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  • 58-60 Avenue de la Grande Armée, 75017, Paris, France
  • +33 1 44 37 17 00
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Rare disease: Identification of new biomarker with 31 patients

Overview

A randomized Phase II study involving 31 patients with Rett Syndrome.
Participants were enrolled in a 7-week study to assess the safety, tolerability, and efficacy of ANAVEX®2-73 treatment.

Impact

    • ● The identification of a new biomarker and its follow-up in the study enabled a Fast Track designation granted by the FDA
    •  
    • ●  Successfully randomized Phase II results
    •  
    • ●  Surrogate biomarker available for Phase 3 study.

Objectives

  •  Identify a new biomarker to define a group of patients who respond better to the treatment.
  •  
  •  Identify a surrogate biomarker of response to measure the treatment’s impact at the biological level.

Method

Ariana had identified a specific variant of SIGMAR1 as a candidate biomarker of response for ANAVEX®2-73 during a study on Alzheimer’s patients. In this context, the treatment or a placebo was randomly administrated to 29 new patients with Rett Syndrome during an additional clinical trial.
KEM® was applied to 8,000 variables, including cognitive tests,Ra omics, and phenotypic data.

Result

● Identification of a new candidate biomarker:
  • – Cognitive Scores significantly improving for patients with a high value of one of the psychometric score evaluated at baseline.
  •  
  • – A longitudinal study confirms the positive interaction across time between the treatment and a high value of one of the psychometric score evaluated at baseline.
● On the top of this, the mRNA expression of SIGMAR1 was identified as surrogate biomarker of response.

Ariana Pharmaceuticals, The AI DRUG Development Company

  • Paris, FRANCE / Cambridge, MA, USA
  • Call Us: +33 144 37 17 00
  • info@arianapharma.com

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