BioMarin has followed through on its shift to external innovation, inking a deal to buy Inozyme for $270 million to add a phase 3 enzyme replacement therapy to its pipeline.
California-based BioMarin signaled a change in its approach to business development last year. The company hired James Sabry, M.D., Ph.D., an ex-Roche dealmaker, as chief business officer and began considering opportunities beyond its traditional focus on early-stage deals. Now, the biopharma has landed on Inozyme as the first big acquisition of its new business development era.
Inozyme completed enrollment in a phase 3 trial of INZ-701 in January, putting it on track to post topline data early next year. The study is testing the enzyme replacement therapy in children with ENPP1 deficiency, a condition associated with increased cardiovascular mortality risk in infants.
Giving INZ-701 to people who are missing or have mutated forms of the enzyme could reverse the effects of pathologic mineralization, such as calcification of the arteries, tendons and ligaments. Inozyme has shared interim biomarker and anti-drug antibody data from the trial but has struggled to convince public investors, who sent its share price down 70% in the 12 months leading up to the BioMarin deal.
BioMarin has agreed to buy Inozyme for $4 a share, more than twice the biotech’s closing price before news of the deal emerged. The deal suggests BioMarin sees merits in the argument Inozyme's CEO Douglas Treco, Ph.D., put forward at a Needham event last month.
Treco admitted that the visibility of ENPP1 deficiency and awareness of the condition among physicians are low. But the CEO said investors are missing that “there's a predictive genetic prevalence” of the rare disease, adding that Inozyme is “finding patients and building a registry.” The ultimate test, Treco said, will be how well INZ-701 sells once it is on the market and “there's good diagnosis and awareness.”
Responsibility for driving diagnoses and awareness will fall on BioMarin, a biopharma with a long history of developing and selling enzyme replacement therapies such as Aldurazyme and Naglazyme. The decision to buy a molecule that aligns with a traditional stronghold of BioMarin fits with the company’s focus on acquiring assets in areas where it has “a strength and a right to win.”
BioMarin CEO Alexander Hardy, who delivered the “right to win” line on an earnings call this month, said in a statement Friday that the company “will continue to evaluate external innovation alongside internal innovation.” Hardy previously said BioMarin was aiming to do “at least one business development deal this year.”