Cellectis, the first biopharmaceutical company to test an allogeneic CAR-T therapy for cancer, has received protocol amendment approval for its Phase 1 clinical trial of UCART123 product candidate in patients with acute myeloid leukemia (AML). The approval is based on the product’s current safety and tolerability profile.
The UCART123 clinical trial initiated in the first half of 2017 is a Phase 1, open label dose-escalation and dose-expansion study to evaluate the safety, expansion, persistence and clinical activity of UCART123 in patients with relapsed/refractory AML, and patients with newly diagnosed high-risk AML.
UCART123 is a gene edited T-cell investigational drug that targets CD123, an antigen expressed on the surface of leukemic cells in AML. It is the first allogeneic, “off-the-shelf” gene-edited CAR T-cell product candidate that the FDA has approved for clinical trial.
The FDA review period for the protocol amendment has passed and Cellectis’ decision to continue the trial with the amended protocol is following approval from the Institutional Review Board. Main changes to the protocol include:
- Increase of dose levels from 6.25×104 to 2.5×105 UCART123 cells per kilogram body weight, with a capping at 80kg equivalent.
- Decrease of dose limiting toxicities (DLT) observation period from 42 to 28 days post-UCART123 infusion, except for patients with aplastic bone marrow for whom the DLT observation period will remain 42 days.
- Decrease of time interval from 42 days to 28 days between the first and the second patient for UCART123 infusion at each new dose level tested. It will remain 42 days in the case of patients with aplastic anemia.
In order to accelerate the product’s clinical development, the trial will also be conducted at a second centre, the MD Anderson Cancer Center in Houston, Texas under the expertise of Prof. Hagop Kantarjian and Dr Naveen Pemmaraju. At present, the trial is conducted only at Weill Cornell Medicine with Prof. Gail J. Roboz as the principal investigator.
Prof. Stéphane Depil, Senior VP of R&D and CMO of Cellectis commented: “This amendment approval for Cellectis’ UCART123 protocol is an important step in the progression of our study, and opening another clinical site at MD Anderson – one of the world’s most premier cancer centers – puts the Company on solid ground to help as many AML patients as possible with this innovative new therapy.”
Source: Cellectis: Approval of UCART123 Amendment in AML to accelerate clinical development. Press Release