US-based Rafael Pharmaceuticals has launched a Phase I clinical trial of its drug candidate CPI-613 to be assessed in combination with chemotherapeutics, gemcitabine, and nab-paclitaxel to treat patients with pancreatic cancer.

CPI-613 is being developed as an altered energy metabolism directed (AEMD) drug to target the mitochondrial metabolism of cancer cells for disrupting their altered energy-production pathways.

To be performed in partnership with Atlantic Health System, the Phase I trial will evaluate the combination therapy in subjects with locally advanced or metastatic pancreatic cancer.

The trial’s primary objective is to determine the maximum tolerated dose of CPI-613 when administered in conjunction with the chemotherapeutics.

Rafael Pharmaceuticals CEO Sanjeev Luther said: “Patients with a low-tolerance for toxicity may not be eligible for the Folfirinox combo.

“Patients with a low-tolerance for toxicity may not be eligible for the Folfirinox combo.”

“We believe that the gemcitabine/nab-paclitaxel regimen has the potential to be a potent treatment with less toxicity.”

The secondary objectives of the Phase I trial are safety and clinical activity of the combination, progression-free survival (PFS) and overall survival (OS).

Trial principal investigator Dr Angela Alistar said: “Due to the low toxicity profile of CPI-613 as a single agent, I anticipate good tolerance in combination with the gemcitabine/nab-paclitaxel regimen.”

Previous trial results are reported to have demonstrated the favourable safety and tolerability profile of CPI-613, which is under assessment in various Phase I, I/II and II trials.

CPI-613 obtained orphan drug designation from the US Food and Drug Administration (FDA) for the treatment of acute myeloid leukaemia (AML), pancreatic cancer and myelodysplastic syndromes (MDS).