Tividenofusp alfa BLA for Hunter syndrome accepted for priority review and assigned PDUFA target action date of January 5, 2026; company preparing for commercial launch DNL126 accelerated approval path for Sanfilippo syndrome Type A aligned with FDA; Phase 1/2 study nearing completion of enrollment; planning underway for a global Phase 3 confirmatory study On track to submit regulatory applications in 2025 to begin clinical... Read More

FDA assigns PDUFA target action date of January 5, 2026, for decision on accelerated approval Tividenofusp alfa is designed to deliver missing enzyme to entire body and cross blood-brain barrier into the brain Tividenofusp alfa leads company’s broader TransportVehicle™-enabled pipeline SOUTH SAN FRANCISCO, Calif., July 07, 2025 (GLOBE NEWSWIRE) -- Denali Therapeutics Inc. (Nasdaq: DNLI) today announced that the U.S. Food and... Read More

SOUTH SAN FRANCISCO, Calif., May 06, 2025 (GLOBE NEWSWIRE) -- Denali Therapeutics Inc. (Nasdaq: DNLI) today reported financial results for the first quarter ended March 31, 2025, and provided business highlights. “The completion of our BLA submission for tividenofusp alfa represents a pivotal milestone—not only in our commitment to delivering a potentially transformative therapy to individuals living with Hunter syndrome,... Read More

Rolling submission of BLA initiated for tividenofusp alfa; preparations ongoing for potential U.S. commercial launch in late 2025 or early 2026 Alignment through recent interactions with CDER on path to accelerated approval and conversion to full approval for tividenofusp alfa Productive collaboration continues under START for an accelerated development and approval path for DNL126 SOUTH SAN FRANCISCO, Calif., April 02, 2025... Read More

SOUTH SAN FRANCISCO, Calif., Feb. 27, 2025 (GLOBE NEWSWIRE) -- Denali Therapeutics Inc . (Nasdaq: DNLI) today reported financial results for the fourth quarter and year ended December 31, 2024, and provided business highlights. “In 2024, we made significant strides across our portfolio, particularly with our Enzyme Transport Vehicle (ETV) programs, achieving a path to a potential accelerated approval for our lead program in... Read More

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 behavior Long-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;... Read More

SOUTH SAN FRANCISCO, Calif., Jan. 30, 2025 (GLOBE NEWSWIRE) -- Denali Therapeutics Inc. (NASDAQ: DNLI), today announced upcoming oral (platform) and poster presentations at the 21 st Annual WORLD Symposium ™, 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).... Read More

FDA Breakthrough Therapy Designation received for tividenofusp alfa for Hunter syndrome (MPS II); on track to submit biologics license application (BLA) in early 2025 Preparing for commercial launch of tividenofusp alfa for Hunter syndrome in late 2025 or early 2026 Enable accelerated approval pathway for DNL126 for Sanfilippo syndrome Type A (MPS IIIA) Expand and advance portfolio of multiple TransportVehicle TM (TV)... Read More

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... Read More

Primary endpoint of overall function (ALSFRS-R) and survival, and key secondary endpoints of muscle strength and respiratory function, were not met at 24 weeks Overall, DNL343 was found to be safe and well tolerated Additional analyses, including neurofilament light (NfL) and other fluid biomarkers, prespecified sub-group analyses and analyses from the active treatment extension period are expected later in 2025 SOUTH SAN... Read More

SOUTH SAN FRANCISCO, Calif., Dec. 05, 2024 (GLOBE NEWSWIRE) -- Denali Therapeutics Inc. (Nasdaq: DNLI) today announced initiation of dosing in a global Phase 2a clinical study, BEACON, of the investigational drug leucine-rich repeat kinase 2 (LRRK2) inhibitor BIIB122 (DNL151) in participants with LRRK2-associated Parkinson’s disease (LRRK2-PD). LRRK2 inhibition is a potential therapeutic approach that may slow progression of... Read More

SOUTH SAN FRANCISCO, Calif., Nov. 06, 2024 (GLOBE NEWSWIRE) -- Denali Therapeutics Inc. (Nasdaq: DNLI) today reported financial results for the third quarter ended September 30, 2024, and provided business highlights. "As leaders in pioneering a new class of therapeutics that cross the blood-brain barrier, we are making significant progress across our Transport Vehicle (TV)-enabled portfolio,” said Ryan Watts, Ph.D., Chief... Read More

Recent successful meeting with the FDA provides path to file for accelerated approval and subsequent conversion to full approval Plan to submit biologics license application (BLA) early in 2025 under the accelerated approval pathway New Phase 1/2 data will be presented this week at SSIEM 2024 demonstrating robust and durable biomarker responses and positive effects on clinical outcomes SOUTH SAN FRANCISCO, Calif., Sept. 03,... Read More

