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A Phase 2 Study of Galunisertib (TGF-β1 Receptor Type I Inhibitor) and Sorafenib in Patients With Advanced

R K Kelley1, E Gane2, E Assenat3

  • 1University of California, San Francisco, San Francisco, California, USA.

Clinical and Translational Gastroenterology
|July 12, 2019
PubMed
Abstract

Insights

The combination of galunisertib and sorafenib demonstrated promising results for advanced hepatocellular carcinoma (HCC) treatment. This therapy showed acceptable safety and improved overall survival (OS) in patients, suggesting a potential new treatment strategy for HCC.

Area of Science:

  • Oncology
  • Pharmacology

Background:

  • Hepatocellular carcinoma (HCC) is a significant global health concern.
  • Tumor growth factor-β (TGF-β) receptor type I inhibition shows potential in preclinical HCC models.
  • Galunisertib, a TGF-β1 receptor type I inhibitor, has demonstrated prior activity in HCC.

Purpose of the Study:

  • To evaluate the safety and efficacy of combining galunisertib with sorafenib in advanced HCC patients.
  • To assess outcomes including time-to-tumor progression, overall survival, and response rates.

Main Methods:

  • A Phase 1/2 clinical trial involving 47 patients with advanced HCC.
  • Patients received galunisertib (80 mg or 150 mg b.i.d.) in combination with sorafenib (400 mg b.i.d.).
  • Key endpoints included safety, pharmacokinetics, time-to-tumor progression, overall survival, and response rate.

Main Results:

  • The combination therapy exhibited an acceptable safety profile, consistent with monotherapy of each drug.
  • Median overall survival (OS) was 18.8 months in the 150 mg galunisertib cohort.
  • Patients responding to TGF-β1 inhibition showed significantly longer OS (22.8 months vs. 12.0 months).

Conclusions:

  • The combination of galunisertib and sorafenib is a safe and potentially effective treatment for advanced HCC.
  • This combination therapy offers a prolonged overall survival benefit for HCC patients.
  • TGF-β1 response may serve as a predictive biomarker for treatment efficacy.

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