{"id":819450,"date":"2025-02-27T16:08:20","date_gmt":"2025-02-27T21:08:20","guid":{"rendered":"https:\/\/www.marketnewsdesk.com\/index.php\/denali-therapeutics-reports-fourth-quarter-and-full-year-2024-financial-results-and-business-highlights\/"},"modified":"2025-02-27T16:08:20","modified_gmt":"2025-02-27T21:08:20","slug":"denali-therapeutics-reports-fourth-quarter-and-full-year-2024-financial-results-and-business-highlights","status":"publish","type":"post","link":"https:\/\/www.marketnewsdesk.com\/index.php\/denali-therapeutics-reports-fourth-quarter-and-full-year-2024-financial-results-and-business-highlights\/","title":{"rendered":"Denali Therapeutics Reports Fourth Quarter and Full Year 2024 Financial Results and Business Highlights"},"content":{"rendered":"
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SOUTH SAN FRANCISCO, Calif., Feb. 27, 2025 (GLOBE NEWSWIRE) — Denali Therapeutics Inc. (Nasdaq: DNLI) today reported financial results for the fourth quarter and year ended December 31, 2024, and provided business highlights.<\/p>\n

\u201cIn 2024, we made significant strides across our portfolio, particularly with our Enzyme Transport Vehicle (ETV) programs, achieving a path to a potential accelerated approval for our lead program in MPS II,\u201d said Ryan Watts, Ph.D., CEO of Denali. \u201cIn 2025, we will continue expanding our capabilities as we prepare for our first potential product launch of tividenofusp alfa. In addition, we are prioritizing opportunities to expand and accelerate our TV portfolio and aim to advance one to two programs to the clinic annually.\u201d<\/p>\n

\n Fourth<\/strong>
\n Quarter 2024 and Recent Program Updates<\/strong>\n <\/p>\n

\n CLINICAL PROGRAMS<\/strong>\n <\/p>\n

\n Tividenofusp<\/strong>
\n alfa (DNL310, ETV:IDS) for Hunter syndrome (MPS II)<\/strong>\n <\/p>\n

In February 2025, Denali presented results of the primary analysis of the Phase 1\/2 study in Hunter syndrome, in addition to long-term data in the ongoing study. The data showed robust and sustained reductions in key biomarkers, along with continued improvements in hearing, cognition, and adaptive behavior. Long-term safety results indicated tividenofusp alfa was generally well tolerated.<\/p>\n

Denali remains on track to submit a Biologics License Application (BLA) under the accelerated approval pathway in early 2025 and is preparing for a U.S. commercial launch in late 2025 or early 2026. The FDA Breakthrough Therapy designation, granted in January 2025, provides enhanced regulatory support, including more intensive FDA guidance and eligibility for rolling submission and priority review.<\/p>\n

Denali is actively advancing prelaunch activities to support tividenofusp alfa and potential future ETV programs. These efforts include engaging with prescribers and payers, developing a suite of patient support services to ensure broad access, and building a targeted commercial and medical affairs team.<\/p>\n

The company continues to enroll patients in the\u00a0global Phase 2\/3 COMPASS study, which aims to support global regulatory approvals. In January 2025, Denali expanded target enrollment for neuronopathic participants (Cohort A) to 42 patients, reflecting steady progress in recruitment and data collection.<\/p>\n

\n DNL126 (ETV:SGSH)<\/strong>
\n for Sanfilippo syndrome Type A (MPS IIIA)<\/strong>\n <\/p>\n

Following initial positive Phase 1\/2 results demonstrating proof of concept for Sanfilippo syndrome, Denali plans to engage with the FDA to align on a pathway for accelerated approval of DNL126. The Phase 1\/2 study continues to enroll participants with Sanfilippo syndrome.<\/p>\n

DNL126 has received FDA Orphan Disease designation and Fast Track status and has been selected for the FDA\u2019s Support for clinical Trials Advancing Rare disease Therapeutics (START) program. This pilot initiative aims to accelerate the development of rare disease treatments.<\/p>\n

\n TAK-594\/DNL593 (PTV:PGRN) for GRN-related frontotemporal dementia<\/strong>\n <\/p>\n

Dosing of participants continues in the Phase 1\/2 study of DNL593 in the treatment of progranulin (GRN)-related frontotemporal dementia (FTD-GRN).<\/p>\n

\n DNL343 (small molecule eIF2B agonist) for the treatment of amyotrophic lateral sclerosis (ALS) <\/strong>\n <\/p>\n

In January 2025, Denali announced topline results that the primary endpoint was not met in the HEALEY ALS platform trial. Further analyses are anticipated later in 2025, including neurofilament light (NfL) and other fluid biomarkers, data from pre-specified subgroups, as well as extended findings from the active treatment extension period.<\/p>\n

\n BIIB122\/DNL151<\/strong> (small molecule<\/strong>LRRK2 inhibitor) for the treatment of Parkinson\u2019s disease (PD)<\/strong><\/p>\n

Denali and Biogen are jointly developing LRRK2 small molecule inhibitors. Biogen is leading the global Phase 2b LUMA study, evaluating BIIB122’s impact on disease progression in early-stage PD, with enrollment of approximately 640 participants expected to complete in 2025. Denali is conducting the Phase 2a BEACON study, specifically enrolling participants with LRRK2-associated PD to assess how LRRK2 inhibition may impact this disease. Dosing in BEACON began in December 2024.<\/p>\n

\n IND-ENABLING STAGE PROGRAMS<\/strong>\n <\/p>\n

Denali is building a broad portfolio of therapeutic candidates by investing in parallel across its TV franchises, i.e., Enzyme TV (ETV), Oligonucleotide TV (OTV), and Antibody TV (ATV), to advance programs for rare diseases, such as lysosomal storage diseases, and common diseases, such as Alzheimer’s disease and Parkinson’s disease. Beginning in 2025, Denali expects to advance one to two additional programs to the clinic per year over the next three years across its TV-enabled franchises (ETV, OTV, and ATV). IND-enabling stage programs include:<\/p>\n

\n ETV<\/strong>\n <\/p>\n