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International Alliance of ALS/MND Associations

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Prilenia Therapeutics – Pridopidine

Background

Pridopidine is an orally administered small molecule that crosses the blood-brain barrier, reaching the brain, and binds to the dopamine D2/D3 and sigma-1 (S1R) receptors. Pridopidine is owned by Prilenia, who describes it as a potent and selective S1R agonist (activator) that rebalances calcium, which reduces intracellular (endoplasmic reticulum) stress, and restores synaptic function. These actions collectively contribute to the hypothesized neuroprotective effects of pridopidine, potentially restoring connectivity between neurons and maintaining their viability (Ref: Gracehv, Meyer et al., 2020; Waters et al., 2018; Prilenia Therapeutics website).

Preclinical studies have shown beneficial effects of pridopidine in cellular and mouse models of several neurodegenerative disorders (Ryskamp et al., 2019). It was originally studied as a treatment for Huntington’s Disease. However, given its characteristics it was thought to potentially also have an effect on ALS/MND disease mechanisms. In SOD1 mice pridopidine showed improvement of motor symptoms but no effect on overall survival (Ref: Estévez‐Silvaet al., 2022; Alzforum).

Trial Design and Results

In 2022, a phase 2/3 clinical trial was conducted through the HEALEY platform in 163 people with ALS/MND. The trial was randomized at a 3:1 ratio to receive active pridopidine or a matching placebo. It was a 6-month, double-blind, placebo-controlled study that rolled over into an open label extension (Ref: Quintana et al., 2023).

The drug or placebo were administered orally twice a day. The measured primary endpoint was change from baseline through 24 weeks in the total ALS functional rating scale revised (ALSFRS-R) score (Ref: Shefner et al., 2024).

In February 2023, a press release from the Healey & AMG Center and the Northeast ALS Consortium (NEALS) indicated that the primary endpoint was not met; however, pridopidine was considered safe and well tolerated. A post hoc analysis revealed that within a very small subset of participants, with definite or probable ALS/MND combined with being early, fast progressors (pridopidine n=20; placebo n=14), those treated with pridopidine had less decline in speech. Additional post hoc analyses in the same subgroup demonstrated ALSFRS-R (Δ5.2, p=0.04) and quality of life measures that favoured pridopidine over placebo (Ref: Shefner et al., 2024). While these results are potentially intriguing, it is important to note that post hoc data from small subgroups can be misleading and require confirmation in larger studies. Furthermore, the SAC feels there has been unclear communication regarding these results and, until the data is released publicly and a larger phase 3 trial is completed, it is not possible to understand if these changes represent a real effect of pridopidine on disease progression.

A global phase 3 clinical trial for pridopidine in ALS/MND has been announced. The trial, scheduled to start in 2025, will examine the effectiveness of pridopidine in ALS/MND on a larger population (Ref: Studna 2024).

Summary

Data shows that pridopidine appears safe and well tolerated at the therapeutic dose. The efficacy of pridopidine was tested through the HEALEY ALS platform but did not meet its primary endpoint (Ref: Neurology live, 2023). Post hoc analyses from the trial suggests there may be some potential benefit to speech, quality of life, and ALSFRS-R score. Due to the very small subset of participants used in these analyses, this potential benefit should be interpreted with caution.  

The Scientific Advisory Council believes that, to date, there is insufficient evidence to conclude that pridopidine provides any clinical benefit to people living with ALS and looks forward to the results of the Phase 3 clinical trial that will provide clearer evidence for/against its efficacy.

Sources

Waters et al., 2018 – https://pubmed.ncbi.nlm.nih.gov/29480206/ 

Gracehv, Meyer et al., 2020 – https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8041674/

PRILENIA WEBSITE – HTTPS://WWW.PRILENIA.COM/ABOUT-PRIDOPIDINE/

Estévez‐Silvaet al., 2022 – https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9305776/

Alzforum – https://www.alzforum.org/therapeutics/pridopidine

Quintanaet al., 2023 – https://onlinelibrary.wiley.com/doi/10.1002/ana.26714

Studna – https://www.appliedclinicaltrialsonline.com/view/prilenia-announces-plans-to-initiate-global-phase-iii-study-of-novel-als-treatment

Ryskamp et al., 2019 – https://pubmed.ncbi.nlm.nih.gov/31551669/

Shefner et al., 2024 – https://www.neurology.org/doi/10.1212/WNL.0000000000206526

clinicaltrials.gov id: NCT04297683 – https://clinicaltrials.gov/study/NCT04297683?Cond=NCT04297683&rank=1

clincaltrials.gov id: NCT04615923 – https://clinicaltrials.gov/study/NCT04615923?Cond=nct04615923&rank=

clinicaltrials.gov id: NCT06069934 – https://clinicaltrials.gov/study/NCT06069934?Cond=ALS%20-%20Amyotrophic%20Lateral%20Sclerosis&intr=Pridopidine&rank=3

Neurology live – https://www.neurologylive.com/view/insights-pridopidine-healey-als-platform-trial

