Phase III FAP-310 trial of CPP-1X/sul in familial adenomatous polyposis published in NEJM.- Cancer Prevention Pharma

Cancer Prevention Pharmaceuticals announced The New England Journal of Medicine (NEJM) has published results from its landmark FAP-310 Phase III clinical trial of CPP-1X/sul (eflornithine + sulindac) as a pharmaco-preventive treatment for adults with familial adenomatous polyposis (FAP). The FAP-310 Phase III trial compared the performance of the combination of CPP-1X (eflornithine) and sulindac (CPP-1X/sul), to each single agent alone in delaying progression to an FAP-related clinical event, such as surgery. The trial enrolled 171 patients and was conducted over many years where patients received daily treatment for 2-4 years. It was the largest prospective controlled study ever performed in FAP, and the only clinical event driven trial ever conducted. Although the trial did not demonstrate that overall disease progression was significantly lower with CPP-1X/sul compared to eflornithine or sulindac alone, the data showed that in a subgroup of patients with intact colons - representing the vast majority of young FAP patients - there was a 70% decreased risk of disease progression with CPP-1X/sul. Based on the results of the trial and observed benefits in a broad group of FAP patients, CPP intends to pursue regulatory approval from the FDA and a Marketing Authorization Application (MAA) with the European Medicines Agency for approval of CPP-1X/sul to delay or prevent surgeries in patients with an intact colon, retained rectum, or surgical pouch. See: "Eflornithine plus Sulindac for Prevention of Progression in Familial Adenomatous Polyposis." Carol A. Burke et al. N Engl J Med September 10, 2020 383:1028-1039 DOI: 10.1056/NEJMoa1916063