, BioMarin will pay $275 million upfront to Alesta shareholders, with the potential for an additional $215 million in development and regulatory milestone payments. BioMarin plans to fund the acquisition using cash on hand, and the transaction is expected to close during the third quarter of 2026, subject to customary closing conditions. Before the acquisition closes, Alesta plans to separate all assets other than ALE1 into a newly formed entity. Alesta employees will also transfer to the spinout company, meaning no Alesta employees are expected to join BioMarin through the transaction. The acquisition has been approved by the boards of directors of both companies. The deal represents a strategic expansion for BioMarin, which focuses on therapies for genetically defined rare diseases. Following completion, ALE1 will become part of BioMarin’s Skeletal Conditions Business Unit and will add a clinical-stage program to the company’s development pipeline.

ALE1 Targets Hypophosphatasia Disease Biology

ALE1 is an orally active small molecule designed to inhibit a novel target regulating levels of inorganic pyrophosphate, or PPi, a metabolite central to HPP disease biology. By lowering excess PPi, the investigational therapy is intended to help restore healthier bone and mineral metabolism across the spectrum of HPP. HPP is caused by mutations in the ALPL gene and can interfere with normal mineralization of bones and teeth. The disease can cause frequent or easy bone fractures, premature tooth loss and, in adults, symptoms such as muscle weakness, fatigue and pain. More than 9,000 people have been diagnosed with HPP in the United States, although the condition is frequently underdiagnosed because its symptoms can resemble more common diseases. ALE1 is currently being evaluated in an ongoing Phase 1/2a clinical trial involving healthy volunteers and adults with HPP. The study is assessing safety, tolerability and pharmacokinetic and pharmacodynamic characteristics. If successfully developed and approved, ALE1 could potentially become the first oral therapy for HPP, offering an alternative to injectable treatment approaches.

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Acquisition Strengthens BioMarin Rare Disease Pipeline

BioMarin said ALE1 provides a strong strategic fit with its focus on rare diseases and could expand the company’s presence in a larger rare disease market. The company plans to continue pursuing clinical-stage opportunities that address significant unmet medical needs and strengthen its long-term development pipeline. For Alesta, the transaction brings the potential to advance ALE1 through later-stage development and toward global commercialization using BioMarin’s experience and international infrastructure in rare disease drug development. Alesta CEO Ilan Ganot said BioMarin’s expertise and global reach make it a suitable partner for advancing the investigational therapy for people living with HPP. The acquisition remains subject to closing conditions, and ALE1 has not been approved for marketing. Its potential clinical benefits will depend on results from ongoing and future clinical studies as well as regulatory review. BioMarin expects to provide updated 2026 financial guidance after the transaction closes.

Source: BioMarin, Alesta Therapeutics press relese