NEW YORK – Houston-based Bellicum Pharmaceuticals on Monday announced that the US Food and Drug Administration had issued a hold on the company's dose-escalation clinical trial evaluating BPX-601, an autologous CAR T-cell therapy designed to target prostate stem cell antigen (PCSA), which is expressed on the surface of certain solid tumors.
The FDA issued the hold on Bellicum's clinical trial after a patient with pancreatic cancer enrolled in the study died. Although Bellicum maintains that the death was not related to the investigational treatment, the company is complying with the FDA to take the steps necessary to resume the clinical trial.
The primary aim of the paused trial is to evaluate the safety and maximum tolerated dose of BPX-601 and rimiducid as a treatment for patients with advanced pancreatic, gastric, or prostate cancer whose tumors express PCSA, as determined by a central test. The study is also powered to assess the pharmacokinetics of the cell therapy, as well as patients' overall response rates.
The therapy is part of Bellicum's GoCAR-T program, which has yielded another product candidate, BPX-603, designed to target HER2-expressing solid tumors. The company still plans to begin enrolling patients in a Phase I/II clinical trial of BPX-603 by year end.
Both investigational therapies incorporate inducible MyD88/CD40, or iMC, a co-activation domain that the company believes will boost T-cell proliferation and allow the CAR-T therapy to overcome tumor resistance mechanisms such as PD-1 and TGF-beta.