On January 30, Bellicum Pharmaceuticals announced it has received notice from the U.S. Food and Drug Administration (FDA) that U.S. studies of BPX-501—an agent being studied to improve outcomes for patients undergoing stem cell transplant who lack a matched donor—have been placed on a clinical hold following three cases of encephalopathy deemed possibly related to BPX-501.
Bellicum is awaiting formal communications from the FDA to determine the requirements for resuming studies and will be working closely with the FDA to address any questions. The FDA clinical hold does not affect the ongoing BP-004 registration trial in Europe.
Encephalopathy has been reported in the allogeneic stem cell transplant literature. Risk factors for encephalitis/encephalopathy after allogeneic stem cell transplants include prolonged immunodeficiency, selected medications, infections, and inflammatory processes such as graft-vs-host disease.
Bellicum has treated more than 240 patients with BPX-501 cells on 3 allogeneic haploidentical stem cell transplantation protocols. These three cases are complex, with a number of potential confounding factors—including prior failed transplants, prior history of immunodeficiency, concurrent infection, and administration of rimiducid (a tacrolimus analog) in combination with other medications.
Bellicum is working with the FDA to evaluate the risk of encephalopathy in patients receiving BPX-501. ■