"We are grateful to the FDA for their recognition of the urgent need for new therapies that could offer a significant improvement in the treatment of Hunter syndrome, as reflected by their priority review designation for our Biologics License Application for tividenofusp alfa," said Carole Ho, M.D., Chief Medical Officer and Head of Development of Denali Therapeutics. "If FDA-approved, tividenofusp alfa would mark the first significant advancement in nearly two decades for enzyme replacement therapy for ind ...

Core Viewpoint - BioMarin Pharmaceutical Inc. has announced the acquisition of Inozyme Pharma, Inc. for $4.00 per share, totaling approximately $270 million, which is expected to enhance BioMarin's enzyme therapies portfolio and provide a potential first-in-class treatment for ENPP1 Deficiency [1][2][3] Acquisition Details - The acquisition is an all-cash transaction, unanimously approved by both companies' Boards of Directors, and is anticipated to close in Q3 2025, pending regulatory approval and other customary conditions [1][7][8] - BioMarin will commence a cash tender offer for all outstanding shares of Inozyme common stock at a price of $4.00 per share, with Inozyme's Board recommending that stockholders tender their shares [7][8] Product Information - INZ-701, the late-stage enzyme replacement therapy being developed for ENPP1 Deficiency, is expected to have pivotal data readout in early 2026, with potential regulatory approval in 2027 [2][4] - ENPP1 Deficiency is a rare genetic condition that leads to serious health complications, including increased cardiovascular mortality risk and severe rickets [6] Clinical Development - INZ-701 is being developed for patients of all ages, with ongoing studies for infants and plans for supportive studies for adolescents and adults [4][5] - A Phase 1/2 study in adults showed a favorable safety profile for INZ-701, with improvements in key health indicators [5] Financial Guidance - BioMarin reaffirmed its full-year 2025 financial guidance and plans to achieve a 40% Non-GAAP Operating Margin in 2026, excluding the impact of the acquisition [9]