SOUTH SAN FRANCISCO, Calif., Aug. 14, 2024 (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 the treatment of neurodegenerative and lysosomal storage diseases, today announced publication of nonclinical data in the August 14, 2024 issue of Science Translational Medicine ( link )... Read More

SOUTH SAN FRANCISCO, Calif., Aug. 01, 2024 (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 the treatment of neurodegenerative diseases and lysosomal storage diseases, today reported financial results for the second quarter ended June 30, 2024, and provided business highlights. "In... Read More

START is a pilot program newly launched jointly by the FDA CDER and CBER divisions to further accelerate the development of novel drug and biological products for rare diseases DNL126 was selected as one of three CDER-regulated products based on eligibility criteria including the potential for clinical benefit for a rare neurodegenerative disease and the drug sponsor’s ability to advance development towards a marketing... Read More

SOUTH SAN FRANCISCO, Calif., May 07, 2024 (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 the treatment of neurodegenerative diseases and lysosomal storage diseases, today reported financial results for the first quarter ended March 31, 2024, and provided business highlights. "It... Read More

SOUTH SAN FRANCISCO, Calif., May 01, 2024 (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 the treatment of neurodegenerative and lysosomal storage diseases, today announced that the Sean M. Healey & AMG Center in collaboration with the Northeast ALS Consortium (NEALS) completed... Read More

SOUTH SAN FRANCISCO, Calif., Feb. 27, 2024 (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 the treatment of neurodegenerative diseases and lysosomal storage diseases, today reported financial results for the fourth quarter and year ended December 31, 2023, and provided business... Read More

SOUTH SAN FRANCISCO, Calif., Feb. 27, 2024 (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 the treatment of neurodegenerative diseases and lysosomal storage diseases, today announced that the company has entered into a securities purchase agreement with certain existing accredited... Read More

New data show additional improvement and stabilization in clinical outcomes in MPS II with 104 weeks of tividenofusp alfa (DNL310) treatment in the open-label, single-arm Phase 1/2 study; sustained normalization of CSF heparan sulfate and robust, sustained reduction in neurofilament light chain (NfL) observed Announcing initiation of dosing with DNL126 (ETV:SGSH) in Phase 1/2 study in MPS IIIA and new supportive data with... Read More

SOUTH SAN FRANCISCO, Calif., Feb. 01, 2024 (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 the treatment of neurodegenerative diseases and lysosomal storage diseases, today announced upcoming presentations from its Enzyme Transport Vehicle (ETV) development programs, tividenofusp... Read More

Complete enrollment in Denali-led, late-stage programs for MPS II and ALS, and establish commercial readiness Advance broad clinical-stage portfolio of seven therapeutic product candidates across neurodegeneration (ALS, Parkinson’s disease, MS, FTD-GRN), lysosomal storage disease (MPS II, MPS IIIA), and inflammatory disease (UC) Advance OTV:MAPT targeting tau for Alzheimer’s disease and OTV:SNCA targeting alpha-synuclein for... Read More

Quotient's Somatic Genomics platform reveals new approaches to treat disease based on the vast genetic variation present in the body's trillions of cells Company emerges from stealth after two years of platform development with an initial commitment of $50 million from Flagship Pioneering CAMBRIDGE, Mass. , Nov. 21, 2023 /PRNewswire/ -- Flagship Pioneering , the bioplatform innovation company, today unveiled Quotient... Read More

SOUTH SAN FRANCISCO, Calif., Nov. 07, 2023 (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 the treatment of neurodegenerative diseases and lysosomal storage diseases, today reported financial results for the third quarter ended September 30, 2023, and provided business highlights.... Read More

CAMBRIDGE, Mass. , Sept. 11, 2023 /PRNewswire/ -- Flagship Pioneering, the bioplatform innovation company, announced today the appointment of Amanda Kay as Senior Partner and Chief Business Development Officer. In this role, she will be responsible for driving and supporting business development across the Flagship ecosystem, leading the effort to forge strong partnerships between Flagship companies and the biopharma... Read More

Additional biomarker data up to two years of treatment continue to demonstrate rapid and sustained normalization of CSF heparan sulfate to normal healthy levels and improvement in lysosomal function biomarkers Additional safety data up to two years of treatment continue to demonstrate that DNL310 is generally well tolerated Previously reported data showed robust and statistically significant reduction of NfL, a marker of... Read More

FDACS approves first registration of Invaio's Trecise™ technology to deliver ArborBiotic™ for the suppression of citrus greening in oranges Citrus growers gain new solution for the protection of young and non-bearing trees Trecise™ technology is a gamechanger for disease treatment and yield recovery CAMBRIDGE, Mass. , Aug. 29, 2023 /PRNewswire/ -- Invaio Sciences , a bio platform company accelerating the leap to... Read More

