Phase 1 study of the ATR inhibitor berzosertib in combination with cisplatin in patients with advanced solid tumours

Br J Cancer. 2021 Aug;125(4):520-527. doi: 10.1038/s41416-021-01406-w. Epub 2021 May 26.

Abstract

Background: Berzosertib (formerly M6620, VX-970) is a highly potent and selective, first-in-class ataxia telangiectasia-mutated and Rad3-related protein kinase (ATR) inhibitor. We assessed the safety, tolerability, pharmacokinetics, and preliminary efficacy of berzosertib plus cisplatin.

Methods: Adult patients with advanced solid tumours refractory or resistant to standard of care therapies received ascending doses of cisplatin (day 1) and berzosertib (days 2 and 9) every 3 weeks (Q3W).

Results: Thirty-one patients received berzosertib (90-210 mg/m2) and cisplatin (40-75 mg/m2) across seven dose levels. The most common grade ≥3 treatment-emergent adverse events were neutropenia (20.0%) and anaemia (16.7%). There were two dose-limiting toxicities: a grade 3 hypersensitivity reaction and a grade 3 increase in alanine aminotransferase. Berzosertib 140 mg/m2 (days 2 and 9) and cisplatin 75 mg/m2 (day 1) Q3W was determined as the recommended Phase 2 dose. Cisplatin had no apparent effect on berzosertib pharmacokinetics. Of the 31 patients, four achieved a partial response (two confirmed and two unconfirmed) despite having previously experienced disease progression following platinum-based chemotherapy.

Conclusions: Berzosertib plus cisplatin is well tolerated and shows preliminary clinical activity in patients with advanced solid tumours, warranting further evaluation in a Phase 2 setting.

Clinical trials identifier: NCT02157792.

Publication types

  • Clinical Trial, Phase I
  • Multicenter Study
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Aged
  • Antineoplastic Combined Chemotherapy Protocols / administration & dosage*
  • Antineoplastic Combined Chemotherapy Protocols / adverse effects
  • Cisplatin / administration & dosage*
  • Cisplatin / adverse effects
  • Cisplatin / pharmacokinetics
  • Drug Administration Schedule
  • Drug Resistance, Neoplasm / drug effects
  • Female
  • Humans
  • Isoxazoles / administration & dosage*
  • Isoxazoles / adverse effects
  • Isoxazoles / pharmacokinetics
  • Male
  • Middle Aged
  • Neoplasms / drug therapy*
  • Pyrazines / administration & dosage*
  • Pyrazines / adverse effects
  • Pyrazines / pharmacokinetics
  • Treatment Outcome

Substances

  • Isoxazoles
  • Pyrazines
  • berzosertib
  • Cisplatin

Associated data

  • ClinicalTrials.gov/NCT02157792