About AIM ImmunoTech

AIM ImmunoTech Inc. and its subsidiaries (AIM) operate as an immuno-pharma company. The company focuses on the research and development of therapeutics to treat multiple types of cancers, viral diseases, and immune-deficiency disorders. The company has established a strong foundation of laboratory, pre-clinical and clinical data with respect to the development of nucleic acids and natural interferon to enhance the natural antiviral defense system of the human body, and to aid the development of therapeutic products for the treatment of certain cancers and chronic diseases. The company's flagship products are Ampligen (rintatolimod), a first-in-class drug of large macromolecular RNA (ribonucleic acid) molecules, and Alferon N Injection (Interferon Alfa-N3). Ampligen has not been approved by the FDA or marketed in the United States. Ampligen is approved for commercial sale in the Argentine Republic for the treatment of severe Chronic Fatigue Syndrome (CFS). The company's primary business focus involves Ampligen. Ampligen represents a dsRNA being developed for globally important cancers, viral diseases, and disorders of the immune system. The company is proceeding primarily in four areas: Conducting a randomized, controlled study to evaluate efficacy and safety of Ampligen compared to a control group to treat locally advanced pancreatic cancer patients. Evaluating Ampligen in other cancers, as a potential therapy that modifies the tumor microenvironment with the goal of increasing anti-tumor responses to check point inhibitors. Exploring Ampligen's antiviral activities and potential use as a prophylactic or treatment for existing viruses, new viruses and mutated viruses thereof. Evaluating Ampligen as a treatment for myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS) and fatigue and/or Post-COVID conditions of fatigue. The company is prioritizing its activities in an order related to the stage of development, with those clinical activities, such as pancreatic cancer, ME/CFS and Post-COVID conditions having priority over antiviral experimentation. The company intends that priority clinical work be conducted in FDA- or EMA-authorized trials, which could support a potential future New Drug Application (NDA). However, the company's antiviral experimentation is designed to accumulate additional preliminary data supporting its hypothesis that Ampligen is a powerful, broad-spectrum prophylaxis and early-onset therapeutic that may confer enhanced immunity and cross-protection. Accordingly, the company conduct its antiviral programs in those venues most readily available and able to generate valid proof-of-concept data, including foreign venues. Immuno-Oncology The company focuses on pancreatic cancer because testing results, to date, primarily conducted in the Netherlands, have been very promising. The Netherlands study generated statistically significant data indicating that Ampligen extended survival well beyond the Standard of Care ('SOC'). These data support the proposition that Ampligen, when administered in patients with locally advanced or metastatic pancreatic cancer after systemic chemotherapy showed a statistically significant increase in survival rate. In 2021, the company and its Contract Research Organization, Amarex Clinical Research LLC (Amarex), submitted an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) for a planned Phase 2 study of Ampligen as a therapy for locally advanced or metastatic late-stage pancreatic cancer. In 2022, the company received Institutional Review Board (IRB) approval of the trial protocol and so announced the trial's commencement. Ampligen has also demonstrated in the clinic the potential for standalone efficacy in a number of solid tumors. The company has also seen success in increasing survival rates and efficacy in the treatment of animal tumors when Ampligen is used in combination with checkpoint blockade therapies. In March 2022, the company announced interim data from an investigator-initiated, Phase 2, single-arm, efficacy/safety trial to evaluate the effectiveness of combining intensive locoregional intraperitoneal (IP) chemoimmunotherapy of cisplatin with IP Ampligen (TLR-3 agonist) and IV infusion of the checkpoint inhibitor pembrolizumab (IVP) for patients with recurrent platinum-sensitive ovarian cancer. The company's data from the study, which is being conducted by the University of Pittsburgh Medical Center, demonstrated that when combining three drugs - Ampligen and pembrolizumab, which are both immune therapies, with cisplatin, a chemotherapy - evidence of increased biomarkers associated with T cell chemotaxis and cytolytic function has been seen. Importantly, increases of these biomarkers in the tumor microenvironment have been correlated with favorable tumor responses. These successes in the field of immuno-oncology have guided the company's efforts toward the potential use of Ampligen as a combinational therapy for the treatment of a variety of solid tumor types. The first of the company's patent applications in this space was granted by the Netherlands on March 15, 2021. Products The company's primary pharmaceutical product platform consists of Ampligen (rintatolimod), a first-in-class drug of large macromolecular double-stranded (ds) RNA (ribonucleic acid) molecules, and the company's FDA-approved natural alpha-interferon product, Alferon N Injection. Ampligen Ampligen is approved for sale in Argentina (to 2026) for severe Chronic Fatigue Syndrome (CFS) and is an experimental drug in the United States undergoing clinical development for the treatment of certain cancers and ME/CFS. Over its developmental history, Ampligen has received various designations, including Orphan Drug Product Designation (FDA and European Medicines Agency ['EMA']), Treatment protocol (e.g., 'Expanded Access' or 'Compassionate' use authorization) with Cost Recovery Authorization (FDA) and 