Latest news with #MuSK
Yahoo
3 days ago
- Business
- Yahoo
argenx to Host R&D Webinar Highlighting ARGX-119 on September 16, 2025
August 19, 2025Amsterdam, the Netherlands – argenx (Euronext & Nasdaq: ARGX), a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases, today announced it will host a webinar, titled 'R&D Spotlight | Pioneering MuSK Biology with ARGX-119' on Tuesday, September 16, 2025, at 2:00pm ET. The webinar will be the first of a 'mini' series highlighting the argenx pipeline and research and development strategy. argenx management and scientific leadership will be joined by key opinion leaders, who will discuss the development of the MuSK agonist program, ARGX-119, and its potential to treat neuromuscular diseases, including congenital myasthenic syndromes (CMS), amyotrophic lateral sclerosis (ALS) and spinal muscular atrophy (SMA). A webcast of the event can be accessed on the Investors section of the argenx website at A replay of the webcast will be available on the argenx website for approximately one year following the presentation. About argenx argenx is a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases. Partnering with leading academic researchers through its Immunology Innovation Program (IIP), argenx aims to translate immunology breakthroughs into a world-class portfolio of novel antibody-based medicines. argenx developed and is commercializing the first approved neonatal Fc receptor (FcRn) blocker and is evaluating its broad potential in multiple serious autoimmune diseases while advancing several earlier stage experimental medicines within its therapeutic franchises. For more information, visit and follow us on LinkedIn, Instagram, Facebook, and YouTube. Contacts Media: Colin McBeancmcbean@ Investors: Alexandra Royaroy@
Yahoo
13-05-2025
- Business
- Yahoo
NMD Pharma to present compelling preclinical data highlighting the potential of ClC-1 ion channel inhibition to improve disease symptoms and progression in MuSK myasthenia gravis at the MGFA International Conference on Myasthenia and Relate
NMD Pharma to present compelling preclinical data highlighting the potential of ClC-1 ion channel inhibition to improve disease symptoms and progression in MuSK myasthenia gravis at the MGFA International Conference on Myasthenia and Related Disorders The Company continues to progress its Phase 2b clinical study in generalized myasthenia gravis (gMG) patients who are AChR and MuSK antibody positive, investigating the potential of NMD670 and its unique skeletal-muscle-targeted, non-immunomodulatory mechanism of action (MoA) Aarhus, Denmark, 13 May 2025 – NMD Pharma A/S, a clinical-stage biotech company dedicated to developing novel and improved treatments for patients living with neuromuscular diseases, today announces that one abstract has been accepted for oral presentation, and multiple abstracts selected for poster presentation, at the influential 15th Annual Myasthenia Gravis Foundation of America (MGFA) International Conference on Myasthenia and Related Disorders, taking place from 13-15 May in The Hague, Netherlands. In the oral presentation, NMD Pharma will present data detailing the positive effects of its novel, orally bioavailable, skeletal muscle-specific chloride ion channel (ClC-1) inhibitor in a muscle-specific kinase (MuSK) myasthenia gravis (MG) rat model. MuSK-MG is a rare and frequently more severe type of MG, with an acute onset that primarily affects the facial-bulbar muscles and can cause frequent occurrences of respiratory crisis. As expected, and demonstrated in previous animal models of MG and other neuromuscular diseases, NMD Pharma's small molecule ClC-1 inhibitor had a positive effect on electromyography (EMG), as well as muscle function in a blinded study. Additionally, evidence of improved lung function, with higher body weight and increased survival, were observed in the ClC-1 treated group relative to vehicle. The presentation will also describe interesting evidence supporting ClC-1 inhibition as a direct or indirect facilitator of reinnervation or attenuation of the denervation process. Supported by findings detailed in poster presentations, these results confirm the translational potential of ClC-1 inhibition in improving symptoms for neuromuscular diseases. In the poster presentations, NMD Pharma authors provide additional post-hoc responder data from the completed Phase 2a proof-of-mechanism clinical study with NMD670 in MG and present the study design and status update of the ongoing phase 2b dose finding study. Posters will also describe the ClC-1 ion channel as a therapeutic