Charcot–Marie–Tooth disease

ORYZON Reports Financial Results and Corporate Update for Quarter Ended December 31, 2023

Retrieved on: 
Lundi, février 26, 2024

Dr Carlos Buesa, Oryzon’s Chief Executive Officer, said: “Oryzon continued with a strong path in its clinical programs in the fourth quarter.

Key Points: 
  • Dr Carlos Buesa, Oryzon’s Chief Executive Officer, said: “Oryzon continued with a strong path in its clinical programs in the fourth quarter.
  • Research and development (R&D) expenses were $3.9 and $16.6 million for the quarter and twelve months ended December 31, 2023, respectively, compared to $5.0 and $18.1 million for the quarter and twelve months ended December 31, 2022.
  • General and administrative expenses were $1.2 and $4.2 million for the quarter and twelve months ended December 31, 2023, respectively, compared to $1.2 and $4.8 million for the quarter and twelve months ended December 31, 2022.
  • Net losses were $1.4 and $5.0 million for the quarter and twelve months ended December 31, 2023, respectively, compared to $1.6 and $5.9 million for the quarter and twelve months ended December 31, 2022.

Curi Bio, Genetox, and DreamCIS Sign MOU for Strategic Collaboration on 3D Neuromuscular Junction Model for Botox Potency Assay

Retrieved on: 
Jeudi, février 22, 2024

Key Points: 
  • View the full release here: https://www.businesswire.com/news/home/20240222795787/en/
    Curi Bio, Genetox, and DreamCIS Sign MOU for Strategic Collaboration on 3D Neuromuscular Junction Model for Botox Potency Assay (Photo: Business Wire)
    BoT is renowned for its therapeutic applications, including the treatment of chronic pain, autonomic disorders, congenital neuromuscular conditions, and cosmetic enhancements.
  • The project has supported Curi Bio’s development of a next-generation 3D human neuromuscular junction model used for disease modeling, for pharmaceutical and cosmetics potency assays, and for testing broad pathogenicity.
  • The project will deliver a functional potency assay for botox research and manufacture in a turnkey, scalable format.
  • With this MOU, Genetox shows its commitment to making Curi Bio’s NMJ-based potency assay a key part of BOTAONE’s manufacturing, quality control, and product release processes.

Orphan designation: Govorestat Treatment of Charcot-Marie-Tooth disease, 22/05/2022 Positive

Retrieved on: 
Dimanche, février 4, 2024

Key facts

Key Points: 
  • Key facts
    - Active substance
    - Govorestat
    - Intended use
    - Treatment of Charcot-Marie-Tooth disease
    - Orphan designation status
    - Positive
    - EU designation number
    - EU/3/23/2783
    - Date of designation
    - Sponsor
    Veristat Spain S.L.
  • Patients' organisations
    For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
    European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
  • EU register of orphan medicines
    The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:
    EMA list of opinions on orphan medicinal product designation
    EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:

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

Retrieved on: 
Lundi, janvier 22, 2024

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: 
Mardi, janvier 23, 2024

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: 
Mercredi, novembre 15, 2023

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.

ORYZON Reports Financial Results and Corporate Updates for Quarter Ended September 30, 2023

Retrieved on: 
Vendredi, octobre 27, 2023

Dr Carlos Buesa, Oryzon’s Chief Executive Officer, said: “Oryzon continued with a strong path in its clinical programs in the third quarter.

Key Points: 
  • Dr Carlos Buesa, Oryzon’s Chief Executive Officer, said: “Oryzon continued with a strong path in its clinical programs in the third quarter.
  • The company has started a structured dialogue with corporate pharma to identify a suitable partner to move forward the asset to Phase III, pending positive results.
  • General and administrative expenses were $0.7 and $2.9 million for the quarter and nine months ended September 30, 2023, respectively, compared to $0.7 and $3.3 million for the quarter and nine months ended September 30, 2022.
  • Net losses were $0.8 and $3.4 million for the quarter and nine months ended September 30, 2023, respectively, compared to $0.7 and $3.9 million for the quarter and nine months ended September 30, 2022.

