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Shares of Beam Therapeutics (BEAM) rallied 3% after hours on Wednesday after the company said that its clinical trial evaluating ristoglogene autogetemcel for the treatment of sickle cell disease (SCD) with severe vaso-occlusive crises demonstrated encouraging efficacy.
The company said that risto-cel, its investigational genetically modified cell therapy, demonstrated an acceptable safety profile and encouraging efficacy with changes that resulted in improved laboratory markers for the disease and the resolution of anemia. The data from the trial was published in the The New England Journal of Medicine.
Sickle cell disease (SCD) is a group of inherited blood disorders caused by a genetic mutation leading to abnormal hemoglobin, which causes red blood cells to become rigid, sickle-shaped, and prone to breaking down. SCD with severe vaso-occlusive crises (VOC) involves sickle-shaped red blood cells blocking blood flow, causing intense, often bone-deep pain, ischemia, and organ damage.
“Based on the totality of data generated to date from the BEACON study and the optimized treatment and manufacturing process, we believe risto-cel has the potential to be best-in-class. With an estimated 10,000 patients with SCD in the U.S. eligible for gene therapies, and significant demand expected as these treatments become more widely available, risto-cel is well positioned within this growing market,” said Amy Simon, chief medical officer of Beam.
Beam is now looking to make an application seeking the U.S. Food and Drug Administration’s approval for the therapy as early as 2026 year-end. The FDA previously granted risto-cel orphan drug designation and Regenerative Medicine Advanced Therapy (RMAT) designation.
On Stocktwits, retail sentiment around BEAM stock fell from ‘bullish’ to ‘neutral’ territory over the past 24 hours, while message volume fell from high to normal levels.
BEAM stock has gained 37% over the past 12 months.
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