Acetylcholine receptor

NMD Pharma Receives FDA IND Clearance to initiate a Phase 2b Clinical Trial of NMD670 in Generalized Myasthenia Gravis Patients in the US

Retrieved on: 
Friday, March 22, 2024
MuSK protein, Science Translational Medicine, Charcot–Marie–Tooth disease, GMG, Patient, Myasthenia gravis, IND, Spinal muscular atrophy, Acetylcholine receptor, Safety, Muscle weakness, Fatigue, FDA, Food, Pharmaceutical industry

The Phase 2b clinical trial is a dose range-finding, double-blind, placebo-controlled study of NMD670, a twice daily oral muscle-targeted therapy, in gMG patients experiencing persistent symptoms or fluctuating symptoms despite treatment on current standard of care.

Key Points: 
  • The Phase 2b clinical trial is a dose range-finding, double-blind, placebo-controlled study of NMD670, a twice daily oral muscle-targeted therapy, in gMG patients experiencing persistent symptoms or fluctuating symptoms despite treatment on current standard of care.
  • The trial will take place in both US and European clinical sites.
  • Thomas Holm Pedersen, Chief Executive Officer of NMD Pharma, said: “This is an important milestone for NMD Pharma, enabling us to progress our lead development candidate NMD670 into a Phase 2b trial in the US in generalized myasthenia gravis patients with AChR and MuSK positive antibodies.
  • It follows positive Phase 1/2a data which provided proof-of-mechanism and where clinically and statistically significant effects were seen in patients.

NMD Pharma Receives FDA IND Clearance to initiate a Phase 2b Clinical Trial of NMD670 in Generalized Myasthenia Gravis Patients in the US

Retrieved on: 
Friday, March 22, 2024
MuSK protein, Science Translational Medicine, Charcot–Marie–Tooth disease, GMG, Patient, Myasthenia gravis, IND, Spinal muscular atrophy, Acetylcholine receptor, Safety, Muscle weakness, Fatigue, FDA, Food, Pharmaceutical industry

The Phase 2b clinical trial is a dose range-finding, double-blind, placebo-controlled study of NMD670, a twice daily oral muscle-targeted therapy, in gMG patients experiencing persistent symptoms or fluctuating symptoms despite treatment on current standard of care.

Key Points: 
  • The Phase 2b clinical trial is a dose range-finding, double-blind, placebo-controlled study of NMD670, a twice daily oral muscle-targeted therapy, in gMG patients experiencing persistent symptoms or fluctuating symptoms despite treatment on current standard of care.
  • The trial will take place in both US and European clinical sites.
  • Thomas Holm Pedersen, Chief Executive Officer of NMD Pharma, said: “This is an important milestone for NMD Pharma, enabling us to progress our lead development candidate NMD670 into a Phase 2b trial in the US in generalized myasthenia gravis patients with AChR and MuSK positive antibodies.
  • It follows positive Phase 1/2a data which provided proof-of-mechanism and where clinically and statistically significant effects were seen in patients.

NMD Pharma Publishes Comprehensive Data Package for NMD670 in Science Translational Medicine

Retrieved on: 
Thursday, March 21, 2024
MuSK protein, Exercise, Science Translational Medicine, Charcot–Marie–Tooth disease, NMD, Patient, Brain cell, Human, Myasthenia gravis, Spinal muscular atrophy, Acetylcholine receptor, Facial muscles, QMG, Myasthenia, Safety, Aarhus University, Muscle weakness, Fatigue, Paper, Neuromuscular disease, Pharmaceutical industry

NMD Pharma’s lead development candidate, NMD670, a novel, selective, and orally bioavailable ClC-1 inhibiting small molecule, is used in the preclinical and clinical studies.

Key Points: 
  • NMD Pharma’s lead development candidate, NMD670, a novel, selective, and orally bioavailable ClC-1 inhibiting small molecule, is used in the preclinical and clinical studies.
  • NMD670 is a muscle-targeted therapy which aims to enhance the activation of skeletal muscle fibers and to improve muscle strength and endurance.
  • As a result, patients have impaired skeletal muscle function and often experience severe muscle weakness and fatigue.
  • Access the full paper in Science Translational Medicine online here:
    In October 2022, NMD Pharma announced positive topline data which established the first clinical proof-of-mechanism of NMD Pharma’s novel CIC-1 chloride channel inhibitor approach in patients impacted by MG.