target in NMJ disorders and additional characterization of the MuSK Myasthenia rat model. Oral and poster presentation Session: 8b: Models for MG and Translational ResearchTitle: Skeletal muscle targeted ClC-1 ion channel inhibitor improves skeletal muscle function and respiratory function in a rat model of MuSK-MGPresenter/Authors: M. Skals, J. J. Morgen, M.B. Skov, N. Huus, N. M. Kelly, M. Broch-LipsDate and Time: Wednesday, 14 May, 3:30-5:00pm CEST Poster presentations Session: Welcome Reception and Poster SessionTitle: The Skeletal Muscle Specific Chloride-1 ion channel (ClC-1) regulates the muscle's ability to activate and therefore represents a viable complementary therapeutic target to immunosuppressants and immunomodulatory agentsAuthors: Martin Skov, Jesper Emil Jakobsgaard, Jeanette Morgen, Martin Broch-Lips, Marianne Skals, Nete Huus, Nicholas Kelly, Vera Kiyasova, Thomas Groennebaek, Thomas K. Petersen, Thomas H. Pedersen Date and Time: Tuesday, 13 May, 5:00-7:00pm CEST Session: Welcome Reception and Poster SessionTitle: Phase 2b study design for NMD670, a First-in-Class ClC-1 Inhibitor in Generalized Myasthenia Gravis: the SYNAPSE-MG dose-finding StudyAuthors: Thomas S. Grønnebæk, Teresa Gidaro, Thomas Breuer, Jitendra Gupte, Claire Sampson, Vera Kiyasova, Thomas H. PedersenDate and Time: Tuesday, 13 May, 5:00-7:00pm CEST Session: Welcome Reception and Poster SessionTitle: Post-hoc responder analyses from the proof-of-mechanism study of NMD670 in Patients with Myasthenia GravisAuthors: Thomas S. Grønnebæk, Teresa Gidaro, Thomas Breuer, Jitendra Gupte, Claire Sampson, Vera Kiyasova, Thomas H. Pedersen Date and Time: Tuesday, 13 May, 5:00-7:00pm CEST Session: Poster SessionTitle: Extensive phenotyping of a rat MuSK myasthenia gravis model – From neuromuscular in vivo function to molecular propertiesAuthors: Jesper Emil Jakobsgaard, Martin Brandhøj Skov, Marianne Skals, Pernille Bogetofte Thomasen, Jeanette Jeppesen Morgen, Nete Huus, Anders Findsen, Jeppe Winther, Martin Broch-Lips, Kristian Vissing, Thomas K. Pedersen Johan Palmfeldt, Thomas H. PedersenDate and Time: Wednesday, 14 May, 5:00-7:00pm CEST NMD Pharma's novel, orally bioavailable skeletal muscle specific ClC-1 inhibitor, NMD670 is currently being evaluated in a Phase 2b clinical trial in patients with generalized myasthenia gravis (gMG) following positive results from a Phase 2a proof-of-mechanism study. Additionally, the company is conducting two separate Phase 2 trials in patients living with spinal muscular atrophy and Charcot-Marie-Tooth disease. The gMG Phase 2b study, which initiated in June 2024, is a dose range-finding, double-blinded, placebo controlled study of NMD670 over 21 days in gMG patients who are anti-acetylcholine receptor (AChR), or anti-muscle-specific tyrosine kinase (MuSK) antibody positive. The study will evaluate changes in the Quantitative Myasthenia Gravis Total Score and the Myasthenia Gravis Activities of Daily Living, among other endpoints, and is taking place at clinical sites across both the US and Europe. Further information on the study can be found here: Study Details | Safety and Efficacy of 3 Dose Levels of NMD670 in Adult Patients With Myasthenia Gravis | Generalized myasthenia gravis patients who are AChR or MuSK antibody positive in the US and Europe are encouraged to participate in the study. Further information and a list of currently active investigational sites can be obtained via email at contact@ -END- Contacts NMD Pharma A/SDan Brennan, SVP Corporate and Commercial StrategyE-mail: contact@ ICR HealthcareMary-Jane Elliott / Ashley Tapp / Lindsey Neville E-mail: NMDPharma@ +44 (0)20 3709 5700 About NMD PharmaNMD Pharma A/S is a clinical-stage biotech company developing a first-in-class platform of small molecule therapies selectively targeting the skeletal muscle chloride ion channel (ClC-1) for the treatment of rare neuromuscular disorders and age-related neuromuscular diseases with high levels of patient unmet. The Company was founded on more than 15 years of muscle physiology research with a focus on regulation of skeletal muscle excitability under physical activity. NMD Pharma has built a world-leading muscle electrophysiology platform leveraging the in-depth know-how of muscle physiology and muscular disorders, small molecule modulators, enabling technologies and tools as well as in vivo pharmacology models for discovering and developing proprietary modulators of neuromuscular function. The Company has built significant clinical and development expertise as its programmes have progressed through the clinic. NMD Pharma