Foundation for the National Institutes of Health

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.

Dr. Benjamin Wolozin to Present "Development of Orally Available, Brain Penetrant Compound Reducing Tau Pathology" at CTAD 2023

Retrieved on: 
Tuesday, October 24, 2023
FDA, MIT, Cognition, Vaccine, Neuron, Hope, Takeda, Neurology, LabCentral, Pathology, Boston Park Plaza, Entrepreneurship, Research, Protein, Manhattan Center, Brain damage, Anatomy, Pfizer, ALS, Boston University School of Public Health, Killing Floor 2, Tau, AbbVie, Foundation for the National Institutes of Health, Patient, Kendall Square, Neuroscience, Alzheimer's Association, Memory, Pharmaceutical industry, Medical imaging, Alzheimer's disease

CAMBRIDGE, Mass., Oct. 24, 2023 /PRNewswire/ -- Aquinnah Pharmaceuticals, leaders in stress granule biology that afflict a wide range of neurodegenerative disorders, today announced that the company's Co-Founder and Chief Scientific Officer, Dr. Benjamin Wolozin MD, Ph.D., will be presenting new preclinical research at the 2023 Clinical Trials on Alzheimer's Disease (CTAD) conference. CTAD is the premier industry event highlighting new research and breakthroughs in the areas of disease-modifying agents, gene therapy, vaccines and multimodal interventions.

Key Points: 
  • CTAD is the premier industry event highlighting new research and breakthroughs in the areas of disease-modifying agents, gene therapy, vaccines and multimodal interventions.
  • CTAD 23 is sold out, but attendees can still register for virtual attendance.
  • Our therapeutic removes abnormal tau protein, which is the key protein contributing to loss of memory and cognition in Alzheimer's disease.
  • If successful in clinical trials, Aquinnah's treatment offers the hope of reducing the brain damage and correspondingly restoring brain function for patients suffering from Alzheimer's disease."

Tonix Pharmaceuticals Announces Publication of Data in the Journal Nature Involving TNX-1500 (Fc-modified dimeric anti-CD40L mAb) for the Prevention of Rejection in Kidney Xenotransplantation in Animal Models

Retrieved on: 
Wednesday, October 18, 2023
Massachusetts General Hospital, Faculty, Research, Primate, Gujarat University of Transplantation Sciences, Harvard Medical School, Cadaver, CD40L, Protein engineering, Journal, Tonix Pharmaceuticals, Thrombosis, Columbia, Animal, Transplant, MGH, NIH, Planning, Foundation for the National Institutes of Health, Dietary supplement, Vaccine, CD154, Nature

CHATHAM, N.J., Oct. 18, 2023 (GLOBE NEWSWIRE) -- Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a biopharmaceutical company with marketed products and a pipeline of development candidates, today announced that a study published in the Journal Nature1 by faculty at the Center for Transplantation Sciences, Massachusetts General Hospital (MGH) in collaboration with biotechnology company, eGenesis, utilized TNX-1500 (Fc-modified dimeric anti-CD40L monoclonal antibody [mAb]) as part of the immune modulating regimen to prevent organ transplant rejection. Tonix’s TNX-1500 is in development for the prevention of human kidney organ transplant rejection. The molecular target of TNX-1500 is CD40-ligand (CD40L), which is also known as CD154.

Key Points: 
  • Tonix’s TNX-1500 is in development for the prevention of human kidney organ transplant rejection.
  • TNX-1500 was invented and developed in-house by Seth Lederman, M.D., Chief Executive Officer of Tonix Pharmaceuticals, and colleagues.
  • Preclinical studies in non-human primates demonstrated that TNX-1500 showed activity in preventing allograft and xenograft organ rejection and was well tolerated.
  • In addition, anti-CD40L monoclonal antibodies have demonstrated efficacy in autoimmune diseases like systemic lupus erythematosus and Sjögren’s Syndrome.”