'promising' clinical outcome recognition based on the evaluation of certain summary clinical reports ('AHRQ' or Agency for Healthcare Research and Quality). Based on the results of published, peer-reviewed pre-clinical studies and clinical trials, Ampligen may have broad-spectrum antiviral and anti-cancer properties. Ampligen represents the first drug in the class of large (macromolecular) dsRNA molecules to apply for NDA review. There are two forms of nucleic acids: deoxyribonucleic acid ('DNA') and ribonucleic acid ('RNA'). DNA is a group of naturally occurring molecules found in chromosomes, the cell's genetic machinery. RNA is a group of naturally occurring informational molecules, which orchestrate a cell's behavior which, in turn, regulates the action of groups of cells, including the cells, which comprise the body's immune system. RNA directs the production of proteins and regulates certain cell activities, including the activation of an otherwise dormant cellular defense against viruses and tumors. The company's drug technology utilizes specifically configured RNA and is a selective Toll-like Receptor 3 ('TLR3') agonist that can be administered intravenously, intranasally and intraperitoneally. Ampligen has been assigned the generic name rintatolimod by the United States Adopted Names Council ('USANC') and has the chemical designation poly(I):poly(C12U). Expanded Access Program/Early Access Programs/clinical trials of Ampligen that have been conducted or that are ongoing include studies of the potential treatment of patients with renal cell carcinoma, malignant melanoma, non-small cell lung cancer, ovarian cancer, breast cancer, colorectal cancer, prostate cancer, pancreatic cancer, ME/CFS, Hepatitis B, HIV, COVID-19 (the disease caused by SARS-CoV-2) and Post-COVID conditions. The company has received approval of its NDA from the Administracion Nacional de Medicamentos, Alimentos y Tecnologia Medica (ANMAT) for the commercial sale of Ampligen in the Argentine Republic for the treatment of severe CFS. The product will be marketed by GP Pharm, the company's commercial partner in Latin America. Shipment of the drug product to Argentina was initiated in 2018 to complete the release testing by ANMAT needed for commercial distribution. In 2019, the company received clearance from the FDA to ship Ampligen to Argentina for the commercial launch and subsequent sales. In 2020, the company received import clearance from ANMAT to import the first shipment of commercial grade vials of Ampligen into Argentina. The company is working with GP Pharm on the commercial launch of Ampligen in Argentina. Commercialization in Argentina will require, among other things, GP Pharm to establish disease awareness, medical education, creation of an appropriate reimbursement level, design of marketing strategies and completion of manufacturing preparations for launch and ANMAT conducting a final inspection of the product and release tests before granting final approval to begin commercial sales. AIM has supplied GP Pharm with the Ampligen required for testing and ANMAT release. This testing and approval process is delayed due to the COVID-19 pandemic and ANMAT's internal processes. The ongoing impact of COVID-19 in Argentina is taxing the nation's health care system and is, understandably, the main priority of its regulators. Once final approval by ANMAT is obtained, GP Pharm will begin distributing Ampligen in Argentina. The company continues to pursue its Ampligen NDA, for the treatment of CFS with the FDA. The FDA has authorized an open-label expanded access treatment protocol ('AMP-511') allowing patient access to Ampligen in a study under which severely debilitated CFS patients have the opportunity to be on Ampligen to treat this very serious and chronic condition. The AMP-511 protocol is ongoing. The data collected from the AMP-511 protocol through clinical sites provide safety information regarding the use of Ampligen in patients with CFS. The company is establishing an enlarged database of clinical safety information, which it provides further documentation regarding the absence of autoimmune disease associated with Ampligen treatment. Continued efforts to understand existing data, and to advance the development of new data and information, ultimately supports the company's future filings for Ampligen and/or the design of future clinical studies that the FDA requested in a CRL. In 2020, the company received IRB approval for the expansion of the AMP-511 Expanded Access Program clinical trial for ME/CFS to include patients previously diagnosed with SARS-CoV-2 following clearance of the virus, but who still demonstrate chronic fatigue-like symptoms that it refers to as Post-COVID conditions. As of December 31, 2022, there were 10 patients enrolled in this open-label expanded access treatment protocol), with seven Post-COVID patients receiving or having received treatment. AIM previously reported positive preliminary results based on data from the first four Post-COVID Condition patients enrolled in the study. The data show that, by week 12, compared to baseline, there was what the investigators considered a clinically significant decrease in fatigue-related measures. In 2016, the company entered into a five-year agreement with myTomorrows, a Netherlands based company, for the commencement and management of an Early Access Program ('EAP') in Europe and Turkey (the 'Territory') related to ME/CFS. Pursuant to the agreement, as amended, myTomorrows is also managing all Early Access Programs and Special Access Programs in Europe, Canada and Turkey to treat pancreatic cancer and ME/CFS patients. The agreement was automatically extended for a period of 12 months on May 20, 2021, and automatically extend for an additional period of 12 months on May 20, 2022. In June 2018, Ampligen was cited as outperforming two other TLR3 agonists poly IC and natural double stranded RNA