NMD

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.

NMD Pharma announces poster and oral presentations on skeletal muscle targeted ClC-1 therapy at two leading neuromuscular disease conferences

Retrieved on: 
Friday, March 1, 2024
EVP, Exercise, Hubli railway division, Conference, Phase II, SMA, Safety, Muscular Dystrophy Association, Science, Fatigue, Muscle weakness, Patient, NMD, Poster, Myasthenia gravis, Phase, MDA, Trial of the century, Congress, Pharmaceutical industry

ClC-1 is a chloride ion channel specifically expressed in skeletal muscle cells that normally dampens muscle fiber excitability and is involved in regulating muscle fiber excitability during exercise.

Key Points: 
  • ClC-1 is a chloride ion channel specifically expressed in skeletal muscle cells that normally dampens muscle fiber excitability and is involved in regulating muscle fiber excitability during exercise.
  • ClC-1 inhibition could be a possible mechanism to improve muscle fiber activation to address muscle weakness and fatigue in neuromuscular diseases.
  • Details of NMD Pharma’s poster presentations are below:
    The 4th Scientific International Congress on SMA is taking place in Ghent, Belgium, from 14-16 March.
  • : NMD Pharma be presenting one poster and one oral presentation at the congress.

Muscular Dystrophy Association Announces Seven Recipient Organizations of Advocacy Collaboration Grants

Retrieved on: 
Thursday, February 1, 2024
Policy, Occupancy, CFO, Hospital, Neuromuscular disease, Travel, Wheelchair, MDA, Therapy, Muscular Dystrophy Association, Muscular dystrophy, Caregiver, Child, Safety, Patient, CMD, Atmosphere, Cure Rare Disease, Disease, Public policy, Air travel, NMD, VCP, DMD, Empowerment, Organization, Nursing

“Muscular Dystrophy Association is proud to announce the recipients of the MDA Advocacy Collaboration Grants.

Key Points: 
  • “Muscular Dystrophy Association is proud to announce the recipients of the MDA Advocacy Collaboration Grants.
  • “The Child Neurology Foundation is thrilled to be a recipient of Muscular Dystrophy Association’s Advocacy Collaboration Grants program to support the development of a comprehensive Needs Assessment Survey,” said Katie Hentges, Director of Programs.
  • “The Muscular Dystrophy Association Advocacy Collaboration Grant will enable Cure CMD to continue its legislative advocacy work and provide skills training, resources, and advocacy opportunities for the CMD community,” said Lani Knutson, Cure CMD Advocacy Team Lead.
  • Other innovative programs, campaigns, or initiatives that can measurably impact one or more of MDA and the collaborating organizations’ common advocacy goals.

NMD Pharma Appoints Morten Bull as SVP, General Counsel and Head of People & Business Services

Retrieved on: 
Monday, January 22, 2024
Senior, Patient, General counsel, IP, Corporate services, NMD, Culture, Spinal muscular atrophy, TDC, M&A, Law, IPO, Myasthenia gravis, Charcot–Marie–Tooth disease, Leo Pharma, SVP, Pharmaceutical industry

NMD Pharma Appoints Morten Bull as SVP, General Counsel and Head of People & Business Services

Key Points: 
  • NMD Pharma Appoints Morten Bull as SVP, General Counsel and Head of People & Business Services
    Aarhus, Denmark, 22 January 2024 – NMD Pharma A/S, a clinical-stage biotech dedicated to developing novel and improved treatments for patients living with neuromuscular diseases, announces the appointment of Morten Bull as Senior Vice President, General Counsel and Head of People & Business Services with effect from 1 January 2024.
  • He has been working with NMD Pharma since June 2023 as General Counsel on a consultancy basis prior to this appointment.
  • Before joining NMD Pharma, Morten was Executive Director and Global Head of IP and R&D Legal of LEO Pharma, where he was part of the Legal and R&D leadership teams and business partner to the M&A group.
  • Morten Bull, newly appointed SVP, General Counsel and Head of People & Business Services at NMD Pharma, said: “I have been working closely with NMD Pharma since June 2023 and am very pleased to now be joining the Company on a fulltime basis as SVP, General Counsel and Head of People & Business Services.

