Alpha-synuclein

Vanqua Bio Announces First Patient Dosed in Phase 1 Clinical Trial Evaluating VQ-101, its Small Molecule GCase Activator for GBA-Parkinson’s Disease and Related Disorders

Retrieved on: 
Tuesday, April 9, 2024
Cell, Recycling, PD, Patient, Glucocerebrosidase, Doctor of Philosophy, Alpha-synuclein, Trial of the century, Protein, Neuron, Safety, Pharmacokinetics, Lysosome

CHICAGO, April 09, 2024 (GLOBE NEWSWIRE) -- Vanqua Bio, a clinical-stage biopharmaceutical company dedicated to discovering and developing next-generation medicines for the treatment of neurodegenerative diseases, announced that the first patient has been dosed in a first-in-human Phase 1 clinical study evaluating VQ-101 in healthy individuals and patients with various forms of Parkinson’s disease (PD). VQ-101 is an orally administered brain-penetrant small molecule allosteric activator of the lysosomal enzyme glucocerebrosidase (GCase).

Key Points: 
  • VQ-101 is an orally administered brain-penetrant small molecule allosteric activator of the lysosomal enzyme glucocerebrosidase (GCase).
  • “VQ-101 demonstrated promising efficacy, safety, pharmacokinetics, and target engagement in preclinical studies.
  • Mutations in GBA1 are the most common genetic risk factor for PD, representing approximately 10% of patients with PD worldwide.
  • In developing VQ-101, Vanqua is adopting a precision-medicine approach that focuses initially on patients with GBA-PD, the largest genetically defined segment of PD.

CervoMed Reports Fourth Quarter and Full Year 2023 Financial Results and Provides Corporate Updates

Retrieved on: 
Monday, April 1, 2024
Lewy body, Research, GBA, Administration, Plasma, Conference, MRI, DLB, GFAP, Basal forebrain, Patient, Journal, Chairperson, Sale, TID, Manuscript, Public expenditure, Biomarker, Glial fibrillary acidic protein, MD, University College London, Atrophy, Publication, Frontotemporal dementia, Alpha-synuclein, NIA, Research and development, Vertex Pharmaceuticals, Axonal transport, Dementia, CRVO

BOSTON, April 01, 2024 (GLOBE NEWSWIRE) -- CervoMed Inc. (NASDAQ: CRVO), a clinical stage company focused on developing treatments for age-related neurologic disorders, today reported its financial results for the fourth quarter and full year ended December 31, 2023.

Key Points: 
  • Moreover, the neflamapimod treatment effects on GFAP were correlated to clinical outcomes, assessed by the CDR-SB.
  • Cash Position: As of December 31, 2023, CervoMed had $7.8 million in cash and cash equivalents as compared to $4.1 million as of December 31, 2022.
  • This increase was attributed to the RewinD-LB Phase 2b clinical study in DLB which began in the first quarter of 2023.
  • Net Loss: Net Loss was $2.2 million for the year ended December 31, 2023, compared to a net loss of $5.8 million for year ended December 31, 2022.

Breakthrough Results of NIH-Sponsored Study of Syn-One Test® as a Skin-Based Diagnostic Tool for Parkinson’s Disease and Related Disorders Published in the Journal of the American Medical Association

Retrieved on: 
Wednesday, March 20, 2024
Research, Neurology, Clinical Trials, Biotechnology, Other Health, Health, Pharmaceutical, Other Science, Science, JAMA, Lewy body, Diagnosis, Harvard Medical School, American Medical Association, DLB, MSA, Disease, PD, Multiple system atrophy, Alpha-synuclein, Physician, Journal, CND, MD, NIH, Synucleinopathy, Skin, Pathology, Bangladesh Technical Education Board, Medicare, PAF, Dementia, Phosphorylation, Medical imaging

The primary goal of this study was to define the positivity rate of skin biopsies to detect P-SYN deposition in clinically confirmed cases of PD, MSA, DLB, and PAF.

Key Points: 
  • The primary goal of this study was to define the positivity rate of skin biopsies to detect P-SYN deposition in clinically confirmed cases of PD, MSA, DLB, and PAF.
  • The proportion of patients with P-SYN was 92.7% in those with PD, 98.2% in those with MSA, 96.0% in those with DLB, and 100% in those with PAF.
  • Future disease-modifying therapies will require convenient, accurate, and precise diagnostic tools to reach the right patients at the right time across a wide array of communities.
  • “The publication of this important NIH-sponsored study is one big step forward with many more milestones to come.”

BioArctic: New data on lecanemab presented at the 2024 AD/PD™ congress

Retrieved on: 
Monday, March 11, 2024
Diagnosis, Brain, Conference, Lecanemab, Disease, Incidence, Alpha-synuclein, Eisai, Patient, CAA, ARIA, DSM-IV codes, Antibody, Safety, Clarity, Pharmaceutical industry

BioArctic's founder, Professor Lars Lannfelt, presented data on the binding properties of the anti-amyloid-beta (Aβ) antibody lecanemab and other anti Aβ antibodies.

