Background: Immune Modulation and Gemcitabine Evaluation-1, a randomised, open-label, phase II, first-line, proof of concept
study (NCT01303172), explored safety and tolerability of IMM-101 (heat-killed Mycobacterium obuense; NCTC 13365) with
gemcitabine (GEM) in advanced pancreatic ductal adenocarcinoma.
Methods: Patients were randomised (2 : 1) to IMM-101 (10 mg ml l intradermally) þ GEM (1000 mg m 2 intravenously; n ¼ 75), or
GEM alone (n ¼ 35). Safety was assessed on frequency and incidence of adverse events (AEs). Overall survival (OS), progressionfree
survival (PFS) and overall response rate (ORR) were collected.
Results: IMM-101 was well tolerated with a similar rate of AE and serious adverse event reporting in both groups after allowance for
exposure. Median OS in the intent-to-treat population was 6.7 months for IMM-101þ GEM v 5.6 months for GEM; while not significant,
the hazard ratio (HR) numerically favoured IMM-101þ GEM (HR, 0.68 (95% CI, 0.44–1.04, P¼ 0.074). In a pre-defined metastatic subgroup
(84%), OS was significantly improved from 4.4 to 7.0 months in favour of IMM-101þ GEM (HR, 0.54, 95% CI 0.33–0.87, P¼ 0.01).
Conclusions: IMM-101 with GEM was as safe and well tolerated as GEM alone, and there was a suggestion of a beneficial effect on
survival in patients with metastatic disease. This warrants further evaluation in an adequately powered confirmatory study.