Progressive supranuclear palsy

Amylyx Pharmaceuticals Reports Fourth Quarter and Full Year 2023 Financial Results

Retrieved on: 
Thursday, February 22, 2024
Biotechnology, Neurology, Health, Pharmaceutical, Clinical Trials, Week, Regulation, Research, Investment, University, Regulation Fair Disclosure, Webcast, CRP, Intellectual property, Progressive supranuclear palsy, Medication, Plasma, ALS, Marketing, Disclosure, AMX0035, Neurosurgery, Publication, Psychiatry, C-reactive protein, Safety, Biomarker, Amylyx Pharmaceuticals, Clinical trial, Survival, Journal of Neurology, Mutation, IND, Wolfram syndrome, PSP, Otsuka Pharmaceutical, Brain cell, Pharmaceutical industry

Amylyx Pharmaceuticals, Inc. (Nasdaq: AMLX) (“Amylyx” or the “Company”) today reported financial results for the fourth quarter and full year ended December 31, 2023.

Key Points: 
  • Amylyx Pharmaceuticals, Inc. (Nasdaq: AMLX) (“Amylyx” or the “Company”) today reported financial results for the fourth quarter and full year ended December 31, 2023.
  • Net product revenue for the three months ended December 31, 2023 was $108.4 million, compared to net product revenue of $102.7 million for the three months ended September 30, 2023.
  • Amylyx announced the first patient dosed in the Global Phase 3 ORION Study of AMX0035 in Progressive Supranuclear Palsy (PSP).
  • The first participant was dosed in December 2023, and topline results are anticipated in 2025 or 2026.

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.

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.

Amylyx Pharmaceuticals to Present Clinical Trial Design of ORION, a Phase 3 Global Study of AMX0035 in Progressive Supranuclear Palsy (PSP), at the Neuro2023 PSP and CBD International Research Symposium

Retrieved on: 
Thursday, October 19, 2023
Biotechnology, Pharmaceutical, Health, Clinical Trials, PSP, ORION, Progressive supranuclear palsy, Movement disorder, Pan Pacific Hotels and Resorts, Medication, MDS, Neurology, AMX0035, Disease, Aggregation, Ludwig Maximilian University of Munich, Unfolded protein response, HIV disease progression rates, LMU, Quality of life, Department of Neurology, Xuanwu hospital, Caregiver burden, Safety, Hospital, Pharmaceutical industry, Medical imaging, Amylyx Pharmaceuticals, MD

Amylyx Pharmaceuticals, Inc. (Nasdaq: AMLX) (“Amylyx” or the “Company”) today announced an upcoming presentation at Neuro2023 on the design of ORION, a planned global, Phase 3 clinical trial of AMX0035 (sodium phenylbutyrate and taurursodiol [PB&TURSO]) in progressive supranuclear palsy (PSP).

Key Points: 
  • Amylyx Pharmaceuticals, Inc. (Nasdaq: AMLX) (“Amylyx” or the “Company”) today announced an upcoming presentation at Neuro2023 on the design of ORION, a planned global, Phase 3 clinical trial of AMX0035 (sodium phenylbutyrate and taurursodiol [PB&TURSO]) in progressive supranuclear palsy (PSP).
  • Neuro2023: The PSP and CBD International Research Symposium will take place on October 19-20, 2023, at the Pan Pacific Hotel in London, UK.
  • The ORION Phase 3 trial was designed and planned in collaboration with key global academic leaders, people living with PSP, and industry advocacy groups.
  • Additional details regarding trial enrollment and eligibility criteria will be shared upon initiation of the trial, which is anticipated to start by the end of 2023.

CurePSP Awards Latest Pathway and Pipeline Grants and Urso Student Fellowship Grants Looking to Take Advantage of Recent Neuroscience Breakthroughs

Retrieved on: 
Thursday, September 28, 2023
Molecule, Columbia University, Translation, Mario Negri Institute for Pharmacological Research, Columbia University Irving Medical Center, Database, Pedunculopontine nucleus, University, Cell death, Therapy, Icahn School of Medicine at Mount Sinai, Stanford University, Epidemiology, Incyte, Role, ISR, Progressive supranuclear palsy, Allied Social Science Associations, Rossy, Research, Brain, Mount Sinai, Biomarker, Tauopathy, Neuron, Student, Patient, Pharmacological Research, SUNY, Integrated stress response, Neuroscience, CBD, Mouse, Diagnosis, Cognitive neuroscience, Gene, TIA1, Alzheimer's disease, PSP, Nursing, Medical device, Park So-yeon

