Despite recent advances in the treatment of pancreatic cancer there is still room for considerable therapeutic improvements (9, 10). Axcentua is evaluating the potential utility of a specific and patented crystalline form of the natural compound genistein in combination with standard gemcitabine (Gemzar®) therapy for patients with locally advanced or metastatic pancreatic cancer. Genistein has been shown in pre-clinical animal models of pancreatic cancer to inhibit metastasis (11, 12) and enhance the effect of gemcitabine (3) and other chemotherapies (14, 15) when given as a combination therapy. The purpose of the current phase Ib/IIa study (18) is to assess the safety and efficacy of Axcentua’s most advanced clinical development candidate called genistein sodium salt dihydrate (genistein-SSDH) or AXP107-11) alone and in combination with gemcitabine.
All sixteen patients in the phase Ib study have now been recruited in April 2014 and the study is still ongoing. An interim analysis of the data from the first 14 patients has not shown any dose-limiting toxicity (DLT). In fact, the maximal tolerated dose (MTD) for the compound has not been reached despite adequate pharmacokinetics, oral bioavailability and systemic exposure. The objective of the phase Ib part of the study is to evaluate the safety of genistein SSDH. However, different efficacy endpoints were also measured and intriguing results have been observed suggesting that genistein-SSDH is safe and may show early effects also on efficacy. The therapeutic efficacy will be assessed in the phase IIa part of the study.