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BEAM Stock Falls 7% Despite Meeting Primary Endpoints in SCD Study
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Key Takeaways
Beam Therapeutics achieved primary efficacy and safety endpoints in its phase I/II BEACON study.
Risto-cel demonstrated freedom from severe VOCs for at least 12 consecutive months in SCD patients.
BEAM plans to submit a BLA for risto-cel by year-end 2026, targeting its first commercial product.
Beam Therapeutics (BEAM - Free Report) has announced positive top-line results from the phase I/II BEACON study evaluating its investigational gene-editing therapy, ristoglogene autogetemcel (risto-cel, formerly BEAM-101), in patients with sickle cell disease (SCD) who experience severe vaso-occlusive crises (VOCs).
Risto-cel is a one-time, genetically modified stem cell therapy designed to increase fetal hemoglobin (HbF) production, reduce sickling of red blood cells and prevent painful crises in patients with SCD.
Sickle cell disease is an inherited blood disorder caused by a beta-globin gene mutation that makes red blood cells sickle-shaped, leading to anemia, severe pain crises and potentially life-threatening organ damage.
Key Highlights of BEAM’s Phase I/II BEACON Study
The BEACON Study achieved its primary efficacy endpoint by demonstrating freedom from severe VOCs for at least 12 consecutive months (VF12), based on independent adjudication in patients with SCD. Treatment with risto-cel also met all primary safety endpoints at the prespecified interim analysis. Additionally, the study achieved all secondary efficacy endpoints, including freedom from inpatient hospitalization for severe VOCs for at least 12 consecutive months.
As of the October 2026 data cutoff, the majority of the patients treated with risto-cel had completed the required follow-up period for the VF12 endpoint. The safety profile remained consistent with previously reported findings and the known risks associated with stem cell collection, busulfan-based conditioning and autologous hematopoietic stem cell transplantation.
Following the positive results, Beam Therapeutics plans to submit a biologics license application (BLA) for risto-cel to the FDA as early as the end of 2026. If successful, the application could become the first BLA for a base-editing therapy, and it could bring the company closer to its first potential commercial product. BEAM plans to present detailed pivotal results from the BEACON study at a medical meeting in 2027.
Despite the encouraging clinical results, BEAM’s shares declined 7% yesterday. The drop may reflect concerns over weaker-than-expected third-quarter 2026 results, profit-taking following the announcement and lingering uncertainty surrounding regulatory approval and the eventual commercialization of risto-cel.
Year to date, shares of Beam Therapeutics have lost 9.3% against the industry’s 12.3% growth.
Image Source: Zacks Investment Research
Beam Therapeutics is evaluating another candidate, BEAM-103, an anti-CD117 monoclonal antibody for the treatment of patients with SCD in an ongoing phase I healthy-volunteer study. In June, the company completed patient dosing in the study.
BEAM’s Zacks Rank & Stocks to Consider
Beam Therapeutics currently carries a Zacks Rank #3 (Hold).
Over the past 30 days, earnings per share estimates for Amneal Pharmaceuticals have increased from $1.03 to $1.04 for 2026. Over the same period, estimates for 2027 earnings per share have risen from $1.16 to $1.19. AMRX’s shares have climbed 69.7% year to date.
Amneal Pharmaceuticals’ earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 27.21%.
Over the past 30 days, loss per share estimates for AC Immune have narrowed from 60 cents to 54 cents for 2026. Over the same period, estimates for 2027 earnings per share have increased from 17 cents to 43 cents. ACIU’s shares have gained 27.1% year to date.
AC Immune’s earnings beat estimates in each of the trailing four quarters, with an average surprise of 37.79%.
Over the past 30 days, loss per share estimates for Adaptive Biotechnologies have narrowed from 55 cents to 49 cents for 2026. Over the same period, estimates for 2027 loss per share have also improved from 17 cents to 13 cents. ADPT’s shares have surged 80.8% year to date.
Adaptive Biotechnologies’ earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 53.62%.
