Cellectis Revives Alemtuzumab with FDA Orphan Drug Status in Leukemia

The US Food and Drug Administration (FDA) has granted orphan drug designation to Cellectis's alemtuzumab (CLLS52) for the treatment of a certain type of leukemia. This designation is for alemtuzumab's use as part of the lymphodepletion regimen in conjunction with another CAR-T therapy by Cellectis, UCART22, aimed at patients with relapsed or refractory B cell acute lymphoblastic leukemia (ALL). This announcement came via a press release on August 1st.

Alemtuzumab, produced by Sanofi under the brand names Campath and Lemtrada, is used to treat chronic lymphocytic leukemia (CLL) and multiple sclerosis (MS). Campath was withdrawn from the market in 2012 to pave the way for Lemtrada and to prevent its off-label use at lower costs. Currently, Lemtrada is available only under a restricted program due to its potential risks, which include autoimmune reactions, infusion-related reactions, and malignancies. Sanofi is supplying the drug for Cellectis's clinical trials based on an agreement made in May 2021, which also included discussions for commercial supply under predetermined financial terms.

In the Phase I BALLI-01 trial (NCT04150497), Cellectis used alemtuzumab as part of a lymphodepletion regimen that also included fludarabine and cyclophosphamide, collectively known as the FCA regimen. This was in combination with their UCART22 therapy, which was administered intravenously following the lymphodepletion regimen to determine the maximum tolerated dose and the recommended Phase II dose. UCART22 has also received orphan drug designation from the European Commission (EC).

UCART22 is derived from healthy donor cells and expresses anti-CD22, a molecule commonly found on cancer cells. The product has the CD52 gene inactivated, making it resistant to anti-CD52 antibodies such as alemtuzumab. Preliminary findings from the trial, reported in August 2023, indicated positive efficacy and safety results.

Mark Frattini, Chief Medical Officer of Cellectis, noted that the inclusion of alemtuzumab to the fludarabine and cyclophosphamide regimen resulted in sustained lymphodepletion and significantly improved UCART22 cell expansion. This enhancement allowed for greater clinical activity.

Cellectis is actively developing up to ten new cell and gene therapy products thanks to an equity investment and research partnership with AstraZeneca, initiated in November 2023. Concluded in May this year, AstraZeneca now holds a 44% equity stake in Cellectis.

This article highlights the promising developments in the field of cell and gene therapy, specifically focusing on the FDA's recent orphan drug designation for Cellectis's alemtuzumab and the positive results from their ongoing Phase I trial. The collaboration with Sanofi and the equity partnership with AstraZeneca underline the significant strides being made by Cellectis in the realm of innovative cancer treatments.

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