Denali Therapeutics Announces Q2 2024 Financial Results and Highlights

Denali Therapeutics Inc. (Nasdaq: DNLI), headquartered in South San Francisco, announced the financial results for its second quarter ending on June 30, 2024. The biopharmaceutical company is known for developing a portfolio of product candidates designed to cross the blood-brain barrier (BBB) for the treatment of neurodegenerative and lysosomal storage diseases. According to CEO Ryan Watts, Denali's Enzyme Transport Vehicle (ETV) franchise showed substantial progress, particularly with tividenofusp alfa (DNL310, ETV:IDS) for MPS II and DNL126 (ETV:SGSH) for MPS IIIA being selected for the FDA's START program.

Program Highlights

Denali provided updates on its various clinical and preclinical programs:

Late-Stage and Mid-Stage Programs:

- Tividenofusp Alfa (DNL310): In April, Denali completed the enrollment of 47 participants in a Phase 1/2 open-label study for the treatment of MPS II (Hunter syndrome). By July, a review co-authored by Denali's Chief Medical Officer, Carole Ho, M.D., was published, summarizing accelerated approval pathways using heparan sulfate as a biomarker. This followed a workshop facilitated by the Reagan-Udall Foundation. Denali is in communication with the FDA’s Center for Drug Evaluation and Research (CDER) regarding the potential accelerated approval of tividenofusp alfa.

- DNL343: The Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital announced in May that enrollment was complete for Regimen G (DNL343), which is being developed for the treatment of amyotrophic lateral sclerosis (ALS).

- SAR443820/DNL788: Sanofi's evaluation of this CNS-penetrant RIPK1 inhibitor for multiple sclerosis (MS) is fully enrolled in a Phase 2 study.

- BIIB122/DNL151: Biogen is conducting a global Phase 2b LUMA study on BIIB122 for early-stage Parkinson’s disease. Denali plans a global Phase 2a study in 2024 to assess safety and biomarkers in Parkinson's patients with LRRK2 mutations.

- Eclitasertib (SAR443122/DNL758): Sanofi continues its Phase 2 study on this peripheral RIPK1 inhibitor for patients with ulcerative colitis (UC).

Early-Stage Programs:

- DNL126: Selected by the FDA for the START Pilot Program, this ETV-enabled SGSH replacement therapy is being developed for MPS IIIA. Phase 1/2 biomarker and safety data are anticipated by the end of 2024.

- TAK-594/DNL593: This PTV-enabled progranulin replacement therapy for frontotemporal dementia-granulin (FTD-GRN) is in the prescreening phase for Cohort B2, following a protocol amendment.

- Oligonucleotide Transport Vehicle (OTV) Platform: Denali is advancing OTV:MAPT, targeting tau for Alzheimer’s disease, and OTV:SNCA, targeting alpha-synuclein for Parkinson’s disease, in the IND-enabling stage of development.

ATV:Abeta Program:
Denali announced it has regained rights to its ATV:Abeta program, which was previously licensed to Biogen. The program focuses on developing anti-amyloid beta therapeutics for Alzheimer’s disease. Preclinical data indicate a potential for improved efficacy and safety, showing superior plaque reduction and low rates of amyloid-related imaging abnormalities (ARIA).

Discovery Programs:
Denali continues to leverage its expertise to develop BBB-crossing therapeutics for neurodegenerative diseases like Alzheimer’s and Parkinson’s, as well as lysosomal storage diseases. In July, Denali posted a manuscript on the distinct biodistribution profiles of its TfR and CD98hc transport vehicle platforms on bioRxiv.

Financial Summary:
For Q2 2024, Denali reported a net loss of $99.0 million, compared to a net income of $183.4 million in Q2 2023. This decline was primarily due to an absence of collaboration revenue, which had been exceptionally high in the previous year owing to Biogen’s licensing of the ATV:Abeta program. Research and development expenses decreased slightly to $91.4 million due to reduced personnel and external costs. General and administrative expenses also saw a minor reduction. Denali’s cash, cash equivalents, and marketable securities totaled approximately $1.35 billion as of June 30, 2024. 

Denali Therapeutics continues to pioneer BBB-crossing treatments, contributing to advancements in the field of neurodegenerative and lysosomal storage diseases.

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