in creating an enhanced tumor microenvironment for checkpoint blockade therapy in the journal of Cancer Research (http://cancerres.aacrjournals.org/content/early/2018/05/31/0008-5472.CAN-17-3985). The company activates the TLR3 pathway and promoted an accumulation of killer T cells but, unlike the other two TLR3 agonists, it did so without causing regulatory T cell (Treg) attraction. These findings were considered important because they indicate that Ampligen selectively reprograms the tumor microenvironment by inducing the beneficial aspects of tumor inflammation (attracting killer T cells), without amplifying immune-suppressive elements, such as regulatory T cells. The study was conducted at the University of Pittsburgh and Roswell Park as a part of the NIH-funded P01 CA132714 and Ovarian Cancer Specialized Program of Research Excellence (SPORE). In 2018, the company completed production of two commercial-size batches of more than 16,000 vials of Ampligen, following its 'Fill & Finish' at Jubilant HollisterStier, the Contract Manufacturing Organization. These lots passed all required testing for regulatory release for human use and are being used for multiple programs, including the treatment of ME/CFS, the pancreatic cancer EAP in the Netherlands, and will continue to be used for ongoing and future clinical studies in oncology. Additionally, two lots of Ampligen were manufactured in December 2019 and January 2020 at Jubilant HollisterStier. The manufactured lots of Ampligen have been fully tested and released for commercial product launch in Argentina and for clinical trials. Additionally, in 2020, the company added Pharmaceutics International Inc. ('Pii') as a 'Fill & Finish' provider. The contracts augment the company's active and in-process fill and finish capacity. Alferon N Injection Alferon N Injection is the registered trademark for the company's injectable formulation of natural alpha interferon. Alferon N Injection is the only natural-source, multi-species alpha interferon approved for sale in the United States and Argentina for the intralesional (within lesions) treatment of refractory (resistant to other treatment) or recurring external genital warts in patients 18 years of age or older. Alferon N Injection is also approved in Argentina for the treatment of refractory patients that failed or were intolerant to treatment with recombinant interferons. Certain types of human papilloma viruses ('HPV') cause genital warts, a sexually transmitted disease ('STD'). Patents and Non-Patent Exclusivity Rights As of December 31, 2022, the company had 46 patents worldwide with 68 additional pending patent applications comprising its intellectual property. The company has received Orphan Drug Designation (ODD) from the FDA for Ampligen used in the treatment of Chronic Fatigue Syndrome, HIV, Metastatic Melanoma, Renal Cell Carcinoma, Pancreatic Adenocarcinoma and Ebola Virus Disease. In the European Union, ODD carries ten years of market exclusivity after receiving marketing authorization. The company has received ODD from the EU for Ampligen used in the treatment of Ebola Virus Disease and Pancreatic Adenocarcinoma and for Alferon used in the treatment of Middle East Respiratory Syndrome. Manufacturing The company is working with GP Pharm on the commercial launch of Ampligen in Argentina. Following the company's approval in Argentina, in 2017 it engaged Jubilant HollisterStier ('Jubilant') to be its authorized CMO for Ampligen. In addition, the company has supplied GP Pharm with the Ampligen required for testing and ANMAT release. Once final approval by ANMAT is obtained, the company anticipates that GP Pharm will begin distributing Ampligen in Argentina. In 2020, the company added Pharmaceutics International Inc. ('Pii') as a 'Fill & Finish' provider to enhance the company's capacity to produce Ampligen. In 2021, the company approved a proposal from Polysciences Inc. ('Polysciences') for the manufacture of its Poly I and Poly C12U polynucleotides and associated test methods at Polysciences' Warrington, PA location to enhance its capacity to produce the polymer precursors to the drug Ampligen. The company is utilizing Polysciences' expertise to refine its approach to polymer production. Additionally, it continues to be open to the possibility of agreements with other CMOs, so as to create redundancy and to meet the potential need for larger quantities of API. Marketing/Distribution In May 2016, the company entered into a five-year exclusive Renewed Sales, Marketing, Distribution and Supply Agreement (the 'Agreement') with GP Pharm. Under this Agreement, GP Pharm was responsible for gaining regulatory approval in Argentina for Ampligen to treat severe CFS in Argentina and for commercializing Ampligen for this indication in Argentina. The company granted GP Pharm the right to expand rights to sell this experimental therapeutic into other Latin America countries based upon GP Pharm achieving certain performance milestones. The company also granted GP Pharm an option to market Alferon N Injection in Argentina and other Latin America countries. The GP Pharm contract was extended in May 2021, and will end on May 24, 2024. In August 2021, ANMAT granted a five-year extension to a previous approval to sell and distribute Ampligen to treat severe CFS in Argentina. This extends the approval until 2026. Competition The major pharmaceutical competitors for Ampligen include Pfizer, GlaxoSmithKline, Merck & Co., Novartis and AstraZeneca. Biotech competitors include Baxter International, Fletcher/CSI, AVANT Immunotherapeutics, AVI BioPharma and Genta. Research and Development For the year ended December 31, 2022, the company's research and development expenses included $6,990,000. History The company was founded in 1966. It was incorporated in 1966. The company was formerly known as Hemispherx Biopharma, Inc. and changed its name to AIM ImmunoTech Inc. in 2019.