FDA clears Servier and Pfizer for clinical development of UCART19 for acute lymphoblastic leukemia

Servier and Pfizer Inc. have received Investigational New Drug (IND) clearance from the U.S. Food and Drug Administration (FDA) to proceed with the clinical development of UCART19, an innovative gene-edited therapy for the treatment of relapsed/refractory acute lymphoblastic leukemia (ALL).

Groundbreaking Gene-Edited Therapy

UCART19, a chimeric antigen receptor (CAR) T-cell therapy, targets CD19-expressing hematological malignancies and is crafted using Talen gene-editing technology. This allogeneic CAR T-cell product is primarily being developed for acute lymphoblastic leukemia and is currently in a phase 1 trial.

Positive Clinical Outcomes Spur Development

The decision by the FDA was influenced by preliminary positive results from earlier clinical studies involving autologous products based on CAR technology. These findings have paved the way for the further exploration of UCART19’s potential in a larger clinical setting.

Statements from the Collaborators

Dr. Patrick Thérasse, Director of Clinical Development Oncology at Servier, expressed enthusiasm about the approval, noting, “We are very pleased that Servier’s first IND approval has been granted for such an innovative approach as allogeneic CAR T therapy. B-ALL is a devastating disease and this study is key to gaining greater insight into the efficacy and safety profile of this new immuno-oncology approach in patients with B-ALL.”

Barbara Sasu, Vice President of CAR T Research at Pfizer, also commented on the clearance, stating, “Pfizer is excited by the potential of this investigational CAR T approach to treating ALL and other B-Cell malignancies. We are looking forward to having the opportunity to investigate this approach in the U.S.”

Expanding Clinical Trials

Following the IND clearance, Servier is set to expand the UCART19 clinical trial to include additional centers, such as the prestigious MD Anderson Cancer Center in Houston. The CALM trial, an open-label, dose-escalation study initiated in August 2016 in the United Kingdom, evaluates the safety, tolerability, and antileukemic activity of UCART19.

Global Rights and Future Prospects

While Pfizer has obtained exclusive rights to develop and market UCART19 in the U.S., Servier retains exclusive rights for the treatment outside of the U.S. This strategic partnership underscores the global potential of UCART19 as a transformative treatment for lymphoblastic leukemia.

The FDA’s approval for the further development of UCART19 marks a significant step forward in the fight against acute lymphoblastic leukemia. By harnessing cutting-edge gene-editing technology, UCART19 could potentially redefine treatment paradigms and offer new hope to patients suffering from this aggressive form of leukemia.