PARMA, Italy and CARMIEL, Israel, Oct. 17, 2025 (GLOBE NEWSWIRE) -- Chiesi Global Rare Diseases, a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people living with rare diseases, and Protalix BioTherapeutics, Inc. (NYSE American:PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx® plant cell-based protein expression system, acknowledge that th ...

Core Insights - Denali Therapeutics Inc. announced that the FDA has accepted the Biologics License Application (BLA) for tividenofusp alfa, seeking accelerated approval for the treatment of Hunter syndrome, with a target action date of January 5, 2026 [1][7]. Company Overview - Denali Therapeutics is a biotechnology company focused on developing therapies that can cross the blood-brain barrier (BBB) for neurodegenerative and lysosomal storage diseases [9]. - The company utilizes a proprietary TransportVehicle™ platform designed to deliver large therapeutic molecules across the BBB, enhancing drug efficacy [8]. Product Details - Tividenofusp alfa is an investigational enzyme replacement therapy designed to address both neurological and physical symptoms of Hunter syndrome by delivering the iduronate 2-sulfatase (IDS) enzyme into the brain and body [2][4]. - The therapy has received Fast Track and Breakthrough Therapy designations from the FDA, as well as Priority Medicines designation from the European Medicines Agency [4]. Clinical Studies - The BLA submission is supported by data from a Phase 1/2 study involving 47 participants with Hunter syndrome [3]. - Denali is conducting an ongoing Phase 2/3 COMPASS study to support global regulatory approvals, with participants randomized to receive either tividenofusp alfa or idursulfase [5]. Disease Context - Hunter syndrome (MPS II) is a rare genetic disorder caused by a deficiency in the IDS enzyme, leading to the accumulation of glycosaminoglycans and resulting in cognitive decline, behavioral issues, and physical complications [6]. - Current therapies do not effectively address neurological symptoms due to their inability to cross the BBB, highlighting the unmet need for new treatments [6].

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]