Viralgen Vector Core and Elpida Therapeutics partner to manufacture gene therapy medicines for anticipated Spastic Paraplegia 50 (SPG50) and Charcot-Marie-Tooth disease type 4J (CMT4J) clinical trials

Retrieved on: 
Jeudi, septembre 28, 2023

SAN DIEGO, Sept. 28, 2023 (GLOBE NEWSWIRE) -- Viralgen Vector Core (Viralgen) and Elpida Therapeutics (Elpida Tx) have partnered to manufacture gene therapies for use in clinical trials sponsored by Elpida Tx involving patients living with SPG50 or CMT4J.

Key Points: 
  • SAN DIEGO, Sept. 28, 2023 (GLOBE NEWSWIRE) -- Viralgen Vector Core (Viralgen) and Elpida Therapeutics (Elpida Tx) have partnered to manufacture gene therapies for use in clinical trials sponsored by Elpida Tx involving patients living with SPG50 or CMT4J.
  • It is anticipated that these trials will take place at various sites in North America and Europe and will explore potential safety and efficacy of the new treatments to be manufactured, as well as potential quality of life improvements.
  • The same CMT4J genotype can present symptoms ranging from mild clinical signs to severe disability.2
    The partnership between Viralgen and Elpida Tx is expected to enable the efficient manufacture of the adeno-associated vector serotype 9 (AAV9) based therapies for use in Elpida Tx's anticipated multi-site clinical trials.
  • We feel so grateful and honored to be working with such an incredible partner."

PurMinds Acquires Novel Chemical Entity HDAC6 Inhibitor for Neurological Disorders

Retrieved on: 
Jeudi, septembre 28, 2023

Toronto, Ontario--(Newsfile Corp. - September 28, 2023) - PurMinds NeuroPharma ("PurMinds" or the "Company"), a neuroscience company with a robust clinical pipeline combining small molecules, psychedelic compounds and other modalities, pursuing breakthrough therapeutic solutions for neurological disorders hallmarked by neurodegeneration, today announced completion of acquisition of a small molecule novel chemical entity (NCE) known as SW-200, a non-hydroxamic acid that shows superior histone deacetylase 6 (HDAC6) selectivity.

Key Points: 
  • Toronto, Ontario--(Newsfile Corp. - September 28, 2023) - PurMinds NeuroPharma ("PurMinds" or the "Company"), a neuroscience company with a robust clinical pipeline combining small molecules, psychedelic compounds and other modalities, pursuing breakthrough therapeutic solutions for neurological disorders hallmarked by neurodegeneration, today announced completion of acquisition of a small molecule novel chemical entity (NCE) known as SW-200, a non-hydroxamic acid that shows superior histone deacetylase 6 (HDAC6) selectivity.
  • Drugs that inhibit the molecular target HDAC6 are a novel class of therapeutics that are able to functionally enhance the axonal transport of nutrients, growth factors and neurotransmitters in neurons.
  • Encouraged by scientific studies that have shown efficacy in rodent models of neurodegenerative disorders, PurMinds NeuroPharma anticipates the successful development of an HDAC6 inhibitor for Charcot-Marie Tooth Disease (CMT) - a progressive neurodegenerative disorder, which causes disabling deficits in nerve function affecting 1 in 2500 people with no current cure.
  • "HDAC6 inhibition represents an innovative therapeutic target to address a range of neurological conditions currently without a cure.

Muscular Dystrophy Association Announces Research Grants Totaling $8.4 Million

Retrieved on: 
Mercredi, septembre 13, 2023

New York, Sept. 13, 2023 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) announced today the awarding of 37 new grants with a total funding commitment of $8.4 million toward neuromuscular disease research.

Key Points: 
  • New York, Sept. 13, 2023 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) announced today the awarding of 37 new grants with a total funding commitment of $8.4 million toward neuromuscular disease research.
  • The newly funded projects aim to advance research discoveries and new therapy development in multiple areas including amyotrophic lateral sclerosis (ALS), Charcot-Marie-Tooth disease (CMT), Duchenne muscular dystrophy (DMD), Emery-Dreifuss muscular dystrophy (EDMD), limb-girdle muscular dystrophy (LGMD), spinal muscular atrophy (SMA), myotonic dystrophy (DM), and many more.
  • “The Muscular Dystrophy Association has consistently been a leader in advancing treatments for people living with muscular dystrophy, ALS and related neuromuscular diseases,” said Donald S. Wood, Ph.D., President and CEO, Muscular Dystrophy Association.
  • Internship recipients and mentors included:
    These are just a few of the many grants the Muscular Dystrophy Association has pledged to support.