StemEx regulatory update

Gamida Cell said FDA advised the company to conduct an additional Phase III trial to support a BLA submission for StemEx to treat hematologic malignancies. The company had previously expected data from its open-label, international Phase II/III ExCell trial would support the submission and allow it to launch the product in "a couple of years." Earlier this year, Gamida Cell reported that StemEx met the primary endpoint in ExCell of improving the morality rate at 100 days post-transplantation vs. a historical control group, but missed secondary endpoints of reducing the mortality rate at 180 days post-transplantation, the incidence of acute graft-versus-host disease (GvHD) grades III-IV and the rate of neutrophil engraftment failure vs. a historical control group (see BioCentury, Feb. 11 & April 22). The company said it "will now regroup and determine how to proceed with our clinical development program" of StemEx, which consists of ex vivo expanded cord blood stem/progenitor cells in combination with non-expanded cells from the same cord blood unit. ...