December 16, 2016
By Alex Keown, BioSpace.com Breaking News Staff
NEW YORK – Cellectis is ending 2016 on a roll. The company is closing out the year with a series of successful runs of its Phase I candidate for the treatment of acute myeloid leukemia (AML) and blastic plasmacytoid dendritic cell neoplasm (BPDCN), UCART123. The runs met cGMP standards.
UCART123 is an engineered T-cell product candidate that targets CD123, an antigen that is located on CD123-expressing leukemic cells in AML as well as leukemic and other tumoral cells in BPDCN.
Cellectis said it plans to file an Investigational New Drug Application with the U.S. Food and Drug Administration.
Dr. André Choulika, chief executive officer of Cellectis, told BioSpace that barring any unforeseen problems, Cellectis could begin clinical trials in the early part of 2017. If the trials begin, he said Cellectis hopes to see “some safety data” and also have “a sense of efficacy.”
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 U. S. alone, there are an estimated 19,950 new AML cases per year and 10,430 deaths per year.
BPDCN is a rare and aggressive disease of the bone marrow and blood cells. It can also affect skin and lymph nodes.
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 term ‘universal’ for the U of UCARTs means that it is one product for all patients within a therapeutic indication. This is to differentiate from autologous CART that are more personalized medicine, one product made for each patient,” Choulika said in an email. “Our UCART product candidates can, however, target several types of tumor cells given the fact that they do present the appropriate tumor associated antigen the CAR can target, such as CD123 for UCART123. We believe they will be usable by all patients adapted to the product.”
UCART123 is Cellectis first wholly-owned product, one that could lead to additional designations if it is ultimately approved. Choulika said 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.”
One of those programs Cellectis is developing jointly with another organization is UCART19 used in treatment for leukemias and lymphomas. In February 2014, Cellectis signed an agreement with Servier to develop UCART19. The agreement also included research, development, and potentially the commercialization of five other product candidates targeting solid tumors.
In addition to the deal with Servier, Cellectis and Pfizer partnered last year to develop CAR-T therapies.
While Cellectis is using gene editing techniques to develop treatments for cancers, the company is also able to use similar techniques in crop science. While genetically modified organisms (GMOs) have their critics, Choulika said gene editing techniques for crops represent a new level of sophistication for agriculture beyond chemical or radiation induced mutagenesis.
“Gene editing expedites this long and uncertain process to a higher refined sophistication. Without putting any foreign materials in the plant, we can rapidly and safely bring what nature has best to offer for human nutrition without losing centuries of breeding and the gains in yields,” Choulika said.