NMD Pharma Publishes Comprehensive Data Package for NMD670 in Science Translational Medicine

Retrieved on: 
Thursday, March 21, 2024
MuSK protein, Exercise, Science Translational Medicine, Charcot–Marie–Tooth disease, NMD, Patient, Brain cell, Human, Myasthenia gravis, Spinal muscular atrophy, Acetylcholine receptor, Facial muscles, QMG, Myasthenia, Safety, Aarhus University, Muscle weakness, Fatigue, Paper, Neuromuscular disease, Pharmaceutical industry

NMD Pharma’s lead development candidate, NMD670, a novel, selective, and orally bioavailable ClC-1 inhibiting small molecule, is used in the preclinical and clinical studies.

Key Points: 
  • NMD Pharma’s lead development candidate, NMD670, a novel, selective, and orally bioavailable ClC-1 inhibiting small molecule, is used in the preclinical and clinical studies.
  • NMD670 is a muscle-targeted therapy which aims to enhance the activation of skeletal muscle fibers and to improve muscle strength and endurance.
  • As a result, patients have impaired skeletal muscle function and often experience severe muscle weakness and fatigue.
  • Access the full paper in Science Translational Medicine online here:
    In October 2022, NMD Pharma announced positive topline data which established the first clinical proof-of-mechanism of NMD Pharma’s novel CIC-1 chloride channel inhibitor approach in patients impacted by MG.

Cartesian Therapeutics Announces Positive Long-Term Follow-Up Data from Phase 2a Study of Lead mRNA Cell Therapy Candidate Descartes-08 in Patients with Myasthenia Gravis

Retrieved on: 
Monday, January 8, 2024
Lancet Neurology, Patient, Quality of life, Acetylcholine receptor, Peer review, Food, Autoimmune disease, Risk, IND, Messenger, Cytokine release syndrome, The Lancet, Data, Fatigue, Lupus, BCMA, Muscle weakness, Orphan, Therapy, Clinical trial, Investigational New Drug, Quality, Pharmacokinetics, Neurotoxicity, Manuscript, Month, MedRxiv, Autoimmunity, Pharmaceutical industry

GAITHERSBURG, Md., Jan. 08, 2024 (GLOBE NEWSWIRE) -- Cartesian Therapeutics, Inc. (NASDAQ: RNAC), (“the Company”) a clinical-stage biotechnology company pioneering mRNA cell therapies for autoimmune diseases, today announced positive twelve-month follow-up data from its Phase 2a trial of Descartes-08 in patients with generalized myasthenia gravis (MG), a chronic autoimmune disorder that causes disabling muscle weakness and fatigue. The manuscript titled, “Twelve-Month Follow-Up of Patients With Generalized Myasthenia Gravis Receiving BCMA-Directed mRNA Cell Therapy,” has been submitted for peer-review and can be accessed on the online preprint server, medRxiv.

Key Points: 
  • The manuscript titled, “Twelve-Month Follow-Up of Patients With Generalized Myasthenia Gravis Receiving BCMA-Directed mRNA Cell Therapy,” has been submitted for peer-review and can be accessed on the online preprint server, medRxiv.
  • Descartes-08, Cartesian’s lead mRNA cell therapy candidate and a potential first-in-class mRNA-engineered chimeric antigen receptor T-cell therapy (mRNA CAR-T), is an autologous anti-B-cell maturation antigen (BCMA) mRNA CAR-T.
  • “Notably, most patients maintained robust, clinically meaningful improvements across all four standard MG severity scores approximately 10 months after the last infusion.
  • Enrollment is ongoing in a Phase 2b randomized, double-blind, placebo-controlled trial (NCT04146051) in patients with MG. Topline results are expected in mid-2024.

ZILBRYSQ® (zilucoplan) Is Now Commercially Available in the U.S. for the Treatment of Generalized Myasthenia Gravis (gMG) in Adult Patients Who Are Anti-Acetylcholine Receptor (AChR) Antibody Positive

Retrieved on: 
Wednesday, January 3, 2024
Food, Acetylcholine receptor, GMG, Risk, Health care, MuSK protein, Caregiver, Diarrhea, FDA, Eyelid, Disease, Muscle weakness, REMS, Myasthenia gravis, UCB, Insurance, Patient, Pharmacy, Pharmaceutical industry

ZILBRYSQ® (zilucoplan) is now commercially available by prescription in the United States and dispensed by UCB's selected exclusive specialty pharmacy PANTHERx® Rare Pharmacy.