Image: Bigstock
BEAM Stock Falls 7% Despite Meeting Primary Endpoints in SCD Study
Key Takeaways
Beam Therapeutics (BEAM - Free Report) has announced positive top-line results from the phase I/II BEACON study evaluating its investigational gene-editing therapy, ristoglogene autogetemcel (risto-cel, formerly BEAM-101), in patients with sickle cell disease (SCD) who experience severe vaso-occlusive crises (VOCs).
Risto-cel is a one-time, genetically modified stem cell therapy designed to increase fetal hemoglobin (HbF) production, reduce sickling of red blood cells and prevent painful crises in patients with SCD.
Sickle cell disease is an inherited blood disorder caused by a beta-globin gene mutation that makes red blood cells sickle-shaped, leading to anemia, severe pain crises and potentially life-threatening organ damage.
Key Highlights of BEAM’s Phase I/II BEACON Study
The BEACON Study achieved its primary efficacy endpoint by demonstrating freedom from severe VOCs for at least 12 consecutive months (VF12), based on independent adjudication in patients with SCD. Treatment with risto-cel also met all primary safety endpoints at the prespecified interim analysis. Additionally, the study achieved all secondary efficacy endpoints, including freedom from inpatient hospitalization for severe VOCs for at least 12 consecutive months.
As of the October 2026 data cutoff, the majority of the patients treated with risto-cel had completed the required follow-up period for the VF12 endpoint. The safety profile remained consistent with previously reported findings and the known risks associated with stem cell collection, busulfan-based conditioning and autologous hematopoietic stem cell transplantation.
Following the positive results, Beam Therapeutics plans to submit a biologics license application (BLA) for risto-cel to the FDA as early as the end of 2026. If successful, the application could become the first BLA for a base-editing therapy, and it could bring the company closer to its first potential commercial product. BEAM plans to present detailed pivotal results from the BEACON study at a medical meeting in 2027.
Despite the encouraging clinical results, BEAM’s shares declined 7% yesterday. The drop may reflect concerns over weaker-than-expected third-quarter 2026 results, profit-taking following the announcement and lingering uncertainty surrounding regulatory approval and the eventual commercialization of risto-cel.
Year to date, shares of Beam Therapeutics have lost 9.3% against the industry’s 12.3% growth.
Image Source: Zacks Investment Research
Beam Therapeutics is evaluating another candidate, BEAM-103, an anti-CD117 monoclonal antibody for the treatment of patients with SCD in an ongoing phase I healthy-volunteer study. In June, the company completed patient dosing in the study.
BEAM’s Zacks Rank & Stocks to Consider
Beam Therapeutics currently carries a Zacks Rank #3 (Hold).
Some better-ranked stocks in the biotech sector are Amneal Pharmaceuticals (AMRX - Free Report) , currently sporting a Zacks Rank #1 (Strong Buy), and AC Immune (ACIU - Free Report) and Adaptive Biotechnologies (ADPT - Free Report) , carrying a Zacks Rank #2 (Buy) each. You can see the complete list of today’s Zacks #1 Rank stocks here.
Over the past 30 days, earnings per share estimates for Amneal Pharmaceuticals have increased from $1.03 to $1.04 for 2026. Over the same period, estimates for 2027 earnings per share have risen from $1.16 to $1.19. AMRX’s shares have climbed 69.7% year to date.
Amneal Pharmaceuticals’ earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 27.21%.
Over the past 30 days, loss per share estimates for AC Immune have narrowed from 60 cents to 54 cents for 2026. Over the same period, estimates for 2027 earnings per share have increased from 17 cents to 43 cents. ACIU’s shares have gained 27.1% year to date.
AC Immune’s earnings beat estimates in each of the trailing four quarters, with an average surprise of 37.79%.
Over the past 30 days, loss per share estimates for Adaptive Biotechnologies have narrowed from 55 cents to 49 cents for 2026. Over the same period, estimates for 2027 loss per share have also improved from 17 cents to 13 cents. ADPT’s shares have surged 80.8% year to date.
Adaptive Biotechnologies’ earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 53.62%.