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Mustang Bio's MB-102 (CD123 CAR) CAR T Therapy Achieves Complete Response in Acute Myeloid Leukemia and Blastic Plasmacytoid Dendritic Cell Neoplasm in Phase 1 Clinical TrialTrial investigators at City of Hope announce first-reported complete response from a CAR T therapy in a BPDCN patient, additional complete response achieved in AML Investigators found infusions of MB-102 are safe and well tolerated Data presented by City of Hope in oral session at ASH Annual Meeting NEW YORK, Dec. 11, 2017 (GLOBE NEWSWIRE) -- Mustang Bio, Inc. (“Mustang”) (NASDAQ:MBIO), a Fortress Biotech (NASDAQ:FBIO) Company focused on the development of novel immunotherapies based on proprietary chimeric antigen receptor engineered T cell (“CAR T”) technology, today announced that investigators at City of Hope have reported that Mustang’s MB-102 (CD123 CAR) CAR T therapy is safe and well tolerated and achieved the first-ever complete response (CR) from a CAR T therapy in blastic plasmacytoid dendritic cell neoplasm (BPDCN), as well as a CR in acute myeloid leukemia (AML), in an ongoing Phase 1 clinical trial (NCT02159495). The data were presented by City of Hope today in an oral session at the 59th American Society of Hematology (ASH) Annual Meeting. Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, “According to investigators at City of Hope, these data demonstrate MB-102’s potential to be a safe, well tolerated and effective CAR T therapy that can achieve complete disease response. In addition, MB-102’s promising anti-leukemic activity in both AML and BPDCN supports further evaluation in clinical trials in transplant eligible and ineligible patients. We are thrilled to report that our CAR Ts have now achieved complete responses in three disease areas, MB-102 in AML and BPDCN, and our MB-101 IL13Ra2-specific CAR T in glioblastoma, which was published in December 2016 in the New England Journal of Medicine.” Elizabeth Lihua Budde, M.D., Ph.D., assistant professor in the department of Hematology & Hematopoietic Cell Transplantation at City of Hope and principal investigator for the Phase 1 trial, said, “Current treatment options in AML are associated with low rates of complete response and limited progression to allogeneic hematopoietic stem cell transplantation. Moreover, BPDCN is a rare and incurable blood cancer with no standard of care. CD123 is overexpressed in both AML and BPDCN, making it an attractive target in these diseases, which have clear unmet therapeutic needs. We are encouraged by these interim data that demonstrate MB-102’s potential to be a new or improved treatment option in BPDCN and AML, and look forward to continuing to evaluate the clinical benefits of MB-102 in our ongoing Phase 1 clinical trial.” Key Efficacy and Safety Findings This single center, first-in-human Phase 1 dose-escalation clinical trial is evaluating the safety and activity of escalating doses of MB-102 in patients with relapsed or refractory AML (cohort 1) and BPDCN (cohort 2). Patients receive a single dose of MB-102 with an option for a second infusion if they continue to meet safety and eligibility criteria and still have CD123+ disease. To date, 14 patients have been enrolled and seven have been treated (six with AML, one with BPDCN) in this first in-human trial for AML and BPDCN patients using a CD123 CAR T therapy. In the AML cohort, two patients were treated at dose level 1 (50M CAR+ T). Trial investigators reported that one achieved a morphologic leukemic-free state at day 28 post-infusion. Four patients received dose level 2 (200M CAR+ T), with a CR observed at day 28 in one patient, and a CR with incomplete blood count recovery demonstrated at day 28 in a second patient. Both patients proceeded to a second allogeneic hematopoietic stem cell transplantation. In the BPDCN cohort, ne patient received a single dose of 100M CAR+ T and achieved a CR at day 28, which lasted at least 60 days, according to investigators. Of note, this patient had previously experienced disease progression following five cycles of treatment with a CD123-targeted recombinant fusion protein. Investigators found MB-102 infusions of up to 200M CAR T cells were safe, with no graft-versus-host disease, myeloablative effects, neurologic toxicity or dose-limiting toxicities. Adverse events (AEs) included: cytokine release syndrome (six grade 1, one grade 2), neurotoxicity (dizziness: one grade 1, two grade 2; headache: five grade 1, two grade 2; somnolence: one grade 1, two grade 2), three cases of infection (lung infection: two, other: one). The most common = grade 3 AEs included lymphopenia (seven), thrombocytopenia (seven) and febrile neutropenia (six). About Acute Myeloid Leukemia CD123 is overexpressed on AML blasts and leukemic stem cell-enriched cell subpopulations compared to normal hematopoietic stem cells and myeloid progenitors, making CD123 an attractive target for T cell-based adoptive immunotherapy. About Blastic Plasmacytoid Dendritic Cell Neoplasm About MB-102 (CD123 CAR) MB-102 has demonstrated anti-AML activity in preclinical studies (Mardiros, Blood 2013), and is currently being evaluated in a Phase 1 clinical trial in AML and BPDCN at City of Hope (NCT02159495). 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