NEW YORK – AvenCell Therapeutics on Tuesday said the European Medicines Agency has approved its clinical trial application to study the allogeneic CAR T-cell therapy AVC-201 in CD123-positive hematologic malignancies.
The agency's decision has cleared the way for Cambridge, Massachusetts-based AvenCell to begin a Phase I trial involving up to 35 patients with CD123-positive acute myeloid leukemia at multiple sites in Germany. In the trial, researchers will assess the safety of AvenCell's CD123-directed AVC-201 and establish its maximum tolerated dose. CD123 is overexpressed in around 80 percent of acute myeloid leukemia cases, as well as in some other hematologic malignancies.
AVC-201 comprises a cellular component (Allo-RevCAR01-T) and a recombinant antibody derivative (R-TM123), which form the active drug when combined. R-TM123 functions as a bridge between Allo-RevCAR01-T and the CD123 epitope on the cancer cell.
Patients in the Phase I trial will receive a continuous infusion of R-TM123 recombinant antibody derivative following lymphodepleting therapy from the first to the 20th day of treatment. After four hours of infusion, a single dose of Allo-RevCAR01-T cellular component will be given intravenously. In addition to determining the treatment's safety, dose-limiting toxicities, and maximum tolerated dose, researchers will also measure its efficacy by tracking complete remission, overall response rate, progression-free survival, and overall survival in patients.
AVC-201 is the result of combining a CRISPR-Cas9 allogeneic cell therapy platform licensed from Intelia with AvenCell's switchable CAR T-cell technology. "With the application of both platform technologies, we are the first company in this space to completely separate the manufacture of cells from the ultimate patients and cancer indications they will be targeted to," AvenCell President and CEO Andrew Schiermeier said in a statement.