A phase II study of ENMD-2076 in advanced soft tissue sarcoma (STS)

Sci Rep. 2019 May 14;9(1):7390. doi: 10.1038/s41598-019-43222-6.

Abstract

ENMD-2076, an aurora-A kinase inhibitor with anti-angiogenic properties, has shown activity in solid and hematologic malignancies. We investigated oral ENMD-2076 in an open-label, single-arm phase II study using 275 mg daily on a 28-day cycle in patients with advanced soft-tissue sarcomas (STS) receiving ≤1 line of prior therapy. Primary endpoint was 6-month progression-free survival (PFS) with ≤15% indicating no interest, and ≥40% indicating further interest in ENMD-2076. Secondary/exploratory endpoints included clinical benefit (CBR ≥6-months) and objective response (ORR) rates, PFS, OS, safety, and whole-exome sequencing (WES) for potentially associated biomarkers. Overall, 23/25 (92%) patients receiving ENMD-2076 were efficacy evaluable with median follow-up of 14 months (range 2.2-39.5). Common subtypes were leiomyosarcoma (n = 10), undifferentiated pleomorphic sarcoma (n = 3), angiosarcoma (n = 3), and alveolar soft-part sarcoma (n = 3). The 6-month PFS was 20.8% (95% CI:3.2-38.4) with a CBR of 17% (95% CI:1.55-33.23) and ORR of 9% (95% CI:3.08-20.46). Median PFS was 2.5 months (95% CI:2.20-4.47) and OS was 14.1 months (95% CI:6.07-20.07). The most common high-grade treatment-related adverse event was hypertension (60%). WES identified PTPRB mutations in 3/4 patients (p = 0.018) benefiting from ENMD-2076. Although this study failed to meet its primary endpoint, occasional responses and prolonged stable disease was noted. ENMD-2076 evaluation in PTPRB mutated tumors and/or angiosarcoma is warranted.

Publication types

  • Clinical Trial, Phase II
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Aged
  • Aurora Kinase A / antagonists & inhibitors
  • Biomarkers, Tumor / genetics
  • Drug Administration Schedule
  • Exome Sequencing
  • Female
  • Follow-Up Studies
  • Humans
  • Hypertension / chemically induced
  • Hypertension / epidemiology*
  • Male
  • Middle Aged
  • Mutation
  • Neoplasm Grading
  • Progression-Free Survival
  • Protein Kinase Inhibitors / administration & dosage*
  • Protein Kinase Inhibitors / adverse effects
  • Pyrazoles / administration & dosage*
  • Pyrazoles / adverse effects
  • Pyrimidines / administration & dosage*
  • Pyrimidines / adverse effects
  • Receptor-Like Protein Tyrosine Phosphatases, Class 3 / genetics
  • Sarcoma / drug therapy*
  • Sarcoma / genetics
  • Sarcoma / mortality
  • Sarcoma / pathology
  • Young Adult

Substances

  • Biomarkers, Tumor
  • ENMD 2076
  • Protein Kinase Inhibitors
  • Pyrazoles
  • Pyrimidines
  • AURKA protein, human
  • Aurora Kinase A
  • PTPRB protein, human
  • Receptor-Like Protein Tyrosine Phosphatases, Class 3