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
Fortress Biotech (FBIO) has initiated dosing in a Phase 2 clinical trial evaluating Triplex, a cytomegalovirus (CMV) vaccine, in stem cell donors. The trial aims to reduce CMV events in patients undergoing hematopoietic stem cell transplantation (HSCT).
The multicenter, placebo-controlled study is funded by the National Cancer Institute and builds upon encouraging pilot study results. Triplex works by inducing CMV-specific immune responses in donors, which is then transferred to transplant recipients through adoptive immunity.
CMV reactivation affects 60-70% of CMV-seropositive HSCT recipients, with primary infection occurring in 20-30% of CMV-seronegative recipients. The trial is being conducted across three major transplant centers in the United States. A second NCI-funded trial is planned for higher-risk transplant recipients with partial HLA matches.
Fortress Biotech (FBIO) ha iniziato a somministrare il farmaco in uno studio clinico di Fase 2 che valuta Triplex, un vaccino contro il citomegalovirus (CMV), nei donatori di cellule staminali. Lo studio mira a ridurre gli eventi legati al CMV nei pazienti sottoposti a trapianto di cellule staminali ematopoietiche (HSCT).
Lo studio multicentrico, controllato con placebo, è finanziato dal National Cancer Institute e si basa su risultati incoraggianti di uno studio pilota. Triplex agisce inducendo risposte immunitarie specifiche per il CMV nei donatori, che vengono poi trasferite ai riceventi del trapianto attraverso l'immunità adottiva.
La riattivazione del CMV colpisce il 60-70% dei riceventi di HSCT positivi per CMV, con un'infezione primaria che si verifica nel 20-30% dei ricevitori negativi per CMV. Lo studio si svolge in tre importanti centri di trapianto negli Stati Uniti. Un secondo studio finanziato dal NCI è previsto per i riceventi di trapianto ad alto rischio con corrispondenze parziali di HLA.
Fortress Biotech (FBIO) ha iniciado la dosificación en un ensayo clínico de Fase 2 que evalúa Triplex, una vacuna contra el citomegalovirus (CMV), en donantes de células madre. El ensayo tiene como objetivo reducir los eventos de CMV en pacientes que se someten a un trasplante de células madre hematopoyéticas (HSCT).
El estudio multicéntrico, controlado con placebo, es financiado por el Instituto Nacional del Cáncer y se basa en resultados alentadores de un estudio piloto. Triplex funciona al inducir respuestas inmunitarias específicas contra el CMV en los donantes, que luego se transfieren a los receptores del trasplante a través de la inmunidad adoptiva.
La reactivación del CMV afecta al 60-70% de los receptores de HSCT seropositivos para CMV, y la infección primaria ocurre en el 20-30% de los receptores seronegativos para CMV. El ensayo se lleva a cabo en tres importantes centros de trasplante en Estados Unidos. Se planea un segundo ensayo financiado por el NCI para receptores de trasplante de mayor riesgo con coincidencias parciales de HLA.
포트리스 바이오텍 (FBIO)가 줄기세포 기증자에서 사이토메갈로바이러스(CMV) 백신인 Triplex를 평가하는 2상 임상 시험의 용량 투여를 시작했습니다. 이 시험은 조혈모세포 이식(HSCT)을 받는 환자에서 CMV 관련 사건을 줄이는 것을 목표로 합니다.
이 다기관, 위약 대조 연구는 국립 암 연구소(NCI)에서 자금을 지원받았으며, 고무적인 파일럿 연구 결과를 기반으로 합니다. Triplex는 기증자에서 CMV 특이적 면역 반응을 유도하여, 이를 통해 이식 수혜자에게 면역을 전달하는 방식으로 작용합니다.
CMV 재활성화는 CMV 양성 HSCT 수혜자의 60-70%에 영향을 미치며, CMV 음성 수혜자에서는 20-30%에서 1차 감염이 발생합니다. 이 시험은 미국 내 3개의 주요 이식 센터에서 진행되고 있습니다. NCI에서 자금을 지원받아 HLA 일치도가 부분적인 고위험 이식 수혜자를 위한 두 번째 임상 시험이 계획되고 있습니다.
