A phase 1/2a dose-finding study and biomarker assessment of oral lisavanbulin in patients with high-grade glioma or glioblastoma

Cell Rep Med. 2025 Jun 17;6(6):102165. doi: 10.1016/j.xcrm.2025.102165.

Abstract

Lisavanbulin is a prodrug of the microtubule-targeting agent avanbulin. Both avanbulin and lisavanbulin have demonstrated significant antitumor activity in several preclinical tumor models including glioblastoma. Previous human studies demonstrated that 48-h infusions of intravenous lisavanbulin were well tolerated with preliminary activity in recurrent glioblastoma. The current phase 1/2a study evaluates the safety and tolerability of once-daily oral lisavanbulin in patients with solid tumors or recurrent glioblastoma or high-grade glioma. Lisavanbulin is associated with profound, durable responses in a subset of patients with recurrent refractory grade 4 astrocytoma or glioblastoma. We present here the clinical and translational results from this trial, including a description of a response-predictive molecular signature that warrants further exploration in these tumor types of significant unmet need. The study is registered at ClinicalTrials.gov (NCT02490800).

Keywords: astrocytoma; glioblastoma; glioma; lisavanbulin.

Publication types

  • Clinical Trial, Phase I
  • Clinical Trial, Phase II

MeSH terms

  • Administration, Oral
  • Adult
  • Aged
  • Antineoplastic Agents* / administration & dosage
  • Antineoplastic Agents* / therapeutic use
  • Biomarkers, Tumor* / metabolism
  • Brain Neoplasms* / drug therapy
  • Brain Neoplasms* / pathology
  • Dose-Response Relationship, Drug
  • Female
  • Glioblastoma* / drug therapy
  • Glioblastoma* / metabolism
  • Glioblastoma* / pathology
  • Glioma* / drug therapy
  • Glioma* / metabolism
  • Glioma* / pathology
  • Humans
  • Male
  • Middle Aged
  • Neoplasm Grading

Substances

  • Biomarkers, Tumor
  • Antineoplastic Agents

Associated data

  • ClinicalTrials.gov/NCT02490800