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Proceeds from transaction to support advancement of Denali’s broad TransportVehicle™-enabled clinical portfolio for lysosomal storage disorders and neurodegenerative diseases
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Denali was awarded Priority Review Voucher following FDA approval of AVLAYAH™, the first FDA-approved biologic specifically designed to cross blood-brain barrier
SOUTH SAN FRANCISCO, Calif.,June 18, 2026(GLOBE NEWSWIRE) --Denali Therapeutics Inc.(Nasdaq: DNLI) today announced it has entered into a definitive agreement to sell its Rare Pediatric Disease Priority Review Voucher (PRV) for gross proceeds of$195 million. TheU.S. Food and Drug Administration(FDA) awarded the PRV to Denali following accelerated approval of the enzyme replacement therapy AVLAYAH™ (tividenofusp alfa-eknm) for the treatment of Hunter syndrome (mucopolysaccharidosis type II; MPS II) inMarch 2026. AVLAYAH is the first FDA-approved medicine in an emerging class of biotherapeutics designed to cross the blood-brain barrier via transferrin receptor (TfR)-mediated transport.
"The Priority Review Voucher program is an important and effective mechanism to support the development of medicines for rare pediatric diseases. Monetizing this PRV strengthens our financial flexibility at a pivotal moment as we build on the momentum created by the FDA approval of AVLAYAH, the first FDA-approved biotherapeutic designed to reach the whole body, including the brain," saidAlexander Schuth, M.D., Chief Operating and Financial Officer ofDenali Therapeutics. "The proceeds will fuel the advancement and acceleration of our broad clinical pipeline, including additional Enzyme TransportVehicle programs for lysosomal storage disorders and Oligonucleotide and Antibody TransportVehicle programs targeting Alzheimer's and other neurodegenerative diseases."
Denali's clinical-stage portfolio includes DNL126 (ETV:SGSH) for Sanfilippo syndrome type A (MPS IIIA), DNL593 (PTV:PGRN) for GRN-related frontotemporal dementia, DNL952 (ETV:GAA) for Pompe disease and DNL628 (OTV:MAPT) for Alzheimer's disease. Denali also has multiple programs in the Investigational New Drug (IND)-enabling stage, including DNL921 (ATV:Abeta) for Alzheimer's disease, DNL111 (ETV:GCase) for Parkinson's disease and Gaucher disease, DNL622 (ETV:IDUA) for Hurler syndrome (MPS I) and DNL422 (OTV:SNCA) for Parkinson's disease.
The PRV transaction is subject to customary closing conditions, including expiration of the applicable waiting period under the Hart-Scott Rodino Antitrust Improvements Act.
About the Denali TransportVehicle™ Platform
The blood-brain barrier (BBB) is essential in maintaining the brain’s microenvironment and protecting it from harmful substances and pathogens circulating in the bloodstream. Historically, the BBB has posed significant challenges to drug development for central nervous system diseases by preventing most drugs from reaching the brain in therapeutically relevant concentrations. Denali’s TransportVehicle™ (TV) platform is a proprietary technology designed to effectively deliver large therapeutic molecules such as antibodies, enzymes and oligonucleotides throughout the whole body, including the brain, by crossing the BBB after intravenous administration. The TV platform is based on engineered Fc domains that bind to specific natural transport receptors, such as transferrin receptor and CD98 heavy chain amino acid transporter, which are expressed at the BBB and deliver the TV and its therapeutic cargo to the brain through receptor-mediated transcytosis. In animal models, antibodies and enzymes engineered with the TV platform demonstrate more than 10- to 30-fold greater brain exposure than similar antibodies and enzymes without this technology. Oligonucleotides engineered with the TV platform demonstrate more than a 1,000-fold greater brain exposure in primates than systemically delivered oligonucleotides without this technology. Improved exposure and broad distribution in the brain may increase therapeutic efficacy by enabling widespread achievement of therapeutically relevant concentrations of product candidates. The TV platform has been clinically validated, with AVLAYAH™ (tividenofusp alfa-eknm) as the first FDA-approved medicine leveraging transferrin receptor to cross the BBB.
About Denali Therapeutics
Denali Therapeutics Inc. is a biotechnology company pioneering a new class of biotherapeutics designed to cross the blood-brain barrier (BBB) using its proprietary TransportVehicle™ platform. With the first FDA-approved biologic specifically designed to cross the BBB, a clinically validated delivery platform and a growing portfolio of therapeutic candidates across all stages of development, Denali is advancing toward its goal of delivering effective medicines to transform life for people with neurodegenerative diseases, lysosomal storage disorders and other serious diseases.