The designation follows durable responses seen in a phase 1 study of the agent.
This content originally appeared on our sister site, Targeted Oncology.
GC012F (Gracell Biotechnologies), a FasTCAR-enabled BCMA/CD19 dual targeted chimeric antigen receptor (CAR) T-cell therapy, has received orphan drug designation for the treatment of multiple myeloma.
The cell therapy is designed to induce deep and durable responses in patients with multiple myeloma by targeting both malignant plasma cells expressing BCMA along with CD19-expressing early progenitor cells.
"As our lead candidate currently being developed on Gracell's FasTCAR next-day manufacturing technology platform, GC012F is a unique BCMA and CD19 dual-targeting CAR-T cell therapy," said Martina Sersch, MD, PhD, chief medical officer of Gracell, in a press release. "GC012F has demonstrated fast, deep and durable responses in patients with Relapsed/Refractory Multiple Myeloma in an ongoing IIT study in China with most patients on study being high risk according to mSMART 3.0 criteria, a difficult-to-treat patient population. We are very excited about being granted orphan drug designation for the treatment of multiple myeloma by the US FDA, another key milestone in advancing our program globally. Multiple myeloma patients are in need of more efficacious and tolerable therapies providing deep and durable responses and ultimately extending progression-free and overall survival."
The CAR T-cell therapy is currently being evaluated in a phase 1 study. The primary end point of the trial is the rate of incidence and severity of adverse events after GC012F infusion up to 24 weeks after infusion. Secondary end points include the percentage of patients with minimal residual disease 12 and 24 weeks after infusion, overall response rate (ORR), progression-free survival, duration of response, and overall survival.
During the study, all patients received a single infusion of GC012F at a dose of (1-5)10E5/kg cells.
According to the analysis, between October 2019 and July 2020, 19 heavily pretreated patients with multiple myeloma were given a single infusion of GC012F. The median number of prior lines of therapy was 5 (range, 2-9). At a data cutoff of January 12, 2012, 19 patients were evaluated for response. The ORR was 94.7%. One hundred percent of patients saw a reduction of paraprotein, with 18 of the 19 patients experiencing a 100% reduction.
In terms of safety, grade 1/2 cytokine release syndrome was seen in 84.2% of patients and grade 3 in 10.5% of patients. No grade 4/5 cytokine release syndrome was observed. Two deaths were observed over the course of the study. However, neither were found to be related to study treatment.
In order to participate in the study, patients must have a confirmed diagnosis of relapsed/refractory multiple myeloma a life expectancy of 3 more or more, adequate liver, kidney, and cardiopulmonary function, and have received at least 2 prior lines of therapy and be refractory to both an immunomodulatory drug and a proteasome inhibitor. Patients with other uncontrolled malignancies, convulsion or stroke within the past 6 months, clinical evidence of dementia, or who have pulmonary fibrosis are not eligible to participate.