Tauopathy

Amylyx Pharmaceuticals Announces Formal Intention to Remove RELYVRIO®/ALBRIOZA™ from the Market; Provides Updates on Access to Therapy, Pipeline, Corporate Restructuring, and Strategy

Retrieved on: 
Thursday, April 4, 2024
Health, FDA, Neurology, Clinical Trials, Pharmaceutical, Biotechnology, Cell death, Medication, Survival, Wolfram syndrome, ALS, Partnership, Metabolism, Patient, AMX0035, Research, American Academy, OLE, Tauopathy, Health Canada, Biology, AAN, Clinical trial, Endoplasmic reticulum, Palsy, Workforce, Amylyx Pharmaceuticals, Division, Publication, Food, Vance DeGeneres, Pharmaceutical industry

Amylyx will continue to evaluate and share learnings from PHOENIX to help inform future ALS research.

Key Points: 
  • Amylyx will continue to evaluate and share learnings from PHOENIX to help inform future ALS research.
  • At this time, Amylyx intends to continue to collect available data on survival at the encouragement of ALS specialists.
  • “Our pipeline is supported by compelling clinical and preclinical science demonstrating the potential of AMX0035 and AMX0114 in neurodegenerative diseases.
  • “We are so thankful and grateful to our Amylyx team for their contributions and steadfast dedication,” said Cohen and Klee.

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

Retrieved on: 
Wednesday, March 13, 2024
Health, Neurology, Genetics, Clinical Trials, Pharmaceutical, Biotechnology, STAC, Gene, Orphan drug, ALTA, Haemophilia A, BBB, Patient, Health care, Quality of life, IND, Male, ERT, Investment, Therapy, Fabry disease, Plasma, Kite, Gene expression, Partnership, Central nervous system, Novartis, CNS, Pivotal, EMA, Prion, STAAR, Enzyme replacement therapy, Biogen, Tauopathy, Workforce, BLA, AAV, Sanofi, Research, DRG, Neuron, Hematology, FDA, DSM-IV codes, Brain, Sangamo Therapeutics, MAA, Haemophilia, CTA, Mouse, Female, Traffic barrier, Sodium, MHRA, Excipient, Society, Spinal cord, Toxicology, Sigma-Aldrich, Liver, Pharmaceutical industry

Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, today reported recent business highlights and fourth quarter and full year 2023 financial results, including meaningful data to support advancement of its neurology pipeline.

Key Points: 
  • Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, today reported recent business highlights and fourth quarter and full year 2023 financial results, including meaningful data to support advancement of its neurology pipeline.
  • “In 2023, Sangamo announced the prioritization of its pipeline programs that support our focus as a neurology-focused genomic medicine company,” said Sandy Macrae, Chief Executive Officer of Sangamo.
  • STAC-BBB was well tolerated in NHPs, with no notable treatment related pathological findings in brain, spinal cord or peripheral tissues.
  • Revenues for the fourth quarter ended December 31, 2023 were $2.0 million, compared to $27.2 million for the same period in 2022.

Sangamo Therapeutics Announces Data From Novel Proprietary Neurotropic AAV Capsid Demonstrating Industry-leading Blood-brain Barrier Penetration and Brain Transduction in NHPs

Retrieved on: 
Wednesday, March 13, 2024
Research, Neurology, Genetics, Clinical Trials, Health, Pharmaceutical, General Health, Other Science, Science, STAC, AAV, Sangamo Therapeutics, Gene, Brain, Capsid, Gene expression, ALS, BBB, Central nervous system, Novartis, CNS, CTA, DRG, Traffic barrier, Prion, NHP, Tropism, IND, Nav1.7, Neuron, Biogen, Tauopathy, Excipient, Therapy, DSM-IV codes, Spinal cord, Liver, Animal, Vaccine

Sangamo is exploring avenues to resume development of these programs internally, subject to receipt of adequate funding, or with new potential collaborators.

Key Points: 
  • Sangamo is exploring avenues to resume development of these programs internally, subject to receipt of adequate funding, or with new potential collaborators.
  • In NHP studies when administered intravenously at clinically relevant doses, STAC-BBB demonstrated its potential to be a leading neurotropic capsid.
  • Exhibited 700-fold higher transgene expression in neurons compared to the benchmark capsid AAV9 and outperformed all other known published neurotropic capsid variants evaluated in the study.
  • STAC-BBB was well tolerated in NHPs, with no notable treatment related pathological findings in brain, spinal cord or peripheral tissues.

Biosplice Therapeutics Announces Department of Defense (DoD) Award to Fund Collaboration with The Roskamp Institute to Advance its Neurology Program

Retrieved on: 
Friday, March 8, 2024
CFO, SAN, ALS, Roskamp Institute, TBI, DYRK, Therapy, Patient, DSM-IV codes, CLK, CBO, Research, Tauopathy, Pharmaceutical industry

As part of the collaboration, the DoD TBIPHRP Award will fund Roskamp to undertake preclinical development in their well characterized TBI models using Biosplice’s DYRK inhibitors.