NEW YORK, Sept. 28, 2023 /PRNewswire/ -- CurePSP has awarded its latest Pathway and Pipeline Grants and Urso Student Fellowships, totaling over $422,000. Recipients of the grants are: Dr. Kurt Farrell, assistant professor at the Icahn School of Medicine at Mount Sinai; Dr. Blas Couto, researcher at the Institute of Translational and Cognitive Neuroscience (INCyT) in Buenos Aires; Dr. Luana Fioriti, head of laboratory at the Mario Negri Institute for Pharmacological Research in Milan, alongside Dr. Carmela Tartaglia, associate professor at the University of Toronto and clinician at the Rossy PSP Centre; and Dr. Joseph B. Rayman, assistant professor of medical sciences at Columbia University Irving Medical Center. Recipients of the Urso Student Fellowship Grants are: Hania Qamar (mentor: Dr. Naomi Visanji), a graduate student at the University of Toronto; Hasnat Nuri (mentor: Dr. Stewart Clark), an undergraduate student at SUNY at Buffalo; and Soyeon Park (mentor: Dr. Jonathan Lin), an undergraduate student at Stanford University. The studies will make progress in identifying biomarkers, studying pathways for therapeutics and creating a new database of patients in Argentina.

Key Points: 
  • NEW YORK, Sept. 28, 2023 /PRNewswire/ -- CurePSP has awarded its latest Pathway and Pipeline Grants and Urso Student Fellowships, totaling over $422,000.
  • Recipients of the Urso Student Fellowship Grants are: Hania Qamar (mentor: Dr. Naomi Visanji), a graduate student at the University of Toronto; Hasnat Nuri (mentor: Dr. Stewart Clark), an undergraduate student at SUNY at Buffalo; and Soyeon Park (mentor: Dr. Jonathan Lin), an undergraduate student at Stanford University.
  • The studies will make progress in identifying biomarkers, studying pathways for therapeutics and creating a new database of patients in Argentina.
  • The newest Urso Student Fellowship grants were awarded to students conducting projects that focus on the complex barriers facing research of PSP and CBD.

Rainwater Charitable Foundation Announces Fourth-Annual Rainwater Prize Winners for Brain Research

Retrieved on: 
Thursday, January 26, 2023
Miller, Conference, Presidential Early Career Award for Scientists and Engineers, Research group, Neurology, Washington University School of Medicine, FDA, Huntington's disease, Brain, Molecular medicine, Disease, Engineering, Innovation, DSM-IV codes, Tauopathy, DZNE, Biophysics, ASO, Nervous system, DRS, SMA, Biology, Neurodegenerative disease, Progressive supranuclear palsy, Neuron, Messenger, DNA, University of California, San Diego, UCSD, Alzheimer's disease, Washington University in St. Louis, Drug discovery, Cell, Tau, Parkinson's disease, University, ALS, German Center for Neurodegenerative Diseases, Distinguishing, Nusinersen, Rain, SOD1, Wegmann, Pharmaceutical industry, Research, Ionis

FORT WORTH, Texas, Jan. 26, 2023 /PRNewswire/ -- The Rainwater Charitable Foundation, one of the largest independent funders of neurodegenerative disease research, today announced this year's recipients of the Rainwater Annual Prize for Outstanding Innovation in Neurodegenerative Disease Research and for Innovative Early Career Scientist. This year's Outstanding Innovation Prize will be awarded to a team: Dr. C. Frank Bennett, Ionis; Dr. Don W. Cleveland, University of California San Diego (UCSD); and Dr. Timothy M. Miller, Washington University. Dr. Susanne Wegmann, German Center for Neurodegenerative Diseases (DZNE) will be awarded the Rainwater Prize for Innovative Early-Career Scientist. The prizes will be presented during the Eurotau 2023 Conference on April 27, 2023 in Lille, France.

Key Points: 
  • FORT WORTH, Texas, Jan. 26, 2023 /PRNewswire/ -- The Rainwater Charitable Foundation, one of the largest independent funders of neurodegenerative disease research, today announced this year's recipients of the Rainwater Annual Prize for Outstanding Innovation in Neurodegenerative Disease Research and for Innovative Early Career Scientist.
  • Dr. Susanne Wegmann, German Center for Neurodegenerative Diseases (DZNE) will be awarded the Rainwater Prize for Innovative Early-Career Scientist.
  • Rainwater Prize for Outstanding Innovation in Neurodegenerative Disease Research will be shared by:
    Collaborators Drs.
  • "I could not be more excited about this year's winners of the Rainwater Prize," said Todd Rainwater, Trustee of the Rainwater Charitable Foundation.

