14 December 2017 - - Suresnes, France-based international pharmaceutical company Servier, New York, New York-based biopharmaceutical and healthcare products specialist Pfizer Inc. (NYSE: PFE), and Paris, France-based gene editing company Cellectis (NASDAQ: CLLS) have released 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 CD19-positive B-cell acute lymphoblastic leukemia (B-ALL), the companies said.
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.
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.
Five out seven patients treated achieved molecular remission, defined by negative minimal residual disease (MRD), a measurement of the number of residual leukemic cells that remain after treatment, at Day 28 post UCAR19.
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 at Day 28 to enable allogeneic stem cell transplantation in pediatric patients with high-risk R/R B-ALL.
Results showed all five children achieved MRD negativity, enabling them to proceed to allogeneic stem cell transplant.
Servier is the sponsor of both studies that are active in Europe and the United States.
UCART19, an allogeneic CAR T-cell product candidate being developed for treatment of CD19-expressing hematological malignancies, is gene edited with Cellectis' Talen technology.
Servier, governed by a non-profit foundation, has an international presence in 148 countries, a turnover of EUR4 bn in 2016, and employs 21,000 people worldwide.
It reinvests 25% of its turnover (excluding generic drugs) in research and development and uses all its profits for development. The company focuses on five areas: cardiovascular, immune-inflammatory and neuropsychiatric diseases, cancers and diabetes, in addition to activities in high-quality generic drugs.
Pfizer discovers, develops, manufactures, and sells healthcare products worldwide. Its global portfolio includes medicines and vaccines as well as consumer health care products.
The company collaborates with health care providers, governments, and local communities to support and expand access to reliable, affordable health care around the world.
Cellectis is focused on developing a new generation of cancer immunotherapies based on gene-edited T-cells (UCART). Using its life-science-focused, pioneering genome engineering technologies, the company's goal is to create innovative products in multiple fields and with various target markets.