February 7, 2017
By Alex Keown, BioSpace.com Breaking News Staff
NEW YORK – Gene editing company Cellectis is moving forward with its engineered T-cell product candidate UCART123. On Monday, the company announced it received an Investigational New Drug approval from the U.S. Food and Drug Administration (FDA) to conduct Phase I clinical trials.
Cellectis will use UCART123 for the treatment of acute myeloid leukemia (AML) and blastic plasmacytoid dendritic cell neoplasm (BPDCN). The manufacturing process for Cellectis’ allogeneic CAR T-cell product line, Universal CARTs or UCARTs, yields frozen, off-the-shelf, engineered CAR T-cells. UCARTs are meant to be readily available CAR T-cells for a large patient population. Their production can be industrialized and standardized with defined pharmaceutical release criteria. The FDA IND approval marks the first off-the-shelf gene-edited CAR T-cell product candidate the agency has approved for clinical trials. Cellectis intends to initiate Phase 1 trials in the first half of 2017, the company said.
Other companies are developing autologous CAR-T therapies, including Kite and Novartis . Both companies are looking to file for approval of their therapies late this year, but Cellectis thinks its off-the-shelf therapies will be a cheaper option for patients due to easier manufacturing methods.
Acute myeloid leukemia is characterized by uncontrolled proliferation and accumulation of leukemic blasts in the bone marrow, peripheral blood and occasionally in other tissues. These cells disrupt normal hematopoiesis and rapidly cause bone marrow failure and death. In the United States alone, there are an estimated 19,950 new AML cases per year and 10,430 estimated deaths per year. BPDCN is a very rare and aggressive hematological malignancy that is derived from plasmacytoid dendritic cell precursors. BPDCN is a disease of bone marrow and blood cells but also often affects skin and lymph nodes.
The approval fulfills expectations that Dr. André Choulika, chief executive officer of Cellectis, told BioSpace about in December. UCART123 is Cellectis first wholly-owned product, a product that could lead to additional designations if it is ultimately approved. Choulika told BioSpace in December that the ultimate value of UCART123 is “in the product and its clinical benefit to the patient.” But, he said it has the potential to be used for other indications for AML and BPDCN.
“UCART123 is the first in a list of a strong self-owned product pipeline which will follow UCARTCS1, UCART22, UCART38 and others in a series of therapeutic indications,” Choulika said. “Beyond that, we strongly believe in all of the product candidates we develop and work hard with our partners or on a stand-alone basis to push them into clinic if preclinical data show promising results.”
The UCART123 clinical program for BPDCN will be led, at the MD Anderson Cancer Center.
“Cellectis’ allogeneic UCART products have the potential to create an important shift with regard to availability, and cost-effectiveness, to make these therapies widely accessible to patient population across the world,” Loan Hoang-Sayag, Cellectis chief medical officer, said in a statement.
UCART123 is not the only UCART program Cellectis has in development. The company is also developing UCART19 as a therapy for leukemias and lymphomas. In February 2014, Cellectis signed an agreement with Servier and Pfizer to develop UCART19. The agreement also included research, development, and potentially the commercialization of five other product candidates targeting solid tumors.