Key Points: 
  • BioArctic's founder, Professor Lars Lannfelt, presented data on the binding properties of the anti-amyloid-beta (Aβ) antibody lecanemab and other anti Aβ antibodies.
  • Lecanemab was designed to preferentially bind soluble (protofibrils), as well as insoluble Aβ aggregates (fibrils), to reduce both Aβ protofibrils and Aβ plaques in the brain.
  • The difference in binding to CAA[1] was presented, with lecanemab having lower binding to CAA than most other antibodies.
  • This could explain the difference in the adverse event ARIA[2] seen between different antibodies, with lecanemab showing relatively low incidence of ARIA.

CervoMed Announces Presentation of Biomarker Data from the AscenD-LB Phase 2a Trial and Preclinical Data Supporting Potential of Neflamapimod in Tau-Mediated Disease at AD/PD ™ 2024

Retrieved on: 
Tuesday, March 5, 2024
Axonal transport, Tauopathy, Neuron, CRVO, Cell biology, DLB, Plasma, Medical laboratory, MB, Phosphorylation, Overalls, FTD, GS, Vrije Universiteit Amsterdam, Publication, MAPK, Glial fibrillary acidic protein, GFAP, Axon, Brain, CNR, IW, EM, SDL, UCL, RAF kinase, Alpha-synuclein, Conference, Biomarker, AG, Lewy body, GBA, MD, Neurochemistry, PDF, JC, CP, Patient, Dementia, Basal forebrain, JA, Medical imaging

BOSTON, March 05, 2024 (GLOBE NEWSWIRE) -- CervoMed Inc. (NASDAQ: CRVO), a clinical stage company focused on developing treatments for degenerative diseases of the brain, today announced the presentation of biomarker data from the AscenD-LB Phase 2a trial of neflamapimod in patients with dementia with Lewy bodies (DLB), demonstrating that neflamapimod reduces plasma levels of glial fibrillary acidic protein (GFAP) compared placebo, and that the effects of neflamapimod on GFAP were inversely correlated to change in CDR-SB (reduction in GFAP associated with improvement in CDR-SB, and increase in GFAP associated with worsening in CDR-SB). These data will be featured in a poster presentation at the 18th International Conference on Alzheimer’s and Parkinson’s Diseases (AD/PD™) 2024, being held both virtually and in Lisbon, Portugal from March 5–9, 2024. In addition, academic researchers from UCL will be presenting data in a separate poster at the meeting demonstrating that p38MAPK inhibition, including with neflamapimod specifically, improves tau-induced axonal transport defects both in vitro and in a tauopathy mouse model.

Key Points: 
  • These data will be featured in a poster presentation at the 18th International Conference on Alzheimer’s and Parkinson’s Diseases (AD/PD™) 2024, being held both virtually and in Lisbon, Portugal from March 5–9, 2024.
  • “The effects on GFAP, particularly the association between GFAP response and clinical outcomes, further support that neflamapimod is clinically efficacious in patients with DLB,” said John Alam, MD, Chief Executive Officer of CervoMed.
  • A PDF copy of the GFAP poster presentation will be available on the “ Presentations and Publications ” section of the CervoMed website.
  • Title: NEW INSIGHTS IN THE DEVELOPMENT OF BIOMARKERS, IMAGING, AND THERAPY OF ALPHA-SYNUCLEIN, LRKK2, AND GBA PATHOLOGIES

Biogen Highlights New Data at the International Conference on Alzheimer’s and Parkinson’s Diseases (AD/PD™) 2024 Annual Meeting

Retrieved on: 
Monday, March 4, 2024
OGA, Research, BIIB, Investment, Eisai, Alpha-synuclein, Amyloid, Conference, Biogen, Quantitative systems pharmacology, Dementia, MCI, QSP, Clarity, MAPT, Brain, Vaccine

The presentations include new data for its oral small molecule inhibitor of tau aggregation (BIIB113), as well as presentations providing insights into the underlying mechanisms of Alzheimer’s disease.

Key Points: 
  • The presentations include new data for its oral small molecule inhibitor of tau aggregation (BIIB113), as well as presentations providing insights into the underlying mechanisms of Alzheimer’s disease.
  • In addition to BIIB113, Biogen is researching the potential of tau reduction in AD with its investigational antisense oligonucleotide targeting the microtubule associated protein tau (MAPT) gene (BIIB080).
  • Oral presentation: Results of the first in-human, randomized, blinded, placebo-controlled, single- and multiple-ascending dose study of BIIB113 in healthy volunteers.
  • Oral presentation: Lecanemab for the treatment of early Alzheimer’s disease; the extension of efficacy results from Clarity AD.

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.

Annovis Bio Announces Participation in Forum Discussion at AD/PD™ 2024

Retrieved on: 
Tuesday, February 27, 2024
Master of Science, PD, Doctor of Philosophy, Medical school, GBA, Alpha-synuclein, Conference, EMBA, Parkinson's disease, Medical University of Innsbruck, Biomarker, Mayo Clinic, Multiple system atrophy, Patient, Dementia, Synucleinopathy, Development, DSM-IV codes, Lewy body, Biogen, Management

MALVERN, Pa., Feb. 27, 2024 (GLOBE NEWSWIRE) -- Annovis Bio, Inc. (NYSE: ANVS), a clinical-stage drug platform company developing novel therapies for neurodegenerative diseases today announced panel participation in the International Conference on Alzheimer’s and Parkinson’s Diseases (AD/PD™ 2024) , taking place in Lisbon, Portugal March 5-9, 2024.

