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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.

Trial Design & Results

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 was subsequently tested in the HEALEY Platform Trial, where 161 participants were randomized to 30 mg CNM-Au8, 60 mg CNM-Au8, or placebo, with a 3:1 treatment to placebo ratio, and measured over 24 weeks. The primary endpoint of change from baseline in ALSFRS-R was not met, nor were secondary endpoints of the Combined Assessment of Function and Survival joint rank test (CAFS) or change in respiratory function as measured by slow vital capacity (SVC). An exploratory analysis of survival is described in press as >90% reduction in risk of death or permanently assisted ventilation in the 30 mg group but it is important to note that this represents one individual of 59 in the treated arm vs. four individuals of 41 in the placebo arm. One individual less in the placebo group or one more in the treatment group would significantly shift this statistic so the  percentage benefit should be received with caution. It is not abnormal to have only zero or one individuals in the treated group across most six-month ALS trials.

On March 8, 2023, an additional press release described delays to clinical worsening in the treatment group of the HEALEY Platform trial. Another described a reduction in neurofilament light (NfL) levels for people treated with CNM-Au8 vs. those on placebo, suggesting this is indicative of neuroprotection as less NfL in blood has been used to demonstrate less neurons dying. Usually, this data is expressed as a change in NfL levels after treatment compared to the same group before treatment so the value or strength of an effect described here is unclear. It is also important to note that these are still small numbers and should be interpreted with caution.

In April 2022, Clene announced the intent to pursue a multi-national 300 participant, Phase 3 clinical trial which would be critical to determining whether any signals seen in the RESCUE-ALS and HEALEY trials can be confirmed in a larger study.

Summary

 The SAC believes that there is insufficient evidence at this time to conclude that CNMAu8 provides any benefit to people with ALS and looks forward to a potential Phase 3 clinical trial that will provide clearer evidence for/against its efficacy.

International Alliance of ALS/MND Associations
June 2023


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

Primary Sidebar

Drugs in Development

  • AB Science – Masitinib
  • BrainStorm Cell Therapeutics – NurOwn
  • Clene Nanomedicine – CNM-Au8
  • Collaborative Medicinal Development – CuATSM
  • ILB – Tikomed
  • Kadimastem – AstroRx
  • Methylcobalamin
  • Mitsubishi Tanabe Pharma America – Oral Edaravone
  • Neuronata-R/Lenzumestrocel
  • NeuroSense – PrimeC
  • Neuvivo – NP001
  • T Regulatory Cell Therapies
  • Prilenia Therapeutics – Pridopidine
  • SOD1 Therapies & Trials

  • MNDaSG Group PALS & CALS, Motor Neurone Disease Association, Singapore (MNDaSG)

    MNDaSG Group PALS & CALS, Motor Neurone Disease Association, Singapore (MNDaSG)

