Orgenesis’ CAR-T ORG-101 Demonstrates Efficacy and Safety in Acute Lymphoblastic Leukemia in Real-World Study in China
Among the treated patients, the complete response (CR) rate in adults was 82% and the CR rate in children was 93%.
Orgenesis’ ORG-101, a CD19-directed chimeric antigen receptor T-cell (CAR-T) therapy, has demonstrated efficacy and safety in patients with CD19+ B-cell acute lymphoblastic leukemia (ALL) treated in real-world data that came from 233 patients treated at a leading hematology center in China.1
Among the treated patients, the complete response (CR) rate in adults was 82% and the CR rate in children was 93%. In terms of safety, severe cytokine release syndrome (CRS) was observed in 2% of treated adults and 6% of treated children. Orgenesis stated that these severe CRS rates are low in comparison to approved CAR-T treatments.
ORG-101, which is produced with a third-generation lentiviral vector coding for a proprietary CAR construct, is manufactured and undergoes analytic testing onsite via a decentralized approach that differs from the centralized approach to manufacturing used for a “traditional” CAR-T therapies. Orgenesis notes that it has held meetings on the topic of ORG-101 with the FDA, the Ministry of Health of Israel, and Germany's Paul-Ehrlich-Institute, and that it intends to bring ORG-101 into a multicenter phase 1/2 clinical trial. With the support of its Enterprise Greece Grant, it will aim to use General University Hospital of Patras in Greece as the first site for the trial.
“We believe that these clinical results combined with our Good Manufacturing Practice-Validated Platform are a significant step forward for our strategy to combine our strong capabilities in decentralized cell therapy production with our regional partnerships,” Vered Caplan, the CEO of Orgenesis, said in a statement.1 “Not only has the product shown initial signs of positive clinical outcomes, but our production data also validated that Orgenesis’ cost-effective decentralized cell processing has the potential to improve access to this treatment and reduce costs. We remain committed to bringing this and other potentially life-saving treatments to patients in need worldwide.”
The news comes on the heels of other positive updates from CAR-T products being used to treat hematologic malignancies in China. Notably, earlier this week Legend Biotech's ciltacabtagene autoleucel (cilta-cel; marketed as Carvykti in the United States and Europe), a BCMA-directed CAR-T therapy was approved by China’s National Medical Products Administration (NMPA) for the treatment of adults with relapsed or refractory (r/r) multiple myeloma (MM) who previously received treatment with 3 or more lines of therapy, including 1 or more proteasome inhibitor and 1 immunomodulatory agent.2
“The approval of cilta-cel in China market marks a key milestone and will bring significant benefits to many patients,” Ying Huang, PhD, the CEO of Legend Biotech, said in a statement at the time.2 “Moving forward, we will continue to pursue our goal of curing patients, expand our clinical research, and enhance the accessibility of this innovative product to benefit more patients.”
In addition, also earlier this week, JW Therapeutics’ relmacabtagene autoleucel injection (relma-cel; marketed as Carteyva), another CAR-T therapy, received approval of its supplemental biologics license application for the treatment of adults with r/r mantle cell lymphoma (MCL) from China’s NMPA.3 The approved indication specifically covers adults with r/r MCL who have previously been treated with at least 2 lines of systemic therapy including bruton tyrosine kinase inhibitors.
“We are delighted to have a product that can deliver meaningful efficacy in this disease, nearly 70% of patients with r/r MCL have achieved complete remission after treatment with Carteyva, and the overall safety data demonstrated that the treatment was generally well-tolerated,” Sophia Yang, the senior vice president and head of regulatory, research, and development at JW Therapeutics, said in a statement at the time.3 “Carteyva becomes the first commercial CAR T-cell product for the treatment of r/r MCL in China.”
