BioMarin Acquires Inozyme for $270M to Enhance Enzyme Expertise

BioMarin, a prominent biotech company known for its focus on enzyme therapies, recently announced its acquisition of Inozyme Pharma, a specialist in rare diseases, for $270 million. This strategic move aims to strengthen BioMarin's portfolio of enzyme therapies. BioMarin plans to buy each outstanding share of Inozyme for $4. The acquisition is expected to be completed in the third quarter, subject to standard closing conditions such as anti-trust clearance and the majority tender of Inozyme shares. Inozyme's board has unanimously advised its shareholders to tender their shares.

The acquisition's primary highlight is the investigational enzyme replacement therapy INZ-701, currently in advanced development stages for ENPP1 deficiency. ENPP1 deficiency is a rare, progressive condition resulting from mutations in the ENPP1 gene, affecting various organs and presenting differently among patients. Notable symptoms include early-onset osteoporosis, increased fracture risk, and tendon calcification. Individuals with this deficiency also face heightened risks for cardiovascular issues, severe rickets, and bone softening. Presently, there are no approved treatments for this condition.

INZ-701 is delivered subcutaneously and is designed to replace the deficient ENPP1 enzyme in the body. This therapy combines the active site of the ENPP1 enzyme with a specific fragment of a human antibody, which allows it to distribute throughout the body effectively. According to Inozyme’s website, preclinical studies have demonstrated the potential of INZ-701 to prevent abnormal mineralization, supporting its mechanism of action. In addition to addressing ENPP1 deficiency, Inozyme is also examining the efficacy of INZ-701 for other rare diseases like ABCC6 deficiency and calcific uremic arteriopathy.

The acquisition comes after a challenging period for BioMarin, marked by significant restructuring. Last year, the company reshaped its executive team, reduced its workforce by 225 employees, and discontinued four molecular projects as part of a wider reorganization strategy. Despite these challenges, BioMarin remains committed to evaluating both external and internal innovations as part of its corporate strategy. Alexander Hardy, BioMarin’s president and CEO, expressed confidence in the company's financial position, stating that they are well-equipped to acquire additional assets and accelerate the development of treatments for patients with unmet medical needs.

The deal with Inozyme follows BioMarin’s first-quarter earnings report, where Hardy indicated the company's intention to engage in at least one business development transaction this year. The company reported revenue of $745 million in the first quarter, reflecting a 15% increase compared to the previous year and surpassing analyst expectations of $738.1 million. A significant portion of BioMarin's revenue comes from its enzyme therapy portfolio, which contributed $484 million, an 8% increase from the previous year. Notably, Voxzogo, BioMarin's C-type natriuretic peptide analog for achondroplasia, saw a 40% increase year-on-year, generating $214 million.

BioMarin has started the year on a strong note and has reaffirmed its fiscal forecasts for 2025. The company projects total revenues to reach $3.1 billion, with Voxzogo expected to contribute at least $900 million. However, analysts at William Blair have expressed caution about BioMarin's prospects. In a note to investors dated May 2, the analysts highlighted uncertainties surrounding BioMarin’s current revenue generators and questioned their ability to continue driving significant value creation in the near term.