Beam Therapeutics Announces Positive Topline Pivotal Data from BEACON Phase 1/2 Trial of Risto-cel in Patients with Sickle Cell Disease
Globe Newswire•10/11/2026•07:00 ET
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Key Highlights
- ➤BEACON met primary efficacy endpoint: freedom from severe VOCs for 12 consecutive months
- ➤BEACON met all primary safety and secondary efficacy endpoints at interim analysis
- ➤49 patients treated; 31 completed required VF12 follow-up as of October 20, 2026
- ➤Beam expects U.S. risto-cel BLA submission as early as year-end 2026
- ➤Detailed pivotal BEACON results planned for presentation at a 2027 medical meeting
Expert Statements
John Evans, Chief executive officer of Beam Therapeutics
“These positive topline results represent a defining milestone for risto-cel and for Beam’s base editing technology more broadly, bringing us an important step closer to delivering a potentially transformative, one-time treatment for people living with sickle cell disease.”
John Evans, Chief executive officer of Beam Therapeutics
“For patients with SCD, severe vaso-occlusive crises can be debilitating and life-threatening, making sustained freedom from these events an important clinical outcome.”
John Evans, Chief executive officer of Beam Therapeutics
“The achievement of our primary efficacy endpoint, supported by the broader clinical safety and efficacy profile observed in the BEACON trial, reinforces our confidence in risto-cel.”
John Evans, Chief executive officer of Beam Therapeutics
“With these results in hand, we are focused on submitting our planned BLA as early as year-end, which has the potential to be the first BLA for a base editing therapy.”
BEACON Trial Achieved Primary Efficacy Endpoint of Freedom from Severe Vaso-occlusive Crises (VOCs) for at Least 12 Consecutive Months, Based on Independently Adjudicated Results, and Met All Primary Safety Endpoints at the Prespecified Interim Analysis
Study Met All Secondary Endpoints Including Freedom from Inpatient Hospitalization for Severe VOCs for 12 Consecutive Months
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