Cellectis, a clinical-stage biopharmaceutical company, has submitted an Investigational New Drug (IND) application to the US Food and Drug Administration (FDA) requesting approval to initiate a phase 1 clinical trial for UCART22, Cellectis’ second wholly controlled TALEN gene-edited product candidate, for the treatment of B-cell acute lymphoblastic leukemia (B-ALL) in adult patients.

Pending regulatory clearance, Cellectis plans to initiate a phase I clinical trial in the third quarter of 2018. The clinical research will be led by Dr. Nitin Jain, Assistant Professor, and Prof. Hagop Kantarjian, Chairman in the Department of Leukemia and University Chair in Cancer Medicine at The University of Texas MD Anderson Cancer Center in Houston.

“This IND application for UCART22 is an important regulatory milestone for the Company,” said Stephan Reynier, chief regulatory and compliance officer, Cellectis. “The first ever FDA approval for a CAR T-cell therapy, directed against CD19, for pediatric and young patients with R/R B-ALL occurred in 2017. However, further CART approaches are needed, as some limitations of the CD19-CART treatment appear to be due to the expansion of CD19-negative leukemia clones. The UCART22 product candidate will be evaluated in relapsed or refractory CD22 B-ALL, including relapses after CD19 CAR-T administration.”

Acute lymphoblastic leukemia (ALL) is a rapidly progressing form of leukemia that is characterized by the presence of a large number of immature white blood cells in the blood and bone marrow. In 2016, an estimated 6,590 new cases were diagnosed in the US, with over 1,400 deaths due to ALL.  Approximately 85 per cent of ALL cases involve precursor B-cells (B-ALL).

UCART22 is an allogeneic, off-the-shelf gene-edited T-cell product candidate designed for the treatment of B-ALL. Like CD19, CD22 is a cell surface antigen expressed from the pre B-cell stage of development through mature B-cells and CD22 expression occurs in more than 90 percent of patients with B-ALL.

“Given the high unmet medical need for patients who suffer from B-ALL, filing the IND is the first vital step to potentially creating a treatment to be manufactured on an industrial scale, allowing these patients to get the help that they need much faster,” added Prof. Stéphane Depil, senior vice president research & development and chief medical officer. “We are dedicated to making this a reality as soon as possible and look forward to hitting the ground running with the clinical trial once we obtain regulatory clearance.”

The manufacturing process of Cellectis’ allogeneic CAR T-cell product line, Universal CARTs or UCARTs, yields frozen, off-the-shelf, non-alloreactive engineered CAR T-cells. UCARTs are meant to be readily available CAR T-cells for a large patient population. Their production is industrialized with defined pharmaceutical release criteria.