On June 3, 2018 The Janssen Pharmaceutical Companies of Johnson & Johnson reported findings today from a Phase 2 study that showed treatment with erdafitinib resulted in durable responses in patients with metastatic or surgically unresectable urothelial cancer (mUC) and fibroblast growth factor receptor alterations (FGFRalt) (Press release, Johnson & Johnson, JUN 3, 2018, View Source [SID1234527110]). This is a patient population with high unmet need based on poor outcomes when treated with available therapies. Erdafitinib is a once-daily pan-FGFR inhibitor.1 FGFRs are cell proteins that, if altered, can contribute to the development of cancer.1 Alterations occur in approximately 20 percent of mUC patients.1 The results were presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2018 Annual Meeting in Chicago (Abstract #4503) and have been selected for the Best of ASCO (Free ASCO Whitepaper) Meetings.1
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"These study results are very promising, particularly as this is an area of high unmet need with patients who otherwise have very limited treatment options remaining. We hope that the response rates shown by erdafitinib could eventually give patients with metastatic or surgically unresectable urothelial cancer a new treatment option"
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"I am very encouraged by these Phase 2 data showing that erdafitinib had promising response rates and progression-free survival in a patient population with such high unmet need," said Dr. Yohann Loriot, Senior Consultant, Department of Cancer Medicine & INSERM, Institut Gustave Roussy, University of Paris Sud, Villejuif, France. "Currently there are no targeted therapies approved for specific subsets of patients with urothelial cancer who have genetic alterations. While immune checkpoint inhibitors have led to improvements in outcomes for these patients, we are still finding that many patients do not respond to treatment."
BLC2001 (NCT02365597) is a multicentre, open-label Phase 2 study evaluating the efficacy and safety of erdafitinib in the treatment of adult patients with locally advanced or metastatic urothelial cancer, whose tumours have certain FGFR alterations.1 Ninety-nine patients were treated with an optimised dosing schedule using pharmacodynamically guided dose up-titration: a starting dose of erdafitinib at 8 mg daily, with the possibility to increase the dose to 9 mg daily based on serum phosphate levels.2 Twelve percent of patients were chemo-naïve, 89 percent of patients had received one or more lines of therapy, 43 percent of patients had received two or more prior lines of therapy, and 78 percent of patients had visceral metastases.2 There was a 40 percent confirmed overall response rate1 (RECIST 1.1;* 3% Complete Response, 37% Partial Response), a median progression-free survival of 5.5 months and median overall survival of 13.8 months.2 In patients who experienced grade 3 adverse events (AEs), the most common were, stomatitis (9%) and diarrhoea (4%).1 Seven patients discontinued due to treatment-related AEs.2
"These study results are very promising, particularly as this is an area of high unmet need with patients who otherwise have very limited treatment options remaining. We hope that the response rates shown by erdafitinib could eventually give patients with metastatic or surgically unresectable urothelial cancer a new treatment option," said Dr Ivo Winiger-Candolfi, Europe, Middle East and Africa (EMEA) Oncology Therapeutic Area Lead, Janssen. "The successful development of new oncology therapies, such as erdafitinib, is an example of our precision medicine approach: providing the right patient, with the right treatment, at the right time. We recognise that every patient is unique and that by accounting for individual differences in people’s genes, environments and lifestyles, we can optimise the therapeutic benefit for particular groups of patients. We look forward to understanding the potential efficacy and broader safety profile of erdafitinib in both Phase 3 development as well as in combination with anti-PD1 therapy."
*RECIST (version 1.1) refers to Response Evaluation Criteria in Solid Tumors which is a standard way to measure how well a cancer patient responds to treatment and is based on whether tumours shrink, stay the same, or get bigger.3
About Urothelial Cancer
Europe has among the highest incidence rates of bladder cancer in the world and mortality rates for men are by far the highest recorded worldwide.4 It is the fifth most frequently diagnosed cancer in the EU, with about 124,000 new cases each year for both sexes.5 The majority (90%) of bladder cancer consists of urothelial carcinoma in Western Europe.6 Urothelial bladder cancer starts in the bladder lining (urothelial cells or transitional cells) and can be non-invasive or invasive.7 For patients with metastatic disease, outcomes can be poor due to the often rapid progression of the tumour and the lack of efficacious treatments.8 The relative five-year survival rate for patients with metastatic disease is five percent.9
About erdafitinib
Erdafitinib is a once-daily oral pan-fibroblast growth factor receptor (FGFR) tyrosine kinase inhibitor being evaluated by Janssen Research & Development in Phase 2 and 3 clinical trials in patients with advanced urothelial cancer.10 FGFRs are a family of receptor tyrosine kinases which may be upregulated in various tumour cell types and may be involved in tumour cell proliferation, tumour angiogenesis and tumour cell survival.11 In 2008, Janssen entered into an exclusive worldwide license and collaboration agreement with Astex Therapeutics Ltd. to develop and commercialise erdafitinib.
Erdafitinib received Breakthrough Therapy Designation from the U.S. Food and Drug Administration in March 2018.12 The aim is to move towards regulatory submission with the Phase 2 data and continue to pursue erdafitinib in Phase 3 clinical development, as well as in combination with anti-PD-1 therapy.