Neuromuscular junction

NIH Awards Hesperos Research Grant to Develop Animal-Free Botox Testing Platform

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星期二, 五月 7, 2024
Health, FDA, Stem Cells, Research, Science, Pharmaceutical, Biotechnology, NMJ, NIH, Safety, Food, Cell, Animal, Acetylcholine, Spasticity, Botulinum toxin, MLB, Santhanam, Neurotoxin, Brain cell, Strabismus, Biology, Paralysis, Drug development, NCATS, Migraine, Drug discovery, Neuromuscular junction, Pharmaceutical industry

The goal of the effort is to develop an advanced testing platform capable of assessing the potency of the Botulinum Toxin (BoT) without the need for animal testing.

Key Points: 
  • The goal of the effort is to develop an advanced testing platform capable of assessing the potency of the Botulinum Toxin (BoT) without the need for animal testing.
  • Despite its therapeutic benefits, BoT is one of the most potent neurotoxins known to man, necessitating stringent batch testing to ensure consistent potency.
  • By successfully developing a high-throughput, human-based platform, Hesperos endeavors to obviate the need for animal testing in this process while simultaneously enhancing the safety profile of BoT.
  • https://fda.gov
    The mission of National Center for Advancing Translational Sciences (NCATS) at NIH is to turn research observations into health solutions through translational science.

Curi Bio, Genetox, and DreamCIS Sign MOU for Strategic Collaboration on 3D Neuromuscular Junction Model for Botox Potency Assay

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星期四, 二月 22, 2024
Biotechnology, Health, Genetics, Stem Cells, Pharmaceutical, Korean, Therapy, Bangladesh Technical Education Board, ALS, Multimedia, Small business, NIH, FDA, US FDA, Food, Sale, Drug discovery, Small Business Innovation Research, Quality control, Charcot–Marie–Tooth disease, University, Chronic pain, CMT, Neuromuscular junction, Patient, NCATS, MOU, NMJ, Partnership, Pharmaceutical industry

View the full release here: https://www.businesswire.com/news/home/20240222795787/en/

Key Points: 
  • View the full release here: https://www.businesswire.com/news/home/20240222795787/en/
    Curi Bio, Genetox, and DreamCIS Sign MOU for Strategic Collaboration on 3D Neuromuscular Junction Model for Botox Potency Assay (Photo: Business Wire)
    BoT is renowned for its therapeutic applications, including the treatment of chronic pain, autonomic disorders, congenital neuromuscular conditions, and cosmetic enhancements.
  • The project has supported Curi Bio’s development of a next-generation 3D human neuromuscular junction model used for disease modeling, for pharmaceutical and cosmetics potency assays, and for testing broad pathogenicity.
  • The project will deliver a functional potency assay for botox research and manufacture in a turnkey, scalable format.
  • With this MOU, Genetox shows its commitment to making Curi Bio’s NMJ-based potency assay a key part of BOTAONE’s manufacturing, quality control, and product release processes.

Athira Pharma Announces Publication in Frontiers in Neuroscience Highlighting Therapeutic Potential of ATH-1105 in Amyotrophic Lateral Sclerosis (ALS)

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星期四, 二月 8, 2024
Human, Inflammation, Survival, ALS, Athira Pharma, Pathology, Sympathetic nervous system, Frontiers, Vance DeGeneres, AFL, HGF, Doctor of Philosophy, Neuromuscular junction, Plasma, Research, Biomarker

ATH-1105 is a next-generation, orally administered small molecule drug candidate designed to enhance the neurotrophic hepatocyte growth factor (HGF) system.

Key Points: 
  • ATH-1105 is a next-generation, orally administered small molecule drug candidate designed to enhance the neurotrophic hepatocyte growth factor (HGF) system.
  • “These data demonstrate that ATH-1105 treatment results in significant, consistent beneficial effects both in cell culture and in vivo models of ALS.
  • “There is an urgent need for new ALS treatment options, particularly those aimed at slowing or stopping neurodegeneration,” said Mark Litton, Ph.D., President and Chief Executive Officer of Athira.
  • These findings further support our plans to progress ATH-1105 into first-in-human studies in the first half of 2024.”

