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Denali Therapeutics Reports First Quarter 2024 Financial Results and Business Highlights

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Mardi, mai 7, 2024
FDA, Amyloid beta, Part, Total, PIPE, CDER, Transferrin receptor, Nerve, BBB, CSF, Biomarker, Gamma, Enzyme replacement therapy, Traffic barrier, Hunting, OTV, PTV, Food, MGH, IDS, Conference, MAPT, ALS, Therapy, Cerebrospinal fluid, SAN, MPS II, Hospital, Safety, AFL, SGSH, UC, Alpha-synuclein, LUMA, SNCA, Clinical trial, Glycogen storage disease, Neurodegenerative disease, MPS, COMPASS, Patient, Alfa, Acquisition, ETV, ATV, Hunter syndrome, Sanofi, Massachusetts General Hospital, Pharmaceutical industry

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.

Key Points: 
  • 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.
  • There was no collaboration revenue for the quarter ended March 31, 2024, compared to $35.1 million for the quarter ended March 31, 2023.
  • Total research and development expenses were $107.0 million for the quarter ended March 31, 2024, compared to $128.8 million for the quarter ended March 31, 2023.
  • General and administrative expenses were $25.2 million for the quarter ended March 31, 2024, compared to $27.1 million for the quarter ended March 31, 2023.

Aligos Therapeutics Reports Recent Business Progress and First Quarter 2024 Financial Results

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Mardi, mai 7, 2024
Doctor of Philosophy, Infection, EASL, Investment, Safety, NIH, Research, ESCMID, PIPE, SHBG, Part, Therapy, Data, Patient, AASLD, Hepatitis B virus, Hepatology, ETV, CHB, G&A, MBA, Interim, Annual general meeting, SAN, Interest

SOUTH SAN FRANCISCO, Calif., May 07, 2024 (GLOBE NEWSWIRE) -- Aligos Therapeutics, Inc. (Nasdaq: ALGS, “Aligos”), a clinical stage biopharmaceutical company focused on developing novel therapeutics to address unmet medical needs in liver and viral diseases, today reported recent business progress and financial results for the first quarter 2024.

Key Points: 
  • SOUTH SAN FRANCISCO, Calif., May 07, 2024 (GLOBE NEWSWIRE) -- Aligos Therapeutics, Inc. (Nasdaq: ALGS, “Aligos”), a clinical stage biopharmaceutical company focused on developing novel therapeutics to address unmet medical needs in liver and viral diseases, today reported recent business progress and financial results for the first quarter 2024.
  • “We have executed on a number of key deliverables to position Aligos for success,” stated Lawrence Blatt, Ph.D., MBA, Chairman, President & CEO of Aligos Therapeutics.
  • “Importantly, we began dosing in the Phase 2a HERALD study of our THR- drug candidate, ALG-055009, and we expect topline data in Q4 2024.
  • In addition, we recently presented data from our three clinical programs at scientific conferences, showcasing our robust pipeline of potentially best-in-class small molecule drug candidates.

Aligos Therapeutics Presents Positive Clinical Data at APASL 2024 from ALG-055009 and ALG-000184 Phase 1 Studies

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Mercredi, mars 27, 2024
Multiple, Professor, ETV, SAN, Patient, Hepatology, Southern Medical University, SHBG, Doctor of Science, University, Doctor of Philosophy, MBBS, MASH, MBA, Science, Therapy, Hepatitis, Part, Division, University of Hong Kong, Infection, CHB, Pharmaceutical industry

The poster presentation highlights ALG-055009 Phase 1 data that showed multiple-ascending doses (MAD) over 14 days in hyperlipidemic subjects produced favorable, dose-dependent pharmacodynamic effects on atherogenic lipids and sex hormone binding globulin (SHBG), an indicator of target engagement in the liver.

