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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.

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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

  • Francisco Perez Palop, Diagnosed 2013 , FUNDELA, Spain

    Francisco Perez Palop, Diagnosed 2013 , FUNDELA, Spain

  • Robbie Caliste, UK

    Robbie Caliste, UK

  • Denis Blais, Diagnosed 2015 , ALS Canada

    Denis Blais, Diagnosed 2015 , ALS Canada

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

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

  • Claudia Cominetti, Associazione conSLAncio Onlus,  Italy

    Claudia Cominetti, Associazione conSLAncio Onlus, Italy

  • Alan Liz Ogg 29042016 000799 lo res

    Alan Liz Ogg 29042016 000799 lo res

  • Duncan Bayly , MND Australia

    Duncan Bayly , MND Australia

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

    Guoqiang Xu, Diagnosed 2016 , Shaanxi ALS Association, China

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

    Angie Bordaen, Diagnosed 2014, ALS Liga België, Belgium

  • Daniel Hare

    Daniel Hare

  • Feng Gin Sun, Diagnosed 2014 , Shaanxi ALS Association, China

    Feng Gin Sun, Diagnosed 2014 , Shaanxi ALS Association, China

  • Mark Miller

    Mark Miller

  • Wilfried Leusing

    Wilfried Leusing

  • Leon Ryba, Argentina

    Leon Ryba, Argentina

  • Lucy Lintott, Diagnosed 2013 , MND Scotland, UK

    Lucy Lintott, Diagnosed 2013 , MND Scotland, UK

  • Kirsty Gerlach, MND New Zealand, Diagnosed 2017

    Kirsty Gerlach, MND New Zealand, Diagnosed 2017

  • Hans Dieter Olszewski, Diagnosed 2010 , DGM, Germany

    Hans Dieter Olszewski, Diagnosed 2010 , DGM, Germany

  • Chen Chun-Chin

    Chen Chun-Chin

  • Timmy, ALS Liga

    Timmy, ALS Liga

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

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

  • IMG_2658

    IMG_2658

  • Carlos Alberto Báez Murillo, ACELA, Colombia

    Carlos Alberto Báez Murillo, ACELA, Colombia

  • Liam Dwyer, England

    Liam Dwyer, England

  • Ada Garrido Benavidez, Diagnosed 2016,  FYADENMAC, Mexico

    Ada Garrido Benavidez, Diagnosed 2016, FYADENMAC, Mexico

  • Sam Hayden-Harler, Motor Neurone Disease (MND) Association, UK

    Sam Hayden-Harler, Motor Neurone Disease (MND) Association, UK

  • Carlos Gomez Matallanas, Diagnosed 2014 , FUNDELA, Spain

    Carlos Gomez Matallanas, Diagnosed 2014 , FUNDELA, Spain

  • Jan Zuring, Diagnosed 2010 , The Netherlands

    Jan Zuring, Diagnosed 2010 , The Netherlands

  • Jean

    Jean
    jean

  • Christian Bär, Germany

    Christian Bär, Germany

  • Susan Keldani, Les Turner ALS Foundation, USA

    Susan Keldani, Les Turner ALS Foundation, USA

  • Malcolm Buck, Australia

    Malcolm Buck, Australia

  • Eddy LeFrançois, Diagnosed 1992,  ALS Canada

    Eddy LeFrançois, Diagnosed 1992, ALS Canada

  • Conny van der Meijden, Diagnosed 2001,  ALS Netherlands

    Conny van der Meijden, Diagnosed 2001, ALS Netherlands

  • Cassio Fernando da Silva, Diagnosed 2013 , ABrELA, Brazil

    Cassio Fernando da Silva, Diagnosed 2013 , ABrELA, Brazil

  • Dick Dayton, USA

    Dick Dayton, USA

  • Natalya Rybakova, Russia

    Natalya Rybakova, Russia

  • João Marcos Andrietta, Diagnosed 2008 , ABrELA, Brazil

    João Marcos Andrietta, Diagnosed 2008 , ABrELA, Brazil

  • Brigitte Wernli,  Association ALS Switzerland,  Diagnosed 2014

    Brigitte Wernli, Association ALS Switzerland, Diagnosed 2014

  • Jo Knowlton and her dog, Scotland

    Jo Knowlton and her dog, Scotland

  • Brian Parsons

    Brian Parsons

  • 393647_2252248542053_984912751_n

    393647_2252248542053_984912751_n

  • Jeff Sutherland

    Jeff Sutherland
    jspic

  • Alberto Baez Murillo, Colombia

    Alberto Baez Murillo, Colombia

  • Alfredo Santos, Diagnosed 2013 , ACELA, Colombia

    Alfredo Santos, Diagnosed 2013 , ACELA, Colombia

  • Gudjon Sigurdsson, Diagnosed 2004 , MND Association of Iceland

    Gudjon Sigurdsson, Diagnosed 2004 , MND Association of Iceland

  • Jean Waters, Diagnosed 2004, MND Association of England, Wales and N Ireland

    Jean Waters, Diagnosed 2004, MND Association of England, Wales and N Ireland

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

    Sharon Corosanite, Diagnosed 2014 , ALS Hope Foundation, USA

  • Jose Rivero Muñoz, Diagnosed 2015, FYADENMAC, Mexico

    Jose Rivero Muñoz, Diagnosed 2015, FYADENMAC, Mexico

  • Cath Muir

    Cath Muir
    Cath

  • Dad

    Dad

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