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/refractory B acute lymphoblastic leukaemia (ALL)

Therapeutic area: Cancer - Oncology

Country: Belgium France, UK

Trial details:

• The PALL (Pediatric Acute Lymphoblastic Leukemia) study is a phase 1, open label study designed to evaluate the safety and ability of UCART19 to induce molecular remission defined by MRD negativity atDay 28 to enable allogeneic stem cell transplantation in pediatric patients with high-risk R/R B-ALL. PALL was initiated in the UK in June 2016. Servier is the sponsor of the study. (NCT02808442)

Latest news:

• • On December 12, 2017,  Servier, Pfizer and Cellectis  presented at the 59th American Society of Hematology (ASH) Annual Meeting and Exposition inAtlanta preliminary results from two phase 1 studies of UCART19, an investigational allogeneic anti-CD19CAR T-cell product, in adult and pediatric patients with relapsed or refractory (R/R) CD19-positive B-cellacute lymphoblastic leukemia (B-ALL).
• These first-in-human data demonstrated the safety and tolerabilityof UCART19, resulting in an 83% complete remission rate across the adult and pediatric patient population.
• Results from the PALL (Pediatric Acute Lymphoblastic Leukemia) showed all five children achieved MRD negativity, enabling them to proceed to allogeneic stem cell transplant. Only one Grade 1 cutaneous acute GvHD occurred. No severe neurotoxicity was observed. Cytokine release syndromes were mild in the majority of cases and were all manageable.
• The results have been presented by Waseem Qasim, principal investigator of the PALL study and consultant in pediatric immunology and reader in cell and gene therapy at Great Ormond Street Hospital for Children.
• • On June 20, 2016, Cellectis announced that the first patient has been treated in the Phase I study of UCART19 in pediatric acute B lymphoblastic leukemia (B-ALL) at the University College of London (UCL). This UCART19 clinical trial is sponsored by Servier in close collaboration with Pfizer. The pediatric Phase I is an open label, non-comparative, monocenter study to evaluate the safety and ability of UCART19 to induce molecular remission in pediatric patients with relapsed or refractory CD19 positive B-cell acute lymphoblastic leukemia ahead of planned allogeneic haematopoeitic stem cell transplantation (allo-HSCT). Cellectis will receive a milestone payment from Servier of an undisclosed amount.
• • On December 23, 2015, Cellectis announced the submission of a clinical trial application (CTA) to the Medicines & Healthcare products Regulatory Agency (MHRA) requesting approval to initiate UCART19 First-inHuman clinical investigation in leukemia in the United Kingdom. This study aims to include CD19-positive Acute Lymphoblastic Leukemia (ALL) patients. Other eligibility criteria to enter clinical trials will be assessed by the investigators.

Is general: Yes