Rainwater Charitable Foundation Announces Fourth-Annual Rainwater Prize Winners for Brain Research

Retrieved on: 
Thursday, January 26, 2023
Miller, Conference, Presidential Early Career Award for Scientists and Engineers, Research group, Neurology, Washington University School of Medicine, FDA, Huntington's disease, Brain, Molecular medicine, Disease, Engineering, Innovation, DSM-IV codes, Tauopathy, DZNE, Biophysics, ASO, Nervous system, DRS, SMA, Biology, Neurodegenerative disease, Progressive supranuclear palsy, Neuron, Messenger, DNA, University of California, San Diego, UCSD, Alzheimer's disease, Washington University in St. Louis, Drug discovery, Cell, Tau, Parkinson's disease, University, ALS, German Center for Neurodegenerative Diseases, Distinguishing, Nusinersen, Rain, SOD1, Wegmann, Pharmaceutical industry, Research, Ionis

FORT WORTH, Texas, Jan. 26, 2023 /PRNewswire/ -- The Rainwater Charitable Foundation, one of the largest independent funders of neurodegenerative disease research, today announced this year's recipients of the Rainwater Annual Prize for Outstanding Innovation in Neurodegenerative Disease Research and for Innovative Early Career Scientist. This year's Outstanding Innovation Prize will be awarded to a team: Dr. C. Frank Bennett, Ionis; Dr. Don W. Cleveland, University of California San Diego (UCSD); and Dr. Timothy M. Miller, Washington University. Dr. Susanne Wegmann, German Center for Neurodegenerative Diseases (DZNE) will be awarded the Rainwater Prize for Innovative Early-Career Scientist. The prizes will be presented during the Eurotau 2023 Conference on April 27, 2023 in Lille, France.

Key Points: 
  • FORT WORTH, Texas, Jan. 26, 2023 /PRNewswire/ -- The Rainwater Charitable Foundation, one of the largest independent funders of neurodegenerative disease research, today announced this year's recipients of the Rainwater Annual Prize for Outstanding Innovation in Neurodegenerative Disease Research and for Innovative Early Career Scientist.
  • Dr. Susanne Wegmann, German Center for Neurodegenerative Diseases (DZNE) will be awarded the Rainwater Prize for Innovative Early-Career Scientist.
  • Rainwater Prize for Outstanding Innovation in Neurodegenerative Disease Research will be shared by:
    Collaborators Drs.
  • "I could not be more excited about this year's winners of the Rainwater Prize," said Todd Rainwater, Trustee of the Rainwater Charitable Foundation.

SYNTap Biomarker Test helps doctors distinguish Parkinson's Disease from Parkinsonism in early stages

Retrieved on: 
Wednesday, April 6, 2022
Linda, Substantia nigra, Chittagong Veterinary and Animal Sciences University, Southern Illinois University School of Medicine, Neurology, Diagnosis, Neurodegeneration, Oregon Health & Science University, Movement disorders, Dopamine, Body, Synuclein, School, Oregon Health Plan, PD, Basal ganglia, History, Alzheimer's disease, Neurological disorder, Progressive supranuclear palsy, Autopsy, Parkinson's disease, Time, Physician, Amprion, Movement, SAN, FDA, Patient, Disease, Medical imaging, Palladium, Pharmaceutical industry, Medical device, Parkinsonism

SAN FRANCISCO, April 6, 2022 /PRNewswire-PRWeb/ -- PD affects 10 million people globally. About one million Americans live with PD, and 60,000 new cases are added each year. Unfortunately, despite billions of spending in drug R&D over the past three decades, there is no cure for Parkinson's–yet.

Key Points: 
  • For the first time in history, Amprion's breakthrough SYNTap Biomarker Test can help doctors effectively distinguish Parkinson's Disease (PD) from Parkinsonism early.
  • Clinical diagnosis of PD is challenging, particularly at early stages when treatments are likely to be more effective.
  • For the first time in history, Amprion's breakthrough SYNTap Biomarker Test can help doctors effectively distinguish PD from Parkinsonism in early stages.
  • Our SYNTap Biomarker Test helps doctors diagnose Parkinson's, Lewy Body Dementia, and Alzheimer's with Lewy Bodies.

Worldwide Progressive Supranuclear Palsy (PSP) Therapies Industry to 2030 - ResearchAndMarkets.com

Retrieved on: 
Thursday, December 16, 2021
Health, Other Health, Palliative care, Curative, Element, 2X2, Population, III, Industry, Initial public offering, D, Adoption, Lists of people executed in the United States, Progressive supranuclear palsy, Research, Patient, R, PSP, Investment, IP, Partnership, II, Type, Healthcare, Pharmaceutical industry, Risk management, Financial services

The report features an extensive study on the marketed, clinical and preclinical molecules available/being developed for the treatment of Progressive Supranuclear Palsy.

Key Points: 
  • The report features an extensive study on the marketed, clinical and preclinical molecules available/being developed for the treatment of Progressive Supranuclear Palsy.
  • One of the key objectives of the report was to estimate the existing market size and the future opportunity of therapies for Progressive Supranuclear Palsy, over the next decade.
  • What are the prevalent R&D trends related to Progressive Supranuclear Palsy?
  • What are the key geographies where research on Progressive Supranuclear Palsy is being conducted?