Global Myeloproliferative Disorders Drugs Research Report 2023: Market is Expected to Surpass $10.5 Bn in 2027 - Focus on JAK2 Inhibitors, Anti-Neoplastics, Demethylation Agents, Imatinib Mesylate - ResearchAndMarkets.com

Retrieved on: 
Thursday, October 5, 2023
Biotechnology, Pharmaceutical, Health, Incidence, Imago, Bristol Myers Squibb, Nilotinib, Pancreatic serous cystadenoma, Primary myelofibrosis, FDA, Ageing, Radiation exposure, Merk, Growth, Bone marrow, Polycythemia, DSM-IV codes, Chronic myelogenous leukemia, Foundation for the National Institutes of Health, Pharmaceutical industry

The "Myeloproliferative Disorders Drugs Global Market Report 2023" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Myeloproliferative Disorders Drugs Global Market Report 2023" report has been added to ResearchAndMarkets.com's offering.
  • In the rapidly advancing pharmaceutical landscape, the significance of myeloproliferative disorder drugs is more pronounced than ever.
  • Driving the surge in the market for these drugs is the escalating incidence of myeloproliferative disorders.
  • The countries covered in the myeloproliferative disorder drugs market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Russia, South Korea, UK, USA.

National Institute of Health to Fund Preclinical Development of Trethera Lead Drug, TRE-515, for Treatment of Crohn’s Disease

Retrieved on: 
Monday, September 25, 2023
Transfer, NIDDK, Stenosis, Antigen, Diarrhea, STTR, Weight loss, Cancer, National Institute of Diabetes and Digestive and Kidney Diseases, Research, Abdominal pain, Steroid, Deoxycytidine kinase, Kidney, Patient, Colorectal cancer, Immune system, Cell division, CD4, Lymphocyte, Digestive, Plasma protein binding, NIH, Foundation for the National Institutes of Health, Safety, Fatigue, Disease, Pharmaceutical industry, Vaccine

The award provides $400,000 to translate proof-of-principle early research findings of Trethera’s clinical stage first-in-class drug, TRE-515, as an oral therapy for Crohn’s disease.

Key Points: 
  • The award provides $400,000 to translate proof-of-principle early research findings of Trethera’s clinical stage first-in-class drug, TRE-515, as an oral therapy for Crohn’s disease.
  • Crohn’s disease is a chronic, relapsing, inflammatory gastrointestinal disorder that can present with diarrhea, fatigue, severe abdominal pain, and weight loss.
  • Almost 1 million Americans suffer from the disease today, with complications including intestinal fistulas, strictures, and colorectal cancer.
  • The disease is primarily driven by autoreactive CD4 T cells of the patient’s own immune system that target antigens in the gut.

Soligenix Announces Achievement of Two-Year Stability with Bivalent and Trivalent Thermostabilized Filovirus Vaccines when Stored at High Temperatures

Retrieved on: 
Monday, September 25, 2023
Vaccine, Antigen, University of Colorado School of Medicine, John A. Burns School of Medicine, University, Senior, Vaccination, Associate professor, COVID-19, UHM, Immunity, Temperature, Doctor of Philosophy, Bangladesh Technical Education Board, Biology, Foundation for the National Institutes of Health, Ricin, Administration, Public health, Primate, Tropical medicine, Small business, Theodore Fitz Randolph, Particle size, NIH, Element, Pharmacology

This follows the previous successful demonstration of 100% protection of non-human primates (NHPs) against lethal Sudan ebolavirus and Marburg marburgvirus challenge with the bivalent vaccine.

Key Points: 
  • This follows the previous successful demonstration of 100% protection of non-human primates (NHPs) against lethal Sudan ebolavirus and Marburg marburgvirus challenge with the bivalent vaccine.
  • It further demonstrates the broad applicability of the heat stable vaccine platform, and its potential role in the United States (U.S.) Administration's initiative for pandemic preparedness .
  • There are no licensed vaccines for either Sudan or Marburg viruses, while vaccines for Zaire ebolavirus are constrained by cold chain logistics.
  • This most recent milestone confirms the robust thermostabilization provided by this vaccine platform.

Spinogenix Awarded $3 Million NIH Grant to Support Continued Development of SPG302, the First Synaptic Regenerative Therapeutic for Alzheimer’s Disease

Retrieved on: 
Tuesday, September 12, 2023
Cognition, Central nervous system, Therapy, Research, Doctor of Philosophy, Alzheimer's disease, Dementia, Amyloid, Patient, SAN, Synapse, PMCID, Ageing, NIH, Memory, Foundation for the National Institutes of Health, Breast cancer, Prostate cancer, Pharmaceutical industry

The new $3 million grant titled “Development of a Novel, Regenerative Therapy to Reverse Synapse Loss in Alzheimer's Disease” will continue to support the development of SPG302, a patent-protected compound designed by Spinogenix to help restore the brain connections and functions lost in Alzheimer’s disease and related neurodegenerative disorders.

