XBiotech announces data from Phase 1/2 1-BETTER study

XBiotech announced data from its Phase 1/Phase 2 randomized, double-blind, placebo-controlled multi-center study for advanced pancreatic cancer. Known as 1-BETTER, the study examined Natrunix antibody in combination with an established chemotherapy regimen, a regimen that is already widely used for treating pancreatic cancer but is associated with difficult toxicities and less then ideal survival outcomes. Natrunix was being evaluated as an anti-cancer agent for use in cytotoxic chemotherapy combinations where the company believes it might potentially also improve tolerability of the chemotherapy. The Phase 1 portion was a dose escalation study in metastatic pancreatic cancer patients to determine if dose limiting toxicities occurred in combination with the ON+5FU+LV regimen in second- or third-line setting. DLTs were not expected with Natrunix and none were seen. The Natrunix dose in the Phase 2 portion was thus the highest dose used in the Phase 1 portion. Sixty-five subjects were randomized into the Phase 2 study on a 1:1 basis to receive either Natrunix+ ON+5FU+LV or Placebo +ON+5FU+LV. There were 33 subjects enrolled into Arm1 and 32 into Arm2. The Phase 2 treatment period was 24-weeks with subjects receiving therapy once every other week for a total of 12 cycles. Subjects included in the study had confirmed metastatic, unresectable, or recurrent pancreatic adenocarcinoma of exocrine pancreas and were required to have had disease progression after one prior gemcitabine-based therapy or one FOLFIRINOX and gemcitabine containing therapy. All patients were required to have at least one measurable lesion according to Response Evaluation Criteria in Solid Tumor. The primary endpoint for the Phase 2 study was to assess the safety and tolerability of Natrunix when used with the ON+5FU+LV combination. Overall, there were fewer adverse events of any kind during the 24-week treatment period for the Natrunix arm compared to placebo, with markedly fewer events in specific categories of adverse events during that time. There was a 28% reduction in the number of subjects experiencing significant adverse events in the Natrunix arm versus placebo that occurred during the 24-week treatment period. Subjects receiving the Natrunix ON+5FU+LV regimen also had about a 33% reduction in hospitalization during the 24-week treatment period compared to subjects receiving placebo + ON+5FU+LV combination. Subjects receiving the Natrunix combination also reported a 22% reduction in fatigue, 32% improved appetite and 41% reduction in pain as of the last day of the 24-week treatment period compared to subjects receiving the placebo ON+5FU+LV combination.