NMD Pharma

A big thank you to the European CMT Federation for inviting our CEO, Thomas Holm Pedersen, and Clinical Scientist, Thomas Skjærlund Grønnebæk, to participate in a recent #webinar, ‘ClC-1 inhibition for improvement of muscle function in CMT’. The webinar, hosted by Arabela Acalinei, provided an opportunity to introduce the NMD Pharma story and discuss the use of ClC-1 inhibitors to improve muscle function for neuromuscular diseases such as Charcot-Marie-Tooth disease (#CMT), with the aim to improve patients’ quality of life. Our presenters also share detailed information about the on-going phase 2 clinical trial studying the potential therapeutic benefit and safety of NMD670 in patients with genetically confirmed CMT subtypes 1 and 2 recruiting interested patients at sites in the US and Europe. The webinar includes an extensive Question and Answer session with the NMD presenters. For more information, watch the full webinar here: https://lnkd.in/dXdHfWXy For further information and a list of currently active clinical trial sites please email us at contact@nmdpharma.com. #CharcotMarieTooth #NMDPharma #Biotech #Innovation #NeuromuscularDisease

2024 has been an eventful year for NMD Pharma, marked by significant advances in our clinical programs and organizational growth. Highlights include: ·      A strengthened and diverse team, with several new senior recruits to support our growing business and pipeline ·      Initiation of a Phase 2 study of NMD670 in patients with Charcot-Marie-Tooth (#CMT) disease types 1 and 2 and a Phase 2b study in myasthenia gravis (#MG) patients, complementing our ongoing Phase 2 #ClinicalTrial of NMD670 in adults with spinal muscular atrophy (#SMA) ·      Active support for, and learning from, key patient organizations: #CureSMA, #SMAEurope,#FundAME #CMTRF, #HNF, #CMTA, #CMT-France, #ECMTF, #MGFA, #ConquerMG, #EuMGA, #AFM-TELETHON, #AIM, #AMES, #Muskelsvinfonden ·      Presentation of our #research at multiple leading neuromuscular disease conferences, showcasing our innovative approach to key scientific, clinical and investor audiences ·      Multiple published manuscripts in top tier peer-reviewed journals that describe data supporting the potential benefit of our skeletal muscle specific NMD670 and ClC-1 inhibitor platform with important contributions to the overall body of knowledge regarding muscle function in neuromuscular diseases, which can be viewed at: https://lnkd.in/dxVNcTt3 ·      Successful progress of numerous non-clinical safety and toxicology studies, manufacturing/CMC campaigns, and quality assurance programs that support the safe and effective use of NMD670 in our ongoing #RareDisease Phase 2 clinical trials, and also lay the foundations for subsequent Phase 3 studies As we reflect on this year of growth and scientific progress, we look forward to continuing our mission to develop transformative therapies for patients with neuromuscular diseases in the coming year. We are honored and inspired by the strength, abilities, and resilience of those impacted by rare diseases, and we look forward to matching their courage with our ambition in the coming year. Happy holidays to all! #NMDPharma #Innovation #NeuromuscularDisease #HappyHolidays

Today the NMD Pharma team announced that findings from its Phase 1 trial evaluating the safety, pharmacokinetic (PK) and pharmacodynamic (PD) profile of NMD670 in healthy volunteers were published in the peer-reviewed journal American Society for Clinical Pharmacology & Therapeutics. By inhibiting the ClC-1 ion channels, NMD670 has been shown to enhance muscle excitability and improve muscle function by alleviating the neuromuscular transmission dysfunction that is present in various neuromuscular diseases. NMD670 was generally safe and well-tolerated in healthy subjects, with no serious adverse events (SAE). Read the full paper here: https://lnkd.in/dCYn4C2v #NeuromuscularDisease #Publication #Research #Biotech #Innovation #clinicalsafetytrials

We are excited to announce that a new paper, ‘Neuromuscular Transmission Deficits in patients with CMT and ClC-1 Inhibition in CMT Animal Models,’ has been published in Annals of Clinical and Translational Neurology, providing new evidence that targeting the neuromuscular junction (NMJ) with ClC-1 inhibitors, such as lead development candidate NMD670, could enhance muscle function in patients living with Charcot-Marie-Tooth disease (#CMT). The paper, published in collaboration with the Aarhus Universitetshospital, University of Missouri-Columbia, The Ohio State University and Health - Aarhus University, details data from ESTABLISH, an international observational clinical study in CMT type 1 and type 2 and healthy volunteers, as well as animal models of the disease. Results from the former demonstrate that patients with CMT, regardless of genotype, have notable NMJ transmission dysfunction that correlates directly with disease severity, while the latter provides proof-of-mechanism for the recovery of muscle function following partial inhibition of ClC-1 channels and the application of nerve-stimulated muscle force. With no currently approved therapies to provide a cure or alleviate symptoms, these results confirm the therapeutic potential of ClC-1 inhibition to address the muscle weakness, atrophy and fatigue characteristic of CMT, which could enable patients with this #RareDisease to live better, more independent lives. To access the full paper: https://lnkd.in/dCy959hz #CharcotMarieTooth #NeuromuscularDisease #Publication #Research #Biotech #Innovation 

