BioMarin Pharmaceutical has announced its acquisition of Alesta Therapeutics for $275 million upfront, gaining access to Alesta’s oral treatment for hypophosphatasia, a rare genetic bone disease. The deal also includes potential milestone payments of up to $215 million.
The acquired treatment, ALE1, is currently in early-stage clinical trials and has the potential to be the first oral treatment for hypophosphatasia, or HPP. This move strengthens BioMarin’s focus on rare skeletal conditions, building on its existing portfolio anchored by the drug Voxzogo.
BioMarin’s president and CEO, Alexander Hardy, stated that this acquisition is an opportunity to address a significant unmet need, allowing the company to compete in larger rare disease markets. He emphasized the company’s plan to continue seeking similar opportunities to drive growth through clinical-stage innovation.
The acquisition marks BioMarin’s third major deal in the rare disease space over the past year, following the $4.8 billion deal to buy Amicus Therapeutics announced last December. This latest move demonstrates the company’s commitment to expanding its portfolio of rare disease treatments, with rare disease investors likely to take notice.
More than 9,000 people in the United States have been diagnosed with HPP, although the true number of people impacted by the disease is likely higher. The acquisition of Alesta Therapeutics brings BioMarin closer to addressing this unmet need, with ALE1 potentially offering a new treatment option for patients with HPP worldwide.
Ilan Ganot, Alesta’s CEO, noted that BioMarin’s global reach, scale, and expertise in rare disease drug development made it an ideal partner to advance ALE1. They expect the deal to close this quarter, pending regulatory approvals.
In practice, this development could mean significant improvements in the quality of life for patients with HPP, who currently have limited treatment options. As BioMarin continues to expand its portfolio of rare disease treatments, it may be well-positioned to make a meaningful impact on the lives of patients with these conditions.
The acquisition of Alesta Therapeutics is subject to customary closing conditions and is expected to be completed by the end of the quarter. Once the deal is finalized, BioMarin will have access to ALE1, which will be integrated into its existing portfolio of rare disease treatments.
Alesta’s treatment will be advanced through clinical trials and regulatory approvals. With its strong track record in rare disease drug development, BioMarin may be well-positioned to bring this promising treatment to market and make a meaningful difference in the lives of patients with HPP.
It is a significant step forward.
