Bellicum Pharmaceuticals (NASDAQ:BLCM) is up 11% premarket on light volume on the heels of its announcement of encouraging preliminary data from a clinical trial assessing BPX-501 in pediatric patients with acute myeloid leukemia (AML) and primary immunodeficiencies (PIDs).
At year 1, the rates of relapse-free survival and overall survival (OS) in 39 pediatric patients in their first or second complete response who underwent a haploidentical hematopoietic stem cell transplant (haplo-HSCT) followed by BPX-501 were 91.5% and 97.3%, respectively. In the literature, the rates for the same population of patients undergoing alternate-donor HSCT are 60 - 80%.
At year 1, the rates of disease-free survival and OS in pediatric patients with PIDs who underwent haplo-HSCT followed by BPX-501 were 88.1% and 88.6%, respectively.
The results will be submitted for presentation at a future medical conference.
The company is working with investigators and the FDA to develop a protocol for a registration study, expected to launch by year-end.
BPX-501, an adjunct T cell therapy administered after a hematopoietic stem cell transplant, is comprised of genetically modified donor T cells incorporating the company's CaspaCIDe safety switch (safety net against possible uncontrollable GvHD).
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