MuSK protein

Cabaletta Bio Reports First Quarter 2024 Financial Results and Provides Business Update

Retrieved on: 
среда, мая 15, 2024
Patient, Cell, Systemic scleroderma, Movement, Lupus, Pemphigus vulgaris, Food, Myopathy, Investment, Letter, CD19, FDA, Disease, MuSK protein, MPV, Conference, Cytokine release syndrome, Dermatology, Congress, Growth, Gene, Immunoglobulin therapy, Potential, Juvenile dermatomyositis, Cyclophosphamide, Dermatomyositis, Symposia, EULAR, IIM, Society, Health Canada, A1, Observation, Research, Clinical trial, DLT, Myositis, Annual general meeting, CRS, Pharmaceutical industry

PHILADELPHIA, May 15, 2024 (GLOBE NEWSWIRE) -- Cabaletta Bio, Inc. (Nasdaq: CABA), a clinical-stage biotechnology company focused on developing and launching the first curative targeted cell therapies designed specifically for patients with autoimmune diseases, today reported financial results for the first quarter ended March 31, 2024, and provided a business update.

Key Points: 
  • PHILADELPHIA, May 15, 2024 (GLOBE NEWSWIRE) -- Cabaletta Bio, Inc. (Nasdaq: CABA), a clinical-stage biotechnology company focused on developing and launching the first curative targeted cell therapies designed specifically for patients with autoimmune diseases, today reported financial results for the first quarter ended March 31, 2024, and provided a business update.
  • In March 2024, Cabaletta announced the first patient had been dosed in the Phase 1/2 RESET-Myositis trial.
  • Cabaletta expects to report initial clinical data from the Phase 1/2 RESET-SSc™ trial in the second half of 2024.
  • Cabaletta expects to report initial clinical data from the Phase 1/2 RESET-MG™ trial in the second half of 2024.

argenx Reports First Quarter 2024 Financial Results and Provides Business Update

Retrieved on: 
четверг, мая 9, 2024
MuSK protein, FDA, ARDA, IND, PDUFA, Medicine, Autoimmunity, Efgartigimod alfa, Glomerulonephritis, CMS, Inflammation, ITP, IIP, ALS, Alfa Romeo, C2, Therapy, Systemic scleroderma, Safety, Euronext, Nektar, DGF, Sjögren syndrome, CIDP, Biology, IL-6, Nektar Therapeutics, GMG, TED, File, MMN, Lupus nephritis, Kidney, AMR, Multifocal motor neuropathy, Patient, Congenital myasthenic syndrome, Immune system, Dermatomyositis, Growth, Amgen, Pharmaceutical industry

“The team at argenx has made significant progress executing across the ambitious plan we set out at the beginning of the year,” said Tim Van Hauwermeiren, Chief Executive Officer of argenx.

Key Points: 
  • “The team at argenx has made significant progress executing across the ambitious plan we set out at the beginning of the year,” said Tim Van Hauwermeiren, Chief Executive Officer of argenx.
  • “We are driven by our commitment to provide patients with the broadest gMG product offering that consistently delivers on safety and efficacy.
  • VYVGART SC played a key role in our growth over the quarter, expanding the breadth of our prescriber base and reaching new patients.
  • In addition, argenx is evaluating ARGX-119, a muscle-specific kinase (MuSK) agonist in both congenital myasthenic syndrome (CMS) and amyotrophic lateral sclerosis (ALS).

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: 
пятница, марта 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: 
пятница, марта 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.

Cabaletta Bio Reports Fourth Quarter and Full Year 2023 Financial Results and Provides Business Update

Retrieved on: 
четверг, марта 21, 2024
Research, MPV, IIM, MuSK protein, Toxicity, Conference, Disease, Dermatomyositis, Juvenile dermatomyositis, Federation, Patient, NOL, Observation, CRS, SLE, Health Canada, IND, Immunoglobulin therapy, Pemphigus, Investment, Investigational New Drug, Myopathy, Systemic scleroderma, Marketing, FDA, Clinical trial, Cytokine release syndrome, Cyclophosphamide, CD19, Food, Autoimmune disease, Pharmaceutical industry

In October 2023, Cabaletta received Investigational New Drug (IND) application clearance from the U.S. Food and Drug Administration (FDA) for the Phase 1/2 RESET-SSc trial.