has raised ~€155 million from investors including Novo Holdings, Lundbeckfonden BioCapital, INKEF Capital, Roche Venture Fund, and Jeito Capital. Find out more about us online at About NMD670NMD670 is NMD Pharma's lead development program. It is a first-in-class small molecule inhibitor of the skeletal muscle specific chloride ion channel 1 (CIC-1). NMD Pharma has demonstrated that CIC-1 inhibition amplifies the muscle's responsiveness to weak signals, improving neuromuscular transmission and restores skeletal muscle function. This novel treatment approach has resulted in clinical evidence of CIC-1 inhibition in myasthenia gravis (MG) and preclinical evidence in spinal muscular atrophy, Charcot-Marie Tooth (CMT) disease and sarcopenia. NMD670 has also been granted orphan-drug designation by the U.S. FDA for treatment of generalised MG and in to access your portfolio
Yahoo
13-05-2025
- Business
- Yahoo
NMD Pharma to present compelling preclinical data highlighting the potential of ClC-1 ion channel inhibition to improve disease symptoms and progression in MuSK myasthenia gravis at the MGFA International Conference on Myasthenia and Relate
NMD Pharma to present compelling preclinical data highlighting the potential of ClC-1 ion channel inhibition to improve disease symptoms and progression in MuSK myasthenia gravis at the MGFA International Conference on Myasthenia and Related Disorders The Company continues to progress its Phase 2b clinical study in generalized myasthenia gravis (gMG) patients who are AChR and MuSK antibody positive, investigating the potential of NMD670 and its unique skeletal-muscle-targeted, non-immunomodulatory mechanism of action (MoA) Aarhus, Denmark, 13 May 2025 – NMD Pharma A/S, a clinical-stage biotech company dedicated to developing novel and improved treatments for patients living with neuromuscular diseases, today announces that one abstract has been accepted for oral presentation, and multiple abstracts selected for poster presentation, at the influential 15th Annual Myasthenia Gravis Foundation of America (MGFA) International Conference on Myasthenia and Related Disorders, taking place from 13-15 May in The Hague, Netherlands. In the oral presentation, NMD Pharma will present data detailing the positive effects of its novel, orally bioavailable, skeletal muscle-specific chloride ion channel (ClC-1) inhibitor in a muscle-specific kinase (MuSK) myasthenia gravis (MG) rat model. MuSK-MG is a rare and frequently more severe type of MG, with an acute onset that primarily affects the facial-bulbar muscles and can cause frequent occurrences of respiratory crisis. As expected, and demonstrated in previous animal models of MG and other neuromuscular diseases, NMD Pharma's small molecule ClC-1 inhibitor had a positive effect on electromyography (EMG), as well as muscle function in a blinded study. Additionally, evidence of improved lung function, with higher body weight and increased survival, were observed in the ClC-1 treated group relative to vehicle. The presentation will also describe interesting evidence supporting ClC-1 inhibition as a direct or indirect facilitator of reinnervation or attenuation of the denervation process. Supported by findings detailed in poster presentations, these results confirm the translational potential of ClC-1 inhibition in improving symptoms for neuromuscular diseases. In the poster presentations, NMD Pharma authors provide additional post-hoc responder data from the completed Phase 2a proof-of-mechanism clinical study with NMD670 in MG and present the study design and status update of the ongoing phase 2b dose finding study. Posters will also describe the ClC-1 ion channel as a therapeutic target in NMJ disorders and additional characterization of the MuSK Myasthenia rat model. Oral and poster presentation Session: 8b: Models for MG and Translational ResearchTitle: Skeletal muscle targeted ClC-1 ion channel inhibitor improves skeletal muscle function and respiratory function in a rat model of MuSK-MGPresenter/Authors: M. Skals, J. J. Morgen, M.B. Skov, N. Huus, N. M. Kelly, M. Broch-LipsDate and Time: Wednesday, 14 May, 3:30-5:00pm CEST Poster presentations Session: Welcome Reception and Poster SessionTitle: The Skeletal Muscle Specific Chloride-1 ion channel (ClC-1) regulates the muscle's ability to activate and therefore represents a viable complementary therapeutic target to immunosuppressants and immunomodulatory agentsAuthors: Martin Skov, Jesper Emil Jakobsgaard, Jeanette Morgen, Martin Broch-Lips, Marianne Skals, Nete Huus, Nicholas Kelly, Vera Kiyasova, Thomas Groennebaek, Thomas