RAGNAR-trial

Prof Prenen highlights one significant trial, the Ragnar trial, which resulted at ASCO 2024 in three posters for the trial site at the University Hospital Antwerp. The Ragnar trial is a Phase 2 basket trial evaluating the efficacy of erdafitinib, an oral FGFR inhibitor, across various tumour types with FGFR alterations, including fusions and mutations.

The first poster focused on non-small cell lung cancer (NSCLC), and was presented as an oral presentation. This part of the trial included both squamous and non-squamous NSCLC patients with FGFR1-4 alterations, excluding those with other targetable mutations like EGFR, ALK, or ROS. The primary endpoint was the response rate among 23 patients (14 squamous, 9 non-squamous), with 13 having fusions and 10 having mutations. Responses were observed in both mutation and fusion groups, with 6 out of 23 patients responding to treatment. However, the median duration of response was relatively short at 4.6 months. Common side effects included mucositis, diarrhoea, and eye toxicity.

The second poster detailed the trial results in breast cancer, involving 16 chemorefractory patients (10 with fusions and 6 with mutations). Erdafitinib showed efficacy in both groups, with a 31% response rate and a 69% disease control rate. The median duration of response in breast cancer was 6.9 months, indicating a notable impact in this patient group.

The third poster focused on cholangiocarcinoma and included a pooled analysis from the Ragnar trial and another study in Asia. A total of 78 patients were included, with 91% having fusions, reflecting the higher prevalence of FGFR fusions in cholangiocarcinoma compared to mutations. The objective response rate was 55%, with a progression-free survival (PFS) of 8.5 months. These results underscore the efficacy of erdafitinib in cholangiocarcinoma with FGFR alterations.

Overall, the Ragnar trial’s findings highlight erdafitinib’s potential across multiple tumour types with FGFR alterations, particularly in cholangiocarcinoma, where it demonstrated significant activity.