FDA rules studies of its Pfizer-partnered drug can be resumed
Servier's bid for a share of the emerging market for cell-based cancer immunotherapies advanced this week after the FDA approved its phase I trial of leukaemia therapy UCART19.
The French pharma company entered the race to develop chimeric antigen receptor therapies (CAR-T) for cancer after it acquired exclusive rights to UCART19 from Cellectis in 2015.
Servier is co-developing the drug with Pfizer, which has US development and commercialisation rights, and already has a phase I trial of the immunotherapy ongoing at Kings College Hospital in the UK involving patients with relapsed or refractory CD19-positive B-cell acute lymphoblastic leukemia (B-ALL).
The FDA has given the go-ahead to start a phase I trial of UCART19 in the same patient population, and Servier says the CALM study will now be expanded to include several centres in the US, including the MD Anderson Cancer Center in Texas.
Cellectis specialises in the development of ‘off-the-shelf’ CAR-T candidates, which differ from rival CAR-Ts from the likes of Novartis and Kite Pharma that use autologous white blood cells harvested from patients which are then modified to boost their tumour-killing activity and re-infused.
The biotech is trying to develop gene-edited donor (allogeneic) cells that fulfil the same purpose, skipping the harvesting stage. It got a green light from the FDA for another allogeneic CAR-T - UCART123 - last month.
The news comes at a pivotal time for CAR-T, shortly after one of the early leaders - Juno Therapeutics – was forced to give up on its front-runner candidate for B-ALL after patient deaths in its pivotal clinical trial liked to swelling in the brain.
And Cellectis has also faced possible toxicity issues with its allogeneic approach. Early results from the CALM trial have already shown that UCART19 can provide life-saving benefits in two infants, but one developed graft-versus-host disease (GVHD).
On the plus side for the emerging therapeutic category, Kite Pharma and Novartis both have late-stage programmes still on course to be filed for approval this year.
Kite's KTE-C19 candidate for non-Hodgkin lymphoma (NHL) has cleared a phase II trial and could be filed for approval in the US later this month, with an EU filing before year-end, while Novartis has indicated it plans to submit a marketing application for its CD19-targeted therapy CTL109 for B-ALL in the US very shortly.