RAGNAR (NCT04083976) is a Phase 2 clinical study designed to evaluate the efficacy and safety of BALVERSA in patients with advanced or metastatic solid tumors and prespecified FGFR gene alterations, regardless of tumor location or histology (tumor-agnostic). The IA was based on 178 patients with 32 distinct solid tumor histologies.1 Patients in the study were prospectively identified by local molecular testing or central next-generation sequencing (NGS); the most common tumor types were cholangiocarcinoma (bile duct cancer) (n=31), high-grade glioma (tumor of the brain or spinal cord) (n=29), breast cancer (n=14), pancreatic cancer (n=13) and squamous non-small cell lung cancer (n=11).1 The study also included tumors that occur less frequently in the real world such as salivary gland and parathyroid carcinomas (rare endocrine malignancies), as well as tumors of unknown primary origin.1 Study participants were heavily pretreated, with 74.7 percent (n=133) having received two or more prior lines of therapy.1
The primary endpoint of the RAGNAR study is the overall response rate (ORR) as assessed by an independent review committee (IRC). At the IA data cutoff, IRC assessed an ORR of 29.2 percent (95 percent confidence interval [CI], 22.7-36.5) and a disease control rate (DCR) of 72.5 percent (95 percent CI, 65.3-78.9) for the overall tumor-agnostic patient population.1 Investigators observed responses in 14 distinct tumor types. This included responses in hard-to-treat malignancies such as salivary gland cancer (100 percent ORR; treated n=5, responders n=5), pancreatic cancer (31 percent ORR; treated n=13, responders n=4) and glioblastoma (21 percent ORR; treated n=29, responders=6).1 Investigators also observed an overall 7.1-month median duration of response (DOR) (95 percent CI, 5.5-9.3). At the data cutoff, 51.1 percent (n=24) of patients who had responded to treatment continued to show a response.1 The primary analysis for all patients treated in this RAGNAR cohort, known as the broad panel cohort, is anticipated later this year.
The safety profile of BALVERSA observed in RAGNAR was consistent with the known safety profile of BALVERSA in metastatic urothelial carcinoma (mUC). Across tumor types, 44.9 percent of patients experienced adverse events of grade three or above.1 Adverse events were manageable with supportive care and treatment interruptions or reductions, when necessary.1 The discontinuation rate due to drug-related adverse events was 7.3 percent.1
'Diagnostic advances in the identification of FGFR gene alterations have opened the door to targeted, tumor-agnostic treatment approaches for patients,' said Yohann Loriot, M.D., Ph.D.,
In 2019, BALVERSA was granted accelerated approval by the
'Janssen is committed to advancing precision medicine approaches for the treatment of patients with biomarker-driven cancers, an area of clear unmet need,' said
About FGFR Alterations
Fibroblast growth factor receptors are a family of receptor tyrosine kinases that help cells grow, survive and multiply; FGFRs play a key role in several biological processes including tissue repair, inflammatory response and metabolism.[3],[4],[5] Fusions or mutations in the genes that control FGFR (known as FGFR1-4 alterations) may lead to the development and progression of certain cancers by increasing tumor cell growth and survival.5 Patients with advanced, FGFR-driven solid tumors who have exhausted standard treatment options typically face a poor prognosis.
About the RAGNAR Study
RAGNAR (NCT04083976) is a Phase 2 clinical trial evaluating the safety and efficacy of BALVERSA in patients with advanced solid tumors, regardless of cancer type or tumor location (tumor-agnostic), driven by FGFR1-4 alterations. Patients in the trial have progressed on or after at least one line of systemic therapy and have no alternative standard treatment options. Following screening by local molecular testing or central NGS, patients are enrolled in four separate cohorts: a broad panel cohort of patients with pathogenic FGFR mutations or gene fusions (tumor histologies evaluated include but are not limited to cholangiocarcinoma [bile duct cancer], high- and low-grade glioma [a tumor type occurring in the brain or spinal cord], breast, pancreatic, squamous and non-squamous non-small cell lung cancer, colorectal, endometrial, esophageal, salivary gland, ovarian, duodenal [cancer occurring in the first part of the small intestine], thyroid and cancer of unknown primary origin); an exploratory cohort of patients with other FGFR mutations; a cholangiocarcinoma expansion cohort; and a pediatric cohort of patients ages 6 to 17 with FGFR alterations.1
The primary endpoint of RAGNAR is IRC-assessed ORR. Key secondary endpoints include investigator-assessed ORR, DOR, DCR, clinical benefit rate, progression free survival, overall survival and incidence and severity of adverse events.
About BALVERSA
BALVERSA (erdafitinib) is a once-daily, oral FGFR kinase inhibitor that is approved by the
In addition to RAGNAR, BALVERSA is being studied in clinical trials including the Phase 3 THOR (NCT03390504) study evaluating BALVERSA versus standard of care, consisting of chemotherapy (docetaxel or vinflunine) or anti-PD-1 agent pembrolizumab, in participants with advanced urothelial cancer and selected FGFR aberrations with disease progression following one or two prior lines of therapy; and the randomized Phase 2 THOR-2 (NCT04172675) study examining BALVERSA versus investigator choice of intravesical chemotherapy in participants who received Bacillus Calmette-Guerin and recurred with high risk non-muscle-invasive bladder cancer.[7],[8]
In 2008,
About the Janssen Pharmaceutical Companies of
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Cautions Concerning Forward-Looking Statements
This press release contains 'forward-looking statements' as defined in the Private Securities Litigation Reform Act of 1995 regarding product development and the potential benefits and treatment impact of BALVERSA (erdafitinib). The reader is cautioned not to rely on these forward-looking statements. These statements are based on current expectations of future events. If underlying assumptions prove inaccurate or known or unknown risks or uncertainties materialize, actual results could vary materially from the expectations and projections of
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