On October 4, 2021 AIM ImmunoTech Inc. (NYSE American: AIM) reported that it has finalized the protocol for a planned Phase 2 study of the company’s drug Ampligen as a therapy for locally advanced or metastatic late-stage pancreatic cancer (Press release, AIM ImmunoTech, OCT 4, 2021, View Source [SID1234590740]). The company expects to submit both an Investigational New Drug application (IND) and an application for Fast Track status with the U.S. Food and Drug Administration (FDA) no later than October 18, 2021.
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Amarex Clinical Research will manage the AIM-sponsored study. The Buffett Cancer Center at the University of Nebraska Medical Center (UNMC) and Erasmus MC in The Netherlands are expected to be the primary study sites, although additional sites are expected to participate.
AIM’s Pancreatic Cancer Research
The new proposed study is based on statistically significant clinical data in an early-access program where 27 subjects were treated at Erasmus MC in The Netherlands. The overall survival of the Ampligen-treated cohort was 19.2 months from the start of FOLFIRINOX, compared to 12.5 months in the historical control group. This increase of 6.7 months in the Ampligen-treated group was clinically and statistically significant. Additionally, several subjects are still alive more than three years later. These detailed data were filed with and supported the recent approval of orphan drug status for Ampligen by both the FDA and the European Medicines Agency. These detailed data will also be a component of the upcoming Phase 2 IND submission and a justification for the Fast Track application.
Prof. C.H.J. van Eijck, MD, PhD, and his team at Erasmus MC intend to publish a detailed clinical report on their results in a peer-reviewed journal no later than January 2022. AIM will publicly release the detailed data and analysis at that time.
Overview of the Planned AMP-270 Study
The planned AMP-270 clinical trial will be a Phase 2, randomized, open-label, controlled, parallel-arm study with the primary objective of comparing the efficacy of Ampligen when added to SOC (standard of care) versus SOC alone for subjects with advanced pancreatic carcinoma recently treated with FOLFIRINOX chemotherapy regimen. Secondary objectives include comparing safety and tolerability. There will be two parallel arms and approximately 250 eligible subjects will be randomized 1:1 to receive either 1) Ampligen alone or Ampligen combined with SOC, or 2) SOC alone.
The parallel control arm will receive SOC without Ampligen. This will consist of monitoring for disease progression along with active anticancer therapy as determined by the patients’ physicians.
Patients in the Ampligen-plus-SOC arm will be administered twice weekly Ampligen intravenous (IV) infusions. Subjects will be monitored for disease progression and may also receive anticancer SOC therapy (depending on their treating physicians).
Amarex CEO Kazem Kazempour, PhD, states: "The Phase 2 study design includes an interim data analysis intended to allow for the transition from a Phase 2 to a Phase 3 study pending the FDA’s review and approval. The interim data may also allow for a "Breakthrough" drug designation from the FDA, which provides significant advantages to the clinical development program."
Pancreatic Cancer Subject-Matter Experts Discuss the New Study
Prof. Kelsey Klute, MD, of the Buffett Cancer Center at UNMC, and the study’s principal investigator in the United States, states: "Most people diagnosed with pancreatic cancer don’t survive more than a year after their diagnosis. There is a critical need for more effective therapies to treat this lethal disease. Based on the Erasmus data and our preclinical data, we’re optimistic about the activity of Ampligen in treating pancreatic cancer. We’ve designed this clinical trial to test whether Ampligen improves survival compared to the current standard of care. But I think it’s equally important that this study will also help us understand the effect of Ampligen at the cellular level of the tumor and the immune system – to learn why and how it works in certain patients and why it might fail in others – and set the stage to refine the use of Ampligen in the future."
Prof. Michael A. "Tony" Hollingsworth, PhD, also of UNMC, a world-renowned pancreatic cancer researcher who is designing exploratory experimental endpoints to supplement the survival-based primary endpoint, states: "We have designed correlative studies to test the hypothesis that administration of Ampligen in the maintenance setting will improve survival by enhancing tumor-specific immunity and also systemic immunity to opportunistic pathogens that contribute to the patients demise during end stage disease."
Prof. C.H.J. van Eijck, MD, PhD, states: "The overall survival of the experimental group was compared to a well matched historical control cohort matched for age, gender, stage of disease, and number of cycles of FOLFIRINOX chemotherapy. Median survival was significantly higher in the Ampligen arm as compared to the historical controls. Based on these data, I see the potential for Ampligen as a meaningful extension of the standard of care for advanced pancreatic cancer, which we are planning to investigate further in the upcoming randomized control trial."