Related Experiment Video
Updated: Jun 28, 2025
Establishing Dual Resistance to EGFR-TKI and MET-TKI in Lung Adenocarcinoma Cells In Vitro with a 2-step Dose-escalation Procedure
Published on: August 11, 2017
Phase II Study of Erdafitinib in Patients With Tumors With Fibroblast Growth Factor Receptor Mutations or Fusions:
Jun Gong1, Alain C Mita1, Zihan Wei2
1Cedars-Sinai Medical Center, Los Angeles, CA.
Purpose:
Subprotocol K2 (EAY131-K2) of the NCI-MATCH platform trial was an open-label, single-arm, phase II study designed to evaluate the antitumor efficacy of the oral FGFR1-4 inhibitor, erdafitinib, in patients with tumors harboring FGFR1-4 mutations or fusions.
Methods:
Central confirmation of tumor FGFR1-4 mutations or fusions was required for outcome analysis. Patients with urothelial carcinoma were excluded. Enrolled subjects received oral erdafitinib at a starting dose of 8 mg daily continuously until intolerable toxicity or disease progression. The primary end point was objective response rate (ORR) with key secondary end points of safety, progression-free survival (PFS), and overall survival (OS).
Results:
Thirty-five patients were enrolled, and 25 patients were included in the primary efficacy analysis as prespecified in the protocol. The median age was 61 years, and 52% of subjects had received ≥3 previous lines of therapy. The confirmed ORR was 16% (4 of 25 [90% CI, 5.7 to 33.0], P = .034 against the null rate of 5%). An additional seven patients experienced stable disease as best-confirmed response. Four patients had a prolonged PFS including two with recurrent WHO grade IV, IDH1-/2-wildtype glioblastoma. The median PFS and OS were 3.6 months and 11.0 months, respectively. Erdafitinib was manageable with no new safety signals.
Conclusion:
This study met its primary end point in patients with several pretreated solid tumor types harboring FGFR1-3 mutations or fusions. These findings support advancement of erdafitinib for patients with fibroblast growth factor receptor-altered tumors outside of currently approved indications in a potentially tumor-agnostic manner.
Insights
The FGFR inhibitor erdafitinib showed antitumor efficacy in patients with solid tumors harboring fibroblast growth factor receptor (FGFR) mutations or fusions. This study met its primary endpoint, supporting erdafitinib
Area of Science:
- Oncology
- Pharmacology
- Genetics
Background:
- Fibroblast growth factor receptor (FGFR) alterations are implicated in various solid tumors.
- Targeted therapies offer potential treatment avenues for patients with specific genetic mutations.
Purpose of the Study:
- To evaluate the antitumor efficacy of erdafitinib, an oral FGFR1-4 inhibitor.
- To assess erdafitinib in patients with solid tumors harboring FGFR1-4 mutations or fusions, excluding urothelial carcinoma.
Main Methods:
- An open-label, single-arm, phase II study (NCI-MATCH Subprotocol K2).
- Central confirmation of FGFR1-4 mutations/fusions was required.
- Patients received erdafitinib 8 mg daily until toxicity or progression.
Main Results:
- Objective response rate (ORR) was 16% (4/25), meeting the primary endpoint (P = .034).
- Median progression-free survival (PFS) was 3.6 months and overall survival (OS) was 11.0 months.
- Erdafitinib demonstrated manageable safety with no new signals.
Conclusions:
- Erdafitinib showed efficacy in pretreated solid tumors with FGFR1-3 alterations.
- Findings support erdafitinib's potential tumor-agnostic use in fibroblast growth factor receptor-altered cancers.
More Related Videos
09:38Establishment and Characterization of Three Afatinib-resistant Lung Adenocarcinoma PC-9 Cell Lines Developed with Increasing Doses of Afatinib
Published on: June 26, 2019
13:34A Combined 3D Tissue Engineered In Vitro/In Silico Lung Tumor Model for Predicting Drug Effectiveness in Specific Mutational Backgrounds
Published on: April 6, 2016
