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

International Alliance of ALS/MND Associations

  • Members’ Login
  • Contact
  • Join
  • Donate
  • What is ALS/MND?
  • Events
    • Alliance Meeting 2021
    • Allied Professionals Forum 2021
    • ALS/MND Connect 2020
    • International Symposium on ALS/MND 2020
    • Awards
    • Webinars
  • March of Faces
  • Find an Association
  • Support for Members
    • Partnership Programme
    • Mentorship Programme
    • Alliance Support Grant
  • Advocacy
    • Advocacy Toolkit
    • Rights of People with ALS/MND
    • Biogen – Tofersen
    • BrainStorm Cell Therapeutics – NurOwn
    • Collaborative Medicinal Development – CuATSM
    • Cytokinetics – Reldasemtiv
    • Right to Try
    • Unproven (Off-Label) Treatments
    • US FDA Orphan Drug Designation
    • T Regulatory Cell Therapies
    • Amylyx – AMX0035
    • Kadimastem – AstroRx
  • Global Day
  • Resources
    • Submit a Resource
  • Newsletters
  • About
    • About the Alliance
    • Newsletter subscription
    • 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

Click here for Coronavirus (COVID-19) updates

Amylyx – AMX0035

Amylyx – AMX0035

Background

Amylyx Pharmaceuticals Inc. is a company that started in 2013 with the aim of testing a combination product called AMX0035 as a potential treatment for ALS and other neurodegenerative disorders. AMX0035 is an oral drug combining two compounds called sodium phenylbutyrate (PB) and tauroursodeoxycholic acid (TUDCA).

AMX0035 was tested in a phase 2/3 clinical trial called CENTAUR, which consisted of 137 participants recruited across 25 sites in the United States through the Northeast ALS (NEALS) Consortium. The trial was randomized, double-blind and placebo-controlled, and participants were assessed for a 24-week period for both safety and any effect of AMX0035 on disease progression.

All participants in the trial (active drug and placebo) were also provided an option to enroll in an openlabel extension where they would receive AMX0035. This extension study will add important survival data that Amylyx intends to publish in the near future.

The clinical trial had financial support from multiple key organizations in the ALS/MND field including The ALS Association, ALS Finding a Cure and the Northeast ALS Consortium (NEALS).

Results of the trial are published in The New England Journal of Medicine here.

A press release delineated the key findings of the publication as follows:

  • Patients retained function longer on AMX0035 versus placebo; study achieved its primary outcome of a difference on the Revised ALS Functional Rating Scale (ALSFRS-R)
  • AMX0035 is the first investigational therapy to demonstrate statistically significant benefit on this prespecified primary outcome in people with ALS since approved therapy edaravone
  • AMX0035 showed numerical benefits on secondary outcomes including measures of muscle strength, breathing, and hospitalizations
  • AMX0035 was generally well tolerated with similar rates of adverse events recorded in the AMX0035 and placebo groups

The publication further indicates that the effects were seen in addition to those provided by riluzole and edaravone use, though a better understanding of this additive value observation will benefit from further study. While the treatment was considered reasonably safe and tolerable, the publication also outlines that early gastrointestinal adverse events were notable and will need monitoring in future use.

An academic editorial that comments on the trial is also available here. It outlines a cautious approach to interpreting the data while balancing that these results are indeed promising. The key points of the editorial are as follows:

  • Well-designed, multi-center trial with “tantalizing preliminary data”
  • Trial was enriched for individuals with more rapidly progressive disease, making interpretation difficult for the wider population of people living with ALS/MND
  • Secondary outcome measures were not convincingly aligned with the affect on ALSFRS-R
  • Recommendation to proceed to a confirmatory phase 3 trial with wider eligibility criteria

Additional information

a) TUDCA clinical trial

One of the compounds in AMX0035, TUDCA is also in a phase 3 clinical trial alone with 440 participants across 9 sites in Europe supported by the TRICALS initiative. In this trial, TUDCA will be tested for 18 months with twice per day oral dosing. A small phase 2 Italian clinical trial which suggested that TUDCA may positively affect disease progression over 54 weeks, was published here in 2016.

b) Sodium phenylbutyrate clinical trial

Sodium phenylbutyrate was evaluated in a small clinical trial by the NEALS consortium and published here in 2009. It was considered safe and tolerable but was not designed to determine an effect on disease progression.

