A critical unmet medical need exists for patients with locally advanced, unresectable, non-metastatic pancreatic cancer (“LAPC”) who no longer respond to first-line therapy with either Abraxane® plus gemcitabine or the 4-drug combination known as FOLFIRINOX – the current first-line standards of care for advanced pancreatic cancer. These patients have no effective treatment alternative once their tumors no longer respond to these therapies. Two of the most commonly used treatments are 5-fluorouiracil (“5-FU”) or capecitabine (a prodrug of 5-FU) plus radiation. Both treatments are only marginally effective in treating the tumor and result in serious side effects. Other treatments are being tried in an attempt to address this problem, but their success is far from certain. We are developing a therapy that we believe can serve as a “consolidation therapy” with the current standards of care and address the critical unmet medical need discussed above.
Subject to FDA approval, we plan to commence a clinical trial involving patients with LAPC whose tumors have ceased to respond to either Abraxane® plus gemcitabine or FOLFIRINOX. We had a Pre-Investigational New Drug Application (“Pre-IND”) meeting with the Center for Biologics Evaluation and Research of the FDA (“CBER”) in January 2017. At the Pre-IND meeting, the FDA communicated its agreement with certain aspects of our clinical development plan, charged us with completing numerous tasks and provided us with the guidance on the tasks we need to complete for a successful Investigational New Drug Application (“IND”). The proposed clinical trial is designed to show that our Cell-in-a-Box® plus low-dose ifosfamide therapy can serve as an effective and safe consolidation chemotherapy for patients whose tumors no longer respond after four to six months of therapy with Abraxane® plus gemcitabine or FOLFIRINOX. The trial will initially take place in the United States (“U.S.”) with possible study sites in Europe later in the trial.
In a previous Phase 1/2 open-label, single site 14-patient clinical trial using our therapy and in which only two courses of ifosfamide were given, our treatment was shown to be safe and effective.(See Figure 3). In fact, when the results of that trial were compared to historical data for gemcitabine (the “gold standard” for pancreatic cancer at the time that this trial was conducted) the percentage of one-year survivors was double that previously reported for gemcitabine, the median survival of patients was substantially increased as compared to gemcitabine, and in contrast to gemcitabine, no treatment-related side effects were seen with our therapy. Interestingly, in 3 of the 14 patients, their advanced-stage tumors were reduced in size to the point where they would have been operable.
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