Denali Therapeutics Inc. develops medicines for neurodegenerative diseases, lysosomal storage disorders and other serious diseases using its proprietary TransportVehicle™ platform, which is designed to deliver biotherapeutics across the blood-brain barrier. Company news centers on AVLAYAH™ (tividenofusp alfa-eknm), an FDA-approved enzyme replacement therapy for Hunter syndrome (MPS II), and on pipeline programs that use enzyme, protein, oligonucleotide and antibody transport technologies.
Recurring updates cover clinical and preclinical data for programs in Sanfilippo syndrome type A, Pompe disease, Alzheimer’s disease, frontotemporal dementia and Parkinson’s disease, as well as regulatory decisions, conference presentations, collaboration changes, commercial launch activity and quarterly financial results.
Denali Therapeutics (DNLI) reported third-quarter 2026 net income of $70.1 million, supported by a one-time gain from a voucher sale. The $194.0 million net gain helped reverse a $126.9 million year-earlier net loss. AVLAYAH net product revenue reached $17.8 million in its second full commercial quarter; Denali expects $30.0 million to $32.0 million in fourth-quarter revenue. Cash, cash equivalents and marketable securities totaled approximately $1.0 billion at September 30.
Commercial insurance coverage for AVLAYAH exceeded 70%, versus more than 50% previously. Denali completed enrollment in its COMPASS confirmatory study, with data expected in the first half of 2028. The company plans a 2027 application seeking accelerated approval for zafinofusp alfa for Sanfilippo syndrome type A, with additional Phase 1/2 data planned for early 2027. Five TransportVehicle programs are expected to generate data in 2027.
Denali Therapeutics (Nasdaq: DNLI) received the 2026 Prix Galien USA award for Best Biotechnology Product for its Hunter syndrome treatment AVLAYAH.
The Galien Foundation presented the award at the American Museum of Natural History in New York City. AVLAYAH, an enzyme replacement therapy designed to cross the blood-brain barrier, received FDA accelerated approval in March 2026 for neurologic manifestations of Hunter syndrome when initiated in presymptomatic or symptomatic pediatric patients weighing at least 5 kg before advanced neurologic impairment. Continued approval may depend on verification of clinical benefit in a confirmatory study.
Denali Therapeutics (DNLI) will report third-quarter 2026 financial results and business highlights on November 3, 2026. The results cover the quarter ended September 30, 2026. Management will host a conference call and webcast that day at 4:30 p.m. Eastern Time (1:30 p.m. Pacific Time) to discuss results and provide a general business update.
Denali Therapeutics (Nasdaq: DNLI) reported second-quarter 2026 net product revenue of $3.6 million, driven by the first full quarter of the U.S. launch of AVLAYAH for Hunter syndrome. Net loss was $127.6 million versus $124.1 million a year earlier, with R&D expenses of $97.0 million and SG&A of $36.3 million.
According to Denali, third-quarter 2026 AVLAYAH net product revenue is projected at $10–12 million. Cash, cash equivalents and marketable securities totaled about $940 million at June 30, 2026; pro forma liquidity exceeds $1.1 billion after receiving $195 million from the sale of a Priority Review Voucher in July 2026.
Denali advanced its TransportVehicle portfolio, including AVLAYAH’s ongoing Phase 2/3 COMPASS confirmatory study, Phase 3 start-up for zafinofusp alfa in Sanfilippo syndrome type A with a planned 2027 BLA, and multiple investigational programs in lysosomal storage diseases, Alzheimer’s disease and frontotemporal dementia.
Denali Therapeutics (Nasdaq: DNLI) will report its second quarter 2026 financial results and business highlights for the period ended June 30, 2026, on Thursday, August 6, 2026. Management will host a conference call and webcast at 4:30 p.m. ET (1:30 p.m. PT) to discuss the results and provide a business update.
The live webcast and slide presentation will be available via the Investors section of Denali’s website, with a replay archived for a limited time. Denali describes itself as a biotechnology company developing biotherapeutics that cross the blood-brain barrier using its TransportVehicle™ platform.
Denali Therapeutics (Nasdaq: DNLI) announced that co-founder and CEO Ryan Watts, Ph.D., will give the opening plenary address at AAIC 2026 in London on July 12, 2026.
The presentation will cover advances in neurodegeneration biology, biomarkers, and Denali’s TransportVehicle™ technology, including its FDA-approved AVLAYAH™ therapy and investigational Alzheimer’s candidates DNL628 and DNL921.
Denali Therapeutics (Nasdaq: DNLI) agreed to sell its Rare Pediatric Disease Priority Review Voucher for $195 million in gross proceeds. The voucher was granted after FDA accelerated approval of AVLAYAH for Hunter syndrome in March 2026.
Denali expects proceeds to support its TransportVehicle-enabled clinical portfolio for lysosomal storage disorders and neurodegenerative diseases. The transaction is subject to customary closing conditions, including expiration of the Hart-Scott-Rodino waiting period.
Biogen (Nasdaq: BIIB) and Denali Therapeutics (Nasdaq: DNLI) reported topline Phase 2b LUMA results for BIIB122 in early-stage Parkinson’s disease. The drug did not meet primary or secondary endpoints for slowing disease progression versus placebo, leading to discontinuation in idiopathic Parkinson’s disease.
Exploratory biomarkers showed >90% peripheral LRRK2 kinase inhibition and up to ~30% CSF phosphorylated Rab10 reduction, with sustained drug levels. BIIB122 was generally well tolerated. Denali continues the Phase 2a BEACON study in LRRK2 variant carriers, with data expected in the first half of 2027.
Denali Therapeutics (NASDAQ: DNLI) reported Q1 2026 results and key business milestones on May 7, 2026. Highlights include FDA approval and U.S. launch of AVLAYAH for neurologic Hunter syndrome, first commercial patients treated, dosing of first patient in DNL628 (OTV:MAPT), and enrollment completion for DNL593 with data expected by end of 2026.
Q1 net loss was $128.4M, R&D $103.8M, G&A $33.5M, and cash and marketable securities totaled approximately $1.05B as of March 31, 2026.
Denali Therapeutics (Nasdaq: DNLI) regained full rights to investigational therapy DNL593 (PTV:PGRN) after Takeda terminated the collaboration on April 3, 2026. Denali will continue clinical development and expects Phase 1/2 results, including biomarker data, by the end of 2026; enrollment of 40 FTD-GRN participants is complete.
Interim Part A data showed dose-dependent CSF progranulin increases and generally favorable tolerability; the company will lead further development and manage the intellectual property.