European Medicines Agency (EMA) Greenlights Mitochon Pharmaceuticals to Initiate Phase I/IIa Biomarker Study in Neurodegenerative Diseases

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星期二, 一月 9, 2024
Science, Neurology, Biotechnology, Research, Pharmaceutical, Managed Care, Health, Clinical Trials, Patient, TBI, HD, CSO, Survival, Memory, Neuromuscular junction, EMA, MP101, Autoimmunity, Disease, Medication, Central nervous system, Safety, Traumatic brain injury, Biomarker, Concussion

Mitochon Pharmaceuticals today announced that it was awarded approval from EMA to begin enrollment for a Phase I/IIa biomarker study in Amyotrophic Lateral Sclerosis (ALS), Multiple Sclerosis (MS), Huntington’s Disease (HD) and Alzheimer’s Disease (AD) participants.

Key Points: 
  • Mitochon Pharmaceuticals today announced that it was awarded approval from EMA to begin enrollment for a Phase I/IIa biomarker study in Amyotrophic Lateral Sclerosis (ALS), Multiple Sclerosis (MS), Huntington’s Disease (HD) and Alzheimer’s Disease (AD) participants.
  • Mitochon’s MP101, a once-daily, oral brain penetrant mitochondrial stimulator, that improves central nervous system survival and function will be used in this 14-day pilot study.
  • The aim of the study is to show safety in the target patient populations and demonstrate meaningful changes in disease specific biomarkers.
  • “We are delighted for the opportunity to explore this provocative idea that most, if not all neurodegenerative diseases are rooted in mitochondrial dysfunction.

PANTHERx® Rare Selected by UCB as the Exclusive Specialty Pharmacy for ZILBRYSQ® (zilucoplan) for the Treatment of Generalized Myasthenia Gravis (gMG) in Adult Patients Who Are Anti-Acetylcholine Receptor (AChR) Antibody Positive

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星期三, 一月 10, 2024
Patient, Pharmacy, UCB, Health care, REMS, Neuromuscular junction, Communication, FDA, Diarrhea, Myasthenia gravis, Pharmaceutical industry

1

Key Points: 
  • 1
    ZILBRYSQ is available by prescription as a ready-to-use pre-filled syringe that is a once-daily administration.
  • Healthcare providers who prescribe ZILBRYSQ must enroll in the ZILBRYSQ REMS because of the serious meningococcal infections.
  • The most common side effects of ZILBRYSQ include injection site reactions, upper respiratory tract infections, and diarrhea.
  • 1
    Myasthenia gravis is a chronic neuromuscular disease that causes weakness in the voluntary muscles.

Athira Pharma Reports Third Quarter 2023 Financial Results and Pipeline and Business Updates

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星期四, 十一月 9, 2023
ACT, Walter, Investment, Neuromuscular junction, Patient, Pathology, Conference, Cognition, R, ADAS, Inflammation, AFL, Society, Glial fibrillary acidic protein, Food, Toxicity, HGF, IND, Administration, GFAP, Excitotoxicity, Alzheimer's disease, G&A, Microglia, Communication, Neuroinflammation, Athira Pharma, Research and development, Brain cell, Biomarker, Riluzole, ALS, Frontotemporal dementia, Washington Convention Center, Drug development, AAIC, Enzyme inhibitor, Plasma, Mouse, Doctor of Philosophy, FDA, Neurodegenerative disease, Survival, Central nervous system, GST, Fine chemical, Pharmaceutical industry, Medical imaging, Cryptocurrency, Neuroscience, Athira

BOTHELL, Wash., Nov. 09, 2023 (GLOBE NEWSWIRE) -- Athira Pharma, Inc. (NASDAQ: ATHA), a late clinical-stage biopharmaceutical company focused on developing small molecules to restore neuronal health and slow neurodegeneration, today reported financial results for the quarter ended September 30, 2023, and provided pipeline and business updates.

