Drug Development, FDA

Brain damage cases force an FDA hold on Bellicum’s lead cell therapy — shares tank

Bellicum Pharmaceuticals $BLCM just fell even further behind the pack leaders in the cell therapy crowd.

Rick Fair, president and CEO of Bellicum

The Houston-based biotech reported after the markets closed Tuesday that the FDA had slapped a clinical hold on its lead cell therapy after researchers tracked three cases of encephalopathy — a broad term for brain damage — among patients taking the drug to support allogeneic stem cell transplants. The cases were deemed as possibly linked to BPX-501, which was being billed as a safer version of the cell therapies now making their way to the market.

Bellicum’s already suffering stock tanked on the news, instantly dropping 44%.

Some analysts quickly did a double take on Bellicum’s decision to continue a registrational study of BPX-501 in Europe dubbed BP-004. Researchers are investigating the therapy “in children with hematological cancers or orphan inherited blood disorders in which BPX-501 is administered after initial allogeneic HSCT.”

Encephalopathy has been reported before in the allogeneic stem cell transplant literature, Bellicum reports. They added:

These three cases are complex, with a number of potential confounding factors—including, in certain of the cases, prior failed transplants, prior history of immunodeficiency, concurrent infection, and administration of rimiducid in combination with other medications. Bellicum is working with FDA to evaluate the risk of encephalopathy in patients receiving BPX-501.

The biotech has been studying this cell therapy for three key objectives: fighting infection, supporting engraftment, and preventing disease relapse and, should GvHD occur, their CaspaCIDe safety switch can be activated to kill the toxic T cells.

Bellicum has been struggling since going public in 2014, watching about two thirds of its market cap evaporate before today even as the leaders in CAR-T — Kite and Novartis — surged forward, with Gilead buying Kite for a big premium. Juno also has been making a big comeback since its own safety woes, which triggered a $9 billion buyout by Celgene.


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