American Gene Technologies^® HIV Cure Clinical Trial Enters Critical Phase: Withdrawing Participants from Antiretrovirals

American Gene Technologies, a clinical-stage biotechnology company, has reached a turning point in its HIV cure clinical trial: it began withdrawing participants from their antiretroviral drugs earlier this month. This clinical protocol, technically known as analytic treatment interruption (ATI), may ultimately determine if this gene therapy treatment can cure HIV.

“This is the next logical step in the trial’s progression,” explained Dr. Marcus A. Conant, Chief Medical Officer at American Gene Technologies. “By withdrawing these patients’ antiretrovirals we can demonstrate whether our AGT103-T gene therapy will lead to a cure.”

In February 2022, the independent Institutional Review Board (IRB) overseeing the clinical trial approved the company to proceed with the ATI. Seven patients have received AGT103-T so far. None have experienced any serious adverse events, thereby demonstrating the therapy’s safety. Blood markers in the treated patients have provided a view into how the cell therapy is responding in their bodies. So far, the data has confirmed that the cells are properly engrafting, persisting, and seem to be remaining uninfected while reacting to, and likely fighting HIV the way scientists expected.

“This is a critical step in our mission to cure HIV using gene therapy,” said American Gene Technologies CEO Jeff Galvin. “We’ll monitor participants carefully, and within the next few months we’ll begin to know how close we are to reaching our dream of curing HIV. If participants respond as we expect, they may no longer need to take their antiretroviral medications, and could remain immune to HIV for life without further treatment. If we obtain that result, we may have achieved the most exciting development in HIV research in decades. This milestone could lead to a reliable cure for HIV that returns infected individuals to a normal life without risk of ever developing AIDS or infecting another person.”

Participation in the treatment withdrawal study by the Phase 1 clinical trial participants is voluntary. To proceed with a monitored antiretroviral treatment interruption, participants re-enroll with a new informed consent agreement that outlines the risks and details of the updated ATI protocol.

Phase 1 Trial Background

• Overview: Designated the RePAIR trial (Restore Potent Antiviral Immune Responses, NCT04561258), American Gene Technologies’ first-in-human study for AGT103-T is currently underway at trial sites in the Maryland / Washington, D.C. area. Participants in the Phase 1 trial receive a single infusion of their own HIV-specific CD4 T cells after those cells are enriched and genetically modified to resist infection. Without any observed negative or adverse events, the DSMB has allowed the trial to continue without adjustments or delays.
• Focus: The primary endpoints of RePAIR are safety and efficacy. Testing related to secondary endpoints evaluates responses to treatment, including changes in the immune response to HIV.
• Participant Criteria: Participants range in age from 18 to 60 and include males and females. Participants have been diagnosed with HIV for at least three years and must have taken HIV antiretroviral medication for more than two years prior to enrollment. Participants cannot be pregnant and must be available to attend 17 study visits over a 10-month period. Anticipated completion of treatments in the Phase 1 study is Q3 2022, although final data and long-term monitoring will continue. The Phase 1 study will include up to 18 participants. (The recruitment status of the Phase 1 RePAIR clinical trial, along with information on the trial sites, can be found on the official ClinicalTrials.gov website.)
• Timeframe: Participants treated with AGT103-T are followed for 6 months in this safety study before enrolling in an FDA-mandated, 15-year, long-term follow up (required for all gene therapy trials). The first infusion occurred in May 2021, the second in August 2021, the third in September 2021, the fourth and fifth in November 2021, the sixth in February 2022 and the seventh in May 2022. An analytic treatment interruption (ATI) study to determine product efficacy has been approved. American Gene Technologies expects to reveal results of the ATI by the end of this year (2022).

About HIV

According to UNAIDS, approximately 37.7 million people worldwide live with HIV/AIDS. In the United States, government statistics show 1.2 million people have HIV and estimate that 34,800 Americans were newly infected with HIV in 2019. Across the globe, UNAIDS estimates that approximately 1.5 million individuals were newly infected with HIV in 2020. The Washington D.C./Baltimore area is often cited as a ‘hot spot’ for HIV, with Washington, D.C., having the highest rate of infection at nearly 46 cases per 100,000 population and Baltimore City having rates of 17 cases per 100,000. Maryland also ranks sixth among U.S. states and territories in HIV diagnosis rates, with more than 900 new cases in 2019 alone, according to the Maryland Department of Health.

Since the late 1980s, antiretroviral drugs have restored quality of life to persons living with HIV and, in some cases, have even been used to prevent new infections. However, no approved treatment has demonstrated the ability to cure HIV. American Gene Technologies is committed to addressing this unmet medical need.

About AGT103-T

AGT103-T is a genetically modified cell product made from a person's own cells. American Gene Technologies’ unique approach focuses on permanently repairing the key immune system damage caused by HIV. American Gene Technologies’ goal is to develop a gene therapy capable of repairing the immune system so it will provide natural control over HIV replication.

About American Gene Technologies

American Gene Technologies is a gene therapy company with a proprietary gene-delivery platform for rapid development of gene therapies to cure infectious diseases, cancers, and inherited disorders. Its mission is to transform people's lives through genetic medicines that rid the body of disease. American Gene Technologies has been granted four patents for the technology used to make AGT103-T and 11 patents for its unique immuno-oncology approach to stimulate gamma-delta (γδ) T cells to destroy a variety of solid tumors. The company has developed a synthetic gene for treating Phenylketonuria (PKU), a debilitating inherited disease. American Gene Technologies' treatment for PKU has been granted Orphan Drug Designation by the Food and Drug Administration (FDA), and it is expected to reach the clinic in 2023.

Video About American Gene Technologies’ Work: https://youtu.be/fiA2s7JCkJ8

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