Fortress Biotech Announces First Patient Dosed in Phase 2 Clinical Trial of Triplex Vaccination in Stem Cell Donors to Reduce CMV Events in Recipients of HSCT

Study is the first randomized, controlled trial examining a vaccine's effectiveness in controlling CMV in recipients of HSCT from vaccinated donors

Encouraging pilot study results demonstrated the benefit of vaccinating donors with Triplex to convey protective CMV-specific T cell immunity to allogeneic HSCT recipients at risk for CMV reactivation

MIAMI, Jan. 27, 2025 (GLOBE NEWSWIRE) -- Fortress Biotech, Inc. (NASDAQ:FBIO) ("Fortress"), and its majority-owned subsidiary, Helocyte, Inc. ("Helocyte"), today announced that the first patient was dosed in a multicenter, placebo-controlled and randomized Phase 2 clinical trial to evaluate Triplex, a cytomegalovirus ("CMV") vaccine, when administered to human leukocyte antigen ("HLA") matched related stem cell donors to reduce CMV events in patients undergoing hematopoietic stem cell transplantation ("HSCT").

The Phase 2 clinical trial is funded by a grant from the National Cancer Institute ("NCI"). Triplex is anticipated to induce a CMV-specific immune response in the donor's body who then conveys that virus-specific immunity to the intended recipient of the stem cell transplant through the concept of adoptive immunity. Vaccination of donors with Triplex prior to stem cell harvest and transplant is believed to introduce virus-specific immunity sooner, and in turn, reduce CMV events in patients undergoing stem cell transplantation. The objective of the trial is to determine whether Triplex is safe and effective in reducing CMV events in recipients of HSCT from vaccinated donors.

CMV reactivation is one of the most common and life-threatening complications following allogeneic HSCT. The virus reactivates in an estimated 60-70% of CMV-seropositive recipients of HSCT with primary CMV infection occurring in 20-30% of CMV-seronegative recipients of HSCT from CMV-seropositive donors.

"CMV frequently affects patients undergoing transplants, posing significant risks to their recovery and survival. In this Phase 2 clinical trial, we are testing a new treatment approach, offering promise for better CMV management in HSCT recipients. This approach transfers CMV immunity from donors to transplant recipients, thereby potentially reducing the need for antiviral medications that can delay reconstitution of virus-specific immunity," said Don J. Diamond, Ph.D., Professor, Hematology & Hematopoietic Cell Transplantation, City of Hope's Los Angeles campus and the grants' principal investigator. Dr. Diamond and his team developed Triplex in cooperation with the NCI "NExT" program. Dr. Diamond also serves as a paid consultant to Helocyte and holds equity in the company.

The initial Phase 2 clinical trial (NCT03560752) builds upon Phase 1 pilot trial data that evaluated the potential safety and immunological response of Triplex presented at the 2023 Tandem Meetings: Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR and published in the American Journal of Hematology. The study is being conducted across three nationally recognized transplant centers in the United States (City of Hope in Duarte, California; Brigham & Women's Hospital and the Dana-Farber Cancer Institute in Boston; and Northside Medical Center in suburban Atlanta). A second trial, also funded by the NCI award, will formally test the same concept and will include higher risk transplant recipients, who are only partially HLA-matched to their donors. A preliminary study of that transplant population was also recently completed (NCT04060277).

Lindsay A. Rosenwald, M.D., Fortress' Chairman and Chief Executive Officer and Executive Chairman of Helocyte, Inc., said, "This new vaccination paradigm carries the advantage of potentially conveying earlier and more robust CMV immunity to a stem cell transplant recipient, versus vaccinating a HSCT recipient after engraftment has occurred around four weeks following transplant. We are very pleased that this trial is underway and believe that this novel treatment approach could play a significant role in the prevention and treatment of CMV in the future. Triplex is the subject of multiple ongoing, planned clinical trials in the transplant setting and for HIV-1."

Triplex was originally developed by City of Hope, one of the largest and most advanced cancer research and treatment organizations in the United States, and exclusively licensed to Helocyte.