Both compounds have demonstrated some success at modifying disease course in preclinical animal models.

Precautions on Self treatment

Sodium phenylbutyrate is available in some countries through prescription, approved to treat urea cycle disorders. TUDCA is widely available over the counter in many forms and as part of various supplements. It is unknown whether taking these separately will have the same affect as AMX0035, or if it’s the combination that enhances the effect. Furthermore, it is unknown what the purity or active compound level of any over the counter TUDCA and sodium phenylbutyrate sources will be.

Recommendation

The SAC recommends that the Alliance communicates this as a promising set of results that are peer reviewed and achieved in a well-designed clinical trial, but also that much remains to be learned about the effect of AMX0035 in ALS/MND. The trial authors emphasize that these findings will need to be confirmed in “longer and larger trials” and the SAC encourages an approach that balances critical scientific rigour with empathy for the urgent need to have safe and effective therapies for people living with ALS/MD. Whether Amylyx seeks approval for marketing in some countries, combined with a confirmatory trial or moves forward with a larger phase 3 trial alone, the SAC will continue to update this document as our understanding of AMX0035 in ALS/MND evolves. The SAC encourages any member organization to reach out to the company directly to enquire whether any plans exist for their country or region. The SAC will continue to keep the Alliance apprised of any information as it becomes known regarding next steps for Amylyx, including updating this document when open label extension/survival data is released/published in the months ahead. With regard to self treatment regimens, the SAC strongly encourages individuals to speak with their ALS physician before considering. These promising results only pertain to compounded PB and TUDCA at the concentrations and purity tested in AMX0035.

 

 

International Alliance of ALS/MND Associations
September 2020

 


