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Poster display session

2627 - Anti-tumor activity of the pan-FGFR inhibitor rogaratinib in patients with advanced urothelial carcinomas selected based on tumor FGFR mRNA expression levels


10 Sep 2017


Poster display session


Clinical Research;  Urothelial Cancers


Martin Schuler


Annals of Oncology (2017) 28 (suppl_5): v295-v329. 10.1093/annonc/mdx371


M. Schuler1, L. Nogova2, A. Heidenreich3, D. Tai4, P. Cassier5, H. Richly1, B.C. Cho6, C.M. Sayehli7, A. Navarro8, S. Bender9, M. Ocker10, H. Nogai11, A. Wagner12, S. Ince13, P. Ellinghaus9, M. Joerger14

Author affiliations

  • 1 Department Of Medical Oncology, West German Cancer Center, University Hospital Essen, 45122 - Essen/DE
  • 2 Klinik I Für Innere Medizin, University Hospital Cologne, 50924 - Köln/DE
  • 3 Klinik Für Urologie, University Hospital Cologne, 50937 - Köln/DE
  • 4 Division Of Medical Oncology, National Cancer Centre Singapore, 169610 - Singapore/SG
  • 5 Département D'oncologie Médicale Adulte, Centre Léon Bérard, 69008 - Lyon/FR
  • 6 Department Of Internal Medicine, Yonsei Cancer Center, Seoul/KR
  • 7 Early Clinical Trial Unit, University Clinic Würzburg-Medizinische Klinik und Poliklinik II Zentrum fuer Innere Medizin (ZIM), 97080 - Würzburg/DE
  • 8 Deparment Of Oncology, University Hospital Vall d’Hebron, 08035 - Barcelona/ES
  • 9 Biomarker Research, Bayer AG, 42113 - Wuppertal/DE
  • 10 Biomarker Strategy, Bayer AG, 13353 - Berlin/DE
  • 11 Clinical Pharmacology, Bayer AG, 13353 - Berlin/DE
  • 12 Clinical Development, Bayer AG, 13353 - Berlin/DE
  • 13 Project Management, Bayer HealthCare Pharmaceuticals, Whippany/US
  • 14 Medical Oncology & Hematology, Cantonal Hospital, 9007 - St. Gallen/CH


Abstract 2627


Fibroblast growth factor receptor (FGFR) signaling is deregulated in urothelial carcinomas (UC). Rogaratinib is an oral inhibitor of FGFRs 1-4 with demonstrated antitumor activity in bladder cancer xenograft models. We report the results from a rogaratinib phase I trial expansion cohort in UC patients selected based on FGFR1-3 mRNA tumor overexpression and/or presence of activating mutations in the FGFR3 gene.


Patients with locally advanced or metastatic UC who have progressed or ineligible for standard therapy were screened for high FGFR1-3 mRNA expression levels by RNA in situ hybridization (RNAscope®) and Nanostring® assays utilizing fresh or archival FFPE tumor specimens. FGFR3-activating mutations were evaluated by a PCR based assay (Qiagen). Patients were treated with rogaratinib 800mg BID on a continuous regimen. Tumor response was assessed by RECIST, v1.1. Adverse events were classified using CTCAE v4.03 criteria.


Biopsies from a total of 109 patients with advanced UC were screened, with 42.3% found to be FGFR positive; of which 87% due to FGFR3 mRNA overexpression, 4% FGFR1, and 9% mixed FGFR isoform mRNA expression. Co-ocurrence of FGFR3-activating mutations and high FGFR3 mRNA expression was seen in 8% of patients. Among 20 patients with UC treated with rogaratinib, 16 (75%) had tumor shrinkage in target lesions with 9 (45%) showing tumor shrinkage of more than 20%, and 6 (30%) having a partial response (PR). Disease control rate (CR+PR+SD>12w) was 75%. Three patients with a PR had elevated tumor FGFR3 mRNA levels without corresponding genomic alterations. The most frequent AEs were hyperphosphatemia and diarrhea.


Selection of UC patients for treatment with rogaratinib based on FGFR1-3 mRNA expression levels in archival tissue was feasible and identified patients benefitting from treatment without having aberrations of FGFR-encoding genes. Rogaratinib has a favorable safety profile and showed anti-tumor activity in biomarker-selected UC patients which warrants further clinical development.

Clinical trial identification


Legal entity responsible for the study

Bayer AG


Bayer AG


M. Schuler: Consultant: AstraZeneca, Boehringer Ingelheim, Bristol-Myers Squibb, Celgene, Lilly, Novartis, Roche; Honoraria: Alexion, Boehringer Ingelheim, Bristol-Myers Squibb, Celgene, Lilly, Novartis Research: Boehringer Ingelheim, Bristol Myers-Squibb, Novar. S. Bender, M. Ocker, H. Nogai, A. Wagner, P. Ellinghaus: Employment: Bayer AG. S. Ince: Employment: Bayer HealthCare Pharmaceuticals. All other authors have declared no conflicts of interest.

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