Tauopathy

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.

Seelos Therapeutics to Present a Poster on SLS-005 in Alzheimer's Disease at Neuroscience 2023

Retrieved on: 
Friday, October 27, 2023
Washington Convention Center, Alzheimer's disease, AFL, Rare, Spinocerebellar ataxia, HD, Autophagy, Mouse, Tauopathy, Research fellow, Walter, Huntington's disease, Society, Therapy, Dietary supplement, ALS, Neuroscience

NEW YORK, Oct. 27, 2023 /PRNewswire/ -- Seelos Therapeutics, Inc. (Nasdaq: SEEL) ("Seelos"), a clinical-stage biopharmaceutical company focused on the development of therapies for central nervous system disorders and rare diseases, today announced that it has been selected to present a poster from a study of SLS-005 (trehalose injection, 90.5 mg/mL for intravenous infusion) in a tauopathy model of Alzheimer's disease at the Society for Neuroscience's Neuroscience 2023 meeting, to be held on November 11-15, 2023, at the Walter E. Washington Convention Center in Washington, D.C.

Key Points: 
  • In this model, tauopathy was induced in older non-human primates (NHPs) through double-tau mutations introduced in entorhinal cortex bilaterally.
  • NfL is a non-specific biomarker for several neurodegenerative conditions, including Alzheimer's disease, Huntington's disease, and amyotrophic lateral sclerosis (ALS).
  • This has now been demonstrated in two aggressive and fast progressing neurodegenerative disease models of Huntington's disease and Alzheimer's disease across two species, mice and non-human primates, respectively," said Krishna Subramanian, Ph.D., Vice President, Non-Clinical Development and Translational Science at Seelos.
  • Seelos will present a poster on Monday, November 13, 2023 at 9:00 a.m.

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.

Seelos Therapeutics Announces Encouraging Data from an In Vivo Study of SLS-005 in an Aggressive Preclinical Model of Alzheimer's Disease

Retrieved on: 
Wednesday, September 27, 2023
ALS, AFL, Research fellow, Neuroscience, Biomarker, Multiple sclerosis, AAV, CNS, Tauopathy, Harvard Medical School, Data, Hospital, Rare, Massachusetts General Hospital, Therapy, Alzheimer's disease, Pharmaceutical industry, Medical imaging, D.C

NEW YORK, Sept. 27, 2023 /PRNewswire/ -- Seelos Therapeutics, Inc. (Nasdaq: SEEL) ("Seelos"), a clinical-stage biopharmaceutical company focused on the development of therapies for central nervous system disorders and rare diseases, today announced encouraging preclinical data from an in vivo study of SLS-005 (trehalose injection, 90.5 mg/mL for intravenous infusion) in an aggressive Alzheimer's disease non-human primate model. Seelos has been selected to present a poster of these data at Neuroscience 2023 to be held on November 11-15, 2023 in Washington, D.C.

Key Points: 
  • Seelos has been selected to present a poster of these data at Neuroscience 2023 to be held on November 11-15, 2023 in Washington, D.C.
  • In this study, the overexpression of tau in older non-human primates (NHPs) was utilized through bilateral AAV induced tauopathy.
  • NHPs in this study were administered either SLS-005 weekly, a single administration of SLS-009 or control.
  • NfL is a non-specific biomarker for several neurodegenerative conditions, including Alzheimer's disease, multiple sclerosis, and amyotrophic lateral sclerosis (ALS).

Oscotec/ADEL Announce FDA Clearance of IND Application of ADEL-Y01 for the Treatment of Alzheimer's Disease

Retrieved on: 
Thursday, September 14, 2023
Partnership, Lysine, Clinical trial, Mild cognitive impairment, Pharmacokinetics, Brain, Tauopathy, Aggregation, Journal, Patient, Immunoglobulin G, Journal of Clinical Investigation, AD, FDA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, IND, Alzheimer's disease, Food, Safety, Pharmaceutical industry, Vaccine

PANGYO, South Korea, Sept. 14, 2023 /PRNewswire/ -- Oscotec Inc. and ADEL Inc. announced that the U.S. Food and Drug Administration (FDA) has cleared the investigational new drug (IND) application of ADEL-Y01 for the treatment of Alzheimer's disease (AD).

