Gene Therapy Developer Beam Therapeutics Reports One Patient Death In Early-Stage Sickle Cell Disease Trial

On Tuesday, Beam Therapeutics Inc. BEAM announced that 35 patients have cleared screening and enrolled in the BEACON Phase 1/2 trial of BEAM-101, an investigational genetically modified cell therapy for sickle cell disease (SCD).

Eight of these patients have been dosed with BEAM-101, while the other enrolled patients are going through pre-transplant stages, including cell collection and drug product manufacturing.

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In a company presentation, preliminary safety data (N=6) showed an initial safety profile consistent with myeloablative conditioning with busulfan and autologous hematopoietic stem cell transplant (HSCT).

The company said that one patient died due to respiratory failure likely related to busulfan conditioning 4 months after BEAM-101 infusion. However, the investigator determined that the death was unrelated to BEAM-101.

The Data Safety Monitoring Committee and FDA reviewed the case.

In all patients dosed, there were no ≥ Grade 3 adverse events (AEs) or serious AEs related to BEAM-101.

Preliminary efficacy data showed that all patients achieved over 60% fetal hemoglobin (HbF, the main oxygen carrier protein in the human fetus) and less than 40% HbS (an abnormal type of hemoglobin that causes sickle cell disease) of non-transfused hemoglobin at Month 1, sustained through all time points.

Markers of hemolysis (destruction of red blood cells) normalized or improved for all patients.

No vaso-occlusive crises (VOCs, painful complication of sickle cell anemia) were reported by investigators following BEAM-101 treatment.

Beam Therapeutics said patient enrollment in the Phase 1/2 trial of BEAM-302 in patients with alpha-1 antitrypsin deficiency (AATD) is progressing. Site activation globally has continued, and dosing has been completed for the first cohort.

Beam has nominated a development candidate for its ESCAPE technology comprised of two investigational drug products: BEAM-103, an anti-CD117 monoclonal antibody (mAb), and BEAM-104, a cell therapy that includes the same therapeutic edit as BEAM-101 (editing the HBG1/2 genes to elevate fetal hemoglobin), plus an additional edit to CD117 which is designed to prevent binding of BEAM-103, allowing the edited cells to evade suppression by the antibody.

The company intends to advance BEAM-103 and BEAM-104 for development in SCD and beta-thalassemia.

Beam Therapeutics ended the third quarter of 2024 with $925.8 million in cash, cash equivalents, and marketable securities, providing a cash runway into 2027.

Price Action: BEAM stock is down 2.40% at $23.78 at the last check on Tuesday.

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