MPS II

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

Retrieved on: 
Tuesday, February 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.

JCR Pharmaceuticals’ Research Presentations at WORLDSymposium™ 2024 Showcase JR-141 (Pabinafusp Alfa) and Other Investigational Treatments for Lysosomal Storage Disorders

Retrieved on: 
Wednesday, February 14, 2024
Science, Other Science, Biotechnology, Research, Pharmaceutical, General Health, Health, Clinical Trials, Enzyme replacement therapy, BBB, JCR, MPS II, Traffic barrier, Glycogen storage disease, Ministry, MHLW, ERT, Hunter syndrome, Syndrome, Scheie syndrome, MPS, Safety, Developmental psychology, Hurler syndrome, Child, Patient, Hurler–Scheie syndrome, Trial of the century, Consciousness, DSM-IV codes, Hypotension, Mouse, Pharmaceutical industry

JCR Pharmaceuticals Co., Ltd. (TSE 4552; Chairman and President: Shin Ashida; “JCR”) today announced the presentation of several datasets demonstrating the potential benefits of its investigational therapies for lysosomal storage disorders (LSDs).

Key Points: 
  • JCR Pharmaceuticals Co., Ltd. (TSE 4552; Chairman and President: Shin Ashida; “JCR”) today announced the presentation of several datasets demonstrating the potential benefits of its investigational therapies for lysosomal storage disorders (LSDs).
  • JCR is dedicated to address the unmet medical needs for this community,” said Shin Ashida, President and CEO of JCR Pharmaceuticals.
  • Finally, the third JR-141 presentation is pre-clinical and highlights the recovery of retinal function in mice with MPS II.
  • “We are pleased to be able to offer IZCARGO® to patients with MPS II in Japan,” said Yoshikatsu Eto, M.D., Ph.D., Institute of Neurological Disorders, Advanced Clinical Research Center, Kanagawa, Japan.

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

Retrieved on: 
Wednesday, February 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.

Gain Therapeutics’ GT-02287 Completely Restores Motor Function in Mouse Models of Parkinson’s Disease

Retrieved on: 
Tuesday, February 6, 2024
Neuroprotection, Poster, Pharmacokinetics, ALS, AFL, Clinical trial, Patient, Safety, Mutation, FDA, Animal, Food, MPS II, Disease, Therapy, MPS, Neurofilament, SOD1, Glucocerebrosidase, GBA1, Plasma, Pharmaceutical industry

The data was accepted as a late-breaker abstract and will be presented at the 20th Annual WORLDSymposium™ being held in San Diego this week.

Key Points: 
  • The data was accepted as a late-breaker abstract and will be presented at the 20th Annual WORLDSymposium™ being held in San Diego this week.
  • “We believe the data showing complete restoration of motor function in a therapeutic model are remarkable and further support the potential of GT-02287 to slow or stop the progression of Parkinson’s disease, a disease for which only symptomatic treatments are available to patients at this time,” said Matthias Alder, Gain Therapeutics’ Chief Executive Officer.
  • Further, animals in the most challenging treatment group – those that began treatment eight days following onset of the disease – showed motor improvement from day 14 to day 27, which suggests progressive reversal of neuronal deficit associated with continued treatment duration.
  • Further details of the study, including protocol and specific results can be found in the poster, which was presented today and can be accessed here .

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

Retrieved on: 
Thursday, February 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

REGENXBIO Announces Presentations at the 20th Annual WORLDSymposium 2024

Retrieved on: 
Wednesday, January 31, 2024
Hospital, Trial of the century, Syndrome, Central nervous system, Hunter syndrome, MPS II, MPS, Phase, Glycogen storage disease, Webcast, Medical device

ROCKVILLE, Md., Jan. 31, 2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced presentations on its program for the treatment of mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome, will be shared at the 20th Annual WORLDSymposium™ 2024, taking place in San Diego, CA February 3-9, 2024.

Key Points: 
  • ROCKVILLE, Md., Jan. 31, 2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced presentations on its program for the treatment of mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome, will be shared at the 20th Annual WORLDSymposium™ 2024, taking place in San Diego, CA February 3-9, 2024.
  • The presentations include the topline results from the pivotal phase of the Phase I/II/III CAMPSIITE® trial of RGX-121 for the treatment of MPS II.
  • The poster presentations will be presented as follows:
    REGENXBIO will host a conference call Wednesday, February 7 at 4:30 p.m.
  • A replay of the webcast will also be available via the Company's investor website approximately two hours after the call's conclusion.

JCR Pharmaceuticals to Present at the 20th Annual WORLDSymposium™ 2024

Retrieved on: 
Thursday, January 25, 2024
Health, Other Science, General Health, Research, Science, Pharmaceutical, Biotechnology, Hurler, Patient, Enzyme replacement therapy, PST, Hunter syndrome, MPS II, JCR, Eto, BBB, MPS, Medicine, MHLW, Ikeda, ERT, Syndrome, Safety, Traffic barrier, Glycogen storage disease, Scheie syndrome, Ministry, Pharmaceutical industry

JCR Pharmaceuticals Co., Ltd. (TSE 4552; Chairman and President: Shin Ashida; “JCR”) announced today that it will present six presentations at the 20th Annual WORLDSymposium™ 2024, to be held February 4-9, 2024 in San Diego, Calif.

Key Points: 
  • JCR Pharmaceuticals Co., Ltd. (TSE 4552; Chairman and President: Shin Ashida; “JCR”) announced today that it will present six presentations at the 20th Annual WORLDSymposium™ 2024, to be held February 4-9, 2024 in San Diego, Calif.
  • In addition to posters being available for each of the presentations, two of them will be shared as oral presentations given by a clinical investigator and a researcher from JCR Pharmaceuticals.
  • A global phase III study of pabinafusp alfa (JR-141) for neuronopathic mucopolysaccharidosis type II: updated study design (Ikeda, et al.)
  • WORLDSymposium™ attendees who would like to receive more information about JCR Pharmaceuticals can visit JCR’s on-site conference booth (#506) or visit its virtual booth on the WORLDSymposium™ conference website.

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

Retrieved on: 
Monday, January 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.

Denali Therapeutics Reports Third Quarter 2023 Financial Results and Business Highlights

Retrieved on: 
Tuesday, November 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

Retrieved on: 
Wednesday, August 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.