Vanda Pharmaceuticals Receives FDA Approval to Proceed with Investigational New Drug VCA-894A, a Novel Antisense Oligonucleotide Candidate for the Treatment of Charcot-Marie-Tooth Disease, Type 2S

Retrieved on: 
Tuesday, January 23, 2024
Patient, Life, Peripheral neuropathy, NMD, CMT, Genetics, Medicine, IND, Food, Journal, Journal of Biological Chemistry, Central nervous system, FDA, Vanda, Investigational New Drug, Atrophy, Neuromuscular disease, Charcot–Marie–Tooth disease, Asos, Gene expression, Prevalence

CMT2S is a rare subtype of Charcot-Marie-Tooth disease (CMT), an inherited peripheral neuropathy for which there is no available treatment.

Key Points: 
  • CMT2S is a rare subtype of Charcot-Marie-Tooth disease (CMT), an inherited peripheral neuropathy for which there is no available treatment.
  • VCA-894A is a novel antisense oligonucleotide (ASO) with a mechanism of action that specifically targets a cryptic splice site variant within immunoglobulin mu-binding protein 2 (IGHMBP2).
  • Truncating and missense mutations in IGHMBP2 cause Charcot-Marie Tooth disease type 2.
  • https://doi.org/10.1016/j.nmd.2021.08.001
    Crooke, S. T., Liang, X. H., Baker, B. F., & Crooke, R. M. (2021).

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.

NMD Pharma to present NMD670 clinical data from a Phase I/IIa trial in myasthenia gravis at the 28th International Annual Congress of the World Muscle Society

Retrieved on: 
Tuesday, October 3, 2023
SMA, Spinal muscular atrophy, NMD, EVP, Clinical trial, Myasthenia gravis, EDT, Congress, MG, Patient, Type III, International Congress on Tuberculosis, Pharmaceutical industry

The World Muscle Society Congress is taking place at the Charleston Convention Center in Charleston, South Carolina, US from 3-7 October.

Key Points: 
  • The World Muscle Society Congress is taking place at the Charleston Convention Center in Charleston, South Carolina, US from 3-7 October.
  • Details of NMD Pharma’s poster presentation are below:
    Date and time: Thursday, 5 October from 3:30 pm to 4:30 pm EDT.
  • "The International Congress of the World Muscle Society is one of the most significant scientific gatherings in our field, globally.
  • We eagerly anticipate the opportunity to share data from our clinical trial involving patients with myasthenia gravis," remarked Jorge A. Quiroz, EVP, and Chief Medical Officer at NMD Pharma.

NMD Pharma to present NMD670 clinical data from a Phase I/IIa trial in myasthenia gravis at the 28th International Annual Congress of the World Muscle Society

Retrieved on: 
Tuesday, October 3, 2023
SMA, Spinal muscular atrophy, NMD, EVP, Clinical trial, Myasthenia gravis, EDT, Congress, MG, Patient, Type III, International Congress on Tuberculosis, Pharmaceutical industry

The World Muscle Society Congress is taking place at the Charleston Convention Center in Charleston, South Carolina, US from 3-7 October.

Key Points: 
  • The World Muscle Society Congress is taking place at the Charleston Convention Center in Charleston, South Carolina, US from 3-7 October.
  • Details of NMD Pharma’s poster presentation are below:
    Date and time: Thursday, 5 October from 3:30 pm to 4:30 pm EDT.
  • "The International Congress of the World Muscle Society is one of the most significant scientific gatherings in our field, globally.
  • We eagerly anticipate the opportunity to share data from our clinical trial involving patients with myasthenia gravis," remarked Jorge A. Quiroz, EVP, and Chief Medical Officer at NMD Pharma.

PepGen Inc. Announces Clearance of CTA by Health Canada to Begin the FREEDOM-DM1 Phase 1 Study of PGN-EDODM1 in Patients with Myotonic Dystrophy Type 1

Retrieved on: 
Wednesday, September 6, 2023
Orphan Drug Act of 1983, Neuromuscular disease, PGN, Myotonic Dystrophy Foundation, NOL, NMD, DM1, Health Canada, CTA, SAD, Patient, FDA, Conference, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, MDF, IND, Food, Safety, D.C, Disease, Pharmaceutical industry, Biology

In addition, the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to PGN-EDODM1 for the treatment of myotonic dystrophy type 1.

Key Points: 
  • In addition, the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to PGN-EDODM1 for the treatment of myotonic dystrophy type 1.
  • The FREEDOM-DM1 clinical trial is a randomized, placebo-controlled, single ascending dose (SAD) study, intended to enroll approximately 24 adult patients with DM1 to evaluate the safety and tolerability of PGN-EDODM1.
  • We expect to report initial results from this study in 2024,” said James McArthur, Ph.D., President and CEO of PepGen.
  • PepGen continues to work closely with the FDA to resolve the clinical hold in the United States as quickly as feasible.