American Gene Technology’s Cell Therapy Continues to Show Safety in HIV

An independent Data Safety and Monitoring Board has recommended that the phase 1 RePAIR trial continue at an accelerated pace.

An independent Data Safety and Monitoring Board (DSMB) has recommended that the phase 1 RePAIR trial (NCT04561258) assessing American Gene Technology’s (AGT) human immunodeficiency virus (HIV) cell therapy AGT-103T continue at a faster pace following a third patient being dosed in the trial without serious adverse events (AEs).1

Following the DSMB’s recommendation, safety reviews will now be quarterly instead of after every initial patient dosing. The trial’s pace of enrollment of treatment will be increased, although investigators will monitor and report any AEs to the DSMB.

“This is a very exciting moment in our trial,” said Jeff Galvin, chief executive officer, AGT, in a statement.1 “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.”

The RePAIR trial, part of the HIV Cure program, is currently being conducted at sites in Maryland and Washington, DC and plans to enroll 15 to 18 participants that have been diagnosed with HIV for at least 3 years and have been taking antiretroviral medication for at least 2 years. Initial completion of the trial is planned for the second quarter of 2022.

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The study is primarily assessing safety of AGT-103T as measured by AEs. Immune response to HIV will also be evaluated for exploratory efficacy. In the study, participants receive a single infusion of their own genetically modified HIV-specific CD4 T-cells. They will then be followed up for 6 months before enrolling in a 15-year, long-term follow-up study. To date, the 3 patients were treated in May, August, and September 2021.

AGT-103T is an autologous cell therapy designed to repair the T helper cells and provide durable virus control. It can withstand HIV strains that vary in sequence or use alternate ways to enter and infect T cells. AGT previously announced that the DSMB voted unanimously in July 2021 to continue the trial without modifications, after a safety analysis revealed no adverse events arising from the therapy.2,3

“Safety represents a critical milestone in the project and gives us additional confidence that HIV can be cured,” Galvin said in an earlier statement.3 “This milestone supports AGT’s mission to relieve suffering and premature death from serious human diseases. AGT’s HIV cure program is based on a platform that has the capacity to treat other chronic viral infections, as well as monogenic disorders and cancers... A success in HIV would provide sustained funding to accelerate dozens of therapeutics that are within the scope of AGT’s technology platform.”

On the heels of AGT is Excision BioTherapeutics’s EBT-101 which received FDA investigational new drug (IND) clearance in September for the potential treatment of HIV.4 EBT-101 is an investigational, CRISPR-based, in-vivo gene therapy as opposed to AGT’s autologous cell therapy approach.

1. American Gene Technologies passes major milestone in potential HIV cure clinical trial with third patient showing no serious adverse effects (SAEs). News release. American Gene Technologies. November 9, 2021.
2. AGT announces progress with HIV Cure program, phase 1 clinical trial. News release. American Gene Technologies. May 27, 2021.
3. HIV Cure program releases initial clinical trial data. News release. American Gene Technologies. July 16, 2021.
4. Excision receives FDA Clearance of IND for phase 1/2 Trial of EBT-101 CRISPR-based therapeutic for treatment of HIV. News release. Excision BioTherapeutics. September 15, 2021.
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