Key Points: 
  • As part of the collaboration, the DoD TBIPHRP Award will fund Roskamp to undertake preclinical development in their well characterized TBI models using Biosplice’s DYRK inhibitors.
  • The data from this collaboration are anticipated to inform the development of Biosplice’s DYRK inhibitor class in the treatment of degenerative neurological conditions, including various tauopathies.
  • This collaboration with Roskamp will help accelerate the development of our novel neurology therapies, ranging from TBI to ALS to Alzheimer’s disease.”
    This award builds upon more than four years of scientific collaboration between Roskamp and Biosplice.
  • Previous research conducted by Roskamp has demonstrated efficacy in TBI animal models with Biosplice’s first-generation DYRK inhibitors.

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

The Rainwater Charitable Foundation Announces 2024 Rainwater Prize Winners, Dr. Virginia Man-Yee Lee and Dr. Cristian Lasagna-Reeves, for Ongoing Contributions to Scientific Understanding of Tau in the Brain

Retrieved on: 
Thursday, February 15, 2024
Yee, DNA-binding protein, Tauopathy, BSN, ALS, Research, Dementia, TAR, Nature Neuroscience, PSP, Rain, Pathology, Alpha-synuclein, Neurodegenerative disease, Conference, Frontotemporal dementia, University, Medical laboratory, Drug discovery, Innovation, Associate, Movement, Biology, Brain, Pharmaceutical industry

FORT WORTH, Texas, Feb. 15, 2024 /PRNewswire/ -- The Rainwater Charitable Foundation, one of the largest independent funders of neurodegenerative research that enables field-advancing programs, resources and breakthrough solutions for primary tauopathies, today announced Virginia Man-Yee Lee, Ph.D., University of Pennsylvania Perelman School of Medicine, will be awarded the 2024 Outstanding Innovation in Neurodegenerative Disease Research Prize of $400,000, and Cristian Lasagna-Reeves, Ph.D., M.S., Indiana University School of Medicine will be awarded the Rainwater Prize for Innovative Early-Career Scientist of $200,000. The prizes will be presented during the Tau2024 Global Conference on March 25-26, 2024, in Washington, D.C.

Key Points: 
  • The Rainwater Prize Program , now in its fifth year, aims to highlight and support scientific progress toward addressing critical gaps for neurodegenerative diseases associated with the accumulation of the tau protein in the brain.
  • Currently, Dr. Lasagna-Reeves' lab focuses on understanding the role of tau in Alzheimer's disease and related dementias.
  • Dr. Lasagna-Reeves winning this year's Early-Career Prize recognizes the scientific discoveries that could lead to a potential new therapeutic approach for tauopathies.
  • We're proud to support their research and provide resources that may contribute to potential future breakthroughs in our understanding of tau."

AC Immune’s Targeted Anti-pTau Active Immunotherapy for Alzheimer’s Disease Advances into Phase 2b Trial

Retrieved on: 
Friday, December 15, 2023
Partnership, AC, Deficit spending, Johnson & Johnson, Janssen Pharmaceuticals, Function (mathematics), Biomarker, FDA, Janssen, Injection, Biologics license application, Clinical trial, CHF, Dementia, JAMA, Pathology, Neurotoxicity, Patient, MCI, Memory, Tauopathy, BLA, Vaccine

ACI-35.030 is an investigational targeted active immunotherapy, selective for pathological phosphorylated Tau (pTau).

Key Points: 
  • ACI-35.030 is an investigational targeted active immunotherapy, selective for pathological phosphorylated Tau (pTau).
  • Studies have shown that pTau correlates with AD progression and the trial aims to show that ACI-35.030 can prevent or slow down the progression of tau pathology and onset of clinical symptoms.
  • The partnership with Janssen aims to develop and commercialize therapeutic anti-Tau active immunotherapies for the treatment of AD and potentially other Tauopathies.
  • It is sensitive enough to detect early changes in cognitive function, even before the first clinical signs of mild cognitive impairment (MCI) are apparent2.

Amylyx Pharmaceuticals Announces First Participant Dosed in the Global Phase 3 ORION Study of AMX0035 in Progressive Supranuclear Palsy (PSP)

Retrieved on: 
Friday, December 22, 2023
Professional Services, Health, Finance, Clinical Trials, Pharmaceutical, Biotechnology, Disease, Quality of life, LMU, Neurology, Hospital, Șaeș, PB, Medication, AMX0035, European, Tauopathy, Caregiver burden, MDS, Clinical trial, Movement disorder, Amylyx Pharmaceuticals, PSP, Safety, Pharmaceutical industry

Amylyx Pharmaceuticals, Inc. (NASDAQ: AMLX) (“Amylyx” or the “Company”) today announced that the first participant has been dosed in ORION, a randomized, double-blind, placebo-controlled Phase 3 clinical trial of AMX0035 (sodium phenylbutyrate [PB] and taurursodiol [TURSO]) for the treatment of progressive supranuclear palsy (PSP).

