• Skip to primary navigation
  • Skip to main content
  • Skip to primary sidebar
  • Skip to footer
  • Email
  • Facebook
  • Instagram
  • Twitter
  • YouTube

International Alliance of ALS/MND Associations

  • Members' Login
  • Contact
  • Join the Alliance
  • Donate
  • What is ALS/MND
  • Find a Member Association
  • Support for PALS & CALS
    • Fundamental Rights of People with ALS/MND and Caregivers
    • Research
      • Open Science
      • Expanded Access
      • Understanding ALS/MND Research
      • Improving Regulatory Pathways
      • Right to Try
      • US FDA Orphan Drug Designation
      • Unproven (Off-Label) Treatments
      • Open Label Extension
    • Advocacy
      • Advocacy Toolkit
    • Clinical Care
      • Genetic Counselling & Testing
      • Mental Health Support
      • Nursing and Symptom Management
      • Nutrition and Swallowing
      • Occupational Therapy and Activities of Daily Living
      • Physiotherapy and Mobility
      • Respiratory Care
      • Speech Therapy and Communication
      • Support for Family & Caregivers
      • Technology
    • Drugs in Development
      • AB Science – Masitinib
      • Amylyx – AMX0035
      • Biogen – Tofersen
      • BrainStorm Cell Therapeutics – NurOwn
      • Clene Nanomedicine – CNM-Au8
      • Collaborative Medicinal Development – CuATSM
      • Cytokinetics – Reldesemtiv
      • Kadimastem – AstroRx
      • Mitsubishi Tanabe Pharma America – Oral Edaravone
      • Orphazyme – Arimoclomol
      • T Regulatory Cell Therapies
    • Approved Drugs
      • Radicava/Edaravone
      • Neudexta
      • Riluzole/Tiglutik
  • Support for Health Professionals
    • How to Break the News in ALS/MND
  • Events/Programs
    • Calendar of Events/Programs
    • Alliance Webinars
    • Alliance Meeting
    • Allied Professionals Forum
    • ALS/MND Connect
    • Global Day
    • March of Faces
    • International Symposium on ALS/MND
  • About
    • Who We Are
    • Board of Directors
    • Committees/Advisory Councils
      • Scientific Advisory Council
      • PALS and CALS Advisory Council
      • Innovation and Technology Council
      • Governance Committee
      • Budget and Finance Committee
    • Staff
    • History
    • Archives
      • Newsletters
      • Meetings
    • Awards
      • Student Award Nomination
      • Forbes Norris Award Nomination
      • Humanitarian Award Nomination
      • Allied Health Professional Award Nomination
  • Members
    • Member Registration
    • Forgot Password

Orphazyme – Arimoclomol

Orphazyme – Arimoclomol

Background

Arimoclomol is an oral capsule drug that enhances a mechanism known as the heat shock response. When a cell of the body is exposed to stress or damage, the proteins required for the cell’s normal functions can change their shape or “fold” and either lose their ability to do their job or become toxic. Cells protect themselves from this misfolding of proteins by stimulating production of heat shock proteins (Hsps) that are designed to manage the proper protein refolding.

Misfolded and clumped proteins have long been a hallmark of ALS/MND and it is believed that these may contribute to multiple disease processes. Unlike other cells, neurons, and particularly motor neurons, have an impaired ability to produce an effective heat shock response. Therefore, drugs that can enhance the production of Hsps may have therapeutic value in ALS/MND.

Arimoclomol was first published to have an effect on elevating Hsps and delaying disease progression in an ALS/MND mouse model in 2004. In 2008, it was tested for safety, tolerability and pharmacokinetics in 84 people living with ALS/MND by the Northeast ALS Consortium (NEALS), indicating it could be dosed safely up to three times daily at 100 mg per dose and that it effectively crossed the blood-brain barrier. A follow up academic (non pharma, investigator-initiated) clinical trial led by Dr. Michael Benatar, examined arimoclomol in a double-blind, placebo-controlled study of 38 people with fast-progressing ALS caused by SOD1 mutations at 200 mg/day over 12 months. Again arimoclomol was deemed safe and tolerable but the indication that the drug may slow down disease progression and prolong survival was not statistically significant, and they could not draw conclusions about its efficacy in ALS/MND. It was considered safe and well-tolerated, with only one person stopping treatment due to skin rash.

