• 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

Clene Nanomedicine – CNM-Au8

Clene Nanomedicine – CNM-Au8

Background

Clene is a biopharmaceutical company developing nanotherapeutics aimed at treating failure in cellular energy production. Their lead candidate is CNM-Au8, an oral liquid aimed at enhancing energy production in neurons and oligodendrocytes (cells that produce insulation for neurons to conduct signals). It is reported by the company to also increase resistance to oxidative stress and decrease the levels of potentially toxic misfolded proteins. CNM-Au8 is currently being tested in clinical trials as a potential treatment to slow progression of ALS/MND.

On November 2, 2021, Clene announced the results from their phase 2 RESCUE-ALS clinical trial. The study was a randomized, placebo-controlled, trial of 45 participants, dosed daily with 30mg, and evaluated over 36 weeks. The primary outcome for this clinical trial was percent change in a measure called MUNIX, which estimates the number of motor neuron/muscle connections. There were several other secondary and exploratory outcomes.

There was no significant change between treated and placebo groups for MUNIX, forced vital capacity, ALSFRS-R at 36 weeks. However, this trial was small and trends in each of these measures favouring CNM-Au8 suggest that a larger trial, better powered to see subtle, but potentially meaningful effects, will be valuable. Some exploratory endpoints, including a responder analysis, defined here as someone with less than a six point decline in the ALSFRS-R over 36 weeks, demonstrated a significant difference for CNM-Au8 over placebo, as did the quality of life ALSSQOL-SF score. Futher exploratory endpoints around disease progression as measured by death or initiation of tracheostomy, NIV or gastrostomy tube, and observed versus predicted survival over 96 weeks, including an open label extension, both favoured CNM-Au8 as well. A pre-specified analysis of limb onset participants demonstrated a stronger trend towards efficacy than the full cohort.

CNM-Au8 appears, to date, to be well tolerated without any significant safety concerns.

CNM-Au8 is currently being tested in the HEALEY Platform Trial and has an expected readout of results in the second half of 2022. The announced enrollment is 161 participants, in a double blind, placebo-controlled study of daily dosing at either 30mg or 60mg, measured over 24 weeks. The treatment to placebo ratio is 3:1. The primary outcome measure is change from baseline in ALSFRS-R with secondary measures of slow vital capacity (respiratory), hand-held dynamometry (muscle strength) and survival. Further exploratory endpoints in this platform trial include DNA, neurofilament assessment, speech analysis, blood/urine/CSF biomarkers and home spirometry. It is not clear if a pre-specified subgroup analysis of limb onset participants is planned.

Recommendation

The SAC recommends that there is insufficient evidence at this time to conclude that CNM-Au8 provides any benefit to people with ALS. Given the trial results to date and the ongoing, larger study as an arm of the HEALEY platform trial, there is reason for cautious optimism.

International Alliance of ALS/MND Associations
January 2022


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

Primary Sidebar

  • Philip Brindle, MND Association, Diagnosed 2015, England

    Philip Brindle, MND Association, Diagnosed 2015, England

  • Carlos Alberto Arango, Colombia

    Carlos Alberto Arango, Colombia

  • Chris McCauley, Diagnosed 2015 - ALS Canada

    Chris McCauley, Diagnosed 2015 – ALS Canada

  • Norm MacIsaac, ALS Society of Canada, ALS Society of Quebec, Diagnosed 2014, Canada

    Norm MacIsaac, ALS Society of Canada, ALS Society of Quebec, Diagnosed 2014, Canada

  • Kirsten Harley, Diagnosed 2013 - Australia

    Kirsten Harley, Diagnosed 2013 – Australia

  • Soledad Rodriguez, FUNDELA, Diagnosed 2013, Spain

    Soledad Rodriguez, FUNDELA, Diagnosed 2013, Spain

  • Nicholas (Nic) Bowman, MND Association of South Africa, Diagnosed 2016, Australia

    Nicholas (Nic) Bowman, MND Association of South Africa, Diagnosed 2016, Australia

