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First-in-human Phase 1 open label study of the BET inhibitor ODM-207 in patients with selected solid tumours

Malaka Ameratunga1,2, Irene Braña3, Petri Bono4,5,6

  • 1The Institute of Cancer Research and Royal Marsden, London, UK.

British Journal of Cancer
|September 29, 2020
PubMed
Abstract

Insights

This Phase 1 trial investigated ODM-207, a bromodomain and extra-terminal domain (BET) inhibitor, in solid tumors. While safe up to 2mg/kg, ODM-207 demonstrated a narrow therapeutic window with no observed anti-tumor activity.

Area of Science:

  • Oncology
  • Pharmacology
  • Epigenetics

Background:

  • Bromodomain and extra-terminal domain (BET) proteins are epigenetic targets for anti-cancer drug development.
  • ODM-207 is an investigational BET inhibitor evaluated in this study.

Purpose of the Study:

  • To assess the safety, pharmacokinetics, and preliminary anti-tumor activity of ODM-207.
  • To determine the maximum tolerated dose (MTD) of ODM-207 in patients with solid tumors.

Main Methods:

  • An open-label, Phase 1 study involving dose escalation using a 3+3 design.
  • Oral administration of ODM-207 once daily.
  • Evaluation of the effect of food on pharmacokinetics.

Main Results:

  • Thirty-five patients with solid tumors, including 12 with castrate-resistant prostate cancer, were treated.
  • The highest dose studied was 2 mg/kg due to cumulative toxicity.
  • Common adverse events included thrombocytopenia, asthenia, nausea, and fatigue. No partial or complete responses were observed.

Conclusions:

  • ODM-207 demonstrated dose-dependent exposure and was safe up to 2 mg/kg.
  • The drug exhibited a narrow therapeutic window.
  • Further development may be limited by toxicity and lack of observed anti-tumor response.

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