Bellicum’s stock jumped 16% after hours on the news that the FDA is lifting a clinical hold placed on its lead cell therapy, BPX-501, in January.
The agency put the program on standby when it learned of three cases of encephalopathy that were possibly linked to the treatment. Bellicum is developing BPX-501 to improve outcomes in patients who receive partial-match hematopoietic stem cell transplants to treat blood cancers and inherited blood disorders. It is designed to fight infection, aid engraftment, stop disease relapse and, if needed, kill the toxic cells that trigger graft-versus-host disease.
While hematopoietic stem cell transplants are associated with encephalopathy, the BPX-501 program had not previously run into the adverse event, despite being used before in 240 patients. At the time, Bellicum played it close to the vest, saying only that the three cases of encephalopathy were “complex, with a number of potential confounding factors,” including prior failed transplants and a prior history of immunodeficiency.
Jefferies analysts provided a fuller picture in a note: “One patient had a complex history with a prior history of primary immunodeficiency and had two concurrent viral infections. This patient ultimately died from encephalopathy which was confirmed through a brain biopsy. Two of the three patients had prior transplants. Also, two patients’ symptoms resolved. The cases initially were not characterized as treatment-related adverse events but after the one fatality both cases were reviewed and reclassified as treatment related,” they wrote.
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Bellicum’s stock price took a 38% hit, but the analysts figured the Houston-based biotech would be able to resolve the issue within three months—and so it has.
“The decision follows consultation with the FDA and agreement on amendments to the study protocols including guidance on monitoring and management of neurologic adverse events,” the company said in a statement. “Bellicum will be working with U.S. clinical sites to resume patient recruitment based on the amended protocols.”
The hold did not affect a European trial, which is now fully enrolled. Bellicum is gunning for an EMA approval for BPX-501 in 2019.