  • JP

    JP

  • Mike Rannie,  ALS Canada,  Diagnosed 2017

    Mike Rannie, ALS Canada, Diagnosed 2017

  • Ada Garrido Benavidez, Diagnosed 2016,  FYADENMAC, Mexico

    Ada Garrido Benavidez, Diagnosed 2016, FYADENMAC, Mexico

  • Lucy Lintott, Diagnosed 2013 , MND Scotland, UK

    Lucy Lintott, Diagnosed 2013 , MND Scotland, UK

  • Angie Bordaen, Diagnosed 2014,  ALS Liga België, Belgium

    Angie Bordaen, Diagnosed 2014, ALS Liga België, Belgium

  • Dorette Lüdi, Diagnosed 2014 , ALS Schweiz, Switzerland

    Dorette Lüdi, Diagnosed 2014 , ALS Schweiz, Switzerland

  • João Marcos Andrietta, Diagnosed 2008 , ABrELA, Brazil

    João Marcos Andrietta, Diagnosed 2008 , ABrELA, Brazil

  • Mike Small, Motor Neurone Disease (MND) Association, UK

    Mike Small, Motor Neurone Disease (MND) Association, UK

  • Steven Gallagher, Canada

    Steven Gallagher, Canada

  • Mary Thomas, Diagnosed 2013 , MND Australia

    Mary Thomas, Diagnosed 2013 , MND Australia

  • Margarita Pizarro, Asociacion ELA Argentina, Diagnosed 2017, Argentina

    Margarita Pizarro, Asociacion ELA Argentina, Diagnosed 2017, Argentina

  • Bayley, Australia

    Bayley, Australia

  • Fabio Correia

    Fabio Correia

  • Sharon Corosanite, Diagnosed 2014 , ALS Hope Foundation, USA

    Sharon Corosanite, Diagnosed 2014 , ALS Hope Foundation, USA

  • Irene McCaughey, Diagnosed 2011,  MND Australia

    Irene McCaughey, Diagnosed 2011, MND Australia

  • Alan Liz Ogg 29042016 000799 lo res

    Alan Liz Ogg 29042016 000799 lo res

  • Yolanda Armendariz, Diagnosed 2017 , FYADENMAC, Mexico

    Yolanda Armendariz, Diagnosed 2017 , FYADENMAC, Mexico

  • Eric Von Schaumburg, USA

    Eric Von Schaumburg, USA

  • H. Todd Kelly, Diagnosed 2013 , ALS Hope Foundation, USA

    H. Todd Kelly, Diagnosed 2013 , ALS Hope Foundation, USA

  • Diana Fernandez, Diagnosed 2009 , Asociación ELA Argentina

    Diana Fernandez, Diagnosed 2009 , Asociación ELA Argentina

  • Ian Gale, MND Australia

    Ian Gale, MND Australia

  • Claire Garry, USA

    Claire Garry, USA
    20200117_214643

  • Luis Antonio Pimenta Lima, Brazil

    Luis Antonio Pimenta Lima, Brazil

  • Sally Pauls, Diagnosed 2006 , Les Turner ALS Foundation

    Sally Pauls, Diagnosed 2006 , Les Turner ALS Foundation

  • Rolf Mauch, Association ALS Switzerland, Diagnosed 2015

    Rolf Mauch, Association ALS Switzerland, Diagnosed 2015

  • Wiebke Braach, Deutsche Gesellschaft für Muskelkranke, Germany

    Wiebke Braach, Deutsche Gesellschaft für Muskelkranke, Germany

  • Shay Rishoni

    Shay Rishoni

  • Den Haag, Diagnosed 2016 , The Netherlands

    Den Haag, Diagnosed 2016 , The Netherlands

  • Jette Odgaard Villemoes, Muskelsvindfonden, Denmark

    Jette Odgaard Villemoes, Muskelsvindfonden, Denmark

  • March of Faces Photo Submission_ALEX_ELA ARGENTINA

    March of Faces Photo Submission_ALEX_ELA ARGENTINA

  • Chen Chun-Chin

    Chen Chun-Chin

  • Jon Newsome, Les Turner ALS Foundation, USA

    Jon Newsome, Les Turner ALS Foundation, USA

  • Stephanie Christiansen Hall, Canada

    Stephanie Christiansen Hall, Canada

  • Len Johnrose,  MND Association,  Diagnosed 2017,  England

    Len Johnrose, MND Association, Diagnosed 2017, England

  • Bruno Leanza Mantegna, Diagnosed 1999 , AISLA Onlus, Italy

    Bruno Leanza Mantegna, Diagnosed 1999 , AISLA Onlus, Italy

  • Debbie Craghill, USA

    Debbie Craghill, USA

  • Chris McCauley, Diagnosed 2015 , ALS Canada

    Chris McCauley, Diagnosed 2015 , ALS Canada

  • John and Loretta Russo, USA

    John and Loretta Russo, USA
    final3878

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

    Glen Elison, ALS Hope Foundation, Diagnosed 2019, USA

  • Gudjon Sigurdsson, Diagnosed 2004 , MND Association of Iceland

    Gudjon Sigurdsson, Diagnosed 2004 , MND Association of Iceland

  • Jon Newsome, USA

    Jon Newsome, USA

  • Kirsten Harley,  Diagnosed 2013,  Australia

    Kirsten Harley, Diagnosed 2013, Australia

  • Jorge Melo, ABrELA, Brazil

    Jorge Melo, ABrELA, Brazil

  • Elkin Ramiro Gaviria Muñoz, Diagnosed December 2018

    Elkin Ramiro Gaviria Muñoz, Diagnosed December 2018

  • Fabrice Kamp, Germany

    Fabrice Kamp, Germany

  • Zelina Brito, Diagnosed 2018, Brazil

    Zelina Brito, Diagnosed 2018, Brazil

  • Antonio Ventriglia,  ALS Liga Belgium,  Diagnosed 2013

    Antonio Ventriglia, ALS Liga Belgium, Diagnosed 2013

  • Jack Buzby, USA

    Jack Buzby, USA

  • Claudia Cominetti, Associazione conSLAncio Onlus,  Italy

    Claudia Cominetti, Associazione conSLAncio Onlus, Italy

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