International Alliance of ALS/MND Associations
February 2025


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

  • Jon Newsome, Les Turner ALS Foundation, USA

    Jon Newsome, Les Turner ALS Foundation, USA

  • Carlos Gomez Matallanas, Diagnosed 2014 , FUNDELA, Spain

    Carlos Gomez Matallanas, Diagnosed 2014 , FUNDELA, Spain

  • Steve Lufkin, USA

    Steve Lufkin, USA
    IMG_3993

  • Mark Miller

    Mark Miller

  • Angela Jansen, Deutsche Gesellschaft für Muskelkranke e.V.-DGM, Diagnosed 1995, Germany

    Angela Jansen, Deutsche Gesellschaft für Muskelkranke e.V.-DGM, Diagnosed 1995, Germany

  • Olga Cosentino, Diagnosed 2013,  Asociación ELA Argentina

    Olga Cosentino, Diagnosed 2013, Asociación ELA Argentina

  • Catherine Pearce, Australia

    Catherine Pearce, Australia

  • Steven Gallagher, Canada

    Steven Gallagher, Canada

  • Joanne Pratt, Diagnosed 2011 , MND Australia

    Joanne Pratt, Diagnosed 2011 , MND Australia

  • Anthony (Tony) Lynch, MND New South Wales, Diagnosed 2016, Australia

    Anthony (Tony) Lynch, MND New South Wales, Diagnosed 2016, Australia

  • João Marcos Andrietta, Diagnosed 2008 , ABrELA, Brazil

    João Marcos Andrietta, Diagnosed 2008 , ABrELA, Brazil

  • Oliver Juenke, Germany

    Oliver Juenke, Germany

  • Len Johnrose,  MND Association,  Diagnosed 2017,  England

    Len Johnrose, MND Association, Diagnosed 2017, England

  • Conny van der Meijden, Diagnosed 2001,  ALS Netherlands

    Conny van der Meijden, Diagnosed 2001, ALS Netherlands

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

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

  • Verónica Isabel Castro Molina, Diagnosed 2014, Argentina

    Verónica Isabel Castro Molina, Diagnosed 2014, Argentina

  • Ian Roberts

    Ian Roberts

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

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

  • Eddy LeFrançois, Diagnosed 1992,  ALS Canada

    Eddy LeFrançois, Diagnosed 1992, ALS Canada

  • Carlos Alberto Arango, Colombia

    Carlos Alberto Arango, Colombia

  • Guoqiang Xu, Diagnosed 2016 , Shaanxi ALS Association, China

    Guoqiang Xu, Diagnosed 2016 , Shaanxi ALS Association, China

  • Rolf Mauch, Association ALS Switzerland, Diagnosed 2015

    Rolf Mauch, Association ALS Switzerland, Diagnosed 2015

  • David Bishop

    David Bishop

  • David Solomon, Diagnosed 2015, MND Association of England, Wales and N Ireland

    David Solomon, Diagnosed 2015, MND Association of England, Wales and N Ireland

  • Ana María Zavala, FYADENMAC, Diagnosed 2019, Mexico

    Ana María Zavala, FYADENMAC, Diagnosed 2019, Mexico

  • Chen Chun-Chin

    Chen Chun-Chin

  • Eric Von Schaumburg, USA

    Eric Von Schaumburg, USA

  • Alfredo Santos, Diagnosed 2013 , ACELA, Colombia

    Alfredo Santos, Diagnosed 2013 , ACELA, Colombia

  • Mahmood Anwar, UK

    Mahmood Anwar, UK

  • Leon Ryba, Argentina

    Leon Ryba, Argentina

  • Maurice Leclerc, Canada

    Maurice Leclerc, Canada

  • Brian Parsons

    Brian Parsons

  • Natalya Rybakova, Russia

    Natalya Rybakova, Russia

  • Dawn Morton, Diagnosed 2014 , MND Scotland, UK

    Dawn Morton, Diagnosed 2014 , MND Scotland, UK

  • 83

    83

  • Alan Liz Ogg 29042016 000799 lo res

    Alan Liz Ogg 29042016 000799 lo res

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

    Susan Anderson, Diagnosed 2014 , Hope Loves Company, USA

  • Dick Dayton, USA

    Dick Dayton, USA

  • Andrietta

    Andrietta

  • Luis Antonio Pimenta Lima, Brazil

    Luis Antonio Pimenta Lima, Brazil

  • Marco Antonio Alvarez Mercado, Mexico

    Marco Antonio Alvarez Mercado, Mexico

  • 727747090571358167

    727747090571358167

  • Hanne Stenmose, Muskelsvindfonden, Denmark

    Hanne Stenmose, Muskelsvindfonden, Denmark

  • Anita Forte, Les Turner ALS Foundation, USA

    Anita Forte, Les Turner ALS Foundation, USA

  • Mike Rannie,  ALS Canada,  Diagnosed 2017

    Mike Rannie, ALS Canada, Diagnosed 2017

  • Francisco Perez Palop, Diagnosed 2013 , FUNDELA, Spain

    Francisco Perez Palop, Diagnosed 2013 , FUNDELA, Spain

  • Chris McCauley, Diagnosed 2015 , ALS Canada

    Chris McCauley, Diagnosed 2015 , ALS Canada

  • Joyce Rusinak, Forbes Norris ALS Center, USA

    Joyce Rusinak, Forbes Norris ALS Center, USA

  • Brian Lovell, Diagnosed 2011 . MND Australia

    Brian Lovell, Diagnosed 2011 . MND Australia

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

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

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