SOUTH SAN FRANCISCO, Calif., June 18, 2026
Denali Therapeutics Inc. announced it has entered into a definitive agreement to sell its Rare Pediatric Disease Priority Review Voucher (PRV) for $195 million. The voucher was awarded by the U.S. Food and Drug Administration (FDA) following the accelerated approval of AVLAYAH™ (tividenofusp alfa-eknm) in March 2026 for the treatment of Hunter syndrome (Mucopolysaccharidosis Type II, MPS II). Denali plans to use the proceeds to accelerate development of its TransportVehicle™ (TV)-enabled pipeline, supporting multiple clinical programs targeting lysosomal storage disorders, Alzheimer’s disease, Parkinson’s disease, frontotemporal dementia, and other neurodegenerative conditions.
PRV Sale Strengthens Funding for CNS and Rare Disease Pipeline
The $195 million transaction is expected to significantly strengthen Denali’s financial position while advancing its next generation of blood-brain barrier (BBB)-penetrating therapeutics. According to Alexander Schuth, M.D., Chief Operating and Financial Officer, monetizing the PRV enhances the company’s flexibility following the FDA approval of AVLAYAH, the first FDA-approved biologic specifically engineered to cross the blood-brain barrier using Denali’s proprietary TransportVehicle™ platform. The company intends to invest the proceeds in expanding its clinical portfolio, including Enzyme TransportVehicle (ETV), Oligonucleotide TransportVehicle (OTV), and Antibody TransportVehicle (ATV) programs designed to improve drug delivery into the brain for diseases with significant unmet medical needs.
Broad Clinical Portfolio Targets Neurodegenerative and Rare Disorders
Denali’s expanding pipeline includes several clinical-stage investigational therapies, including 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. Additional IND-enabling programs include DNL921 targeting amyloid-beta in Alzheimer’s disease, DNL111 for Parkinson’s disease and Gaucher disease, DNL622 for Hurler syndrome (MPS I), and DNL422 targeting alpha-synuclein in Parkinson’s disease. The company believes its proprietary platform can substantially improve drug exposure within the central nervous system, potentially enhancing treatment effectiveness for neurological disorders that have historically been difficult to treat because of the protective blood-brain barrier.
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TransportVehicle™ Platform Continues to Differentiate Denali
Denali’s TransportVehicle™ technology utilizes engineered Fc domains that bind to natural transport receptors, including the transferrin receptor (TfR) and CD98 heavy chain transporter, enabling therapeutic molecules such as enzymes, antibodies, and oligonucleotides to cross the blood-brain barrier after intravenous administration. Preclinical studies have demonstrated 10- to 30-fold higher brain exposure for engineered antibodies and enzymes, while TransportVehicle-enabled oligonucleotides achieved more than 1,000-fold greater brain exposure in primate models compared with conventional systemic delivery. With AVLAYAH providing the first clinical validation of this approach, Denali continues to position its TransportVehicle platform as a foundational technology for developing therapies capable of reaching both the body and brain. The PRV transaction remains subject to customary closing conditions, including regulatory clearance under the Hart-Scott Rodino Antitrust Improvements Act.
Source: Denali Therapeutics press release