Key Points: 
  • The poster presentation highlights ALG-055009 Phase 1 data that showed multiple-ascending doses (MAD) over 14 days in hyperlipidemic subjects produced favorable, dose-dependent pharmacodynamic effects on atherogenic lipids and sex hormone binding globulin (SHBG), an indicator of target engagement in the liver.
  • “We are pleased to present these additional Phase 1 data for ALG-055009 and ALG-000184 at APASL 2024.
  • We continue to believe our molecule has the potential to be best-in-class,” said Lawrence Blatt, Ph.D., MBA, Chairman, President, and Chief Executive Officer at Aligos Therapeutics.
  • “In addition, the three ALG-000184 oral presentations demonstrate a consistent effect in the reduction of key viral markers.

Denali Therapeutics Reports Fourth Quarter and Full Year 2023 Financial Results and Business Highlights

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Mardi, février 27, 2024
MPS II, Hearing, ARIA, Antibody, LRRK2, PTV, IDS, Transferrin receptor, MAPT, Traffic barrier, ALS, Hunting, Dermatan sulfate, Therapy, BBB, SAN, Total, Amyloid-related imaging abnormalities, Alpha-synuclein, COMPASS, ETV, Hunter syndrome, Neurodegenerative disease, Conference, MPS, Safety, Biomarker, OTV, LUMA, AFL, SNCA, Amyloid beta, CSF, PIPE, Glycogen storage disease, Research, ATV, FDA, Food, Risk, Sanofi, UC, CD98, Patient, Cognition, Oligonucleotide, Science, Alfa, Pharmaceutical industry

Enrollment continues in the global Phase 2/3 COMPASS study and is expected to be completed in 2024.

Key Points: 
  • Enrollment continues in the global Phase 2/3 COMPASS study and is expected to be completed in 2024.
  • Sanofi intends to present the detailed efficacy and safety results of the ALS Phase 2 HIMALAYA study at a future scientific forum.
  • There was no collaboration revenue for the quarter ended December 31, 2023, compared to $10.3 million for the quarter ended December 31, 2022.
  • Further, for the quarter ended December 31, 2023, there was also a decrease in other unallocated research and development expenses as a result of reduced facility costs.

Denali Therapeutics Announces New Data and Expansion of Its Blood-Brain Barrier (BBB)-Crossing Enzyme Replacement Therapy Programs at WORLDSymposium™

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Mercredi, février 7, 2024
Traffic barrier, Sanfilippo, Genomics, Hearing, Growth, BBB, AFL, Metabolism, Feinberg School of Medicine, Spleen, Dermatan sulfate, Patient, Cerebrospinal fluid, University of North Carolina, FDA, Cognition, COMPASS, Mouse, CSF, Physician, Hunter syndrome, MPS II, Brain, Behavior, MPS, Mucopolysaccharidosis, Hunting, University, SGSH, ETV, Liver, Alfa, Glycogen storage disease, Pharmaceutical industry

Denali’s BBB-crossing enzyme replacement approach has the potential to prevent cognitive and behavioral manifestations in MPS IIIA.

Key Points: 
  • Denali’s BBB-crossing enzyme replacement approach has the potential to prevent cognitive and behavioral manifestations in MPS IIIA.
  • New two-year peripheral biomarker data demonstrate high magnitude and sustained reduction of urine heparan sulfate and dermatan sulfate, including in participants who switched from standard-of-care enzyme replacement therapy to tividenofusp alfa, suggesting enhanced peripheral activity.
  • Denali also announced that dosing has begun in the Phase 1/2 study of DNL126 for the potential treatment of MPS IIIA.
  • “Our goal is to bring effective new medicines to MPS families as soon as possible,” said Carole Ho, M.D., Chief Medical Officer of Denali.

Denali Therapeutics Announces Presentations on Its Investigational Blood-Brain Barrier (BBB)-Crossing Enzyme Replacement Therapies at the Upcoming 2024 WORLDSymposium™

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Jeudi, février 1, 2024
Traffic barrier, SAN, BBB, Biomarker, Poster, Degeneracy, CNS, MPS II, Therapy, MPS, Interim analysis, SGSH, ETV, Glycogen storage disease

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 alfa (DNL310) and DNL126 (ETV:SGSH), to be given at the 20th Annual WORLDSymposium™, which will be held February 4-9, 2024, in San Diego, California.

