FDA Grants Orphan Drug Status to Cellectis’ CLLS52 for ALL Treatment

On August 1, 2024, Cellectis, a clinical-stage biotechnology company listed on Euronext Growth and NASDAQ, announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its Investigational Medicinal Product (IMP), CLLS52 (alemtuzumab). This product is used as part of the lymphodepletion regimen in the BALLI-01 clinical trial, which evaluates UCART22 in patients with relapsed or refractory B-cell acute lymphoblastic leukemia (ALL).

The FDA's ODD status is significant as it aims to support the development of treatments for rare diseases affecting fewer than 200,000 people in the United States. This designation can accelerate the development and reduce the costs associated with bringing a therapeutic agent to market. Mark Frattini, M.D., Ph.D. and Chief Medical Officer at Cellectis, emphasized the importance of incorporating alemtuzumab into the lymphodepletion regimen. The BALLI-01 study demonstrated that adding alemtuzumab to the fludarabine and cyclophosphamide regimen resulted in sustained lymphodepletion and a significant increase in UCART22 cell expansion, which contributed to enhanced clinical activity.

Cellectis is known for developing innovative gene-editing technologies, including TALEN® and the PulseAgile electroporation system. The company specializes in creating life-saving cell and gene therapies. The combination of CD52 knockout UCART cells with a lymphodepleting regimen containing an anti-CD52 antibody like alemtuzumab is a novel approach pioneered by Cellectis. This strategy aims to render UCART product candidates resistant to alemtuzumab, which is part of the lymphodepletion regimen. Specifically, the CD52 gene in the UCART22 product candidate is inactivated using TALEN® gene editing technology.

Cellectis's efforts in the field of gene editing and CAR-T immunotherapies are noteworthy. The company employs an allogeneic approach for CAR-T immunotherapies in oncology, which involves developing off-the-shelf, ready-to-use gene-edited CAR T-cells for cancer treatment. In addition, Cellectis is working on therapeutic gene editing in hematopoietic stem cells to address various diseases with unmet medical needs.

With 25 years of experience in gene editing, Cellectis continues to innovate in the biotechnology sector. Their headquarters are located in Paris, France, and they also have facilities in New York, New York, and Raleigh, North Carolina. The company's stock is traded on the Nasdaq Global Market under the ticker symbol CLLS and on Euronext Growth under the ticker symbol ALCLS.

In conclusion, the FDA's approval of the Orphan Drug Designation for CLLS52 (alemtuzumab) is a pivotal moment for Cellectis, reflecting their cutting-edge research and commitment to addressing rare diseases. Through its pioneering gene-editing platform and innovative therapies, Cellectis continues to advance the field of biotechnology by developing treatments that offer hope to patients with serious and rare medical conditions.

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