K. Petersen, Thomas H. Pedersen Date and Time: Tuesday, 13 May, 5:00-7:00pm CEST Session: Welcome Reception and Poster SessionTitle: Phase 2b study design for NMD670, a First-in-Class ClC-1 Inhibitor in Generalized Myasthenia Gravis: the SYNAPSE-MG dose-finding StudyAuthors: Thomas S. Grønnebæk, Teresa Gidaro, Thomas Breuer, Jitendra Gupte, Claire Sampson, Vera Kiyasova, Thomas H. PedersenDate and Time: Tuesday, 13 May, 5:00-7:00pm CEST Session: Welcome Reception and Poster SessionTitle: Post-hoc responder analyses from the proof-of-mechanism study of NMD670 in Patients with Myasthenia GravisAuthors: Thomas S. Grønnebæk, Teresa Gidaro, Thomas Breuer, Jitendra Gupte, Claire Sampson, Vera Kiyasova, Thomas H. Pedersen Date and Time: Tuesday, 13 May, 5:00-7:00pm CEST Session: Poster SessionTitle: Extensive phenotyping of a rat MuSK myasthenia gravis model – From neuromuscular in vivo function to molecular propertiesAuthors: Jesper Emil Jakobsgaard, Martin Brandhøj Skov, Marianne Skals, Pernille Bogetofte Thomasen, Jeanette Jeppesen Morgen, Nete Huus, Anders Findsen, Jeppe Winther, Martin Broch-Lips, Kristian Vissing, Thomas K. Pedersen Johan Palmfeldt, Thomas H. PedersenDate and Time: Wednesday, 14 May, 5:00-7:00pm CEST NMD Pharma's novel, orally bioavailable skeletal muscle specific ClC-1 inhibitor, NMD670 is currently being evaluated in a Phase 2b clinical trial in patients with generalized myasthenia gravis (gMG) following positive results from a Phase 2a proof-of-mechanism study. Additionally, the company is conducting two separate Phase 2 trials in patients living with spinal muscular atrophy and Charcot-Marie-Tooth disease. The gMG Phase 2b study, which initiated in June 2024, is a dose range-finding, double-blinded, placebo controlled study of NMD670 over 21 days in gMG patients who are anti-acetylcholine receptor (AChR), or anti-muscle-specific tyrosine kinase (MuSK) antibody positive. The study will evaluate changes in the Quantitative Myasthenia Gravis Total Score and the Myasthenia Gravis Activities of Daily Living, among other endpoints, and is taking place at clinical sites across both the US and Europe. Further information on the study can be found here: Study Details | Safety and Efficacy of 3 Dose Levels of NMD670 in Adult Patients With Myasthenia Gravis | Generalized myasthenia gravis patients who are AChR or MuSK antibody positive in the US and Europe are encouraged to participate in the study. Further information and a list of currently active investigational sites can be obtained via email at contact@ -END- Contacts NMD Pharma A/SDan Brennan, SVP Corporate and Commercial StrategyE-mail: contact@ ICR HealthcareMary-Jane Elliott / Ashley Tapp / Lindsey Neville E-mail: NMDPharma@ +44 (0)20 3709 5700 About NMD PharmaNMD Pharma A/S is a clinical-stage biotech company developing a first-in-class platform of small molecule therapies selectively targeting the skeletal muscle chloride ion channel (ClC-1) for the treatment of rare neuromuscular disorders and age-related neuromuscular diseases with high levels of patient unmet. The Company was founded on more than 15 years of muscle physiology research with a focus on regulation of skeletal muscle excitability under physical activity. NMD Pharma has built a world-leading muscle electrophysiology platform leveraging the in-depth know-how of muscle physiology and muscular disorders, small molecule modulators, enabling technologies and tools as well as in vivo pharmacology models for discovering and developing proprietary modulators of neuromuscular function. The Company has built significant clinical and development expertise as its programmes have progressed through the clinic. NMD Pharma has raised ~€155 million from investors including Novo Holdings, Lundbeckfonden BioCapital, INKEF Capital, Roche Venture Fund, and Jeito Capital. Find out more about us online at About NMD670NMD670 is NMD Pharma's lead development program. It is a first-in-class small molecule inhibitor of the skeletal muscle specific chloride ion channel 1 (CIC-1). NMD Pharma has demonstrated that CIC-1 inhibition amplifies the muscle's responsiveness to weak signals, improving neuromuscular transmission and restores skeletal muscle function. This novel treatment approach has resulted in clinical evidence of CIC-1 inhibition in myasthenia gravis (MG) and preclinical evidence in spinal muscular atrophy, Charcot-Marie Tooth (CMT) disease and sarcopenia. NMD670 has also been granted orphan-drug designation by the U.S. FDA for treatment of generalised MG and in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
