Fibroblast growth factor receptor inhibition for succinate dehydrogenase-deficient gastrointestinal stromal tumors: a phase 2 trial - Report - MDSpire

Fibroblast growth factor receptor inhibition for succinate dehydrogenase-deficient gastrointestinal stromal tumors: a phase 2 trial

  • By

  • Priscilla Merriam

  • James J. Morrow

  • Emanuele Mazzola

  • Nicole L. Solimini

  • Prafulla C. Gokhale

  • Ping Chi

  • Alice P. Chen

  • Mark Agulnik

  • Melissa Burgess

  • Scott M. Schuetze

  • Neeta Somaiah

  • Brian A. Van Tine

  • Seth M. Pollack

  • Gabriel Tinoco

  • Jonathan Trent

  • Breelyn A. Wilky

  • Nicola Bothwick

  • Benjamin K. Eschle

  • Vivian Nguyen

  • Jan H. Beumer

  • Noushin Rastkari

  • Shahanawaz Jiwani

  • Peter I-Fan Wu

  • Lorraine Pelosof

  • Matthew L. Hemming

  • Geoffrey I. Shapiro

  • George Demetri

  • Bradley Bernstein

  • Suzanne George

  • May 26, 2026

  • 0 min

Share

Clinical Report: Inhibition of Fibroblast Growth Factor Receptor in GISTs

Overview

This phase 2 study evaluated the efficacy of rogaratinib, a pan-FGFR inhibitor, in patients with advanced SDH-deficient gastrointestinal stromal tumors (GISTs). A partial response rate of 41.7% and a median progression-free survival of 31.0 months were reported.

Background

Gastrointestinal stromal tumors (GISTs) are the most common sarcomas of the gastrointestinal tract, with a subset lacking common oncogenic mutations. These SDH-deficient GISTs exhibit unique epigenetic alterations.

Data Highlights

ResponseNumber of Patients
Partial Response10 (41.7%)
Stable Disease12
Progressive Disease2

Key Findings

  • 41.7% of patients achieved a partial response to rogaratinib.
  • Median progression-free survival (PFS) was 31.0 months.
  • 77.4% of patients had PFS at 1 year.
  • 50% of patients had documented SDHA subunit mutations.
  • 67% of patients were previously treated with systemic therapy.

Clinical Implications

The findings suggest that rogaratinib may provide a new therapeutic option for patients with advanced SDH-deficient GISTs, particularly those who have limited responses to standard therapies. Further studies are warranted to confirm these results and explore the role of FGFR inhibition in this patient population.

Conclusion

The study demonstrates the potential of FGFR inhibition in treating advanced SDH-deficient GISTs, highlighting a promising avenue for targeted therapy in this challenging subset of tumors.

Related Resources & Content

  1. NCI, Gastrointestinal Stromal Tumors Treatment (PDQ®), 2024 -- Current guidance and disease context
  2. Nature, Altered chromosomal topology drives oncogenic programs in SDH-deficient GISTs, 2019 -- Rationale for FGFR inhibition
  3. Dana-Farber Cancer Institute, Phase 2 Study of Rogaratinib in the Treatment of Patients with Sarcoma, 2024 -- Trial details
  4. The ASCO Post — Regorafenib Has ‘Notable Anticancer Activity’ after Patients Develop Resistance to Imatinib and Sunitinib
  5. The ASCO Post — IGF2 May Be Target in Colorectal Cancer With Stable Disease As Response  to Anti-EGFR Therapy
  6. Evaluation of FGFR2 and HER2 Expression in Colorectal and Gastric Cancer Patients: Findings from the DS-Screen Study
  7. The ASCO Post — Phase II Trial Reports Anticancer Activity With Novel FGFR Inhibitor in Advanced Intrahepatic Cholangiocarcinoma
  8. Regorafenib Has ‘Notable Anticancer Activity’ after Patients Develop Resistance to Imatinib and Sunitinib
  9. Targeted therapy in SDH-deficient GIST
  10. Gastrointestinal Stromal Tumors Treatment (PDQ®) - NCI
  11. Altered chromosomal topology drives oncogenic programs in SDH-deficient GISTs | Nature
  12. Phase 2 Study of Rogaratinib (BAY 1163877) in the Treatment of Patients with Sarcoma Harboring Alterations in Fibroblast Growth Factor Receptor (FGFR) 1-4 and SDH-deficient Gastrointestinal Stromal Tumor (GIST) | Dana-Farber Cancer Institute
  13. Pemigatinib for the Treatment of Locally Advanced or Metastatic SDH-Deficient Gastrointestinal Stromal Tumors, PEMIGIST Trial - NCI

Original Source(s)

Fibroblast growth factor receptor inhibition for succinate dehydrogenase-deficient gastrointestinal stromal tumors: a phase 2 trial

  • by Priscilla Merriam, James J. Morrow, Emanuele Mazzola, Nicole L. Solimini, Prafulla C. Gokhale, Ping Chi, Alice P. Chen, Mark Agulnik, Melissa Burgess, Scott M. Schuetze, Neeta Somaiah, Brian A. Van Tine, Seth M. Pollack, Gabriel Tinoco, Jonathan Trent, Breelyn A. Wilky, Nicola Bothwick, Benjamin K. Eschle, Vivian Nguyen, Jan H. Beumer, Noushin Rastkari, Shahanawaz Jiwani, Peter I-Fan Wu, Lorraine Pelosof, Matthew L. Hemming, Geoffrey I. Shapiro, George Demetri, Bradley Bernstein, Suzanne George

  • May 26, 2026

Related Content

Management of toxicities from antibody – drug conjugates in breast cancer

Author Correction: The efficacy of chemotherapy is limited by intratumoral senescent cells expressing PD-L2

Biotech exec Jeremy Levin on the industry’s strategic turning point