Denali Therapeutics to Present New Data on ETV: IDS (DNL310) for Potential Treatment of Hunter Syndrome at WORLDSymposium ™

SOUTH SAN FRANCISCO, Calif., February 5, 2021 (GLOBE NEWSWIRE) – Denali Therapeutics Inc. (DNLI), a biopharmaceutical company developing a broad portfolio of product candidates designed to cross the blood brain barrier (BBB) ​​for neurodegenerative diseases, today announced its participation in the 17^e Annual WORLDSymposium™, a research conference devoted to lysosomal diseases, taking place virtually February 8-12, 2021. Denali will share new clinical and preclinical data from its development program for ETV: IDS (DNL310) with safety and biomarker data ( including lysosomal lipids) from cohort A of an ongoing Phase 1/2 study in patients with Hunter syndrome after up to three months of weekly intravenous doses of DNL310

Platform presentations

• The iduronate-2-sulfatase transport vehicle rescues neurobehavioral and skeletal phenotypes in a mouse model of type II mucopolysaccharidosis (Arguello A., et al.) Thursday February 11^e
• The molecular architecture determines the cerebral delivery of iduronate-2-sulfatase targeted to the transferrin receptor in a mouse model of type II mucopolysaccharidosis (Mahon C., et al.) Friday February 12^e
• Intravenous VTE: IDS (DNL310) significantly reduced heparan sulfate in cerebrospinal fluid in an open label Ph1 / 2 study in MPS II patients (Bakardjiev A., et al.) Friday February 12^e

Virtual posters

• Poster n ° 93: Design of a first 24-week human study on intravenous DNL310 (IDS by brain penetration) in MPS II (Harmatz P., et al.) Wednesday February 10^e
• Poster n ° 21: Evaluation of fluid biomarkers reveals lysosome dysfunction and neurodegeneration in MPS II neuronopathic patients (Bhalla A., et al.) Thursday February 11^e

About the ETV Development Program: IDS (DNL310) for the Potential Treatment of Hunter Syndrome
Hunter syndrome is a rare neurodegenerative disorder of lysosomal storage caused by a mutation in the gene encoding the enzyme iduronate-2-sulfatase (IDS). The resulting reduction or loss of IDS enzyme activity leads to a build-up of glycosaminoglycans, which causes lysosomal dysfunction and neurodegeneration as well as progressive damage to several organs including bones, cartilage, heart, and limbs. lungs. The current standard enzyme replacement therapy does not treat the neuronopathic manifestations of the disease because it does not sufficiently cross the BBB. DNL310 is a fusion protein composed of IDS fused to Denali’s proprietary enzyme transport vehicle (ETV), which is designed to cross BBB via receptor-mediated transcytosis in the brain. Denali previously announced the human biomarker proof of concept for its transport vehicle (VT) technology in cohort A (n = 5) of an ongoing Phase 1/2 study of DNL310 in patients with syndrome. Hunter. The study is currently recruiting cohort B, and a cohort C is planned to further explore the clinical parameters.

Webinar Details
Denali to Host Analyst Webinar to Discuss Data Presentations at WORLDSymposium™ on February 12, 2021, starting at 8:00 a.m. Eastern Time. A registration link will be available on the Events page under the Investors section of the Denali website at https://www.denalitherapeutics.com/investors/events or by clicking here. An archived replay of the webinar will be available for at least 30 days after the event.

About Denali Therapeutics
Denali Therapeutics is a biopharmaceutical company developing a broad portfolio of product candidates designed to cross the blood-brain barrier (BBB) ​​for neurodegenerative diseases. Denali pursues new treatments by rigorously evaluating genetically validated targets, ingesting delivery through the BBB, and guiding development with biomarkers that demonstrate target and pathway engagement. Denali is based south of San Francisco. For more information, please visit www.denalitherapeutics.com.

Investor Relations Contact:
Laura Hansen, Ph.D.
Vice-President, Investor Relations
(650) 452-2747
[email protected]

Media contacts:
Lizzie hyland
(646) 495-2706
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or

Morgan warners
(202) 295-0124
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