SOUTH SAN FRANCISCO, Calif., Aug. 08, 2023 (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 the treatment of neurodegenerative diseases and lysosomal storage diseases, today reported financial results for the second quarter ended June 30, 2023, and provided business highlights. “In... Read More

Empress's Chemilogics™ product platform creates small molecule drugs quickly and cost-effectively The company emerges after two years of platform development with an initial commitment of $50 million from Flagship Pioneering Empress has identified and characterized 15 potential small molecule drugs in less than two years; it expects to file multiple Investigational New Drug (IND) applications within the next 24 months Murray... Read More

Interim results demonstrate average reduction of 64% (p <0.001) from baseline in serum NfL after 2 years of dosing with DNL310 in Phase 1/2 study FDA has recommended assessment of NfL, a marker of neuroaxonal damage, as a possible biomarker in MPS II Additional interim data from the DNL310 Phase 1/2 study will be presented at the SSIEM symposium in August Management will host a webinar for analysts and investors at 8:30 a.m.... Read More

SOUTH SAN FRANCISCO, Calif., May 08, 2023 (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 the treatment of neurodegenerative diseases and lysosomal storage diseases, today reported financial results for the first quarter ended March 31, 2023, and provided business highlights. “In... Read More

CAMBRIDGE, Mass. and SOUTH SAN FRANCISCO, Calif., April 12, 2023 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) and Denali Therapeutics Inc. (Nasdaq: DNLI) today announced that Biogen has exercised the option to license Denali’s Antibody Transport Vehicle (ATV):Amyloid beta program (ATV:Aβ). Accumulation of Aβ plaque in the brain is a defining feature of Alzheimer’s disease (AD). Using Denali’s ATV platform to cross the... Read More

Data show that DNL343 is generally well-tolerated at doses that demonstrate robust inhibition of biomarkers associated with the integrated stress response (ISR) Clinical pharmacokinetic and ISR pharmacodynamic data, along with preclinical data, are consistent with extensive CNS distribution and support once-daily dosing Initiation of DNL343 regimen in the Phase 2/3 HEALEY ALS Platform Trial is expected in mid 2023 SOUTH SAN... Read More

SOUTH SAN FRANCISCO, Calif., Feb. 27, 2023 (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 the treatment of neurodegenerative diseases and lysosomal storage diseases, today reported financial results for the fourth quarter and year ended December 31, 2022, and provided business... Read More

Over 49 weeks of DNL310 (ETV:IDS) treatment in the Phase 1/2 study, positive changes across measures of exploratory clinical outcomes including VABS-II (adaptive behavior) and BSID-III (cognitive capabilities) scores and global impression scales were observed Interim Phase 1/2 data also suggest that DNL310 improves hearing, as assessed by auditory brainstem response testing Additional biomarker data out to 49 weeks continue... Read More

SOUTH SAN FRANCISCO, Calif., Feb. 15, 2023 (GLOBE NEWSWIRE) -- Denali Therapeutics Inc. (Nasdaq: DNLI), a biopharmaceutical company developing a broad portfolio of product candidates engineered to cross the blood-brain barrier for the treatment of neurodegenerative diseases and lysosomal storage diseases, today announced upcoming presentations from Enzyme Transport Vehicle (ETV) development programs, DNL310 (ETV:IDS) and... Read More

Partner Sanofi has commenced dosing in a Phase 2 clinical trial of SAR443820 (DNL788) in individuals with multiple sclerosis Denali to receive a milestone payment of $25 million from Sanofi for Phase 2 clinical trial initiation Development of RIPK1 inhibitor program continues in a broad range of central nervous system and peripheral inflammatory conditions SOUTH SAN FRANCISCO, Calif., Jan. 25, 2023 (GLOBE NEWSWIRE) -- Denali... Read More

Four programs expected to progress in late-stage clinical studies for MPS II (Hunter syndrome), ALS, and Parkinson’s disease Further validation and expansion of Transport Vehicle (TV) technology, including multiple interim clinical data readouts, submission of IND for additional ETV program, and selection of five Oligonucleotide TV (OTV) targets Continued invention and scientific insights in neurodegeneration biology fuel... Read More

DNL343 demonstrated extensive blood-brain barrier penetration and robustly inhibited the integrated stress response pathway implicated in ALS Once-daily oral dosing with DNL343 for 28 days was generally well tolerated in participants with ALS The design phase for entry of DNL343 into a Phase 2/3 trial in the HEALEY ALS Platform Trial is underway SOUTH SAN FRANCISCO, Calif., Dec. 05, 2022 (GLOBE NEWSWIRE) -- Denali... Read More