SOUTH SAN FRANCISCO, Calif., Feb. 05, 2021 (GLOBE NEWSWIRE) – Denali Therapeutics Inc. (NASDAQ: DNLI), a biopharmaceutical company developing a broad package of product candidates designed to overcome the blood-brain barrier (BBB) barrier for neurodegenerative diseases, today announced that it is participate at the 17th THE WORLD annualSymposium™, a research conference dedicated to lysosomal diseases, takes place almost from February 8 – 12, 2021. Denali will share new clinical and preclinical data from its development program for ETV: IDS (DNL310) with data safety and biomarker (including lysosomal lipids) from Cohort A of a Phase 1/2 follow-up study in patients with Hunter syndrome after up to three months of weekly intravenous doses of DNL310.
Stage performances
- Iduronate-2-sulfatase transport vehicle rescues neurobehavioral and skeletal phenotypes in a mouse model of type II mucopolysaccharidosis (Arguello A., et al.) on Thursday, February 11thth
- Molecular architecture validates brain delivery of targeted transferrin iduronate-2-sulfatase receptor in a mouse model of type II mucopolysaccharidosis (Mahon C., et al.) on Friday, February 12thth
- Intravenous ETV: IDS (DNL310) significantly reduces cerebrospinal hepatic sulfate in open Ph1 / 2 study in MPS II patients (Bakardjiev A., et al.) on Friday, February 12thth
Meaningful posters
- Poster # 93: Design of a 24-week first-in-human study of Intravenous DNL310 (brain-penetrant IDS) in MPS II (Harmatz P., et al.) on Wednesday, February 10thth
- Poster # 21: Evaluation of lytic biomarkers reveals lysosome dysfunction and neurodegeneration in MPS II neuronopathic patients (Wall A., et al.) on Thursday, February 11thth
About the ETV: IDS (DNL310) development program for the possible treatment of Hunter syndrome
Hunter syndrome is a rare neurodegenerative lysosomal storage disorder caused by a mutation in the gene encoding for the enzyme iduronate-2-sulfatase (IDS). The reduction or loss due to IDS enzyme activity leads to accumulation of glycosaminoglycans, which causes lysosomal dysfunction and neurodegeneration as well as progressive damage to multiple organs including bone, cartilage, heart and lungs. Conventional treatment enzyme treatment does not address neuronopathic manifestations of the disease as it does not cross the BBC sufficiently. DNL310 is a fusion protein made up of IDS attached to Denali’s Enzyme Transport Vehicle (ETV), which is engineered to cross the BBC through intermediate receptor transcytosis into the brain. Denali previously cited a human biomarker hypothesis for its Transport Vehicle (TV) technology from Cohort A (n = 5) of a Phase 1/2 follow-up study of DNL310 in patients with Hunter syndrome. The study is currently enrolling Cohort B, and Cohort C intends to further investigate clinical points.
Webinar Information
Denali will host a webinar for analysts to discuss the data presentations at WORLDSymposium™ on February 12, 2021, starting at 8:00 a.m. East. A registration link will be available on the Events page under the Investment section of the Denali website at https://www.denalitherapeutics.com/investors/events or by clicking here. Archive 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 package of product candidates designed to cross the blood-brain barrier (BBB) barrier for neurodegenerative diseases. Denali pursues new therapies by rigorously evaluating genetically determined targets, delivering engineering across the BBC and driving development through biotractors featuring target communication and way. Denali is based in South San Francisco. For more information, visit www.denalitherapeutics.com.
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