Key Points: 
  • PANGYO, South Korea, Sept. 14, 2023 /PRNewswire/ -- Oscotec Inc. and ADEL Inc. announced that the U.S. Food and Drug Administration (FDA) has cleared the investigational new drug (IND) application of ADEL-Y01 for the treatment of Alzheimer's disease (AD).
  • Oscotec and ADEL are jointly developing a novel disease-modifying immunotherapy agent (ADEL-Y01) targeting tau protein accumulation in the AD brain.
  • "The IND clearance by the FDA represents a major achievement for us, especially in light of our first candidate entering into clinical trials," said Seung-Yong Yoon, M.D., CEO of ADEL.
  • "We are enthusiastic about commencing the clinical trial, as it brings us closer to providing therapeutic solutions for the patients grappling with tauopathies including AD."

APRINOIA Therapeutics Announces Strategic Investment From The Alzheimer’s Drug Discovery Foundation (ADDF)

Retrieved on: 
Monday, September 11, 2023
Fillit, Drug development, Therapy, Physician, Personalized medicine, DSM-IV codes, Cancer, PET, Traffic barrier, Medicine, Biomarker, CNS, Tauopathy, Eli Lilly, BBB, MD, Patient, Donanemab, Alpha-synuclein, INN, Diagnosis, Entrepreneurship, AD, FDA, Safety, IND, PSP, Medical imaging, Pharmaceutical industry, Neuroscience

Investment Supports APRINOIA’s Effort to Bring Precision Medicine to Neuroscience, Advancing APRINOIA’s Diagnostic and Therapeutic Pipelines

Key Points: 
  • Investment Supports APRINOIA’s Effort to Bring Precision Medicine to Neuroscience, Advancing APRINOIA’s Diagnostic and Therapeutic Pipelines
    CAMBRIDGE, Mass., Sept. 11, 2023 (GLOBE NEWSWIRE) -- APRINOIA Therapeutics (“APRINOIA” or the “Company”), a clinical-stage biopharmaceutical company developing novel therapeutics and precision diagnostics for the treatment of neurodegenerative diseases, headquartered in Cambridge, MA, is pleased to announce the strategic investment by the Alzheimer’s Drug Discovery Foundation (ADDF) of approximately $4.4M.
  • Accumulation of misfolded tau aggregates is a pathological hallmark of AD and several neurodegenerative diseases collectively known as tauopathies, including PSP.
  • There are very few drug development efforts in neuroscience that are pursuing this potentially transformative approach.
  • With the ADDF’s investment, APRINOIA plans to produce clinical-stage protein degraders capable of penetrating the blood-brain-barrier (BBB), to enter brain cells and clear toxic aggregated forms of tau.

APRINOIA Therapeutics Appoints Mark S. Shearman, Ph.D., as Chief Executive Officer

Retrieved on: 
Monday, August 28, 2023
Pharmacy, Medicinal chemistry, Drug development, Therapy, Doctor of Philosophy, Head, DSM-IV codes, Food, Merck Group, Brain, Merck & Co., Strategic planning, Editas Medicine, Tauopathy, Neuroscience, Dementia, Patient, Drug discovery, Preclinical development, INN, Growth, Diagnosis, Merck, Alzheimer's disease, AD, FDA, Applied Genetic Technologies Corporation, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Death, Synucleinopathy, PSP, Safety, Pharmaceutical industry, Management, IND

Dr. Shearman, who holds a Ph.D. in neuroscience, has extensive experience in pharmaceutical research, drug development and strategic partnerships.

