Cellectis throws in the towel on CAR-T candidate

May 5, 2023

Paris-based biotech Cellectis announced this week that after a few years of struggling to get its CAR-T cell therapy UCARTCS1 out of trials, it is scrapping the program altogether.

UCARTCS1, an allogeneic CAR T-cell product candidate that targets CS1, is currently undergoing evaluation in the MELANI-01 phase 1 dose-escalation clinical study for patients with relapsed or refractory multiple myeloma.

In October 2019, dosing was initiated for the phase I trial; however, in June 2020, a safety report was issued concerning a patient who suffered from treatment-related fatal cardiac arrest, and the trial was put on hold. Following conversations with the FDA, Cellectis was able to get the trial on track but was still looking at a long road to approval. With its targeted completion dates pushed back twiceCellectis says it "would have had to invest meaningful amount of resources" to accelerate enrollment.

In its first quarter financial results statement, the company’s CEO André Choulika said, “The company decided to stop enrollment and treatment of patients with UCARTCS1,” adding “to optimize its resources, Cellectis decided to focus its development efforts on the BALLI-01, AMELI-01 and NATHALI-01 studies." 

The three programs feature allogeneic CAR T-cell products being studied in B-cell acute lymphoblastic leukemia, acute myeloid leukemia and B-cell non-Hodgkin lymphoma, respectively.