Key Points: 
  • MALVERN, Pa., Feb. 27, 2024 (GLOBE NEWSWIRE) -- Annovis Bio, Inc. (NYSE: ANVS), a clinical-stage drug platform company developing novel therapies for neurodegenerative diseases today announced panel participation in the International Conference on Alzheimer’s and Parkinson’s Diseases (AD/PD™ 2024) , taking place in Lisbon, Portugal March 5-9, 2024.
  • Maria Maccecchini, PhD, Founder, President, and CEO of Annovis, will join a forum discussion focused on new approaches for alpha-synuclein (αSyn), LRKK2, and GBA pathologies.
  • Aggregated αSyn is the major constituent of Lewy bodies and a pathogenic hallmark of all synucleinopathies, including Parkinson's disease, dementia with Lewy bodies, and multiple system atrophy.
  • It is present in a number of other neurodegenerative diseases and is responsible for axonal impairment and synaptic degeneration.

AC Immune Announces Upcoming Presentations at AD/PD™ 2024

Retrieved on: 
Thursday, February 22, 2024
PD, Alpha-synuclein, Conference, Down syndrome, Medicine, Synucleinopathy, Diagnosis, Primate

Lausanne, Switzerland, February 22, 2024 – AC Immune SA (NASDAQ: ACIU), a clinical-stage biopharmaceutical company pioneering precision medicine for neurodegenerative diseases, today announced upcoming presentations highlighting its precision medicine pipeline at the International Conference on Alzheimer’s and Parkinson’s Diseases (AD/PD™ 2024), taking place in Lisbon, Portugal March 5-9, 2024.

Key Points: 
  • Lausanne, Switzerland, February 22, 2024 – AC Immune SA (NASDAQ: ACIU), a clinical-stage biopharmaceutical company pioneering precision medicine for neurodegenerative diseases, today announced upcoming presentations highlighting its precision medicine pipeline at the International Conference on Alzheimer’s and Parkinson’s Diseases (AD/PD™ 2024), taking place in Lisbon, Portugal March 5-9, 2024.
  • In addition, Andrea Pfeifer, Ph.D., CEO of AC Immune SA will lead an industry symposium exploring the latest clinical advances in the diagnosis and treatment of alpha-synuclein pathologies.
  • Active immunotherapy ACI-7104.056 in early PD: update from the Phase 2 trial VacSYn
    Utility of [18F]PI-2620 PET in clinical trials: insights into tau pathology deposition in Down Syndrome
    Discovery and preclinical development of [18F]ACI-19626, a first-in-class TDP-43 PET tracer
    VacSYn study: An innovative biomarker-based phase 2 clinical trial to evaluate ACI-7104.056, a novel active immunotherapy for Parkinson’s disease
    ACI-7104.056, an active immunotherapy for synucleinopathies, induces a strong and sustained antibody response against alpha-synuclein in non-human primates

CND Life Sciences Partners with Visiopharm on Novel AI-Tool to Detect and Quantify Pathological Alpha-Synuclein in Cutaneous Nerves

Retrieved on: 
Tuesday, March 5, 2024
Parkinson's disease, Marie Cassidy, Algorithm, Ariz, CND, MD, Pathology, Drug development, Alpha-synuclein, Neurology, LDT, Disease, Patient, Clinical trial, Sympathetic nervous system, Synucleinopathy, Diagnosis, Machine learning, Three-body problem, Deep learning, Lewy body, Partnership, Medical imaging

SCOTTSDALE, Ariz.., March 5, 2024 /PRNewswire/ -- CND Life Sciences (CND), a medical technology company pioneering the development of cutaneous neurodiagnostic tests and associated biomarker services, today will unveil its NerValence pathology detection system at the AD/PD 2024 conference in Lisbon, Portugal. NerValence, developed through a strategic collaboration with precision pathology company Visiopharm, significantly enhances CND's biomarker testing services for clinical trials targeting the alpha-synuclein protein in patients with suspected Parkinson's disease, dementia with Lewy bodies, and related disorders.

Key Points: 
  • NerValence aids in the identification, tracking, and measuring the volume of alpha-synuclein in cutaneous nerves.
  • NerValence features machine learning algorithms that recognize and differentiate microscopic structures, nerves, and important disease markers that can be found in cutaneous tissue.
  • "Utilizing our novel deep learning technology to power this crucial advancement in neurodegenerative diseases represents a significant leap forward in analyzing peripheral nerves," said Michael Grunkin , Visiopharm Chief Executive Officer.
  • "Collaborating with CND Life Sciences on its deployment of NerValence will continue to be a major priority as we advance AI-driven precision pathology to enhance biopharmaceutical drug development."