#NMDPharma is #hiring! We’re looking for an experienced DMPK Scientist, based in Aarhus or Copenhagen, Denmark. If you have experience in designing DMPK strategies across all stages of clinical development, we’d love to hear from you. As a maturing #biotech with three Phase 2 trials underway for our lead development candidate, NMD670, a novel skeletal muscle-specific chloride 1 ion channel (ClC-1) inhibitor, we are looking to recruit an experienced DMPK Scientist. Key responsibilities for the role include: · Leading PK/PD and drug metabolism activities including designing, monitoring and analysing studies and using PK/PD modelling and simulation tools · Developing and implementing DMPK strategies · Providing subject matter expertise in our cross-functional teams   Find out more about this position and apply online through the link below: https://lnkd.in/dHwfnChU #NeruomuscularDisease #Innovation #Biotech #Research #Jobs #Recruitment #RareDisease

From 12-17 January 2025, members of the NMD Pharma management team will be in San Francisco, CA for the 43rd Annual J.P. Morgan Healthcare Conference. As the largest global #healthcare investment meeting in the #LifeSciences industry, we look forward to discussing our first-in-class skeletal muscle specific ClC-1 inhibitor approach for the treatment of severe neuromuscular diseases with global #biopharma leaders and key #investors. This includes lead development candidate NMD670, which is currently being investigated in three separate Phase 2 clinical trials in disorders characterised by compromised neuromuscular transmission, including spinal muscular atrophy (#SMA), myasthenia gravis (#MG), and Charcot-Marie-Tooth disease (#CMT). If successful, this therapeutic approach could improve muscle function and endurance in patients living with neuromuscular diseases, enabling them to live better, more independent lives. If you are also attending #JPM25 and are interested in meeting with Thomas Holm Pedersen, CEO, Thomas Kongstad Petersen, COO, Dan Brennan, SVP, Corporate and Commercial Strategy, or Morten Bull, SVP, General Counsel and Head of People & Business Services, please reach out to contact@nmdpharma.com. #JPM25 #NMDPharma #Innovation #Conference #NeuromuscularDisease

NMD Pharma novel skeletal muscle-targeted ClC-1 ion channel has been featured in an article published by @Labiotech.eu, exploring the latest clinical developments that aim to enable those living with Charcot-Marie-Tooth disease (#CMT) to live better, more independent lives. The article highlights our recently initiated Phase 2 #ClinicalTrial of NMD670, an oral first-in-class small molecule inhibitor of the skeletal muscle specific ClC-1 ion channel, in patients with CMT types 1 and 2. By inhibiting this ion channel in those with neuromuscular deficits and disease,  NMD670 has the potential to increase neuromuscular transmission and improve muscle function to address the muscle weakness and fatigue that are characteristic of this #RareDisease, helping muscles to contract in order to provide increased muscle strength and endurance. To find out more, read the full article here: https://lnkd.in/dTJXq7Vz #ICYMI #NeuromuscularDisease #CharcotMarieTooth #Biotech #Innovation

Today, 3 December, is International Day of Persons with Disabilities (#IDPWD), a day to learn from the experiences of people living with a disability and make a commitment to create a world characterised by equal opportunity and rights, regardless of ability. Despite recent advancements in the treatment of severe and rare neuromuscular diseases, there still remains a significant need for novel therapies that can better help patients to manage the associate muscle weakness, fatigue and atrophy. At NMD Pharma, we are dedicated to raising awareness of and removing barriers to #inclusion through the development of our skeletal muscle-targeted chloride 1 ion channel (ClC-1) inhibitor approach, which has the potential improve muscle power and endurance in those living with neuromuscular disorders such as such as myasthenia gravis (#MG), spinal muscular atrophy (#SMA) and Charcot-Marie-Tooth disease (#CMT). We also commit to take our Passion for Movement value into action by finding ways to help turn disability into ability through many ways in addition to developing new medications. Join us in raising awareness of disability inclusion, not only today, but every day. #CMTA #CMTRF #HNF #MGFA #CureSMA #SMAEurope #ECMTF #ConquerMG 

#NMDPharma is #hiring! We’re looking for a Senior Associate of Regulatory Affairs preferably based in Denmark (Aarhus or Copenhagen), the USA (East Coast) or remotely based in Europe. If you have experience in regulatory affairs management, and global regulatory health authorities, we’d love to hear from you. As a growing #biotech with three ongoing Phase 2 trials underway we are looking to expand our team with a Senior Associate of Regulatory Affairs. Key responsibilities include coordinating the content and drafting of regulatory documentation, assisting in the development of regulatory strategy, and preparing routine submissions and related activities. Find out more about this position and apply online through the link below: https://lnkd.in/d58AhiXv #NeruomuscularDisease #Innovation #Biotech #Research #Jobs #Recruitment #RareDisease

#NMDPharma is #hiring! We’re looking for a Regulatory Writer based in Denmark (Aarhus or Copenhagen), the USA (East Coast), or remotely based in Europe. If you have experience in producing regulatory documents for health authority submissions, we’d love to hear from you. As a growing #biotech with three ongoing Phase 2 trials, we are expanding our Regulatory team and therefore looking for a Regulatory Writer on location or remote (full-time or part-time). Key responsibilities include producing investigator brochures, health authority meeting packages/briefing books, annual reports, and other key clinical and regulatory documents. Find out more about this exciting opportunity and apply online through the link below: https://lnkd.in/dnSjNcfE #NeruomuscularDisease #Innovation #Biotech #Research #Jobs #Recruitment #RareDisease