Orphazyme was founded in Denmark in 2009 based on work demonstrating that Hsps could also correct an abnormality in a cellular structure called the lysosome, implicated in diseases called lysosomal storage diseases. In advancing arimoclomol as an Hsp-inducing drug for these diseases, the company also initiated an ALS program, using the substantial groundwork to initiate a phase 3 clinical trial.

The multicenter, randomized, double-blind, placebo-controlled study was started in 2018, enrolling 245 people living with ALS, with a 2:1 treatment to placebo ratio and studied over 76 weeks. A measure called the Combined Assessment of Function and Survival was used as the primary means of determining if arimoclomol is effective in ALS, while other common measures like ALSFRS-R, survival and slow vital capacity (SVC) were also evaluated. Arimoclomol is an oral capsule that was taken three times daily for the duration of the study.

Trial Results

On May 7, 2021, an Orphazyme press release stated that the “pivotal trial did not meet primary and secondary endpoints evaluating impact on function and survival”. This indicates that arimoclomol will not be further pursued as a treatment for ALS/MND. A forthcoming publication of the data, including additional biomarker work, is expected.

Recommendation

Orphazyme is expected to communicate an initial interpretation of their data in press release form, sometime in Q2 of 2021. The SAC will provide more comment at that time.

 

International Alliance of ALS/MND Associations
May 2021

 


The original language of communication is English and any translation cannot be guaranteed for accuracy of messaging.

Primary Sidebar

  • Joanne Pratt, Diagnosed 2011 - MND Australia

    Joanne Pratt, Diagnosed 2011 – MND Australia

  • Frank "Papa" Taylor

    Frank “Papa” Taylor

  • Duncan Bayly - MND Australia

    Duncan Bayly – MND Australia

  • Jack Buzby, USA

    Jack Buzby, USA

  • Steve Lufkin, USA

    Steve Lufkin, USA
    IMG_3993

  • Stephanie Christiansen Hall, Canada

    Stephanie Christiansen Hall, Canada

  • Elkin Ramiro Gaviria Muñoz, Diagnosed  December 2018

    Elkin Ramiro Gaviria Muñoz, Diagnosed December 2018

  • Josée Kolijn-de Man, Diagnosed 2015 - ALS Patients Connected, The Netherlands

    Josée Kolijn-de Man, Diagnosed 2015 – ALS Patients Connected, The Netherlands

  • Elisabeth Zahnd, Switzerland

    Elisabeth Zahnd, Switzerland

  • Alejandro Aquino, Diagnosed 2011 - Asociación ELA Argentina

    Alejandro Aquino, Diagnosed 2011 – Asociación ELA Argentina

  • Dorette Lüdi, Diagnosed 2014 - ALS Schweiz, Switzerland

    Dorette Lüdi, Diagnosed 2014 – ALS Schweiz, Switzerland

  • Glen Elison, ALS Hope Foundation, Diagnosed 2019, USA

    Glen Elison, ALS Hope Foundation, Diagnosed 2019, USA

  • England-Lee-Millard, UK

    England-Lee-Millard, UK

  • Carlos Alberto Arango, Colombia

    Carlos Alberto Arango, Colombia

  • Marcel R. Wernard, Diagnosed 2016 - ALS Patients Connected, The Netherlands

    Marcel R. Wernard, Diagnosed 2016 – ALS Patients Connected, The Netherlands

  • Inta Grubb, Diagnosed 2014 - MND Australia

    Inta Grubb, Diagnosed 2014 – MND Australia

  • Richard Clark, MND New Zealand, Diagnosed 2011, New Zealand

    Richard Clark, MND New Zealand, Diagnosed 2011, New Zealand

  • Sanjay Kumar Srivastava, Asha Ek Hope Foundation for ALS/MND, Diagnosed 2018, India

    Sanjay Kumar Srivastava, Asha Ek Hope Foundation for ALS/MND, Diagnosed 2018, India

