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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

  • England-Lee-Millard, UK

    England-Lee-Millard, UK

  • 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

  • Elisabeth Zahnd, Switzerland

    Elisabeth Zahnd, Switzerland

  • Maria Lucia Wood Saldanha, Associação Pró-Cura da ELA, Brazil

    Maria Lucia Wood Saldanha, Associação Pró-Cura da ELA, Brazil

  • Colm Francis Davis, Ireland

    Colm Francis Davis, Ireland

  • Dad

    Dad

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

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

  • Chun Ju Xiao, China

    Chun Ju Xiao, China

  • Robbie Caliste, UK

    Robbie Caliste, UK

  • João Marcos Andrietta, Diagnosed 2008 , ABrELA, Brazil

    João Marcos Andrietta, Diagnosed 2008 , ABrELA, Brazil

  • Diana Fernandez, Diagnosed 2009 , Asociación ELA Argentina

    Diana Fernandez, Diagnosed 2009 , Asociación ELA Argentina

  • Marco Antonio Alvarez Mercado, Mexico

    Marco Antonio Alvarez Mercado, Mexico

  • Wilfried Leusing

    Wilfried Leusing

  • Bayley, Australia

    Bayley, Australia

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

    Cassio Fernando da Silva, Diagnosed 2013 , ABrELA, Brazil

  • Philip Brindle,  MND Association,  Diagnosed 2015,  England

    Philip Brindle, MND Association, Diagnosed 2015, England

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

    Erwin Coppejans, Diagnosed 2007 , ALS Liga België, Belgium

  • Ana María Zavala, FYADENMAC, Diagnosed 2019, Mexico

    Ana María Zavala, FYADENMAC, Diagnosed 2019, Mexico

  • IMG_1211

    IMG_1211

  • Marcelo Farinelli, Diagnosed 2006, ABrELA, Brazil

    Marcelo Farinelli, Diagnosed 2006, ABrELA, Brazil

  • Steve

    Steve

  • Eddy LeFrançois, Diagnosed 1992,  ALS Canada

    Eddy LeFrançois, Diagnosed 1992, ALS Canada

  • Roxana Canova, Diagnosed 2012 ,  Asociación ELA Argentina

    Roxana Canova, Diagnosed 2012 , Asociación ELA Argentina

  • Fabio Carvalho

    Fabio Carvalho

  • Ali Var, Turkey

    Ali Var, Turkey

  • Conny van der Meijden, Diagnosed 2001,  ALS Netherlands

    Conny van der Meijden, Diagnosed 2001, ALS Netherlands

  • Enzo Maccarrone, AISLA ONLUS, Italy

    Enzo Maccarrone, AISLA ONLUS, Italy

  • Juvenal Bayona Romero

    Juvenal Bayona Romero

  • Leon Ryba, Asociación ELA Argentina

    Leon Ryba, Asociación ELA Argentina

  • Elkin Ramiro Gaviria Muñoz, Diagnosed December 2018

    Elkin Ramiro Gaviria Muñoz, Diagnosed December 2018

  • Den Haag, Diagnosed 2016 , The Netherlands

    Den Haag, Diagnosed 2016 , The Netherlands

  • Francisco Perez Palop, Diagnosed 2013 , FUNDELA, Spain

    Francisco Perez Palop, Diagnosed 2013 , FUNDELA, Spain

  • Graham Johnson, MND Australia

    Graham Johnson, MND Australia

  • Fabrice Kamp, Germany

    Fabrice Kamp, Germany

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

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

  • Lachlan Terry,  MND Australia,  Diagnosed 2015

    Lachlan Terry, MND Australia, Diagnosed 2015

  • Shay Rishoni, Diagnosed 2011 , Prize4Life, Israel

    Shay Rishoni, Diagnosed 2011 , Prize4Life, Israel

  • Frank "Papa" Taylor

    Frank “Papa” Taylor

  • Ian Gale, MND Australia

    Ian Gale, MND Australia

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

    Sharon Corosanite, Diagnosed 2014 , ALS Hope Foundation, USA

  • Willi Klein

    Willi Klein

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

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

  • Dorette Lüdi, Diagnosed 2014 , ALS Schweiz, Switzerland

    Dorette Lüdi, Diagnosed 2014 , ALS Schweiz, Switzerland

  • Monica Soriano, Diagnosed 2011 ,  Asociación ELA , Argentina

    Monica Soriano, Diagnosed 2011 , Asociación ELA , Argentina

  • Jorge Melo, ABrELA, Brazil

    Jorge Melo, ABrELA, Brazil

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

    Margarita Pizarro, Asociacion ELA Argentina, Diagnosed 2017, Argentina

  • Catherine Pearce, Australia

    Catherine Pearce, Australia

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

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

  • Denis Blais, Diagnosed 2015 , ALS Canada

    Denis Blais, Diagnosed 2015 , ALS Canada

  • Brigitte Wernli,  Association ALS Switzerland,  Diagnosed 2014

    Brigitte Wernli, Association ALS Switzerland, Diagnosed 2014

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