Candidate: Ampligen® (rintatolimod)
Type: Immune modulator indicated for severe chronic fatigue syndrome
2021 Status: AIM ImmunoTech said January 29 that it entered into a sponsorship agreement with the Centre for Human Drug Research (CHDR) for the proposed AMP-COV-100 (CHDR2049) clinical study on the safety of Ampligen as an intranasal COVID-19 therapy. CHDR, an independent institute located in Leiden in the Netherlands, will conduct and manage the proposed Phase I study, which AIM is funding.
According to plans, the Phase I trial calls for the enrollment of eight healthy subjects in each of four Ampligen treatment groups and one placebo group, for a total of 40 healthy subjects. They will receive intranasal dosing every other day for 13 days, for a total of seven doses each. AIM said it was working to finalize the trial protocol.
2020 Status: AIM said November 25 that a Roswell Park Comprehensive Cancer Center-sponsored Phase I/II trial (NCT04379518) evaluating the two-drug combination of AIM’s Ampligen and interferon alpha-2b as a potential early-onset treatment for patients with cancer and mild-to-moderate COVID-19 is fully underway, with the first patient enrolled and treated on the study. AIM is providing Ampligen at no charge for the trial, which is funded in part through grants from the National Cancer Institute and AIM, as well as institutional support from Roswell Park.
AIM also said it is working on an intranasal prophylaxis strategy for Ampligen in frontline workers, elderly people, and other high-risk patients, including those with co-morbidities such as cancer. The company is also developing a therapy designed for COVID-19 “long haulers” showing persistent symptoms after their infections have ended.
In October, AIM received institutional review board (IRB) approval to expand its AMP-511 Expanded Access Program (NCT00215813) open label trial of Ampligen in Myalgic Encephalomyelitis/Chronic Fatigue Syndrome (ME/CFS) in order to include patients previously diagnosed with SARS-CoV-2, but who still show chronic fatigue-like symptoms. Those patients, also called “long haulers” because of the persistence of their symptoms, will be able to receive Ampligen treatments alongside the ME/CFS patients in the EAP. Up to 20 of the 100 active participants can be long haulers, according to the new trial protocol amendment. AIM is currently preparing the IRB-approved protocol for submission to the FDA.
In August, AIM said Ampligen decreased SARS-CoV-2 infectious viral yields by 90% at clinically achievable intranasal dosage levels, based on an in vitro model at The Institute for Antiviral Research at Utah State University. The company used the USA-WA1/2020 strain of SARS-CoV-2 in a 3-D, in vitro model of normal, human-derived tracheal/bronchial epithelial cells, citing the lack of an adequate animal model.
The results support developing an intranasal prophylactic approach using Ampligen to prevent COVID-19, according to the company.
In July, AIM entered into a clinical trial agreement with Roswell Park Comprehensive Cancer Center to support the Roswell Park-sponsored Phase I/IIa trial (NCT04379518) of Ampligen in combination with interferon alfa-2b in cancer patients with COVID-19. The trial is designed to assess the safety and effectiveness of the combination regimen to clear the SARS-CoV-2 virus from the upper airway in patients with cancer and mild to moderate COVID-19. AIM reasons that Ampligen with interferon alfa-2b may benefit cancer patients with COVID-19, as Ampligen demonstrated a protective effect in earlier SARS-CoV-1 studies in mouse models.
The initial Phase I portion of the study is set to evaluate 12-24 patients receiving both Ampligen and interferon alfa-2b at escalating doses. Once the Phase I portion is complete, the Phase IIa portion of this study will be initiated with patients randomized into two arms: one receiving the two-drug combination and a control group who will not receive Ampligen or interferon alfa-2b, but will receive best available care. Enrollment of patients is expected to begin within 30 days for the trial, which will be led by on the trial, which will be conducted at Roswell Park by a team headed by Brahm Segal, MD, Chair of Internal Medicine and Chief of Infectious Diseases.
Funding for the clinical trial has been provided in part through grants from the NIH’s National Cancer Institute and AIM, as well as institutional support from Roswell Park.
AIM filed a provisional utility patent application for Ampligen in June as a potential therapy for COVID-19-induced ME/CFS-like illness.
In April, AIM entered into a Material Transfer and Research Agreement (MTA) with Shenzhen Smoore Technology to research in China the efficacy of Smoore’s vaping device using Ampligen, to enable inhalation of the drug deep into the lungs at the first signs of COVID-19.
In March, the company said it was in talks with regulators in the Netherlands, where Ampligen was recently used to treat pancreatic cancer patients, to explore expedited preclinical and clinical trials of Ampligen. Protocols for those trials are in final stages of development. AIM ImmunoTech also said it was actively seeking investigators and sites for clinical trials—and disclosed talks with a potential partner in Argentina, GP-Pharm, to advance Ampligen in COVID-19. The drug is approved in Argentina to treat myalgic encephalomyelitis/chronic fatigue syndrome.
AIM ImmunoTech has stated in a prospectus that it partnered with ChinaGoAbroad, a matchmaking and advisory service for cross-border deals involving China, to facilitate the entry of Ampligen into China for use as a prophylactic/early-onset therapeutic against COVID-19. AIM ImmunoTech also said Japan’s National Institute of Infectious Diseases (NIID) will study Ampligen as a potential treatment for COVID-1, through a study to be conducted at NIID and the University of Tokyo.
The company filed three provisional patent applications with the U.S. Patent and Trademark Office in January.
COVID-19: 300 Candidates and Counting
To navigate through the >300 potential therapeutic and vaccine options for COVID-19, GEN has grouped the candidates into four broad categories based on their developmental and (where applicable) clinical progress:
● FRONT RUNNER – the most promising therapeutics/vaccines based on clinical progress, favorable data or both.
● DEFINITELY MAYBE – earlier phases with promising partners, or more advanced candidates in development that have generated uneven data
● KEEPING AN EYE ON… – interesting technology, attracting notable partners, or both, but preliminary data.
● TOO SOON TO TELL – longshots pending additional experimental and/or clinical data.
GEN has also tagged the most common treatment types: