American Gene Technologies (AGT), a clinical-stage biotechnology company, announced that the independent Data Safety and Monitoring Board (DSMB) overseeing the trial of AGT’s HIV gene therapy found no serious adverse events (SAEs) from the treatment for the third patient and voted unanimously to allow AGT’s HIV cure program to continue at a faster pace, eliminating the requirement of DSMB reviews between each participant and moving to quarterly DSMB safety meetings. AGT and trial sites will monitor participants for any adverse events and report them to the DSMB, however, the pace of enrollment and treatment can be increased.
“This is a very exciting moment in our trial,” said AGT CEO Jeff Galvin. “With no serious adverse effects observed in the third participant, we’re seeing a verifiable trend in the data that gives reasonable confidence that the treatment has low risk of adverse events. Once three more participants are treated in November and December, we can start studying efficacy data. We expect to see objective markers from studies of treated participants’ blood tests by the end of the year, and hopefully will have a functionally cured patient by next summer.”
Designated as the RePAIR trial (Restore Potent Antiviral Immune Responses, NCT04561258), participants receive a single infusion of their own HIV-specific CD4 T cells after those cells were 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.
Phase 1 Trial Status
- Overview: AGT’s first-in-human study for AGT103-T is currently underway at trial sites in the Maryland / Washington, D.C. area. The Phase 1 trial is the company’s first step in clinical testing of cell and gene therapies for HIV, cancer, and rare diseases. Enrollment, product manufacturing and treatments are ongoing at AGT's clinical trial sites. 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.
- Focus: The primary endpoint is safety; 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 Q2 2022, although final data and long-term monitoring will continue. The Phase 1 study will include from 15 to 18 participants.
- 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, and the third in September 2021.
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 the 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. AGT is committed to addressing this unmet medical need.
AGT103-T is a genetically modified cell product made from a person's own cells. AGT's unique approach focuses on permanently repairing the key immune system damage caused by HIV. AGT’s goal is to develop a cell and gene therapy capable of repairing the immune system so it will provide natural control over HIV replication.
About American Gene Technologies
AGT is a gene and cell therapy company with a proprietary gene-delivery platform for rapid development of cell and gene therapies to cure infectious diseases, cancers, and inherited disorders. AGT's mission is to transform people's lives through genetic medicines that rid the body of disease. AGT 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. AGT's 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 2022.