  • Peng Yi-Wen

    Peng Yi-Wen

  • Conny van der Meijden, Diagnosed 2001 - ALS Netherlands

    Conny van der Meijden, Diagnosed 2001 – ALS Netherlands

  • Maurice LeClerc, ALS Canada

    Maurice LeClerc, ALS Canada

  • Steven Gallagher, Canada

    Steven Gallagher, Canada

  • Art Eggert, USA

    Art Eggert, USA

  • John and Loretta Russo, USA

    John and Loretta Russo, USA
    final3878

  • UK-Mahmood-Anwar

    UK-Mahmood-Anwar

  • Jo Knowlton and her dog, Scotland

    Jo Knowlton and her dog, Scotland

  • Zabun Nassar, MND Association, Diagnosed 2016, England

    Zabun Nassar, MND Association, Diagnosed 2016, England

  • Alberto Baez Murillo, Colombia

    Alberto Baez Murillo, Colombia

  • Jeff Sutherland

    Jeff Sutherland
    jspic

  • Colm Francis Davis, Ireland

    Colm Francis Davis, Ireland

  • Stephanie Christiansen Hall, Canada

    Stephanie Christiansen Hall, Canada

  • Sally Pauls, Diagnosed 2006 - Les Turner ALS Foundation

    Sally Pauls, Diagnosed 2006 – Les Turner ALS Foundation

  • David Bishop

    David Bishop

  • Robbie Caliste, UK

    Robbie Caliste, UK

  • Fabio Correia

    Fabio Correia

  • Elisabeth Zahnd, Switzerland

    Elisabeth Zahnd, Switzerland

  • Juvenal Bayona Romero

    Juvenal Bayona Romero

  • Fabrice Kamp, Germany

    Fabrice Kamp, Germany

  • Valdomiro Xavier Honório, Brazil

    Valdomiro Xavier Honório, Brazil

  • Chun Ju Xiao, China

    Chun Ju Xiao, China

  • Christian Bär, Germany

    Christian Bär, Germany

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

    Glen Elison, ALS Hope Foundation, Diagnosed 2019, USA

  • Dorette Lüdi, Diagnosed 2014 - ALS Schweiz, Switzerland

    Dorette Lüdi, Diagnosed 2014 – ALS Schweiz, Switzerland

  • Oliver Juenke, DGM, Germany

    Oliver Juenke, DGM, Germany

  • Antonio Ventriglia, ALS Liga Belgium, Diagnosed 2013, Belgium

    Antonio Ventriglia, ALS Liga Belgium, Diagnosed 2013, Belgium

  • Francisco Perez Palop, Diagnosed 2013 - FUNDELA, Spain

    Francisco Perez Palop, Diagnosed 2013 – FUNDELA, Spain

  • Joy Blakeley, Diagnosed 2017 - MND Australia

    Joy Blakeley, Diagnosed 2017 – MND Australia

  • Luis Antonio Pimenta Lima, Brazil

    Luis Antonio Pimenta Lima, Brazil

  • Denis Blais, Diagnosed 2015 - ALS Canada

    Denis Blais, Diagnosed 2015 – ALS Canada

  • Mark Miller

    Mark Miller

  • Shay Rishoni, Diagnosed 2011 - Prize4Life, Israel

    Shay Rishoni, Diagnosed 2011 – Prize4Life, Israel

  • Steve Lufkin, USA

    Steve Lufkin, USA
    IMG_3993

  • Chen Chun-Chin

    Chen Chun-Chin

  • Daniel Hare

    Daniel Hare

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

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

  • Len Johnrose, MND Association, Diagnosed 2017, England

    Len Johnrose, MND Association, Diagnosed 2017, England

  • David Watson, MND Scotland, Diagnosed 2018, Scotland

    David Watson, MND Scotland, Diagnosed 2018, Scotland

  • Joanne Pratt, Diagnosed 2011 - MND Australia

    Joanne Pratt, Diagnosed 2011 – MND Australia

  • Irene McCaughey, Diagnosed 2011 - MND Australia

    Irene McCaughey, Diagnosed 2011 – MND Australia

  • Guido De Mets, Belgium

    Guido De Mets, Belgium

  • Joyce Rusinak, Forbes Norris ALS Center, USA

    Joyce Rusinak, Forbes Norris ALS Center, USA

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