Key Points: 
  • 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 alfa (DNL310) and DNL126 (ETV:SGSH), to be given at the 20th Annual WORLDSymposium™, which will be held February 4-9, 2024, in San Diego, California.
  • Presentations on DNL310 will include new two-year data on clinical outcomes and previously presented data on biomarkers from the ongoing Phase 1/2 study in MPS II.
  • In addition, an oral presentation will highlight data from preclinical studies of DNL126 in an MPS IIIA mouse model.
  • The presentation schedule at WORLDSymposium™ is as follows:
    Title: DNL310 Phase 1/2 Case Study Demonstrates Properties of Raw, Standard and Growth Scale Scores for Adaptive Behavior Scales (Poster #80)
    Title: DNL310 Normalizes Primary Storage Substrates, Corrects Biomarkers of Lysosomal Dysfunction and Reduces Biomarkers of Neuronal Injury (Neurofilament Light Chain) in MPS II: 2-Year Interim Analysis of a Phase 1/2 Study (Poster #34)
    Title: ETV:SGSH, a Brain-Penetrant Enzyme Transport Vehicle for SGSH, Improves Lysosomal and Microglial Morphology, Degeneration and Cognitive Behavior in MPS IIIA Mice
    Title: 3rd Annual Robert J. Gorlin Symposium – Beyond the Blood Brain barrier: Strategies for Treating the CNS

Denali Therapeutics Announces Key Anticipated 2024 Milestones and Priorities to Further Advance Its Therapeutics Portfolio for Neurodegeneration and Lysosomal Storage Diseases

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Lundi, janvier 8, 2024
CSF, Lysosomal storage disease, Cerebrospinal fluid, Risk, Sanfilippo syndrome, Amyloid, BBB, UC, Sanfilippo, Manuscript, ETV, ALS, OTV, SNCA, IDS, Asos, Conference, Cognition, RIPK1, Fluid mechanics, Neuroinflammation, Premedication, Glycogen storage disease, Amyloid beta, SGSH, Therapy, COMPASS, Traffic barrier, Hunting, N-sulfoglucosamine sulfohydrolase, Hunter syndrome, ISR, NewCo, Amyloid-related imaging abnormalities, Inflammation, CNS, Neurodegenerative disease, Takeda, Biomarker, Alpha-synuclein, K2, Biology, DSM-IV codes, LUMA, Brain, MPS, Investigational New Drug, PTV, ARIA, Nerve, AFL, Pathology, MPS II, Interim, BioRxiv, Plaque, MAPT, LRRK2, Elevator mechanic

The global Phase 2/3 COMPASS study continues to recruit up to 54 participants with neuronopathic and non-neuronopathic MPS II.

Key Points: 
  • The global Phase 2/3 COMPASS study continues to recruit up to 54 participants with neuronopathic and non-neuronopathic MPS II.
  • Upon completion of the ongoing Phase 1/2 study, and together with data from COMPASS, this combined data package is intended to support registration.
  • Increased RIPK1 activity in the CNS is hypothesized to drive neuroinflammation and cell necroptosis and to contribute to neurodegeneration.
  • Denali will maintain ownership of and continue to advance its current portfolio of clinical stage small molecule programs.

Aligos Therapeutics Presents Positive Data at the AASLD Liver Meeting® 2023 Demonstrating that Treatment with ALG-000184 (CAM-E) Results in Significant Multi-log Reductions in Hepatitis B Antigens (HBsAg, HBcrAg and HBeAg)

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Vendredi, novembre 10, 2023
Hepatology, Safety, DNA, SAN, Therapy, PEI, HBsAg, AASLD, Emory University, Liver, Week, Doctor of Philosophy, B, CccDNA, Liver cancer, Hepatitis B virus, CHB, Entecavir, MBA, University, Hepatitis, Hong Kong, Physician, Poster, ETV, RNA, Vaccine, Pharmaceutical industry, HBeAg, Boston, New York University, Gastroenterology, Jacob Aligo

SOUTH SAN FRANCISCO, Calif., Nov. 10, 2023 (GLOBE NEWSWIRE) -- Aligos Therapeutics, Inc. (Nasdaq: ALGS), a clinical stage biopharmaceutical company focused on developing novel therapeutics to address unmet medical needs in liver and viral diseases, today announced that emerging hepatitis B antigen lowering data for its capsid assembly modulator – empty (CAM-E) drug, ALG-000184, are available as a late breaking poster at The Liver Meeting® of the American Association for the Study of Liver Diseases (AASLD), being held in Boston, Massachusetts, November 10 – 14, 2023.

