Cellectis has secured approval from the US Food and Drug Administration (FDA) to launch a phase 1 clinical trial for the evaluation of its gene-edited T-cell product candidate UCARTCS1 as a potential treatment for multiple myeloma (MM).
In this connection, the regulator approved the company’s Investigational New Drug (IND) application to begin the early stage trial for UCARTCS1.
The allogeneic CAR-T therapy is based on a customized manufacturing process developed by the clinical-stage biopharma company. The process is said to help in removing the CS1 antigen and TCR from the T-cell surface using TALEN gene editing technology, prior to the addition of the CS1 CAR construct.
Cellectis claims that the approach has clinical and also operational benefits. The company said that UCART is designed to have a lymphodepleting effect, and the CAR T-cell cross reaction is subdued, thereby enabling successful manufacturing.
The MUNDI-01 trial to be initiated by the company is a dose-escalation and dose-expansion study to assess the safety, expansion, persistence and clinical activity of UCARTCS1 in MM patients.
Dose level 1 will be at 1×10⁶ UCARTCS1 cells per kilogram, while dose levels 2 and 3 will be at 3×10⁶ and 9×10⁶, respectively. The company said that there will be a 28-day dose limiting toxicity (DLT) period in concordance with a staggering of 28 days for the first two patients at each dose level.
According to Cellectis, UCARTCS1 is the first allogeneic CAR-T therapy for MM to enter clinical stage. It is the fourth TALEN gene-edited allogeneic CAR-T product candidate of the company to be approved for clinical trials after UCART19 for Acute Lymphoblastic Leukemia (ALL) patients, UCART123 for Acute Myeloid Leukemia (AML) patients and UCART22 for B-ALL patients.
Cellectis chairman and CEO André Choulika said: “The last quarters have been very productive for Cellectis’ UCARTCS1 product candidate. We successfully manufactured and released GMP batches of UCARTCS1, filed an IND and secured approval from the FDA to start the MUNDI-01 Phase 1 clinical study.
“This is the 4th time in 4 years that Cellectis demonstrates excellence with an allogeneic product candidate. It further demonstrates the strength of our innovation, our manufacturing process and our execution, as we are eager to bring the first allogeneic multiple myeloma CAR T-cell treatment to patients.”