Bellicum Pharma rivo-cel hits primary endpoint in pivotal trial
Bellicum Pharmaceuticals’ (NASDAQ:BLCM) registrational trial of rivo-cel achieved its primary endpoint of event-free survival when the drug was administered to patients receiving stem cell transplants.
Some 20% to 30% of stem cell transplant patients do not have a human leukocyte antigen (HLA)-matched donor. Rivo-cel is a T-cell treatment designed to treat immunodeficiency and reduce the rate of leukemia following a stem cell transplant. T-cell proliferation is triggered with the administration of an additional drug, rimiducid, to reduce morbidity and mortality due to graft-versus-host disease.
The Phase 1/2 registrational study met its primary endpoint of event free survival rate at 180 days of nearly 91%, and demonstrated rivo-cel’s non-inferiority compared with matched unrelated donor transplants.
“These final results support our belief that rivo-cel, if approved, may be a transformational new treatment option for pediatric patients with leukemias, lymphomas and genetic blood diseases,” Rick Fair, Bellicum’s president and CEO, said in a statement.
“Adding rivo-cel to haploidentical stem cell transplantation improved the curative outcomes of these procedures, and may enable them to become more broadly available to patients without access to an HLA-matched donor,” he added.
Bellicum is planning to file European marketing authorization applications for rivo-cel and rimiducid, and is seeking a strategic partner for potential commercialization.