Key Points: 
  • ZILBRYSQ® (zilucoplan) is now commercially available by prescription in the United States and dispensed by UCB's selected exclusive specialty pharmacy PANTHERx® Rare Pharmacy.
  • The most common side effects of ZILBRYSQ include injection site reactions, upper respiratory tract infections, and diarrhea.
  • This support includes refresher injection coaching and information on symptom tracking and ongoing treatment support, as well as help in understanding insurance coverage.
  • If an eligible patient or caregiver is looking for support, please visit http://www.UCBONWARD.com or call 1-844-ONWARD.

Human medicines European public assessment report (EPAR): Zilbrysq, Zilucoplan, Date of authorisation: 01/12/2023, Status: Authorised

Retrieved on: 
Tuesday, January 2, 2024
Patient, Medical Subject Headings, Marketing, Meningococcal disease, Protein, Acetylcholine receptor, Autoantibody, INN, GMG, Immune system, Fatigue, Risk, ATC, Antibody, Pain, International, Disease, Nose, Language, Muscle weakness, Vaccination, Neisseria meningitidis, European Medicines Agency, C5, Select, Bruise, Safety, Time, Anatomy, Contract management, Disorder, IIA, Medical device

Human medicines European public assessment report (EPAR): Zilbrysq, Zilucoplan, Date of authorisation: 01/12/2023, Status: Authorised

Key Points: 


Human medicines European public assessment report (EPAR): Zilbrysq, Zilucoplan, Date of authorisation: 01/12/2023, Status: Authorised

Human medicines European public assessment report (EPAR): Vyvgart, efgartigimod alfa, Date of authorisation: 10/08/2022, Revision: 6, Status: Authorised

Retrieved on: 
Tuesday, January 2, 2024
Urine, Medical Subject Headings, Marketing, Protein, Acetylcholine receptor, Autoantibody, INN, GMG, Immune system, Fatigue, Lower respiratory tract infection, Infection, ATC, Antibody, International, Disease, Nose, Language, Blood, Muscle weakness, Urinary tract infection, Medicine, B cell, Neonatal Fc receptor, European Medicines Agency, Acetylcholine, Select, Patient, Anatomy, IIA, Medical device

Human medicines European public assessment report (EPAR): Vyvgart, efgartigimod alfa, Date of authorisation: 10/08/2022, Revision: 6, Status: Authorised

Key Points: 


Human medicines European public assessment report (EPAR): Vyvgart, efgartigimod alfa, Date of authorisation: 10/08/2022, Revision: 6, Status: Authorised

argenx Announces European Commission Approval of Subcutaneous VYVGART® (efgartigimod alfa) for Generalized Myasthenia Gravis

Retrieved on: 
Thursday, November 16, 2023
Acetylcholine receptor, European Commission, GMG, Civil service commission, Patient, Autoimmune disease, CHMP, Committee, Efgartigimod alfa, Euronext, SE

Following this decision, VYVGART is now approved in Europe for both IV and self-administered SC use.

Key Points: 
  • Following this decision, VYVGART is now approved in Europe for both IV and self-administered SC use.
  • The approval is applicable to all 27 European Union (EU) Member States plus Iceland, Norway and Liechtenstein.
  • argenx will work with local health authorities to secure patient access for VYVGART SC in the region.
  • We are proud to deliver this second formulation to the European gMG community, just 15 months after the initial approval of VYVGART IV,” said Anant Murthy, General Manager of argenx EMEA.

NMD Pharma Raises €75 Million (~$80 million) in a Series B Financing

Retrieved on: 
Wednesday, November 15, 2023
Spinal muscular atrophy, Acetylcholine receptor, Investment, Patient, Myasthenia gravis, Novo Holdings A/S, Therapy, Charcot–Marie–Tooth disease, MuSK protein, NMD, Growth, Pharma, Pharmaceutical industry, Fine chemical

The financing was led by current investor Jeito Capital and includes investments from other current NMD Pharma investors: Novo Holdings, Lundbeckfonden BioCapital, INKEF Capital, and the Roche Venture Fund.

Key Points: 
  • The financing was led by current investor Jeito Capital and includes investments from other current NMD Pharma investors: Novo Holdings, Lundbeckfonden BioCapital, INKEF Capital, and the Roche Venture Fund.
  • NMD Pharma is developing first-in-class small molecule inhibitors of the skeletal muscle specific chloride ion channel (ClC-1) to enhance neuromuscular transmission with the potential to restore muscle function in a range of rare neuromuscular diseases.
  • Proceeds from the financing will be used to complete three Phase 2 studies with NMD670, the Company’s lead ClC-1 inhibitor.
  • Furthermore, NMD Pharma will continue to expand its pipeline of ClC-1 inhibitor molecules and pursue undisclosed targets for the treatment of other neuromuscular diseases.