Acquisition adds ALE1, a potential first oral therapy for hypophosphatasia to clinical pipeline

SAN RAFAEL, Calif., Sept. 1, 2026 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) said today that it completed the previously announced agreement to acquire Alesta Therapeutics. The acquisition will strengthen BioMarin's skeletal conditions portfolio, adding ALE1, an oral small molecule for the potential treatment of hypophosphatasia (HPP), a rare genetic bone disease caused by mutations in the ALPL gene. ALE1 is currently being evaluated in an ongoing Phase 1/2a clinical trial assessing safety, tolerability and pharmacokinetics/pharmacodynamics in healthy volunteers and adults with HPP.

On August 18, BioMarin announced it had entered into a definitive agreement to acquire Alesta Therapeutics to gain Alesta's lead clinical-stage asset, ALE1. Prior to the close of the transaction, Alesta spun out all non-ALE1 assets to a new entity. Alesta Therapeutics is now a wholly owned subsidiary of BioMarin.

About BioMarin

BioMarin is a leading, global rare disease biotechnology company focused on delivering medicines for people living with genetically defined conditions. Founded in 1997, the San Rafael, California-based company has a proven track record of innovation, with nine commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, BioMarin seeks to unleash the full potential of genetic science by pursuing category-defining medicines that have a profound impact on patients.

To learn more, please visit www.biomarin.com.

About Alesta Therapeutics

Alesta Therapeutics is a clinical-stage biotechnology company committed to developing novel oral small-molecule therapies for underserved diseases. The company's lead asset, ALE1, is being developed for hypophosphatasia (HPP), a rare genetic disorder with significant unmet need.

For more information, visit www.alestatherapeutics.com.

Forward-Looking Statements

This press release contains forward-looking statements about, among other things, the acquisition of ALE1, the lead clinical-stage asset, of Alesta Therapeutics (Alesta) by BioMarin Pharmaceutical Inc. (BioMarin) and the business prospects of BioMarin, including, without limitation, statements about: the prospective benefits of the acquisition, including expectations that it will strengthen BioMarin's skeletal conditions portfolio; expectations regarding ALE1 and its ongoing development, including its potential to be a first-in-class oral therapy for the treatment of hypophosphatasia (HPP); and other statements that are not historical facts.

These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: the effects of the acquisition (or the announcement thereof) on BioMarin's stock price and/or BioMarin's operating results; unknown or inestimable liabilities; BioMarin's ability to successfully develop, launch and commercialize products and product candidates such as ALE1, if approved; BioMarin's ability to realize the anticipated benefits of the acquisition, including the possibility that the expected benefits from the acquisition will not be realized or will not be realized within the expected time period and that integration will not be successful or that such integration may be more difficult, time-consuming or costly than expected; the time-consuming and uncertain regulatory approval process for pharmaceutical product development; the costly and time-consuming pharmaceutical product development process and the uncertainty of clinical success, including risks related to failure or delays in successfully initiating or completing clinical trials and assessing patients, including with respect to current and planned future clinical trials; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission, including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended June 30, 2026, as such factors may be updated by any subsequent reports. Investors are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.

BioMarin^® is a registered trademark of BioMarin Pharmaceutical Inc. or its affiliates.

Contacts:

Investors	Media
Traci McCarty	Erin Rau
BioMarin Pharmaceutical Inc.	BioMarin Pharmaceutical Inc.
(415) 455-7558	(925) 683-9622

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SOURCE BioMarin Pharmaceutical Inc.