What is UCART19?
UCART19 is an allogeneic CAR T-cell product candidate developed for the treatment of CD19-expressing hematological malignancies, which has been engineered using TALEN® gene editing technology. This product candidate was developed by Cellectis before entering into a license agreement with Servier, and is initially being developed in relapsed/refractory acute lymphoblastic leukemia (ALL). Cellectis’ approach with UCART19 was based on the preliminary positive results from clinical trials using autologous CAR T-cells, but bringing the potential to overcome the limitation of the current autologous approach by providing an allogeneic, frozen, off-the-shelf T-cell based medicinal product.
On November 18, 2015, we signed with Servier an amendment to our collaboration agreement. Notably, Servier exercised its option to acquire exclusive worldwide rights to further develop and commercialize UCART19, which is now being jointly developed by Servier and Allogene. Allogene has exclusive rights to UCART19 in the U.S. while Servier retains exclusive rights for all other countries
A medical first
In 2015, in a medical first, two young patients were treated with gene-edited off-the-shelf T-cells. The two infants had leukemia and did not respond to previous treatments, according to a description of their cases published in January 2017 in Science Translational Medicine. This first-in-human application of our TALEN® engineered T-cell product candidate was and still is a landmark in the use of new gene engineering technology and provides encouraging data for a ready-made T-cell strategy that is currently tested in clinical investigations. Great Ormond Street Hospital treated these young patients in 2015 under a special license from the Medicines & Healthcare products Regulatory Agency (MHRA) with Cellectis’ TALEN® gene-edited allogeneic UCART19 product candidate because no other therapies were available for refractory/relapsed acute lymphoblastic leukemia (ALL) following mismatched allogeneic stem cell transplantation.
Encouraging data as a solid proof-of-concept for Cellectis’ leading allogeneic approach
Phase 1 clinical trials started in adult and pediatric patients in the U.K. in June 2016 and in the U.S. in 2017. Since then, several clinical sites opened in France, Belgium, Spain and Japan.
Preliminary results from two Phase 1 studies of UCART19 were presented in December 2018 at the 60th American Society of Hematology (ASH) Annual Meeting and Exposition in San Diego. First-in-human data demonstrated the safety and tolerability of UCART19, resulting in an 82% complete remission rate across the adult and pediatric patient population who received a lymphodepletion regimen consisting of fludarabine, cyclophosphamide and an anti-CD52 monoclonal antibody.