April 5, 2016 8:44am

 

These data also demonstrate high BPX-501 cell viability, expansion and persistence, and that the improvement of immune reconstitution is sustained.

 

Future studies are needed to address the role of repeated infusions or higher numbers of BPX-501 cells in malignant patients with resistant disease

 


 

Interim data presented at 42nd Annual Meeting of European Society for Blood & Marrow Transplantation in Spain.

 

In the ongoing BP-004 clinical trial, malignant and nonmalignant pediatric patients receive a haploidentical T cell-depleted hematopoietic stem cell transplant (HSCT) followed by an add-back of BPX-501 donor T cells. The trial is designed to evaluate whether this regimen is safe and improves immune reconstitution, infection control and overall outcomes.

In an oral presentation reviewing the preliminary outcomes from the BP-004 clinical trial in 17 high-risk pediatric patients with blood cancers, the data showed that BPX-501 cells expand in vivo and persist over time, contributing to adaptive immunity. There was no transplant-related mortality due to infections, GvHD or other transplant-related complications. Additionally, the relapse rate to date compares favorably with that of historical controls, with 16 of 17 patients in the trial with acute leukemias showing disease-free outcomes. The median follow-up period to date for these patients was approximately seven months.

 

The Bottom Line: BLCM’s interim data from ongoing BP-004 P1/2 clinical trial in children with genetic diseases and cancers showed that BPX-501 cells expand in vivo and persist over time, contributing to adaptive immunity.

  • No transplant-related mortality due to infections, GvHD or other transplant-related complications
  • Relapse rate to date compares favorably vs historical controls, with 16 of 17 patients in trial with acute leukemias showing disease-free outcomes
  • BLCM plans to meet with FDA, EMA during 2Q

BLCM closed at $10.11, a positive and a preliminary indication of further testing  - BUY