The US Food and Drug Administration (FDA) has lifted the clinical hold on studies of BPX-501 in the US.
BPX-501 is an adjunct T-cell therapy intended to be given after allogeneic hematopoietic stem cell transplant (HSCT).
The product is being tested in phase 1/2 trials of children and adults with hematologic disorders.
The FDA placed all US trials of BPX-501 on hold earlier this year due to 3 cases of encephalopathy that were considered possibly related to BPX-501.
The hold did not affect a registrational trial of BPX-501—known as BP-004—that is underway in Europe.
Now, the FDA has lifted the hold on the US trials after discussions with Bellicum Pharmaceuticals, Inc., the company developing BPX-501.
Bellicum agreed to amend study protocols to include guidance on monitoring and management of neurologic adverse events. The company said it will be working with US clinical sites to resume patient recruitment in BPX-501 trials based on the amended protocols.
BPX-501 is intended to speed immune reconstitution, fight infection, and enhance the graft-versus-leukemia effect of HSCT while also minimizing graft-vs-host disease (GVHD) and other T-cell-mediated transplant complications.
BPX-501 contains a safety switch, CaspaCIDe®, that can be activated with the drug rimiducid. The drug can be used to eliminate alloreactive BPX-501 T cells if uncontrollable GVHD or other T-cell-mediated complications occur.
BPX-501 and rimiducid have demonstrated efficacy in the BP-004 trial. Results from this trial have been presented at the EBMT Annual Meeting in 2016 (abstract WP16 and abstract O007), the 2017 EHA Congress, and the 2017 ASH Annual Meeting.