Clinical Trials

Date: 2017-12-12

Type of information: Presentation of results at a congress

phase: 1

Announcement: presentation of results at the 59th American Society of Hematology (ASH) Annual Meeting in Orlando

Company: Cellectis (France) Servier (France) Pfizer (USA - NY)

Product: UCART19

Action mechanism:

cell therapy/immunotherapy product/gene therapy/CAR-T cell therapy. UCART19 is a potential best-in-class allogeneic TALEN® gene edited T-cell product for treatment of CD19 expressing hematological malignancies, initially developed in Chronic lymphocytic leukemia (CLL) and Acute lymphoblastic leukemia (ALL). Engineered allogeneic CD19 CAR T-cells currently stand out as a real therapeutic innovation for treating various types of leukemia and lymphoma. Cellectis’ approach with UCART19 is based on the preliminary positive results from clinical trials using products based on the CAR technology and has the potential to overcome the limitation of the autologous current approach by providing an allogeneic frozen, “off the shelf” T-cell based medicinal product. On November 18, 2015 Servier exercised its worldwide option to license UCART19 under a collaboration agreement. Cellectis also entered into a global development and commercialization collaboration with Pfizer on UCART19. According to their recent agreement, Cellectis will hand over the clinical development of UCART19 to Servier and their US partner Pfizer. Due to the early exercise, Cellectis is no longer responsible for funding the UCART19 Phase I clinical program.

Disease: relapsed or refractory (R/R) CD19-positive B-cell acute lymphoblastic leukemia (B-ALL)

Therapeutic area: Cancer - Oncology

Country: France, UK, USA

Trial details:

The CALM study (UCART19 in Advanced Lymphoid Malignancies) is an open label, dose-escalation study designed to evaluate the safety, tolerability and anti-leukemic activity of UCART19 in adult patients with R/R B-ALL. The study was initiated in the UK in August 2016. (NCT02746952)

Latest news:

• On December 12, 2017, Servier, Pfizer and Cellectis presented at the 59th American Society of Hematology (ASH) Annual Meeting and Exposition in Atlanta preliminary results from two phase 1 studies of UCART19, an investigational allogeneic anti-CD19 CAR T-cell product, in adult and pediatric patients with relapsed or refractory (R/R) CD19-positive B-cell acute lymphoblastic leukemia (B-ALL). These first-in-human data demonstrated the safety and tolerability of UCART19, resulting in an 83% complete remission rate across the adult and pediatric patient population.
Results from the CALM Trial: Five out seven patients treated achieved molecular remission at Day 28 post UCAR19. Molecular remission is defined by negative minimal residual disease (MRD). MRD is a measurement of the number of residual leukemic cells that remain after treatment. Only one Grade 1 cutaneous acute graft versus host disease (GvHD) occurred. No severe neurotoxicity was observed. Cytokine release syndromes (CRS) were mild and manageable except in one patient treated with UCART19 at the first dose level, who developed CRS Grade 4 and neutropenic sepsis leading to death at Day 15.