Cornerstone Pharmaceuticals has started a Phase I clinical trial of its lead Altered Energy Metabolism Directed (AEMD) drug candidate, CPI-613, in combination with fluorouracil (5-FU), to treat with non-resectable metastatic colorectal cancer who have failed prior therapy.
CPI-613 is designed to disrupt the altered energy-production pathways in cancer cells by targeting their altered mitochondrial metabolism.
The company said that positive results and patient benefit observed in earlier trials including patients diagnosed with metastatic colorectal cancer have led to further evaluation of CPI-613 in this indication.
In earlier trials, the drug was well tolerated providing further support for evaluation of CPI-613 in combination with other drugs to maximise benefit.
Sponsored by Wake Forest Baptist Medical Center in collaboration with the National Cancer Institute (NCI), the open label, dose-escalating Phase I trial is designed to determine the maximum tolerated dose of CPI-613 in combination with 5-FU.
The trial will also evaluate secondary endpoints the pharmacokinetics, safety and efficacy of several doses of CPI-613 when used in combination with 5-FU.
Additionally, the Phase I trial will also evaluate overall response rates, progression-free survival (PFS) and disease control rates.
According to the company, 5-FU has been used as a standard chemotherapeutic agent to treat colon, rectum, and head and neck cancers with commonly accepted response rates of less than 20% and acquired drug resistance.
Cornerstone chief executive officer Steve Carchedi said: "We believe by working with the investigators at Wake Forest Baptist Medical Center in this trial, which will be supported by the National Cancer Institute, we will be able to further demonstrate the capabilities of our lead product candidate, CPI-613, and mark another important step forward for Cornerstone.
"The study is the sixth evaluating CPI-613 in solid tumours and highlights the broad potential utility of our unique cancer metabolism approach in both solid tumours and haematologic malignancies.
"We look forward to the results of this study as we work to enhance data for CPI-613 and are excited about the efficacy demonstrated by our AEMD approach in both haematological and solid tumours thus far."
CPI-613 is the lead drug candidate from the company’s AEMD platform, which disrupts the metabolism (energy producing) pathways that support the growth and development of many types of cancer cells.
Currently, CPI-613 is being evaluated in several Phase I, I/II and II human clinical trials in solid tumours and haematological malignancies.