Key Points: 
  • BOTHELL, Wash., Nov. 09, 2023 (GLOBE NEWSWIRE) -- Athira Pharma, Inc. (NASDAQ: ATHA), a late clinical-stage biopharmaceutical company focused on developing small molecules to restore neuronal health and slow neurodegeneration, today reported financial results for the quarter ended September 30, 2023, and provided pipeline and business updates.
  • R&D expenses were $27.2 million for the quarter ended September 30, 2023, compared with $17.0 million for the quarter ended September 30, 2022.
  • G&A expenses were $7.8 million for the quarter ended September 30, 2023, compared with $7.2 million for the quarter ended September 30, 2022.
  • The increase was primarily due to increases in personnel expenses, partially offset by a decrease in business development expenses.

Cabaletta Bio Receives FDA Clearance of CABA-201 IND Application for Treatment of Generalized Myasthenia Gravis

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星期一, 十一月 6, 2023
GMG, Steroid, FDA, IND, Respiratory failure, Food, Enzyme inhibitor, Myositis, MG, Lupus, Autoantibody, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Protein, Disease, Clinical trial, Acetylcholine receptor, Risk, Neurology, Neuromuscular junction, Autoimmune disease, Scleroderma, NMJ, Patient, Investigational New Drug, Rheumatology, Fatigue, Pharmaceutical industry

PHILADELPHIA, Nov. 06, 2023 (GLOBE NEWSWIRE) -- Cabaletta Bio, Inc. (Nasdaq: CABA), a clinical-stage biotechnology company focused on developing and launching the first curative targeted cell therapies for patients with autoimmune diseases, today announced that the Company’s fourth Investigational New Drug (IND) application for CABA-201, a 4-1BB-containing fully human CD19-CAR T cell investigational therapy, has been allowed to proceed by the U.S. Food and Drug Administration (FDA) for a Phase 1/2 study in patients with generalized myasthenia gravis (gMG). The Company plans to initiate a Phase 1/2 clinical trial of CABA-201 across two parallel gMG cohorts based on autoantibody status – one cohort of six patients with acetylcholine receptor (AChR) antibody-positive gMG and a second cohort of six patients with AChR antibody-negative gMG. Consistent with the previously announced CABA-201 IND application clearances for lupus, myositis and systemic sclerosis, the starting dose for the Phase 1/2 trial evaluating CABA-201 in gMG will be 1 x 106 cells/kg.

Key Points: 
  • Consistent with the previously announced CABA-201 IND application clearances for lupus, myositis and systemic sclerosis, the starting dose for the Phase 1/2 trial evaluating CABA-201 in gMG will be 1 x 106 cells/kg.
  • The announcement of our fourth CABA-201 IND clearance is also our first IND clearance for the product candidate in a predominantly autoantibody mediated disease.
  • Generalized MG affects approximately 85% of the between 50,000 and 80,000 estimated MG patients in the United States.
  • Standard of care therapies include cholinesterase inhibitors, steroids, immunomodulators, and biologics, which often provide modest clinical effect and require chronic administration, increasing the risk of serious long-term side effects.

UCB announces U.S. FDA approval of ZILBRYSQ® (zilucoplan) for the treatment of adults with generalized myasthenia gravis

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星期二, 十月 17, 2023
Neonatal Fc receptor, UCB, MuSK protein, Food, MG, University of North Carolina at Chapel Hill, MAC, System, Disease, Immunoglobulin G, Hospital, Plasmapheresis, Oklahoma Office of the Chief Medical Examiner, Brussels Stock Exchange, NMJ, GMG, Diarrhea, Physician, RAISE, U.S. FDA, Neuromuscular junction, University, Safety, Lancet Neurology, Patient, The Lancet, Week, MD, Allied health professions, Myasthenia, Quality of life, Muscle weakness, Acetylcholine receptor, Travel, QMG, Neuromuscular disease, Fatigue, FDA, Upper respiratory tract infection, Pathology, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Priority review, ACHR, Weakness, Pharmaceutical industry, Vaccine, Medicine, Neurology, C5

It is the only once-daily gMG target therapy for self-administration by adult patients with anti-AChR antibody-positive gMG.

