FDA Clears the IND for UCARTCS1, the First Allogeneic CAR-T to Treat Multiple Myeloma Patients

Cellectis announced that the U.S. Food and Drug Administration (FDA) has approved the Company’s Investigational New Drug (IND) application to initiate a Phase 1 clinical trial for UCARTCS1, in patients with multiple myeloma (MM). The IND for UCARTCS1 was filed on December 28, 2018 and approved by the FDA within a month, on January 25, 2019. Cellectis is the sponsor of the UCARTCS1 clinical study (MUNDI-01) and successfully ensured the manufacturing and release of UCARTCS1 GMP batches, as well as an IRB approval.

UCARTCS1 is based on a tailored manufacturing process developed by Cellectis, which removes both the CS1 antigen and TCR from the T-cell surface using TALEN gene editing technology, before adding the CS1 CAR construct. This approach has both clinical and operational benefits: the UCART is designed to have a lymphodepleting effect, and the CAR T-cell cross reaction is suppressed, allowing for successful manufacturing.

UCARTCS1 is the first allogeneic CAR-T therapy for MM to enter clinical development. This milestone reinforces Cellectis’ leadership in the space, as it represents the fourth TALEN gene-edited allogeneic CAR-T product candidate developed by Cellectis to be approved for clinical trials following UCART19* for ALL patients, UCART123 for AML patients and UCART22 for B-ALL patients. The Phase 1 of the MUNDI-01 study is designed to assess the safety and tolerability at increasing dose levels of UCARTCS1 in patients living with MM.

“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,” said Dr. André Choulika, Chairman and CEO of Cellectis. “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.”

We anticipate the clinical research to be led by Dr. Krina Patel, Principal Investigator, Assistant Professor, Department of Lymphoma/Myeloma, Division of Cancer Medicine at the MD Anderson Cancer Center in Houston, Texas. We plan to have two additional sites enrolling patients for this clinical study: Weill Cornell Medicine under the leadership of Dr. Ruben Niesvizky, Director of the Multiple Myeloma Center at New York Presbyterian Hospital-Cornell Medical Center and Hackensack Meridian under the supervision of Dr. Andre Goy, Chairman and Director of John Theurer Cancer Center (JTCC) at Hackensack University Medical Center.