  • Colm Francis Davis, Ireland

    Colm Francis Davis, Ireland

  • John and Loretta Russo, USA

    John and Loretta Russo, USA
    final3878

  • Guido De Mets, Belgium

    Guido De Mets, Belgium

  • Roy

    Roy
    roy

  • Steven Gallagher, Canada

    Steven Gallagher, Canada

  • Carlos Gomez Matallanas, Diagnosed 2014 - FUNDELA, Spain

    Carlos Gomez Matallanas, Diagnosed 2014 – FUNDELA, Spain

  • Steven Spencer, Diagnosed 2014 - MND New Zealand

    Steven Spencer, Diagnosed 2014 – MND New Zealand

  • Danny Reviers, Diagnosed 1979 - ALS Liga België, Belgium

    Danny Reviers, Diagnosed 1979 – ALS Liga België, Belgium

  • Alberto Baez Murillo, Colombia

    Alberto Baez Murillo, Colombia

  • Ailsa Malcolm-Hutton, Diagnosed 2013 - MND Association of England, Wales and N Ireland

    Ailsa Malcolm-Hutton, Diagnosed 2013 – MND Association of England, Wales and N Ireland

  • David Watson, MND Scotland, Diagnosed 2018, Scotland

    David Watson, MND Scotland, Diagnosed 2018, Scotland

  • Zelina-Brito-Diagnosed-2018-Brazil

    Zelina-Brito-Diagnosed-2018-Brazil

  • Osiel Mendoza, Diagnosed 2016 - ALS Therapy Development Institute, USA

    Osiel Mendoza, Diagnosed 2016 – ALS Therapy Development Institute, USA

  • Art Eggert, USA

    Art Eggert, USA

  • Sally Pauls, Diagnosed 2006 - Les Turner ALS Foundation

    Sally Pauls, Diagnosed 2006 – Les Turner ALS Foundation

  • Roxana Canova, Diagnosed 2012 - Asociación ELA Argentina

    Roxana Canova, Diagnosed 2012 – Asociación ELA Argentina

  • Amparo Muriel Engativa, Colombia

    Amparo Muriel Engativa, Colombia

  • Ian Roberts

    Ian Roberts

  • Denis Blais, Diagnosed 2015 - ALS Canada

    Denis Blais, Diagnosed 2015 – ALS Canada

  • Susan Anderson, Diagnosed 2014 - Hope Loves Company, USA

    Susan Anderson, Diagnosed 2014 – Hope Loves Company, USA

  • Juvenal Bayona Romero

    Juvenal Bayona Romero

  • Daniel Hare

    Daniel Hare

  • Zabun Nassar, MND Association, Diagnosed 2016, England

    Zabun Nassar, MND Association, Diagnosed 2016, England

  • Margreth Burger-Saile, Diagnosed 2011 - ALS Schweiz, Switzerland

    Margreth Burger-Saile, Diagnosed 2011 – ALS Schweiz, Switzerland

  • Wilfried Leusing, Diagnosed 2010 - DGM, Germany

    Wilfried Leusing, Diagnosed 2010 – DGM, Germany

  • Brigitte Wernli, Association ALS Switzerland, Diagnosed 2014, Switzerland

    Brigitte Wernli, Association ALS Switzerland, Diagnosed 2014, Switzerland

  • Erwin Coppejans, Diagnosed 2007 - ALS Liga België, Belgium

    Erwin Coppejans, Diagnosed 2007 – ALS Liga België, Belgium

  • Jo Knowlton and her dog, Scotland

    Jo Knowlton and her dog, Scotland

  • Francisco Perez Palop, Diagnosed 2013 - FUNDELA, Spain

    Francisco Perez Palop, Diagnosed 2013 – FUNDELA, Spain

  • Willi Klein

    Willi Klein

  • Phil Rossall, MND-Association, UK

    Phil Rossall, MND-Association, UK

  • UK-Mahmood-Anwar

    UK-Mahmood-Anwar

Learn more about the March of Faces

Latest Tweets

  • Just now

Subscribe to our Bi-Monthly Newsletter

Fill out the form below to subscribe to the Alliance’s bi-monthly newsletter, packed with updates from members associations from around the world!

Footer

Keep in touch

Sign up to receive updates and to hear what's going on in the International Alliance of ALS/MND Associations.

"*" indicates required fields

 
This field is for validation purposes and should be left unchanged.
  • Email
  • Facebook
  • Instagram
  • Twitter
  • YouTube
Return to top of page

Contact | Disclaimer | Privacy Notice & Cookies | Sitemap

Copyright © 2023 The International Alliance of ALS/MND Associations. All rights reserved.


Registered in England: Charity Number 1079504 · Site built by graphics.coop · Powered by WordPress · Members' login