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 ...

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Core Insights - Denali Therapeutics Inc. (DNLI) has received Breakthrough Therapy Designation from the FDA for its candidate tividenofusp alfa (DNL310) aimed at treating Hunter syndrome (MPS II) [1][2] Group 1: FDA Designation and Implications - The Breakthrough Therapy Designation is intended to expedite the development and review of drugs for serious or life-threatening conditions, indicating that tividenofusp alfa may offer substantial improvement over existing therapies [2] - This designation will provide Denali with more intensive guidance from the FDA, including involvement of senior reviewers and eligibility for rolling and priority review of the marketing application [3] Group 2: Clinical Development and Future Plans - Data from an open-label phase I/II study have shown promising results, with positive effects on surrogate endpoints and early signs of improved clinical outcomes in Hunter syndrome participants [3] - Denali plans to submit a Biologics License Application (BLA) for tividenofusp alfa in early 2025 under the accelerated approval pathway [5] Group 3: Recent Pipeline Updates - Denali has initiated dosing in a global mid-stage study, BEACON, for BIIB122 (DNL151), an investigational LRRK2 inhibitor for Parkinson's disease [6] - BIIB122 is also being evaluated in the ongoing global phase IIb LUMA study, which aims to enroll approximately 640 participants with early-stage Parkinson's disease [8] Group 4: Disappointing Results and Discontinuations - Denali reported disappointing results from the phase II/III HEALEY ALS Platform Trial for DNL343, which did not meet its primary endpoint of efficacy in slowing disease progression [9][10] - Partner Sanofi has discontinued the development of SAR443820/DNL788 for ALS and multiple sclerosis due to failure to meet primary and key secondary endpoints in clinical studies [11][12]

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 ...

Core Findings - Denali Therapeutics Inc (DNLI) announced disappointing top-line results from the phase II/III HEALEY ALS Platform Trial for its pipeline candidate DNL343, which failed to meet the primary endpoint of slowing disease progression compared to placebo [1][3][4] - DNL343, a novel small molecule targeting eIF2B, was well tolerated but did not show statistically significant differences in key secondary endpoints measuring muscle strength and respiratory function at week 24 [4][5] - The HEALEY ALS Platform Trial involved 186 participants receiving DNL343 and 139 receiving placebo, with ALS affecting approximately 30,000 people in the US and 500,000 worldwide [3] Stock Performance - DNLI shares declined 6.5% over the past six months, slightly underperforming the industry's 6.4% decline [2] Pipeline Updates - Denali and partner Sanofi discontinued the development of SAR443820/DNL788 for multiple sclerosis after the phase II K2 study failed to meet primary and key secondary endpoints [6][8] - The companies are co-developing SAR443122/DNL758 (eclitasertib) for ulcerative colitis, currently in phase II studies [8] - Denali's wholly owned program, DNL310 (tividenofusp alfa), is in development for MPS II (Hunter syndrome) [9] - BIIB122 (DNL151), an LRRK2 inhibitor for Parkinson's disease, is being evaluated in a global phase IIa study (BEACON) and a phase IIb study (LUMA) in collaboration with Biogen [9][10][11] Future Plans - Denali will report additional data from the HEALEY ALS Platform Trial in 2025, including neurofilament light (NfL) and other fluid biomarkers, subgroup analyses, and extended findings from the active treatment extension period [5] - The company will evaluate the data before determining next steps for DNL343 [5] Industry Comparison - Immunocore Holdings plc (IMCR), a Zacks Rank 1 (Strong Buy) stock in the biotech sector, has seen narrowing loss per share estimates for 2024 and 2025, with an average earnings surprise of 25.57% in the trailing four quarters [12][13]