ASCO GU 2018: Erdafitinib, (FGFR) a Pan-fibroblast Growth Factor Receptor Inhibitor, in Patients with Metastatic or Unresectable Urothelial Carcinoma and FGFR Alterations

San Francisco, CA (UroToday.com) Although immune checkpoint inhibitors (ICI) have improved outcomes in some patients with platinum-resistant metastatic or unresectable urothelial carcinoma (mUC), many patients (eg, patients with TCGA luminal 1 tumors, many of whom are FGFR alterations (FGFRa)) may not benefit. Erdafitinib (ERDA; JNJ-42756493), a pan-fibroblast growth factor receptor (pan-FGFR) (1-4) inhibitor, demonstrated promising phase 1 activity: 11 partial responses among 24 FGFRa mUC pts. We report efficacy and safety of ERDA in the ongoing global open-label phase 2 study BLC2001 (NCT02365597).

Methods: Pts had measurable mUC with specific FGFR2/FGFR3 mutations or translocations per central lab Janssen assay, ECOG 0-2, and were chemorefractory (progressed during/following ≥ 1 line of prior systemic chemo or ≤ 12 mos of [neo]adjuvant chemo). Cisplatin-ineligible, chemo-naïve pts, and prior ICI treatment were allowed. Pts were randomized 1:1 to 28-d cycles of oral 6 mg/d continuous dosing (6 C) or 10 mg/d intermittent 7 d on/7 d off dosing (10 I) ERDA; the dose was further uptitrated if no significant treatment-related adverse events (TRAEs) were observed. The primary end point was ORR.

Results: 78 pts received 6 C and 33 pts received 10 I (10 I cohort stopped early) ERDA. 31 pts in 6 C arm were further uptitrated. Across arms, 50% had ≥ 2 prior lines of therapy; 93% were chemorefractory. Confirmed ORRs (RECIST 1.1) were 35% and 24%, and disease control rates (CR+PR+SD) were 74% and 73% in the 6 C and 10 I arms, respectively. Adverse events (AEs) were manageable, and there were no treatment-related deaths (Table). Treatment is ongoing in 10 pts.

Conclusions: Erdafitinib (6 C or 10 I) has promising efficacy and tolerability in patients with FGFRa mUC. Based on these results and ERDA pharmacometric modeling, dosing was optimized at 8 mg/d (continuous), and this cohort is ongoing. Phase 3 study is planned. 


Presented by: Yohann Loriot, MD, PhD

Co Authors: Andrea Necchi, Se Hoon Park, Jesús García-Donas, Robert A Huddart, Earle Frederick Burgess, Mark T. Fleming, Arash Rezazadeh, Begona Mellado, Sergei Varlamov, Monika Joshi, Ignacio Duran, Anne OHagan, Anjali Narayan Avadhani, Bob Zhong, Kim Stuyckens, Anne-Gaëlle Dosne, Arlene O. Siefker-Radtke, On Behalf of the BLC2001 Study Group; Institut Gustave Roussy, Villejuif, France; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Korea, Republic of (South); Clara Campal Comprehensive Cancer Center, Madrid, Spain; Institute of Cancer Research, Sutton, United Kingdom; Levine Cancer Institute, Carolinas HealthCare System, Charlotte, NC; Virginia Oncology Associates, US Oncology Research, Norfolk, VA; Norton Healthcare, Louisville, KY; Hospital Clinic de Barcelona, Barcelona, Spain; Altai Regional Cancer Center, Barnaul, Russia; Penn State Health Milton S. Hershey Medical Center, Hershey, PA; Hospital Universitario Virgen del Rocio, Seville, Spain; Janssen Research & Development, LLC, Spring House, PA; Janssen Research & Development, LLC, Beerse, Belgium; UT MD Anderson Cancer Center, Houston, TX

Presented at the 2018 American Society of Clinical Oncology Genitourinary (ASCO GU) Cancers Symposium, February 8-10, 2018 - San Francisco, CA