CT053, a novel cell-based therapy being developed by Carsgen Therapeutics, was given priority medicines (PRIME) status by the European Medicines Agency (EMA) as a potential treatment of relapsed or refractory multiple myeloma.
Early findings from a Phase 1 trial in people with heavily pre-treated disease support the therapy’s potential effectiveness and safety.
PRIME designation is given to candidate therapies with promising clinical data showing a potential to treat diseases of high unmet medical need. It provides CARsgen with enhanced interaction and early dialogue with the European regulatory agency, ultimately speeding its development and review.
“PRIME eligibility is an important regulatory milestone in the continued development and commercialization of CT053 anti-BCMA CAR T cells,” Zonghai Li, MD, PhD, chief executive officer of CARsgen, said in a press release. “PRIME designation is invaluable to the advancement of this cutting-edge therapeutic to potential market approval and being available to patients as quickly as possible”
CT053 is an investigational CAR T-cell therapy that uses a patient’s own immune T-cells, equipped with a receptor for the BCMA protein. BCMA is present in most multiple myeloma cells, making it a suitable target for cell-based therapies.
After being collected and genetically engineered in the lab, the T-cells are expanded to millions and injected back into the patient’s bloodstream, aiming to eliminate malignant cancer cells while leaving healthy cells unharmed.
The PRIME designation was based on clinical data from an ongoing, three-site Phase 1 trial (NCT03716856, NCT03302403, and NCT03380039; each number corresponds to a site where the trial is taking place). The study is exploring the safety and early efficacy of CT053 in 24 people with heavily pre-treated multiple myeloma.
These patients had received a median of 4.5 prior lines of therapy, and were given a single dose of CT053 soon after high-dose chemotherapy.
Preliminary results were discussed at the recent 17th International Myeloma Workshop in Boston, in the oral presentation, “CT053, Anti-BCMA CAR T-cell therapy for Relapsed/Refractory Multiple Myeloma: Proof of Concept Results from a Phase I Study” (page 84).
The findings showed that 88% of patients responded to the treatment, with 79% complete responses, meaning that patients had no signs of active cancer cells after treatment. A total of 16 patients were still in complete response or very good partial response after a median follow-up of 295 days (about 9.8 months).
The most common side effects were blood related, and included low levels of white blood cells, neutrophils, lymphocytes, and platelets. One patient died due to bone marrow failure.
Fifteen patients developed cytokine release syndrome — an adverse event of immunotherapies characterized by an excessive activation of the immune system — but all were mild to moderate.
“This study demonstrated that CT053 had an excellent efficacy and a good safety profile in patients with relapsed-refractory multiple myeloma,” the researchers wrote.
Given the promising results in the trial, CARsgen has been cleared to initiate CT053 clinical studies in the U.S. and Canada. The U.S. Food and Drug Administration has also granted orphan drug designation to CT053 for the treatment of multiple myeloma.