Dresden, Germany, 09 March 2020. GEMoaB, a biopharmaceutical company focused on the development of next generation immunotherapies for hard-to-treat cancers, today announced that it has apheresed the first patient in a Phase IA study with UniCAR-T-CD123, the lead product candidate from its proprietary UniCAR cellular immunotherapy platform. UniCAR has been designed to ensure excellent control over the universal CAR-T effector cell through a rapidly switchable on/off capability. This is combined with high flexibility to effectively target tumor antigens of choice by re-directing and activating CAR-T effector cells through soluble adapters termed Targeting Modules (TMs). UniCAR-T-CD123 is investigated in late stage, relapsed/refractory Acute Leukemias expressing the CD123 antigen.
The Phase IA study includes patients with CD123 positive relapsed/refractory Acute Myeloid Leukemia (AML) post standard treatment as well as CD123 positive relapsed/refractory Acute Lymphoblastic Leukemia (ALL) post CD19-targeting therapies and will examine the feasibility, safety and potential efficacy of the combined application of a single dose of UniCAR-T and the continuous infusion of the CD123 specific Targeting Module TM123. “The ability to rapidly switch on and off the CAR-T effector cells of our UniCAR platform and thereby tightly control their activity may help to overcome many of the limitations that current CAR-T therapies face, especially when targeting less differentially expressed antigens and solid tumors”, said Prof. Dr. Gerhard Ehninger, GEMoaB’s co-founder and Chief Medical Officer. ”The start of the first UniCAR clinical study therefore is not only an important milestone for our company but could also lead to an important advancement in cellular immunotherapy beyond targeting CD19 and BCMA.” According to Prof. Bob Löwenberg, Department of Hematology at Erasmus University Medical Center Rotterdam, The Netherlands, the study could be an important step in the research efforts to improve patient outcomes in relapsed/refractory Acute Leukemias, which belong to the most difficult to treat blood cancers. “Despite recent advances, patients with acute leukemia who progress after currently available treatment approaches, generally have a notoriously poor prognosis with only very limited options. This trial will contribute to the development of a safe and effective cellular immunotherapy against the validated target antigen CD123 in these deadly diseases and potentially offer an alternative to allogeneic hematopoietic stem cell transplants in the future.”
ABOUT THE UNICAR-T-CD123 STUDY
This first-in-human phase I study is an open-label, non-randomized, dose-finding study designed to evaluate the safety and activity of UniCAR-T-CD123 in up to 16 CD123 positive patients with relapsed/refractory Acute Leukemias. Its purpose is to determine the maximum tolerated dose (MTD) as well as Dose limiting toxicities (DLT) of the combined application of a single dose of UniCAR-T and the continuous infusion of TM123 over 25 days. Application will follow post salvage therapy and lymphodepletion. The study will also investigate persistence of UniCAR-T cells over time as well as the ability to rapidly switch UniCAR-T cells on and off in case of side effects through stopping TM infusion. The study will take place at selected Phase I, Acute Leukemia and CAR-T experienced University centers in Germany. The study is jointly conducted with Cellex Patient Treatment and supported by a grant from the German Federal Ministry for Education and Research (project “TurbiCAR”). To learn more about the trial, please visit clinicaltrials.gov.
GEMoaB is developing a rapidly switchable universal CAR-T platform, UniCAR, to improve the therapeutic window and increase efficacy and safety of CAR-T cell therapies in more challenging cancers, including solid tumors. Standard CAR-T cells depend on the presence and direct binding of cancer antigens for activation and proliferation. An inherent key feature of the UniCAR platform is a rapidly switchable on/off mechanism (less than 4 hours after interruption of TM supply) enabled by the short pharmacokinetic half-life and fast internalization of soluble adaptors termed targeting modules (TMs). These TMs provide the antigen-specificity to activate UniCAR gene-modified T-cells (UniCAR-T) and consist of a highly flexible antigen-binding moiety, linked to a small peptide motif recognized by UniCAR-T.
GEMoaB is a privately-owned, clinical-stage biopharmaceutical company that is aiming to become a globally leading biopharmaceutical company. By advancing its proprietary UniCAR, RevCAR and TCE platforms, the company will discover, develop, manufacture and commercialize next generation immunotherapies for the treatment of cancer patients with a high unmet medical need.  GEMoaB has a broad pipeline of product candidates in pre-clinical and clinical development for the treatment of hematological malignancies as well as solid tumors. Its clinical stage assets GEM333, a T-cell engaging bispecific antibody (TCE) with binding specificity to CD33 in relapsed/refractory AML, and GEM3PSCA, a TCE with binding specificity to PSCA for the treatment of castrate-resistant metastatic prostate cancer and other PSCA expressing late stage solid tumors, are currently investigated in Phase I studies and globally partnered with Bristol-Myers Squibb/Celgene. A Phase IA dose-finding study of the first UniCAR asset, UniCAR-T-CD123 for treatment of relapsed/refractory AML and ALL has been initiated, UniCAR-T-PSMA against CRPC and other PSMA-expressing late-stage solid tumors, is planned to be tested in a Phase I study initiated by H2 2020.
Manufacturing expertise, capability and capacity are key for developing cellular immunotherapies for cancer patients. GEMoaB has established a preferred partnership with its sister company Cellex in Cologne, a world leader in manufacturing hematopoietic blood stem cell products and a leading European CMO for CAR-T cells, co-operating in that area with several large biotech companies.
More information can be found at www.gemoab.com.
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