AIM ImmunoTech (AIM) Corporate Connect Webinar Series summary

Strategic focus and clinical progress

• Ampligen is being repurposed from chronic fatigue syndrome to oncology, with a primary focus on pancreatic cancer based on strong safety and efficacy data from multiple solid tumor studies in collaboration with major industry partners.

• The pancreatic cancer program began in 2017 with Dutch government approval for an early access program, treating 57 late-stage patients and progressing to a phase II trial with AstraZeneca's Imfinzi at Erasmus Medical Center.

• Ampligen's mechanism of action involves converting immunosilent (cold) tumors into immunoresponsive (hot) tumors by altering the tumor microenvironment, supported by specific immunological biomarkers.

• The safety profile is robust, with over 100,000 IV doses administered and additional safety data from intranasal and intraperitoneal studies.

• The company is prioritizing new drug approval for late-stage metastatic pancreatic cancer, addressing a significant unmet need.

Intellectual property and market exclusivity

• Ampligen benefits from both patent and orphan drug designations, providing market exclusivity in the US and EU, with a recent US patent covering Ampligen plus PD-L1 valid until 2039.

• Orphan drug exclusivity periods begin upon drug approval, offering up to 10 years of protection in the EU.

• A Netherlands patent has also been granted for the Ampligen plus checkpoint inhibitor combination.

Clinical outcomes and data highlights

• In the Dutch early access program, Ampligen extended median overall survival to 19.7 months versus 12.5 months for standard care, an 8.6-month benefit.

• Patients reported significant improvements in quality of life, a critical factor in late-stage pancreatic cancer.

• Biomarker analysis, including neutrophil-lymphocyte ratio and CA 19-9 levels, correlates with improved survival and progression-free survival.

• Ongoing phase II DURIPANC trial interim results show positive safety, efficacy, and quality of life data, supporting advancement to phase III.