The BALLI-01 study, initiated in 2019, has now dosed its first patient with an in-house manufactured CAR T-cell therapy candidate.
Cellectis has dosed the first patient with relapsed or refractory B-cell acute lymphoblastic leukemia (r/r B-ALL) with its allogeneic, in-house manufactured chimeric antigen receptor (CAR) T-cell therapy UCART22.1 The patient has completed the 28-day observation period and no dose-limiting toxicities (DLTs) were observed as of December 14, 2022.
“First dosing of a patient with a product candidate manufactured in-house is a major milestone for Cellectis. UCART22 has been developed to potentially offer a therapeutic alternative for patients with r/r B-ALL, including patients that have relapsed from or unable to receive CD19-directed therapy. The ability to have our manufacturing completely in-house maximizes the chances that eligible patients can be treated without delay,” Mark Frattini, MD, PhD, chief medical officer, Cellectis, said in a statement.1
UCART22 is being evaluated in the phase 1 BALLI-01 clinical trial (NCT04150497), which dosed its first patient with r/r B-ALL with a non-in-house manufactured UCART22 candidate in late 2019.2 The trial reported interim data in December 2021 that showed that the CAR T candidate was well-tolerated in study participants with no DLTs.3
Eleven participants had been administered UCART222 as of October 1, 2021. Out of 6 participants in the fludarabine, cyclophosphamide and alemtuzumab cohort, 2 (at dose levels 1 and 2) had encouraging anti-leukemic activity with blast reductions to less than 5% (0.4% and 0%, respectively) by day 28. Investigators also observed measurable UCART22 expansion and changes in relevant inflammatory cytokines.
“This is a transformational step forward for Cellectis: our in-house manufacturing capabilities would allow us to move product candidates like UCART22 from R&D to development to a finished UCART product on a timeline that would not have been possible working with a contract manufacturer,” Steven Doares, senior vice president, US Manufacturing, added to the statement.1 “We believe that having this capability in-house is a great competitive advantage as it would give us the ability to swiftly version our product candidates as we monitor clinical responses, resulting in what we expect to be the best product possible.”
This dosing reflects Cellectis’ shift to becoming an end-to-end cell and gene therapy company, with manufacturing facilities located in both Raleigh, North Carolina; and Paris, France. BALLI-01 continues to enroll patients with r/r B-ALL.