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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

  • Mahmood Anwar, UK

    Mahmood Anwar, UK

  • Ismail Gokcek, Turkey

    Ismail Gokcek, Turkey
    ismail_gokcek_alsmnd_tr

  • Chen Chun-Chin

    Chen Chun-Chin

  • Teddy Hanono Annie, Apoyo Integral Gila A.C., Diagnosed 2018, Mexico

    Teddy Hanono Annie, Apoyo Integral Gila A.C., Diagnosed 2018, Mexico

  • Kirsty Gerlach, MND New Zealand, Diagnosed 2017

    Kirsty Gerlach, MND New Zealand, Diagnosed 2017

  • Jason Goodman, Les Turner ALS Foundation, USA

    Jason Goodman, Les Turner ALS Foundation, USA

  • Stephanie Christiansen Hall, Canada

    Stephanie Christiansen Hall, Canada

  • Art Eggert, USA

    Art Eggert, USA

  • Willi Klein

    Willi Klein

  • Olga Cosentino, Diagnosed 2013,  Asociación ELA Argentina

    Olga Cosentino, Diagnosed 2013, Asociación ELA Argentina

  • Inta Grubb, Diagnosed 2014,  MND Australia

    Inta Grubb, Diagnosed 2014, MND Australia

  • Wilfried Leusing

    Wilfried Leusing

  • Richard Clark, MND New Zealand,  Diagnosed 2011

    Richard Clark, MND New Zealand, Diagnosed 2011

  • Mark Miller

    Mark Miller

  • Joanne Pratt, Diagnosed 2011 , MND Australia

    Joanne Pratt, Diagnosed 2011 , MND Australia

  • Andrea Zicchieri, Associazione conSLAncio Onlus, Italy

    Andrea Zicchieri, Associazione conSLAncio Onlus, Italy
    AndreaZicchieri_conSLAncioItaly

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

    Angie Bordaen, Diagnosed 2014, ALS Liga België, Belgium

  • Len Johnrose,  MND Association,  Diagnosed 2017,  England

    Len Johnrose, MND Association, Diagnosed 2017, England

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

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

  • Greg Heydet, ALS Hope Foundation, USA

    Greg Heydet, ALS Hope Foundation, USA

  • Bayley, Australia

    Bayley, Australia

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

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

  • Phil Rossall, MND-Association, UK

    Phil Rossall, MND-Association, UK

  • Mauril Belanger

    Mauril Belanger

  • Alberto Baez Murillo, Colombia

    Alberto Baez Murillo, Colombia

  • Daniel Hare

    Daniel Hare

  • Dick Dayton, USA

    Dick Dayton, USA

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

    Glen Elison, ALS Hope Foundation, Diagnosed 2019, USA

  • Karl Hughes, Diagnosed 2010 , IMNDA,  Ireland

    Karl Hughes, Diagnosed 2010 , IMNDA, Ireland

  • Ian Gale, MND Australia

    Ian Gale, MND Australia

  • Natalya Rybakova, Russia

    Natalya Rybakova, Russia

  • Steve Lufkin, USA

    Steve Lufkin, USA
    IMG_3993

  • Horacio Fritzer, Argentina

    Horacio Fritzer, Argentina

  • Monica Soriano, Diagnosed 2011 ,  Asociación ELA , Argentina

    Monica Soriano, Diagnosed 2011 , Asociación ELA , Argentina

  • Wilfried Leusing, Diagnosed 2010 , DGM, Germany

    Wilfried Leusing, Diagnosed 2010 , DGM, Germany

  • Carlos Gomez Matallanas, Diagnosed 2014 , FUNDELA, Spain

    Carlos Gomez Matallanas, Diagnosed 2014 , FUNDELA, Spain

  • Claudia Gotti, Brazil

    Claudia Gotti, Brazil

  • Liz Ogg, Diagnosed 2013 , MND Scotland, UK

    Liz Ogg, Diagnosed 2013 , MND Scotland, UK

  • Cath Muir

    Cath Muir
    Cath

  • Francisco Perez Palop, Diagnosed 2013 , FUNDELA, Spain

    Francisco Perez Palop, Diagnosed 2013 , FUNDELA, Spain

  • Lin Yong Yi, Taiwan MND Association, Diagnosed 2004

    Lin Yong Yi, Taiwan MND Association, Diagnosed 2004

  • Frank "Papa" Taylor

    Frank “Papa” Taylor

  • Jeff Sutherland

    Jeff Sutherland
    jspic

  • Fabio Carvalho, Associação Pró-Cura da ELA, Brazil

    Fabio Carvalho, Associação Pró-Cura da ELA, Brazil

  • Carlos Alberto Báez Murillo, ACELA, Colombia

    Carlos Alberto Báez Murillo, ACELA, Colombia

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

    Bruno Leanza Mantegna, Diagnosed 1999 , AISLA Onlus, Italy

  • Kris Van Reusel, Belgium

    Kris Van Reusel, Belgium

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

    Erwin Coppejans, Diagnosed 2007 , ALS Liga België, Belgium

  • Malcolm Buck, Australia

    Malcolm Buck, Australia

  • Fernando Ocampo Cardona, Colombia

    Fernando Ocampo Cardona, Colombia

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