FDA halts Bellicum trial over brain disease in participants, shares crash 48%

Texas-based clinical-stage company Bellicum Pharmaceuticals has taken a massive stock hit after the FDA put a hold on its trials of its T-cell therapy BPX-501 due to three cases of encephalopathy in participants that are thought to be treatment-related.

BPX-501, which is currently in trials to assess its efficacy in improving the outcomes of patients undergoing haploidentical hematopoietic stem cell transplants in the treatment of haematological cancers and inherited blood disorders, has been tested in over 240 patients on three allogeneic haploidentical stem cell transplantation protocols. Three of these participants developed encephalopathy during the trial, leading to its shutdown by the FDA because of the potential adverse effects of treatment, but Bellicum asserts that the patients in question had "a number of potential confounding factors," having taken rimiducid in combination with other medications, as well as undergoing previous failed transplants.

While Bellicum did not provide a large amount of detail beyond this, Jefferies analysts noted: "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."

Encephalopathy has been linked to haematopoietic stem cell transplants in the past, but the actual cause in this case is still up in the air. News of the disaster hit the firm hard, stripping away a huge 48% of its stock price. Bellicum is now awaiting further guidance from the FDA in order to get the trial back up and running. The company noted that the FDA’s decision will not affect the therapy’s current registration trial in Europe, where it hopes to secure marketing authorisation later this year.

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