BioMarin snaps up Alesta's lead asset for $275M

BioMarin Pharmaceuticals has snapped up another small rare disease player, buying Alesta Therapeutics for $275 million upfront, plus up to $215 million in potential milestone payoffs.

Although structured as an acquisition, the end result is more like an asset purchase. That's because, prior to the deal closing, Alesta's team will spin out all assets except lead asset ALE1 into a new company, bringing the whole Alesta team along with them.

ALE1 is an oral small molecule under development to treat hypophosphatasia, a rare bone disease caused by mutations in the ALPL gene. Currently in phase 1/2a, the drug has the potential to be the first oral therapy for HPP.

"This is exactly the kind of opportunity to address a significant unmet need that lets us compete in larger rare disease markets – adding an asset that has the potential to reach our largest addressable patient population," Alexander Hardy, president and CEO of BioMarin, said in a statement. "We plan to continue to seek these kinds of opportunities as we focus on clinical-stage innovation to drive durable growth for BioMarin."

Alesta Therapeutics is a fairly new biotech -- the company has only raised a $65 million Series A to date. The company has not shared details of its pipeline beyond ALE1, but it did say in a release in January that it was pursuing a programme in "a major unment need" that would "benefit from similarities to ALE1, including similar clinical and regulatory pathways."

BioMarin's last acquisition was a significantly bigger deal, its $4.8 billion deal to buy Amicus Therapeutics, which closed in April.

The company ran into some trouble this past March when it had to suspend some phase 2 label expansion trials for its achondroplasia drug Voxzogo. 

But Voxzogo continues to do well in its existing indications, with the FDA approving the company's supplemental NDA for the drug just last month. 

BioMarin recently released its Q2 financials, reporting a 20% YoY revenue increase for a quarterly total revenue of $990 million, primarily driven by therapies brought in as part of the Amicus acquisition.