Evidence map›Paper›PMID 40055280›Full record

ArticleCNS drugs2025

The Safety Profile of Pridopidine, a Novel Sigma-1 Receptor Agonist for the Treatment of Huntington's Disease.

Yigal Paul Goldberg, Leehee Navon-Perry, Andrés Cruz-Herranz, Kelly Chen, Gabriele Hecker-Barth, Katrin Spiegel, Yael Cohen, Martin Niethammer, Andrew M Tan, Henk Schuring and 2 more

Abstract read
In one paragraph

Article in CNS drugs, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 10 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
10citing papers in PubMed, 2 pooled it
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

10 citing papers in PubMed, 2 syntheses or guidelines pooled it.

  1. Systematic Review of the Huntington's Disease Drug Development Pipeline, 2014 to 2025.Movement disorders : official journal of the Movement Disorder Society · 2026
    Pooled it
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  3. Trial
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  6. Article
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  8. Choline-An Essential Nutrient with Health Benefits and a Signaling Molecule.International journal of molecular sciences · 2025
    Review
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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

12 authors.

Yigal Paul GoldbergPrilenia Therapeutics B.V., Naarden, The Netherlands.
Leehee Navon-PerryPrilenia Therapeutics B.V., Naarden, The Netherlands.
Andrés Cruz-HerranzPrilenia Therapeutics B.V., Naarden, The Netherlands.
Kelly ChenPrilenia Therapeutics B.V., Naarden, The Netherlands.
Gabriele Hecker-BarthGHB Hecker-Barth Biopharma Consulting GmbH, Konstanz, Germany.
Katrin SpiegelPrilenia Therapeutics B.V., Naarden, The Netherlands.
Yael CohenPrilenia Therapeutics B.V., Naarden, The Netherlands.
Martin NiethammerNorth Shore University Hospital, Northwell Health, Manhasset, NY, USA.
Andrew M TanPrilenia Therapeutics B.V., Naarden, The Netherlands.ORCID 0000-0002-0099-8814
Henk SchuringPrilenia Therapeutics B.V., Naarden, The Netherlands.
Michal GevaPrilenia Therapeutics B.V., Naarden, The Netherlands.
Michael R HaydenPrilenia Therapeutics B.V., Naarden, The Netherlands. Michael.Hayden@prilenia.com.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundHuntington's disease (HD) is a rare, fatal, chronic progressive neurodegenerative disorder with a significant unmet medical need for effective treatments. Pridopidine is a novel, first-in-class, highly selective and potent sigma-1 receptor (S1R) agonist in development for HD. Pridopidine has been extensively studied in adult HD across the full spectrum of disease severity and age ranges, and its safety profile has been characterized in approximately 1600 participants across multiple studies and a broad range of doses. The specific objective of this study was to gain an in-depth understanding of pridopidine's safety profile at the recommended human dose of 45 mg twice daily (bid) in patients with HD.

methodsAn integrated safety analysis of pooled data from 1067 patients with HD enrolled in four double-blind, placebo-controlled studies was performed. The safety profile of pridopidine was compared with placebo.

resultsPridopidine was found to be generally safe and well tolerated with an adverse event (AE) profile comparable to that of placebo. Moreover, there were no significant differences observed in the safety profile of pridopidine compared with placebo when analyzed by age, sex, baseline total functional capacity (TFC), cytosine-adenine-guanine (CAG) repeat length, use of antidopaminergic medications (ADMs), and region.

conclusionsThe integrated analysis replicated and corroborated the good safety profile observed in the individual studies. Despite the larger sample size, no new safety signals emerged. Long-term exposure to pridopidine, up to 6.5 years in open-label extension studies, revealed no new safety concerns, supporting its potential for long-term use in patients with HD.

Indexed as

Huntington DiseasePiperidinesReceptors, sigmaAdultAgedDouble-Blind MethodFemaleHumansMaleMiddle AgedSigma-1 ReceptorYoung AdultPiperidinespridopidineReceptors, sigmaSigma-1 Receptor

Identifiers

PMID40055280
PMCPMC11982116

What Socratic holds

Textmetadata
LicenceCC BY-NC
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the Socratic graph.