About Triplex

Triplex is a universal (non-HLA-restricted) recombinant Modified Vaccinia Ankara viral vector vaccine engineered to induce a robust and durable virus-specific T cell response to three immuno-dominant proteins [UL83 (pp65), UL123 (IE1), UL122 (IE2)] linked to CMV complications in the post-transplant setting. In completed Phase 1 (see NCT01941056) and Phase 2 (see NCT02506933, NCT03383055) studies, Triplex was found to be well-tolerated and highly immunogenic. Triplex is currently the subject of multiple ongoing clinical trials, including: a Phase 2 evaluation for CMV control in recipients of liver transplant (see NCT06075745); a Phase 1/2 trial for CMV control in pediatric recipients of HSCT (see NCT03354728); a Phase 2 trial for safety and immunogenicity in adults living with HIV and CMV (see NCT05099965); a Phase 2 trial for CMV control in recipients of stem cell transplant in which the stem cell donor is vaccinated with Triplex (see NCT06059391) and a Phase 1 trial of Triplex in combination with a bi-specific CMV/CD19 Chimeric Antigen Receptor (CAR) T cell for the treatment of non-Hodgkin lymphoma (see NCT05432635). Triplex is also the subject of several planned studies, including a Phase 2 trial for CMV control in recipients of kidney transplants. Helocyte additionally entered into an option agreement with City of Hope for exclusive worldwide rights to a novel bispecific CMV/HIV CAR T cell therapy (optionally for use in combination with Triplex), which is currently the subject of a Phase 1 trial in adults living with HIV-1 (see NCT06252402).

About Helocyte

Helocyte is a clinical-stage company developing novel immunotherapies for the prevention and treatment of cancer and infectious diseases, including cytomegalovirus ("CMV") and human immunodeficiency virus ("HIV"). The Centers for Disease Control estimate that 50 to 80 percent of Americans are living with CMV by the age of 40. While the virus is asymptomatic in healthy individuals, it can cause severe and life-threatening disease in those with weakened or uneducated immune systems. Patients undergoing allogeneic stem cell and solid organ transplantation are at particularly high risk of experiencing complications associated with CMV. According to the Center for International Blood and Marrow Transplant Research, there were approximately 9,000 allogeneic (unrelated and related) bone marrow and cord blood transplants performed in the United States in 2023. According to preliminary data from the Organ Procurement and Transplantation Network, there were over 46,000 organ transplants performed in the United States in 2023, comprised primarily of kidney and liver transplant procedures. Helocyte's Triplex vaccine is engineered to induce a robust and durable virus-specific T cell response to control CMV in transplant recipients. While current antiviral therapies have reduced the rate of CMV disease-related mortality in transplant recipients, such treatments have been linked to increased toxicity, delayed immune reconstitution and late onset of CMV. The Helocyte vaccines may also educate the body's innate immune system to fight CMV. For more information, please visit www.helocyte.com.

About Fortress Biotech

Fortress Biotech, Inc. ("Fortress") is an innovative biopharmaceutical company focused on acquiring and advancing assets to enhance long-term value for shareholders through product revenue, equity holdings and dividend and royalty revenue. The company has seven marketed prescription pharmaceutical products and over 20 programs in development at Fortress, at its majority-owned and majority-controlled partners and subsidiaries and at partners and subsidiaries it founded and in which it holds significant minority ownership positions. Such product candidates span six large-market areas, including oncology, rare diseases and gene therapy, which allow it to create value for shareholders. Fortress advances its diversified pipeline through a streamlined operating structure that fosters efficient drug development. The Fortress model is focused on leveraging its significant biopharmaceutical industry expertise and network to further expand the company's portfolio of product opportunities. Fortress has established partnerships with some of the world's leading academic research institutions and biopharmaceutical companies to maximize each opportunity to its full potential, including AstraZeneca, City of Hope, Fred Hutchinson Cancer Center, Nationwide Children's Hospital and Sentynl. For more information, visit www.fortressbiotech.com.

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Fortress Biotech, Inc.

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