Key Points: 
  • It is the only once-daily gMG target therapy for self-administration by adult patients with anti-AChR antibody-positive gMG.
  • Patients were randomized in a 1:1 ratio to receive daily subcutaneous injections of 0.3 mg/kg ZILBRYSQ or placebo for 12 weeks.
  • The most common adverse reactions (≥10%) in patients with gMG were injection site reactions, upper respiratory tract infection, and diarrhea.
  • The primary endpoint for the RAISE study was change from baseline to Week 12 in the Myasthenia Gravis-Activities of Daily Living (MG-ADL) score.

Athira Pharma Presents Preclinical Data Further Supporting the Therapeutic Potential of ATH-1105 in Amyotrophic Lateral Sclerosis (ALS)

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星期四, 十月 5, 2023
ALS, Asian literature, Neuromuscular junction, Plasma, Excitotoxicity, Brain cell, Poster, Mouse, AFL, HGF, Athira Pharma, Riluzole, Pharmaceutical industry

BOTHELL, Wash., Oct. 05, 2023 (GLOBE NEWSWIRE) -- Athira Pharma, Inc. (NASDAQ: ATHA), a late clinical-stage biopharmaceutical company focused on developing small molecules to restore neuronal health and slow neurodegeneration, presented new preclinical data supporting the potential benefit of ATH-1105 for the treatment of amyotrophic lateral sclerosis (ALS) at the 22nd Annual Northeast ALS Consortium (NEALS) Meeting, taking place October 4-6, 2023, virtually and in-person in Clearwater, Florida. ATH-1105 is an orally administered small molecule therapeutic candidate designed to enhance the neurotrophic hepatocyte growth factor (HGF) system.

Key Points: 
  • ATH-1105 is an orally administered small molecule therapeutic candidate designed to enhance the neurotrophic hepatocyte growth factor (HGF) system.
  • “There is an urgent need for new ALS treatment options, particularly those aimed at stopping or slowing neurodegeneration.
  • We are encouraged by the consistent benefit of ATH-1105 in these animal models, whether given pre- or post-symptom onset or in combination with riluzole.
  • NEALS is a closed meeting, and the presentation will be available on the Scientific Publications & Presentations page of the company’s website at w ww.at hira.com .

NMD Pharma initiates Phase II trial of NMD670 in Spinal Muscular Atrophy

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星期二, 九月 26, 2023
SMA, Peripheral neuropathy, EVP, Myasthenia gravis, Quality of life, Facial muscles, Doctor of Philosophy, Trial of the century, Muscle weakness, Neuromuscular junction, MD, Patient, MG, Phase, Laboratory, Safety, Fatigue, Pharmaceutical industry

Aarhus, Denmark, 26 September 2023 – NMD Pharma A/S, a clinical-stage biotech company developing first-in-class, small molecule ClC-1 inhibitors directly targeting muscle function within neuromuscular disorders, today announces that the first patient has been dosed in a Phase II clinical trial with the ClC-1 inhibitor NMD670 in patients impacted by spinal muscular atrophy (SMA).

Key Points: 
  • Aarhus, Denmark, 26 September 2023 – NMD Pharma A/S, a clinical-stage biotech company developing first-in-class, small molecule ClC-1 inhibitors directly targeting muscle function within neuromuscular disorders, today announces that the first patient has been dosed in a Phase II clinical trial with the ClC-1 inhibitor NMD670 in patients impacted by spinal muscular atrophy (SMA).
  • The Phase II clinical trial ( NCT05794139 ) is a randomized, double-blind, placebo-controlled, 2-way crossover study to evaluate the efficacy, safety, and tolerability of twice-a-day oral dosing of NMD670 for 21 days in ambulatory adult patients with SMA Type 3.
  • We are very pleased to announce the dosing of the first patient in this Phase II study.
  • NMD Pharma has pre-clinically demonstrated that ClC-1 inhibition can enhance neuromuscular transmission and ultimately skeletal muscle function in multiple animal models of neuromuscular diseases.