Key Points: 
  • Amylyx Pharmaceuticals, Inc. (NASDAQ: AMLX) (“Amylyx” or the “Company”) today announced that the first participant has been dosed in ORION, a randomized, double-blind, placebo-controlled Phase 3 clinical trial of AMX0035 (sodium phenylbutyrate [PB] and taurursodiol [TURSO]) for the treatment of progressive supranuclear palsy (PSP).
  • Safety and tolerability will be evaluated by assessing the frequency of treatment emergent adverse events (TEAEs) and serious adverse events (SAEs).
  • “AMX0035 has a strong scientific rationale in PSP, targeting key upstream pathways in disease pathophysiology including the unfolded protein response and mitochondrial dysfunction.
  • “Given PSP is a relentlessly progressive disease, it is imperative that we respond to the unmet needs of the community with a sense of urgency.

Transposon Announces Interim Results from a Phase 2 Study of TPN-101 for the Treatment of Progressive Supranuclear Palsy to be Presented at the AD/PD™ 2024 International Conference on Alzheimer's and Parkinson's Diseases

Retrieved on: 
Tuesday, November 14, 2023
Alzheimer's disease, Disease, SAN, Disorder, Therapy, ALS, CSF, PSP, Biotechnology, Neurological disorder, Reverse-transcriptase inhibitor, IL-6, Patient, Conference, Frontotemporal dementia, Neuroinflammation, Progressive supranuclear palsy, Biomarker, International Conference on Creationism, Cerebrospinal fluid, Tauopathy, Transposable element, AFL, Medical imaging, Pharmaceutical industry

SAN DIEGO, Nov. 14, 2023 /PRNewswire/ -- Transposon Therapeutics, a biotechnology company developing a platform of novel, orally administered therapies for the treatment of neurodegenerative and aging-related diseases, today announced its abstract of interim results from its Phase 2 study of TPN-101 for the treatment of progressive supranuclear palsy (PSP) has been accepted for poster presentation at the hybrid AD/PD™ 2024: 18th International Conference on Alzheimer's and Parkinson's Diseases. The meeting will take place online and in Lisbon, Portugal, from March 5-9, 2024.

Key Points: 
  • In the treatment group receiving 400 mg of TPN-101 once daily for 24 weeks, TPN-101 showed an 18.4% reduction in NfL levels in CSF as compared to placebo.
  • "The lowering of CSF NfL levels seen in this interim analysis provides biomarker evidence of a treatment effect on neurodegeneration.
  • We are excited about the potential of TPN-101 as a much-needed treatment option for patients with PSP.
  • Transposon will present results from the interim analysis in a poster presentation (abstract #433) at AD/PD 2024 entitled: "A Phase 2a Study of TPN-101, a Nucleoside Reverse Transcriptase Inhibitor, in Patients with Progressive Supranuclear Palsy."

Aquinnah Pharmaceuticals Advances Oral Small Molecule Program Targeting Alzheimer's and Other Tauopathy Diseases

Retrieved on: 
Monday, October 30, 2023
PSP, TBI, FDA, Research, Cognition, Progressive supranuclear palsy, Aquinnah, Massachusetts, CBD, Pathology, Injection, Entrepreneurship, Administration, Dementia, Brain damage, Disease, ALS, Roche, AbbVie, Foundation for the National Institutes of Health, Patient, Tauopathy, Traumatic brain injury, Alzheimer's Association, Memory, FTD, Medical imaging, Pharmaceutical industry, Alzheimer's disease, Takeda, Pfizer

CAMBRIDGE, Mass., Oct. 30, 2023 /PRNewswire/ -- Aquinnah Pharmaceuticals announced preclinical research findings for a novel therapeutic designed to slow or stop the progression of Alzheimer's disease and related disorders. Aquinnah is a leader in pharmaceutical approaches targeting stress granule biology, which underlies the pathology of a wide range of neurodegenerative disorders. The Aquinnah team announced today that their lead compound targets the interaction of tau with stress granules and removes ~70% of tau pathology in an Alzheimer's animal model with advanced stage disease, measured using three different markers of pathological tau that also increase in patients as their disease progresses. Importantly, the Aquinnah compound is expected to be administered orally as a pill, which is generally preferred by patients over injections that are required currently for approved Alzheimer's immunotherapies.

Key Points: 
  • Aquinnah is a leader in pharmaceutical approaches targeting stress granule biology, which underlies the pathology of a wide range of neurodegenerative disorders.
  • Importantly, the Aquinnah compound is expected to be administered orally as a pill, which is generally preferred by patients over injections that are required currently for approved Alzheimer's immunotherapies.
  • Focused on innovative neurodegenerative research, Aquinnah Pharmaceuticals signed a collaboration agreement with Roche in 2022 to advance oral small molecules for ALS and other neurodegenerative diseases, by modulating TDP-43 pathology, which is the hallmark pathology in more than 95% of ALS patients.
  • Aquinnah has received funding from Pfizer, AbbVie and Takeda, with additional grant funding from the National Institute of Health, the Alzheimer's Association, The Rainwater Foundation and the Mass Life Sciences Center.