Fortress Biotech (FBIO) a commencé l'administration dans un essai clinique de Phase 2 évaluant Triplex, un vaccin contre le cytomégalovirus (CMV), chez les donneurs de cellules souches. L'essai vise à réduire les événements liés au CMV chez les patients subissant une transplantation de cellules souches hématopoïétiques (HSCT).
L'étude multicentrique, contrôlée par placebo, est financée par l'Institut National du Cancer et s'appuie sur des résultats préliminaires encourageants. Triplex fonctionne en induisant des réponses immunitaires spécifiques au CMV chez les donneurs, qui sont ensuite transférées aux receveurs de transplantation par le biais de l'immunité adoptive.
La réactivation du CMV touche 60 à 70 % des receveurs HSCT séropositifs au CMV, tandis qu'une infection primaire se produit chez 20 à 30 % des receveurs séro-négatifs. L'essai est mené dans trois grands centres de transplantation aux États-Unis. Un deuxième essai financé par le NCI est prévu pour les receveurs de transplantation à haut risque avec des correspondances HLA partielles.
Fortress Biotech (FBIO) hat mit der Dosierung in einer klinischen Phase-2-Studie begonnen, die Triplex, einen Cytomegalovirus (CMV)-Impfstoff, bei Stammzellspendern bewertet. Die Studie zielt darauf ab, CMV-Ereignisse bei Patienten zu reduzieren, die sich einer hämatopoetischen Stammzelltransplantation (HSCT) unterziehen.
Die multizentrische, placebo-kontrollierte Studie wird vom National Cancer Institute finanziert und baut auf ermutigenden Ergebnissen aus Pilotstudien auf. Triplex wirkt, indem es CMV-spezifische Immunantworten bei Spendern induziert, die dann durch adoptive Immunität auf die Transplantatempfänger übertragen werden.
Die Reaktivierung des CMV betrifft 60–70 % der CMV-positiven HSCT-Empfänger, während eine Primärinfektion bei 20–30 % der CMV-negativen Empfänger auftritt. Die Studie wird an drei großen Transplantationszentren in den Vereinigten Staaten durchgeführt. Eine zweite vom NCI finanzierte Studie ist für Empfänger von Hochrisiko-Transplantationen mit teilweisen HLA-Übereinstimmungen geplant.
- Phase 2 trial initiation represents significant clinical progress
- NCI funding support demonstrates external validation
- Previous pilot study showed encouraging results
- Multiple ongoing and planned clinical trials expand potential applications
- None.
Insights
This Phase 2 trial launch represents a significant milestone in addressing a critical unmet need in stem cell transplantation. The study's innovative approach of vaccinating donors rather than recipients could revolutionize CMV management in HSCT.
The trial's strategic importance is magnified by several key factors:
- The current CMV management landscape relies heavily on antiviral medications, which can impair immune reconstitution. Triplex's approach of transferring pre-existing immunity could provide superior outcomes while reducing antiviral dependency.
- The multicenter trial involves prestigious institutions including City of Hope and Dana-Farber, enhancing credibility and potential for successful execution.
- The NCI funding support validates the scientific merit and reduces financial burden on FBIO.
The market opportunity is substantial, considering:
- Approximately 20,000 allogeneic HSCTs are performed annually in the US
- With 60-70% of CMV-seropositive recipients experiencing reactivation, the addressable patient population is significant
- Current antiviral treatments represent a $500M+ market, suggesting strong commercial potential if Triplex proves successful
The planned expansion into higher-risk, partially HLA-matched transplants and HIV-1 applications demonstrates promising platform potential. However, investors should note that Phase 2 results will be important in validating this novel approach and determining commercial viability.
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
“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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Company Contact:
Jaclyn Jaffe
Fortress Biotech, Inc.
(781) 652-4500
ir@fortressbiotech.com
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6 Degrees
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FAQ
What is the purpose of Fortress Biotech's (FBIO) Phase 2 Triplex vaccine trial?
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What percentage of HSCT recipients are affected by CMV reactivation according to FBIO's data?
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