Denali Therapeutics (DNLI) reported primary analysis data from an early to mid-stage study of its pipeline candidate, tividenofusp alfa (DNL310), in 47 patients with Hunter syndrome (MPS II) over a 24-week treatment period, along with additional long-term follow-up data. DNLI’s wholly-owned program, tividenofusp alfa, is an Enzyme Transport Vehicle-enabled iduronate-2-sulfatase replacement therapy in development for MPS II.Per the additional long-term data readout from the phase I/II study, treatment with t ...

Long-term data demonstrate that robust reductions and normalization in key biomarkers from baseline were maintained over time with continued improvement in hearing, cognition and adaptive behaviorLong-term safety data with median follow-up of two years, and out to more than four years, demonstrate that tividenofusp alfa was generally well tolerated Regulatory submission for accelerated approval is planned for early 2025; U.S. launch preparation is ongoing to deliver tividenofusp alfa to families with MPS II ...

SOUTH SAN FRANCISCO, Calif., Jan. 30, 2025 (GLOBE NEWSWIRE) -- Denali Therapeutics Inc. (NASDAQ:DNLI), today announced upcoming oral (platform) and poster presentations at the 21st Annual WORLDSymposium™, which will be held February 3-7, 2025, in San Diego, California. The oral presentation includes clinical results related to its Hunter syndrome (MPS II) investigational therapeutic, tividenofusp alfa (DNL310). Tividenofusp alfa is enabled by the Denali TransportVehicle™ platform, which is designed to effec ...

SOUTH SAN FRANCISCO, Calif., Jan. 30, 2025 (GLOBE NEWSWIRE) -- Denali Therapeutics Inc. (NASDAQ: DNLI), today announced upcoming oral (platform) and poster presentations at the 21st Annual WORLDSymposium™, which will be held February 3-7, 2025, in San Diego, California. The oral presentation includes clinical results related to its Hunter syndrome (MPS II) investigational therapeutic, tividenofusp alfa (DNL310). Tividenofusp alfa is enabled by the Denali TransportVehicle™ platform, which is designed to effe ...

Denali Therapeutics (DNLI) has performed well in the past 12 months amid a volatile market. Shares of the company have gained 26.6% against the industry’s decline of 14.3%. The stock has also outperformed the sector and the S&P 500 Index in this time frame.Denali is developing a broad portfolio of product candidates engineered to cross the blood-brain barrier for the treatment of neurodegenerative and lysosomal storage diseases.  The upward price trajectory can be attributed to positive pipeline updates.DNL ...

Denali Therapeutics Inc DNLI CEO Ryan Watts presented at the J.P. Morgan Healthcare Conference. The presentation primarily focused on the company’s blood-brain-barrier (BBB) crossing platform technology and the pipeline, including tividenofusp alfa (tivi, DNL310) in Hunter syndrome and DNL126 in Sanfilippo syndrome Type A, as well as ATV:Abeta approach in Alzheimer’s disease.Also Read: Denali Therapeutics’ Amyotrophic Lateral Sclerosis Misses Pivotal Trial, But Analyst Says It Was ExpectedJP Morgan notes th ...

FDA Breakthrough Therapy Designation received for tividenofusp alfa for Hunter syndrome (MPS II); on track to submit biologics license application (BLA) in early 2025Preparing for commercial launch of tividenofusp alfa for Hunter syndrome in late 2025 or early 2026Enable accelerated approval pathway for DNL126 for Sanfilippo syndrome Type A (MPS IIIA)Expand and advance portfolio of multiple TransportVehicleTM (TV) enabled programs for delivery of enzyme, oligonucleotide, and antibody therapeutics for rare a ...

This article is published by Terry Chrisomalis, who runs the Biotech Analysis Central pharmaceutical service on Seeking Alpha Marketplace. If you like what you read here and would like to subscribe to, I'm currently offering a two-week free trial period for subscribers to take advantage of. My service offers a deep-dive analysis of many pharmaceutical companies. The Biotech Analysis Central SA marketplace is $49 per month, but for those who sign up for the yearly plan will be able to take advantage of a 33. ...

Denali Therapeutics Inc. (DNLI) announced that the FDA has granted Breakthrough Therapy Designation to its pipeline candidate, tividenofusp alfa (DNL310), for the treatment of individuals with Hunter syndrome (MPS II).Denali’s wholly owned program, DNL310 or tividenofusp alfa, is an Enzyme Transport Vehicle-enabled iduronate-2-sulfatase (IDS) replacement therapy in development for MPS II (Hunter syndrome).Shares of DNLI have lost 1.9% in the past six months compared with the industry’s 8.3% decline.Image So ...

SOUTH SAN FRANCISCO, Calif., Jan. 08, 2025 (GLOBE NEWSWIRE) -- Denali Therapeutics Inc. (NASDAQ:DNLI), today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for tividenofusp alfa (DNL310) for the treatment of individuals with Hunter syndrome (MPS II). This designation is in addition to Fast Track Designation, Orphan Drug Designation, and Rare Pediatric Disease Designation, all previously granted by the FDA for tividenofusp alfa in Hunter syndrome. Dena ...