First AML Patient Dosed in Phase 1 Trial of Cellectis’ CAR T-cell Therapy, UCART123

First AML Patient Dosed in Phase 1 Trial of Cellectis’ CAR T-cell Therapy, UCART123
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Cellectis has started dosing participants in its Phase 1 trial assessing the safety and clinical activity of an updated version of UCART123, its CAR T-cell therapy for patients with relapsed or refractory acute myeloid leukemia (AML), the company has announced.

The AMELI-01 trial (NCT03190278) is part of an investigational new drug application (IND) that the U.S. Food and Drug Administration (FDA) approved last July, allowing Cellectis to test a new version of the UCART123 construct, and an amended and more efficient protocol.

This new application replaces the company’s previous IND, under which a prior version of UCART123 was being tested in two Phase 1 trials: one called AML123 (NCT03190278) for AML, and the other called ABC123 (NCT03203369) for blastic plasmacytoid dendritic cell neoplasm (BPDCN).

After the death of the first BPDCN patient, which was caused by severe cytokine release syndrome — a common complication of immunotherapies — the trials were suspended by the FDA in September 2017, and terminated in June 2019, even after the FDA agreed to lift the clinical hold on the trials with an amended protocol. AMELI-01 is now replacing AML123 as the first U.S. trial to test UCART123.

“Cellectis invented and has pioneered the allogeneic [donor-derived] approach for many years,” André Choulika, Cellectis chairman and CEO, said in a press release. “Being a leader of the space, it’s important for us to consistently improve our technology and manufacturing expertise to remain at the forefront.”

“With this new IND, we are delivering on our promise of continual innovation in order to advance the efforts of our clinical trials. We hope that with this optimized production process, our UCART123 product candidate will be well equipped to help people living with AML,” Choulika added.

Chimeric antigen receptor T-cell therapy, more commonly known as CAR T-cell therapy, normally involves collecting a patient’s immune T-cells and modifying them in the lab in such a way that they start producing a chimeric antigen receptor (CAR), a protein construct that helps these cells recognize and target specific cancer proteins.

UCART123 is a CAR T-cell therapy designed to target the CD123 molecule found at the surface of AML cells. Unlike more common versions of CAR T-cells — which sometimes fail to be produced because not enough T-cells are harvested from the patient — UCART123 uses immune T-cells harvested from unrelated donors. In addition to bypassing the problem of low T-cell numbers, its donor-derived nature also allows the therapy to be more readily available.

AMELI-01 is a Cellectis-sponsored open-label, dose-escalation, and dose-expansion study that is currently underway at four clinical sites in the U.S. Two of these sites, the MD Anderson Cancer Center in Texas — where the first patient was dosed — and the H. Lee Moffitt Cancer Center & Research Institute in Florida, are currently recruiting participants. More information can be found here.

The study’s main goal is to assess the safety and tolerability of a single infusion of increasing doses of UCART123 and determine the maximum tolerated dose, which will then be administered to a higher number of patients in the study’s dose-expansion phase.

Patricia holds her Ph.D. in Cell Biology from University Nova de Lisboa, and has served as an author on several research projects and fellowships, as well as major grant applications for European Agencies. She also served as a PhD student research assistant in the Laboratory of Doctor David A. Fidock, Department of Microbiology & Immunology, Columbia University, New York.
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Inês holds a PhD in Biomedical Sciences from the University of Lisbon, Portugal, where she specialized in blood vessel biology, blood stem cells, and cancer. Before that, she studied Cell and Molecular Biology at Universidade Nova de Lisboa and worked as a research fellow at Faculdade de Ciências e Tecnologias and Instituto Gulbenkian de Ciência. Inês currently works as a Managing Science Editor, striving to deliver the latest scientific advances to patient communities in a clear and accurate manner.
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Patricia holds her Ph.D. in Cell Biology from University Nova de Lisboa, and has served as an author on several research projects and fellowships, as well as major grant applications for European Agencies. She also served as a PhD student research assistant in the Laboratory of Doctor David A. Fidock, Department of Microbiology & Immunology, Columbia University, New York.
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