Biomarker plan – PI3K disorders

From the public pipeline, it appears that the interest is in vascular anomalies

Screenshot

Hence the biomarker plan would be the following:

  • PD Biomarker and longitudinal disease measurements: Create a sensitive ctDNA/ddPCR assay for mutant PIK3CA detection
  • Target engagement panel for PI3K pathway inhibition: pAKT, pS6, insulin/glucose signaling markers, and inflammatory mediators in blood.
  • Link PK and biomarker data: drug exposure, pathway suppression, and clinical improvement
  • Imaging studies: Link biomarker changes to MRI-based lesion volume reduction or other objective measures
  • Develop functional clinical biomarkers: Evaluate pain scores, bleeding events, swelling, mobility, and quality-of-life measures (PRO) to identify clinical indicators of treatment benefit.
  • Explorations :a) Establish a longitudinal bio-repository b) Identify biomarkers associated with responders c) Confirm PIK3CA mutation subtype (H1047R, E542K, E545K, etc.) to determine whether mutation burden = disease severity or treatment response. d) Characterize enrolled patients from other studies
  • ETC: Learn the current status of Translational medicine ( assays, biomarkers, sample collected etc) Personnel management, Operational (vendor contracts, negotiation) and plan for future

The website is : https://relaytx.com

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