AIM ImmunoTech Inc (NYSE:AIM) announced that it has finalized the protocol for a planned Phase 2 study of Ampligen as a therapy for locally advanced or metastatic late-stage pancreatic cancer.
The company expects to submit both an Investigational New Drug application (IND) and a request for Fast Track status with the US Food and Drug Administration (FDA) by no later than October 18.
Amarex Clinical Research will manage the AIM-sponsored study. The Buffett Cancer Center at the University of Nebraska Medical Center and Erasmus MC in The Netherlands are expected to be the primary study sites, although additional sites are expected to participate, the company added.
READ: AIM ImmunoTech submits Pre-IND application to FDA for Phase 2 trials of Ampligen as potential therapeutic for early-onset coronavirus
AIM said the 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, a chemotherapy regimen for treatment of advanced pancreatic cancer, compared to 12.5 months in the historical control group.
It noted that the 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,” AIM said in a statement.
“These detailed data will also be a component of the upcoming Phase 2 IND submission and a justification for the Fast Track application.”
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 standard of care (SOC) 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 Ampligen alone or Ampligen combined with SOC, or 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 infusions. Subjects will be monitored for disease progression and may also receive anticancer SOC therapy, depending on their treating physicians.
“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," said Amarex CEO Dr Kazem Kazempour.
"The interim data may also allow for a “Breakthrough” drug designation from the FDA, which provides significant advantages to the clinical development program.”
AIM ImmunoTech is focused on the research and development of therapeutics to treat multiple types of cancers, immune disorders, and viral diseases, including coronavirus.
Contact the author at stephen.gunnion@proactiveinvestors.com