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

Primary Sidebar

  • Steven Spencer, Diagnosed 2014 - MND New Zealand

    Steven Spencer, Diagnosed 2014 – MND New Zealand

  • Eddy LeFrançois, Diagnosed 1992 - ALS Canada

    Eddy LeFrançois, Diagnosed 1992 – ALS Canada

  • David Watson, MND Scotland, Diagnosed 2018, Scotland

    David Watson, MND Scotland, Diagnosed 2018, Scotland

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

    Angie Bordaen, Diagnosed 2014 – ALS Liga België, Belgium

  • Frank "Papa" Taylor

    Frank “Papa” Taylor

  • 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

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

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

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

    Brigitte Wernli, Association ALS Switzerland, Diagnosed 2014, Switzerland

  • Conny van der Meijden, Diagnosed 2001 - ALS Netherlands

    Conny van der Meijden, Diagnosed 2001 – ALS Netherlands

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

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

  • Liong Ting Ngu, MND Malaysia, Diagnosed 2014, Malaysia

    Liong Ting Ngu, MND Malaysia, Diagnosed 2014, Malaysia

  • Shay Rishoni, Diagnosed 2011 - Prize4Life, Israel

    Shay Rishoni, Diagnosed 2011 – Prize4Life, Israel

  • 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

  • Lachlan Terry, MND Australia, Diagnosed 2015, Australia

    Lachlan Terry, MND Australia, Diagnosed 2015, Australia

  • Maurice LeClerc, ALS Canada

    Maurice LeClerc, ALS Canada

  • Joyce Rusinak, Forbes Norris ALS Center, USA

    Joyce Rusinak, Forbes Norris ALS Center, USA

  • Chris McCauley, Diagnosed 2015 - ALS Canada

    Chris McCauley, Diagnosed 2015 – ALS Canada

  • Joanne Pratt, Diagnosed 2011 - MND Australia

    Joanne Pratt, Diagnosed 2011 – MND Australia

  • Sally Pauls, Diagnosed 2006 - Les Turner ALS Foundation

    Sally Pauls, Diagnosed 2006 – Les Turner ALS Foundation

  • Zelina-Brito-Diagnosed-2018-Brazil

    Zelina-Brito-Diagnosed-2018-Brazil

  • Brian Lovell, Diagnosed 2011 - MND Australia

    Brian Lovell, Diagnosed 2011 – MND Australia

  • Francisco Perez Palop, Diagnosed 2013 - FUNDELA, Spain

    Francisco Perez Palop, Diagnosed 2013 – FUNDELA, Spain

  • Alejandro Aquino, Diagnosed 2011 - Asociación ELA Argentina

    Alejandro Aquino, Diagnosed 2011 – Asociación ELA Argentina

  • Wilfried Leusing, Diagnosed 2010 - DGM, Germany

    Wilfried Leusing, Diagnosed 2010 – DGM, Germany

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

    Erwin Coppejans, Diagnosed 2007 – ALS Liga België, Belgium

  • Carlos Gomez Matallanas, Diagnosed 2014 - FUNDELA, Spain

    Carlos Gomez Matallanas, Diagnosed 2014 – FUNDELA, Spain

  • Dorette Lüdi, Diagnosed 2014 - ALS Schweiz, Switzerland

    Dorette Lüdi, Diagnosed 2014 – ALS Schweiz, Switzerland

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

    Susan Anderson, Diagnosed 2014 – Hope Loves Company, USA

  • Duncan Bayly - MND Australia

    Duncan Bayly – MND Australia

  • 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

  • Mike Rannie, ALS Canada, Diagnosed 2017, Canada

    Mike Rannie, ALS Canada, Diagnosed 2017, Canada

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

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

  • Oliver Juenke, DGM, Germany

    Oliver Juenke, DGM, Germany

  • Rolf Mauch, Association ALS Switzerland, Diagnosed 2015, Switzerland

    Rolf Mauch, Association ALS Switzerland, Diagnosed 2015, Switzerland

  • Mary Thomas, Diagnosed 2013 - MND Australia

    Mary Thomas, Diagnosed 2013 – MND Australia

  • Roxana Canova, Diagnosed 2012 - Asociación ELA Argentina

    Roxana Canova, Diagnosed 2012 – Asociación ELA Argentina

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

    Antonio Ventriglia, ALS Liga Belgium, Diagnosed 2013, Belgium

  • Denis Blais, Diagnosed 2015 - ALS Canada

    Denis Blais, Diagnosed 2015 – ALS Canada

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

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

  • Karl Hughes, Diagnosed 2010 - IMNDA, Ireland

    Karl Hughes, Diagnosed 2010 – IMNDA, Ireland

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

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

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

    Danny Reviers, Diagnosed 1979 – ALS Liga België, Belgium

  • Soledad Rodriguez, FUNDELA, Diagnosed 2013, Spain

    Soledad Rodriguez, FUNDELA, Diagnosed 2013, Spain

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

    Glen Elison, ALS Hope Foundation, Diagnosed 2019, USA

  • Len Johnrose, MND Association, Diagnosed 2017, England

    Len Johnrose, MND Association, Diagnosed 2017, England

  • Irene McCaughey, Diagnosed 2011 - MND Australia

    Irene McCaughey, Diagnosed 2011 – MND Australia

  • Charlie “Hark” Dourney, Diagnosed 2007 - Hark ALS, USA

    Charlie “Hark” Dourney, Diagnosed 2007 – Hark ALS, USA

  • Jon Newsome, Les Turner ALS Foundation, USA

    Jon Newsome, Les Turner ALS Foundation, USA

  • Orlando Ruiz, Diagnosed 2001 - ACELA, Colombia

    Orlando Ruiz, Diagnosed 2001 – ACELA, Colombia

Learn more about the March of Faces

Advocacy

  • Advocacy Toolkit
  • Rights of People with ALS/MND
  • Biogen – Tofersen
  • BrainStorm Cell Therapeutics – NurOwn
  • Collaborative Medicinal Development – CuATSM
  • Cytokinetics – Reldasemtiv
  • Right to Try
  • Unproven (Off-Label) Treatments
  • Amylyx – AMX0035
  • Kadimastem – AstroRx

Allied Professionals Forum Registration

The International Alliance of ALS/MND Associations will host a virtual Allied Professionals Forum spread over two days to accommodate time zones on 3 – 4 December 2020.

Click here to register for the Allied Professionals Forum

Footer

Support the Alliance

Please donate

Please consider making a donation to support our work. Thank you!

Latest Tweets

  • January 2021 Newsletter - https://t.co/658q8pNWbM January 22, 2021 8:59 pm
  • An earlier tune by @RoyTaylor10 "Watch Your Back MND" https://t.co/Q4AsjcAEvR We see you there too… https://t.co/9Bcb9E9Dj9 January 17, 2021 4:24 pm
  • Congratulations! https://t.co/r2lwGwEKWC January 6, 2021 1:31 am

Keep in touch

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

Return to top of page
  • Disclaimer
  • Privacy notice & Cookies
  • Sitemap

Copyright © 2021 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