Key Points: 
  • Dr. Shearman, who holds a Ph.D. in neuroscience, has extensive experience in pharmaceutical research, drug development and strategic partnerships.
  • Prior to APRINOIA, he held executive leadership positions at Editas Medicine, Applied Genetic Technologies Corporation, Merck KGaA and Merck & Co., and has served as Chair of APRINOIA’s Scientific Advisory Board since 2015.
  • “It is a great honor to assume the post of CEO of APRINOIA.
  • APRINOIA is focusing on tauopathies and synucleinopathies, that affect approximately 50 million and 10 million people worldwide, respectively.

Oligomerix Publishes Preclinical Data on Novel Small Molecule Therapeutic in Development for Alzheimer’s Disease and Rare Neurodegenerative Diseases

Retrieved on: 
Wednesday, August 9, 2023
Nursing, Biotechnology, FDA, Managed Care, Health, Pharmaceutical, Mental Health, Research, Hospitals, Science, Clinical Trials, Alzheimer's disease, Disease, Memory, Feinstein Institutes for Medical Research, Pathology, MBA, Tauopathy, National Institute on Aging, Bangladesh Technical Education Board, Development, HIV disease progression rates, Patient, Frontotemporal dementia, Mouse, PLOS, Principal, FTD, NIH, News, PSP, Pharmaceutical industry, Eisai

Earlier this year, Oligomerix announced first-in-human dosing of OLX-07010 in a Phase 1a clinical trial.

Key Points: 
  • Earlier this year, Oligomerix announced first-in-human dosing of OLX-07010 in a Phase 1a clinical trial.
  • The self-association of tau protein into progressively larger aggregates and tau tangles is a common pathological process in AD and multiple rare inherited neurodegenerative diseases such as PSP and FTD.
  • Since tau tangles and amyloid plaques are hallmarks of AD, treating both could be more beneficial to patients than targeting a single pathology.
  • Compared to controls, oral treatment with the drug over four months significantly prevented the development of tau aggregates in this mouse model.

Prothena Reports Second Quarter 2023 Financial Results and Business Highlights

Retrieved on: 
Thursday, August 3, 2023
Biotechnology, Pharmaceutical, Health, Society, Failure, PD, ASH, Survival, Pathology, VITAL, CTE, Plasma, AAIC, Acquisition, Patient, Risk, Epitope, R, Fast track (FDA), Therapy, Brain, American Society of Hematology, Alpha-synuclein, MAD, CSF, FDA, IND, Roche, Organ dysfunction, SOC, Neuroscience, Chronic traumatic encephalopathy, Amyloid, AL, Standard of care, Donanemab, Frontotemporal dementia, SPA, European Medicines Agency, Mortality, Research, PSP, Central nervous system, Clinical trial, SAD, Bristol Myers Squibb, CNS, Tauopathy, TTR, Fluid mechanics, Amyloidosis, Novo Nordisk, Orphan, FTD, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Food, Pharmaceutical industry, Vaccine, Lithium, Fine chemical, Security (finance), G&A, Blood, ATTR

Prothena Corporation plc (NASDAQ:PRTA), a late-stage clinical biotechnology company with a robust pipeline of investigational therapeutics built on protein dysregulation expertise, today reported financial results for the second quarter and first six months of 2023 and provided business highlights.

Key Points: 
  • Prothena Corporation plc (NASDAQ:PRTA), a late-stage clinical biotechnology company with a robust pipeline of investigational therapeutics built on protein dysregulation expertise, today reported financial results for the second quarter and first six months of 2023 and provided business highlights.
  • For the second quarter and first six months of 2023, Prothena reported net loss of $54.6 million and $101.5 million, as compared to a net loss of $41.2 million and $77.5 million for the second quarter and first six months of 2022.
  • Research and development (R&D) expenses totaled $56.0 million and $100.8 million for the second quarter and first six months of 2023, as compared to $31.6 million and $58.8 million for the second quarter and first six months of 2022, respectively.
  • Total non-cash share-based compensation expense was $10.1 million and $18.9 million for the second quarter and first six months of 2023, as compared to $8.3 million and $15.9 million for the second quarter and first six months of 2022.