Key Points: 
  • The data will be presented by Dr. Man-Fung Yuen, Chair and Professor of Gastroenterology and Hepatology at the University of Hong Kong, in Poster Hall C on Monday November 13 from 1-2 pm ET.
  • “ALG-000184 appears to have best-in-class antiviral properties which are also unique compared to other drug classes being evaluated for the treatment of chronic hepatitis B (CHB).
  • If the trends observed to date continue, ALG-000184 has the potential to become a cornerstone therapy in the treatment of CHB.
  • The antiviral effects of ALG-000184 presented at AASLD indicate that this drug is achieving significant viral suppression, likely via inhibition of cccDNA synthesis.

Denali Therapeutics Reports Third Quarter 2023 Financial Results and Business Highlights

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Mardi, novembre 7, 2023
Neurofilament light polypeptide, MPS II, CLE, Biomarker, Therapy, MS, BioRxiv, ETV, Ulcerative colitis, BBB, ALS, PTV, ATV, ROFN, Definitive, Traffic barrier, Hunter syndrome, Phase 10, Denali, MPS, Antibody, Biology, Safety, Patient, AFL, Plaque, Fluid mechanics, ASO, ARIA, RIPK1, Asos, Research, Cerebrospinal fluid, ISR, SGSH, DSM-IV codes, Nerve, Brain, Society, Total, OTV, N-sulfoglucosamine sulfohydrolase, Hunting, LUMA, Glycogen storage disease, Manuscript, UC, CSF, Amyloid-related imaging abnormalities, Takeda, COMPASS, Pathology, CNS, Sanfilippo, Neuroinflammation, Collaboration, Multiple sclerosis, Sanfilippo syndrome, IDS, Cognition, Mutation, SAN, Pharmaceutical industry, Medical imaging, Fine chemical, Cryptocurrency, Biogen, LRRK2, Metabolism, Sanofi

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.

Key Points: 
  • 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.
  • Denali has selected five ASO targets for further development and is focused on advancing two OTV candidates towards clinical development.
  • 2023 Guidance on Operating Expenses:
    Cash, cash equivalents, and marketable securities were approximately $1.12 billion as of September 30, 2023.
  • General and administrative expenses were $25.3 million and $23.3 million for the three months ended September 30, 2023 and 2022, respectively.

Denali Therapeutics Announces New Interim Data from Phase 1/2 Study of DNL310 (ETV:IDS) in MPS II (Hunter Syndrome) at SSIEM 2023

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Mercredi, août 30, 2023
Cognition, Learning, Partnership, Society, Event, AFL, Serum, Genomics, ABR, Glycogen storage disease, Hunting, MPS II, Traffic barrier, COMPASS, Biomarker, MPS, Hunter syndrome, BBB, Anne, Patient, Hearing, CSF, ETV, PDF, Infant, Nerve, Report of the 1st Annual Symposium on Relaxed Improvisation, Safety, Disease, Medical imaging, Pharmaceutical industry, Dietary supplement, Metabolism, Genetics, MD

DNL310 is an investigational enzyme replacement therapy designed to cross the BBB and address the behavioral, cognitive, and physical manifestations of MPS II.

Key Points: 
  • DNL310 is an investigational enzyme replacement therapy designed to cross the BBB and address the behavioral, cognitive, and physical manifestations of MPS II.
  • The interim data from the Phase 1/2 study of DNL310 were highlighted in an oral presentation at the Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium 2023 in Jerusalem, Israel.
  • A PDF of the Phase 1/2 presentation is available on Denali’s website on the Events page of the Investor section.
  • We are encouraged to see positive changes across multiple clinical outcomes measures in the ongoing Phase 1/2 study.