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05.25.2021

New research supports pridopidine's neuroprotective properties in Huntington disease models

  • Newly published papers further elucidate the mechanisms underlying pridopidine's neuroprotective properties through activation of the Sigma-1 Receptor (S1R).
  • Pridopidine enhances mitochondrial function and reduces mHTT-induced ER stress, which are impaired in Huntington disease, mediated by the S1R.
  • Three new peer-reviewed publications highlight pridopidine's therapeutic potential and provide data supporting the role of the S1R in neurodegenerative diseases

Prilenia Therapeutics BV, a clinical stage biotech company focused on developing novel treatments for neurodegenerative and neurodevelopmental disorders, today announces the publication of three peer-reviewed journal articles, highlighting key aspects of the mechanism of action of its lead asset, pridopidine, and the importance of S1R activation as a mechanism to attenuate biological features of neurodegenerative diseases.

S1R is a protein highly expressed in the brain where it regulates several cellular mechanisms common to neurodegenerative diseases. Activation of the S1R improves energy production, reduces cellular stress, enhances clearance of toxic proteins, and mitigates inflammation, thus supporting the continued function and survival of neurons.

These latest publications are the result of long-term collaborations with researchers from leading institutions across the globe and provide novel insights into the mechanisms driving pridopidine neuroprotective effects.

Highlights from the published articles include the following:

1. The Sigma-1 Receptor Mediates Pridopidine Rescue of Mitochondrial Function in Huntington Disease Models - Naia et al., Neurotherapeutics, 2021

* This article describes how pridopidine enhances mitochondrial functions by activation of the S1R, contributing to its neuroprotective effects and supporting its therapeutic potential in Huntington disease

2. Pridopidine reduces mutant huntingtin-induced endoplasmic reticulum stress by modulation of the Sigma-1 receptor - Shenkman et al., Journal of Neurochemistry, 2021

* This article demonstrates the effect of pridopidine on reducing mutant HTT-induced ER stress via activation of the S1R, providing additional evidence of its therapeutic potential as a selective and potent S1R agonist

3. Sigma-1 Receptor (S1R) Interaction with Cholesterol: Mechanisms of S1R Activation and Its Role in Neurodegenerative Diseases - Zhemkov et al., International Journal of Molecular Sciences, 2021

* This review article investigates the biological interactions between the S1R and cholesterol and looks into the therapeutic benefit of pridopidine as a selective and potent S1R agonist in neurodegenerative diseases.

Pridopidine is a first-in-class small molecule in clinical development for the treatment of Huntington disease and amyotrophic lateral sclerosis. It is a highly selective and potent S1R agonist in clinical development for both indications. Pridopidine demonstrates neuroprotective effects in preclinical models of Huntington disease, amyotrophic lateral sclerosis, and other neurodegenerative diseases.

Michael R. Hayden MD PhD CEO of Prilenia and world-renowned expert in Huntington Disease research, commented:

" These new insights on pridopidine and how it affects S1R continues to advance our knowledge on its mechanism of action, which supports the development of this drug for Huntington disease and amyotrophic lateral sclerosis."

Pridopidine is a safe and well-tolerated orally administered drug currently being assessed in a Global Phase 3 study (PROOF-HD), evaluating the effect of pridopidine 45 mg bid on Total Functional Capacity (TFC) in patients with early-stage Huntington disease. The study is being conducted in collaboration with the Huntington Study Group (HSG). Pridopidine is also being assessed in the first platform trial for amyotrophic lateral sclerosis in collaboration with the Healey Center for amyotrophic lateral sclerosis at Mass General Hospital.

Source: News Release
Prilenia
May 25, 2021

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