Key Points: 
  • In October 2023, Cabaletta received Investigational New Drug (IND) application clearance from the U.S. Food and Drug Administration (FDA) for the Phase 1/2 RESET-SSc trial.
  • In November 2023, Cabaletta announced that its IND application for CABA-201 was allowed to proceed by the FDA for the Phase 1/2 RESET-MG trial.
  • Cabaletta anticipates reporting initial clinical data from the Phase 1/2 RESET-MG trial in the second half of 2024.
  • As of December 31, 2023, Cabaletta had cash, cash equivalents and short-term investments of $241.2 million, compared to $106.5 million as of December 31, 2022.

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

Retrieved on: 
четверг, марта 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: 
четверг, марта 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.

IASO Bio Announces U.S. FDA Approval of Investigational New Drug Application for BCMA CAR-T Equecabtagene Autoleucel for Generalized Myasthenia Gravis

Retrieved on: 
четверг, апреля 4, 2024
Vital capacity, MuSK protein, Glucocorticoid, NMPA, Hormone, GMG, Disease, Chinese, MRS, BCMA, Nervous system, Patient, SAN, Enzyme inhibitor, History, Female, Multiple myeloma, CRS, B cell, Biotechnology, IND, Phenotype, Investigational New Drug, QMG, Safety, Thymectomy, FDA, Food

The Chinese IND for this indication of Equecabtagene Autoleucel was approved by the NMPA in January this year.

Key Points: 
  • The Chinese IND for this indication of Equecabtagene Autoleucel was approved by the NMPA in January this year.
  • The 2 subjects were treated with a single infusion of Eque-cel at the doses of 1.0×106 CAR-T/Kg, respectively.
  • No immunomodulatory therapy other than low dose pyridostigmine (90 mg/day and 60 mg/day, respectively) was used during the follow-up period.
  • Anti-AChR antibodies, anti-Titin antibodies, and anti-MuSK antibodies decreased rapidly and maintained at very low levels in both subjects after infusion.

argenx Reports Full Year 2023 Financial Results and Provides Fourth Quarter Business Update

Retrieved on: 
четверг, февраля 29, 2024
DGF, Neonatal Fc receptor, ALS, Congenital myasthenic syndrome, CMS, CIDP, MMN, Alfa Romeo, IIP, IL-6, FDA, Inflammation, MHLW, PDUFA, Euronext, Neurology, MuSK protein, Kidney, ARDA, Health care, C2, GMG, Ministry of Health, Dermatomyositis, Patient, Efgartigimod alfa, MHRA, Medicine, Multifocal motor neuropathy, Immune system, Pharmaceutical industry

“argenx reached thousands of new patients and their families in 2023 by delivering on our commitment to make VYVGART available to the global MG community,” said Tim Van Hauwermeiren, Chief Executive Officer of argenx.

Key Points: 
  • “argenx reached thousands of new patients and their families in 2023 by delivering on our commitment to make VYVGART available to the global MG community,” said Tim Van Hauwermeiren, Chief Executive Officer of argenx.
  • Clinically, we generated significant data through multiple study readouts, achieving key milestones for both the CIDP and MMN patient communities and importantly advancing our second molecule, empasiprubart.
  • Looking forward to 2024, we will act with a continued sense of purpose to expand our patient reach.
  • In addition, argenx is evaluating ARGX-119, a muscle-specific kinase (MuSK) agonist in both congenital myasthenic syndrome (CMS) and amyotrophic lateral sclerosis (ALS).

argenx Highlights 2024 Strategic Priorities

Retrieved on: 
понедельник, января 8, 2024
Immunoglobulin therapy, GMG, FDA, PRV, Congenital myasthenic syndrome, ARDA, Research Councils UK, Euronext, Inflammation, Cohort, PGIC, NRDL, Autoimmunity, Efgartigimod alfa, Dermatomyositis, Conference, MuSK protein, IND, CIDP, Pharmacokinetics, MMN, Safety, Therapy, Patient, ALS, Immune system, IL-6, Medicine, International Myeloma Foundation, IIP, FSS, Neonatal Fc receptor, Multifocal motor neuropathy, Growth, Pharmaceutical industry

argenx is planning to reach more patients commercially in 2024 through its multi-dimensional expansion efforts.

Key Points: 
  • argenx is planning to reach more patients commercially in 2024 through its multi-dimensional expansion efforts.
  • In addition, ARGX-119, a muscle-specific kinase (MuSK) agonist, will initiate Phase 1b/2a studies in congenital myasthenic syndrome and amyotrophic lateral sclerosis in 2024.
  • As of December 31, 2023, argenx had approximately $3.2 billion in cash, cash equivalents and current financial assets*.
  • Based on its current operating plans, argenx expects its combined R&D and SG&A expenses in 2024 to be less than $2 billion.