AIM ImmunoTech Inc. has reported positive mid-year safety and efficacy data from its ongoing Phase 2 clinical study evaluating the combination of Ampligen (rintatolimod) with AstraZeneca's anti-PD-L1 immune checkpoint inhibitor Imfinzi (durvalumab) for treating metastatic pancreatic cancer patients with stable disease following FOLFIRINOX chemotherapy.
The DURIPANC study, conducted in collaboration with AstraZeneca and Erasmus Medical Center in the Netherlands, represents a significant development in pancreatic cancer treatment, a disease known for its limited responsiveness to immunotherapy approaches.
Study Design and Enrollment
The investigator-initiated, exploratory, open-label, single-center study is designed to enroll up to 25 subjects in the Phase 2 portion. As of the mid-year report, 14 subjects have been enrolled in the trial. The primary objective focuses on the clinical benefit rate of the combination therapy, while secondary and exploratory objectives include assessing overall survival, progression-free survival, and immune-monitoring through tissue biopsies and peripheral immune profiling.
Encouraging Safety and Efficacy Signals
The mid-year data revealed several promising outcomes that distinguish this combination from historical immunotherapy approaches in pancreatic cancer. The study demonstrated no significant toxicity, presenting an encouraging safety profile for patients in a post-chemotherapy setting.
Efficacy results showed that approximately 21% of patients (3 out of 14) achieved progression-free survival greater than 6 months, with an additional 21% not yet having progressed. Perhaps most notably, overall survival exceeded 6 months in the majority (64%) of eligible patients, representing outcomes better than expected in this challenging treatment setting.
Prof. Casper van Eijck, MD, PhD, of Erasmus MC, commented on the preliminary findings: "Our preliminary data suggests that the combination of Ampligen and durvalumab is well-tolerated in post-FOLFIRINOX pancreatic cancer patients, with encouraging preliminary survival data, especially given the historical difficulty of improving outcomes in this setting."
Clinical Context and Unmet Need
Pancreatic cancer presents significant therapeutic challenges, with limited immunotherapy responsiveness particularly in unselected patient populations. Following FOLFIRINOX treatment, maintenance or second-line immunotherapies have historically demonstrated limited survival benefit in comparison trials. The DURIPANC study results suggest potential superiority over these historical benchmarks.
Thomas K. Equels, CEO of AIM ImmunoTech, emphasized the clinical significance: "DURIPANC builds on that foundation and these results suggest a clear path forward and identify a promising potential benefit of combining the selective innate immune activation of Ampligen with the checkpoint inhibition of durvalumab in pancreatic cancer maintenance therapy."
The disease represents a substantial global health burden, killing more than 100,000 people annually in American and European Union markets combined, and over 500,000 worldwide.
Foundation in Monotherapy Experience
The combination study builds upon extensive experience with Ampligen as monotherapy in pancreatic cancer. Since 2017, more than 50 pancreatic cancer patients have received Ampligen treatment as immuno-oncology monotherapy under a Dutch government-approved Compassionate Use/Early Access Program at Erasmus MC.
This experience has generated multiple peer-reviewed publications demonstrating Ampligen's potential mechanisms of action, including enhancement of peripheral B cell numbers associated with longer survival, and enhancement of antitumor immunity through dendritic cell-mediated T-cell responses.
Intellectual Property and Regulatory Strategy
AIM has developed a comprehensive intellectual property strategy for pancreatic cancer applications, including a recently issued U.S. patent for Ampligen as an oncology treatment in combination with anti-PD-L1 agents, extending protection until August 9, 2039. The company has also secured orphan drug designations for pancreatic cancer in both the United States and European Union, providing years of market exclusivity following potential commercial approval.
Next Steps and Future Analysis
Prof. van Eijck noted that immunologic correlatives and further follow-up remain essential to determine biological activity and response durability. The research team plans to identify which patients are most likely to benefit from the combination treatment, potentially enabling personalized therapy approaches to maximize clinical outcomes.
The ongoing immune-monitoring data analysis may provide additional mechanistic insights or identify predictive biomarkers that could further advance this potential therapeutic approach for pancreatic cancer patients.
