Fortress Biotech Subsidiary Helocyte Announces Option Agreement with City of Hope for Exclusive Worldwide Rights to use a Novel Bispecific CMV/HIV CAR T Cell Therapy (optionally in combination with Triplex) for the Treatment of Adults Living with HIV-1 - Seite 2

“City of Hope made a major advancement when our transplant team helped a patient achieve remission for both HIV and leukemia,” said John A. Zaia, M.D., the Aaron D. Miller and Edith Miller Chair for Gene Therapy at City of Hope. “We are hoping to evaluate a CAR T therapy for HIV in a Phase 1 clinical trial so that one day more people with HIV might be able to achieve long-term HIV remission.”

The clinical study will build upon preclinical data published in Molecular Therapy - Methods & Clinical Development which demonstrated the potential efficacy of combining a CMV vaccine and CMV/HIV CAR T cell therapy to eradicate HIV. The study illustrated the potential long-term durability of the combination therapy, which induced therapeutic immune cells to take hold in bone marrow. The objective of this approach is to target and eradicate latent viral T cell reservoirs in immune cells to achieve complete HIV clearance.

The current standard of care for the treatment of HIV relies upon the use of ART, which is a non-curative, life-long therapy. Optimal use of ART can effectively control HIV replication; prevent the onset of Acquired Immuno-Deficiency Syndrome (“AIDS”); prolong survival; and reduce the risk of transmission to others. However, there are several challenges associated with the use of ART, including: chronic treatment adherence; drug resistance, particularly in those who do not fully adhere to treatment; adverse events; and the persistence of immune dysfunction during treatment. There remains a significant unmet medical need to provide a functional cure for HIV and eliminate the need for ART.

To date, the engineering of T cells to express HIV-specific CAR T cells has failed to demonstrate meaningful clinical benefits. This is believed to be due in part to the effectiveness of highly active ART, which reduces HIV viral load to a level that prevents the activation of CAR T cells. The use of Triplex is believed to stimulate CMV/HIV-CAR T cells to proliferate within patients’ bodies. In the aforementioned preclinical study, CAR T cells were engineered to target and kill cells tagged with a particular protein called gp120, which is expressed in all HIV viruses, frequently with mutations that cause the virus to evade natural immunity without affecting healthy cells.