Benzinga | Feb 5, 2021 09:03AM EST

Denali Therapeutics Says To Present New Data On ETV:IDS For Potential Treatment Of Hunter Syndrome At WORLDSymposium

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

SOUTH SAN FRANCISCO, Calif., Feb. 05, 2021 (GLOBE NEWSWIRE) -- Denali Therapeutics Inc. (NASDAQ:DNLI), a biopharmaceutical company developing a broad portfolio of product candidates engineered to cross the blood-brain barrier (BBB) for neurodegenerative diseases, today announced its participation at the 17th Annual WORLDSymposium(tm), a research conference dedicated to lysosomal diseases, taking place virtually from 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

* Iduronate-2-sulfatase transport vehicle rescues neurobehavioral and skeletal phenotypes in a mouse model of mucopolysaccharidosis type II (Arguello A., et al.) on Thursday, February 11th

* Molecular architecture determines brain delivery of transferrin receptor targeted iduronate-2-sulfatase in a mouse model of mucopolysaccharidosis type II (Mahon C., et al.) on Friday, February 12th

* Intravenous ETV:IDS (DNL310) significantly reduces cerebrospinal fluid heparan sulfate in an open label Ph1/2 study in MPS II patients (Bakardjiev A., et al.) on Friday, February 12th

Virtual Posters

* Poster #93: Design of a First-in-Human, 24-week study of Intravenous DNL310 (brain-penetrant IDS) in MPS II (Harmatz P., et al.) on Wednesday, February 10th

* Poster #21: Evaluation of fluid biomarkers reveals lysosome dysfunction and neurodegeneration in neuronopathic MPS II Patients (Bhalla A., et al.) on Thursday, February 11th

About the ETV:IDS (DNL310) Development Program for the Potential Treatment of Hunter syndrome

Hunter syndrome is a rare neurodegenerative lysosomal storage disorder caused by a mutation in the gene that encodes for the enzyme iduronate-2-sulfatase (IDS). The resultant reduction or loss of 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 lung. Current standard of care enzyme replacement treatment does not address neuronopathic manifestations of the disease as 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 engineered to cross the BBB via receptor-mediated transcytosis into the brain. Denali previously announced human biomarker proof-of-concept for its Transport Vehicle (TV) technology from Cohort A (n=5) of an ongoing Phase 1/2 study of DNL310 in patients with Hunter syndrome. The study is currently enrolling Cohort B, and a Cohort C is planned to further explore clinical endpoints.

Webinar Details

Denali will host a webinar for analysts to discuss the data presentations at WORLDSymposium(tm) on February 12, 2021, starting at 8:00 a.m. Eastern Time. A registration link will be available on the Events page under the Investor section of Denali's 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 following the event.

About Denali Therapeutics

Denali Therapeutics is a biopharmaceutical company developing a broad portfolio of product candidates engineered to cross the blood-brain barrier (BBB) for neurodegenerative diseases. Denali pursues new treatments by rigorously assessing genetically validated targets, engineering delivery across the BBB and guiding development through biomarkers that demonstrate target and pathway engagement. Denali is based in South San Francisco. For additional information, please visit www.denalitherapeutics.com.

Investor Relations Contact:

Laura Hansen, Ph.D.

Vice President, Investor Relations

(650) 452-2747

hansen@dnli.com

Media Contacts:

Lizzie Hyland

(646) 495-2706

Lizzie.Hyland@FGH.com

or

Morgan Warners

(202) 295-0124

Morgan.Warners@FGH.com