Rafael Pharmaceuticals has partnered with Michigan Medicine to start a Phase Ib/II clinical trial of devimistat (CPI-613) combination for locally advanced, unresectable or metastatic biliary tract cancer.

Devimistat targets the mitochondrial tricarboxylic acid (TCA) cycle, which is required for tumour cell multiplication and survival. The drug is said to boost the sensitivity of cancer cells to different chemotherapies.

The randomised, multi-centre Phase Ib/II study will assess the drug in combination with gemcitabine and cisplatin as first-line therapy for patients who did not receive any previous treatment.

A maximum tolerated dose (MTD) of the devimistat combination without any side effects will be determined during the Phase Ib part of the study. The identified dose will establish the recommended dose for the Phase II part.

In the Phase II part, the combination’s efficacy will be compared to gemcitabine plus cisplatin alone. A total of 68-78 patients will be enrolled in the trial.

Rafael Pharmaceuticals president and CEO Sanjeev Luther said: “Biliary tract cancer is a rare and aggressive cancer that affects approximately 15,000 people in the US each year. Launching this trial aligns with our mission to help patients with significant unmet medical needs.”

The company obtained approval from the US Food and Drug Administration (FDA) to conduct Phase III studies of devimistat in pancreatic cancer and acute myeloid leukaemia patients.

Also, the drug holds the FDA orphan drug for designation in pancreatic cancer, acute myeloid leukaemia, peripheral T-cell lymphoma, Burkitt’s lymphoma and myelodysplastic syndrome.

The European Medicines Agency (EMA) also awarded orphan drug status in pancreatic cancer and acute myeloid leukaemia indications.

Earlier this month, the company launched a Phase II trial of CPI-613 in combination with modified folfirinox (mFFX) in patients with localised borderline and unresectable pancreatic cancer.