Beam Therapeutics’ investigational gene-edited therapy for sickle cell disease met its primary efficacy endpoint in a Phase 1/2 clinical trial, bringing the company closer to a planned FDA submission.
The biotech said Nov. 10 that its BEACON study of ristoglogene autogetemcel, or risto-cel, achieved its primary endpoint of freedom from severe vaso-occlusive crises for at least 12 consecutive months.
As of the Oct. 20 data cutoff, 39 adults and 10 adolescents had received the one-time cell therapy, with 31 patients completing the required follow-up period for the primary endpoint. The trial also met all primary safety endpoints at the prespecified interim analysis and all secondary efficacy endpoints, including freedom from inpatient hospitalization for severe vaso-occlusive crises for at least 12 consecutive months.
Safety findings were consistent with previously reported trial data and the known effects of the treatment process and underlying sickle cell disease. Beam did not disclose the percentage of patients who achieved the primary endpoint in its topline results.
Beam expects to submit a biologics license application to the FDA as early as the end of 2026. The company plans to present detailed pivotal results from the BEACON study at a medical meeting in 2027.
