Beam Therapeutics’ Phase I/II trial of its cell therapy for sickle cell disease (SCD) has met its primary and secondary endpoints, potentially teeing the therapy up for regulatory approval.
In the Phase I/II BEACON trial (NCT05456880), ristoglogene autogetemcel (risto-cel) was investigated in patients with SCD with severe vaso-occlusive crises (VOCs).
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The study achieved the primary efficacy endpoint of freedom from severe VOCs for at least 12 consecutive months (VF12) based on independently adjudicated results and met all primary safety endpoints at the prespecified interim analysis.
As of the 20 October data cutoff, a total of 39 adults and 10 adolescents were treated in the BEACON trial with the one-time therapy, and 31 patients had completed the required follow-up period for the VF12 endpoint.
The study also met all secondary efficacy endpoints, including freedom from inpatient hospitalisation for severe VOCs for at least 12 consecutive months. The safety findings observed as of the data cutoff date were consistent with previously reported data.
CEO of Beam Therapeutics, John Evans, said: “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.
“For patients with SCD, severe vaso-occlusive crises can be debilitating and life-threatening, making sustained freedom from these events an important clinical outcome. 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. 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.”
Based on this data, Beam expects to submit a biologics license application (BLA) for risto-cel to the US Food and Drug Administration (FDA) as early as the end of 2026.
Detailed pivotal results from the BEACON study are planned for presentation at a medical meeting in 2027.
Risto-cel is a one-time, genetically modified cell therapy that consists of autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) that have been base-edited in the promoter regions of the HBG1/2 genes and are administered via a haematopoietic stem cell transplant procedure. The HBG1 and HBG2 genes encode foetal haemoglobin (HbF).
It is designed to inhibit the transcriptional repressor BCL11A from binding to the promoter without disrupting BCL11A expression, which is intended to lead to increased production of non-sickling and anti-sickling HbF to mimic the effects of naturally occurring variants seen in hereditary persistence of foetal haemoglobin.
According to the World Health Organization (WHO), SCD ranks as the 12th leading cause of under-five mortality when looking at total mortality burden. Common complications of SCD are acute pain episodes, anaemia, stroke, infections, kidney failure, and pregnancy-related risks.
GlobalData’s epidemiology forecast for the 16 major markets (16MM: the US, France, Germany, Italy, Spain, the UK, Japan, Australia, Brazil, Canada, China, India, Mexico, Russia, South Africa, and South Korea) predicted the burden of SCD to be at approximately 400,000 diagnosed prevalent cases in 2025. While current therapies primarily offer symptomatic relief, there are no cures on the market.
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