OBJECTIVES: To compare the efficacy and safety of algenpantucel-L 
[HyperAcute-Pancreas algenpantucel-L (HAPa); IND# 12311] immunotherapy combined 
with standard of care (SOC) chemotherapy and chemoradiation to SOC chemotherapy 
and chemoradiation therapy alone in patients with borderline resectable or 
locally advanced pancreatic ductal adenocarcinoma (PDAC).
SUMMARY BACKGROUND DATA: To date, immunotherapy has not been shown to benefit 
patients with borderline resectable or locally advanced unresectable PDAC. HAPa 
is a cancer vaccine consisting of allogeneic pancreatic cancer cells engineered 
to express the murine α(1,3)GT gene.
METHODS: A multicenter, phase 3, open label, randomized (1:1) trial of patients 
with borderline resectable or locally advanced unresectable PDAC. Patients 
received neoadjuvant SOC chemotherapy (FOLFIRINOX or gemcitabine/nab-paclitaxel) 
followed by chemoradiation (standard group) or the same standard neoadjuvant 
regimen combined with HAPa immunotherapy (experimental group). The primary 
outcome was overall survival.
RESULTS: Between May 2013 and December 2015, 303 patients were randomized from 
32 sites. Median (interquartile range) overall survival was 14.9 (12.2-17.8) 
months in the standard group (N = 158) and 14.3 (12.6-16.3) months in the 
experimental group (N = 145) [hazard ratio (HR) 1.02, 95% confidence intervals 
0.66-1.58; P = 0.98]. Median progression-free survival was 13.4 months in the 
standard group and 12.4 months in the experimental group (HR 1.33, 95% 
confidence intervals 0.72-1.78; P = 0.59). Grade 3 or higher adverse events 
occurred in 105 of 140 patients (75%) in the standard group and in 115 of 142 
patients (81%) in the experimental group (P > 0.05).
CONCLUSIONS: Algenpantucel-L immunotherapy did not improve survival in patients 
with borderline resectable or locally advanced unresectable PDAC receiving SOC 
neoadjuvant chemotherapy and chemoradiation.
TRIAL REGISTRATION: ClinicalTrials.gov Identifier: NCT01836432.