Key Points: 
  • The new $3 million grant titled “Development of a Novel, Regenerative Therapy to Reverse Synapse Loss in Alzheimer's Disease” will continue to support the development of SPG302, a patent-protected compound designed by Spinogenix to help restore the brain connections and functions lost in Alzheimer’s disease and related neurodegenerative disorders.
  • Spinogenix anticipates that this NIH grant support will accelerate its efforts to bring this novel treatment to patients and potentially slow and reverse the fundamental process of synaptic degeneration at work in Alzheimer’s disease.
  • Spinogenix is advancing SPG302 as the first synaptic regenerative therapy in Alzheimer’s disease.
  • NIH previously awarded Spinogenix a phase one grant (number 1R43AG058278) for its research and development of this unique treatment for Alzheimer’s patients.

The FNIH Announces Study Results Published in Nature Medicine Showing That Liver Disease Can Be Diagnosed Using Noninvasive Blood Tests

Retrieved on: 
Thursday, September 7, 2023
Health, Clinical Trials, Research, Pharmaceutical, Science, Biotechnology, Knowledge, FNIH, Partnership, Drug development, Therapy, NASH, Liver disease, Research fellow, AUROC, Non-alcoholic fatty liver disease, Biomarker, Doctor of Philosophy, Bangladesh Technical Education Board, DSM-IV codes, Patient engagement in Canada, Liver cancer, Foundation for the National Institutes of Health, Kidney, Patient, Risk, Blood, Liver, Fatty liver disease, Mortality, United, Digestive, FDA, MPH, Death, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Cirrhosis, Food, Safety, Biopsy, Medical imaging, Nature Medicine, MD

The research is part of the Biomarkers Consortium’s Noninvasive Biomarkers of Metabolic Liver Disease (NIMBLE) project, a public-private partnership bringing together the FDA, academic researchers, and industry partners to rigorously evaluate blood and imaging tests to measure the presence of liver disease.

Key Points: 
  • The research is part of the Biomarkers Consortium’s Noninvasive Biomarkers of Metabolic Liver Disease (NIMBLE) project, a public-private partnership bringing together the FDA, academic researchers, and industry partners to rigorously evaluate blood and imaging tests to measure the presence of liver disease.
  • Although several companies have developed such tests, none have met the burden of proof needed for FDA regulatory approval.
  • This lack of approval limits the use of these tests for diagnosing NASH and recruiting patients into studies to develop therapies.
  • For more information, visit Non-Invasive Biomarkers of Metabolic Liver Disease (NIMBLE) | FNIH .

Exavir Therapeutics Receives $3M Award from NIH / NIAID to Advance Ultra-Long-Acting Integrase Inhibitor XVIR-110

Retrieved on: 
Tuesday, August 22, 2023
White House, Federal Government, AIDS, Public Policy, Government, Health, Infectious Diseases, Pharmaceutical, Biotechnology, Therapy, HIV, National Institute of Allergy and Infectious Diseases, Pharmacokinetics, CYP3A, CNS, Patient, Central nervous system disease, NIAID, IND, NIH, Infection, Foundation for the National Institutes of Health, Conditional sentence, Pharmaceutical industry

Exavir Therapeutics, a company dedicated to transforming the lives of patients with chronic viral infections and CNS disorders with ultra-long-acting therapeutics, today announced that the company has received a $3M award from the National Institute of Allergy and Infectious Diseases (NIAID), a division of the National Institute of Health (NIH), to support the development of XVIR-110.

Key Points: 
  • Exavir Therapeutics, a company dedicated to transforming the lives of patients with chronic viral infections and CNS disorders with ultra-long-acting therapeutics, today announced that the company has received a $3M award from the National Institute of Allergy and Infectious Diseases (NIAID), a division of the National Institute of Health (NIH), to support the development of XVIR-110.
  • XVIR-110 is an ultra-long-acting HIV integrase inhibitor with a preclinical profile that suggests best-in-class potential for HIV pre-exposure prophylaxis (PrEP), and for HIV treatment as the cornerstone of a next-generation multi-drug regimen.
  • “We are thankful for the NIH’s support of American companies and innovative biotechnology, particularly in an area like HIV / AIDS that affects so many millions of people worldwide,” said Alborz Yazdi, co-founder and CEO of Exavir.
  • “This award speaks to XVIR-110’s transformative potential, but also the importance of American agencies and public-private partnerships in improving population health outcomes.