On Saturday, Beam Therapeutics Inc. (NASDAQ:BEAM) revealed new safety and efficacy data from its BEACON Phase 1/2 trial of BEAM-101 in patients with sickle cell disease (SCD) with severe vaso-occlusive crises (VOCs).
The data were featured at the American Society of Hematology (ASH) Annual Meeting and Exposition.
Consistent with Beam's previously announced data, updated data from seven patients treated with investigational base-editing therapy BEAM-101 demonstrated robust and durable increases in fetal hemoglobin (HbF) and reductions in sickle hemoglobin (HbS), rapid neutrophil and platelet engraftment, and normalized or improved markers of hemolysis.
No VOCs were reported post-engraftment.
Key highlights include the following:
- Rapid and Sustained Increases in Protective Fetal Hemoglobin (HbF): All patients achieved endogenous HbF levels exceeding 60% and reduction in corresponding sickle hemoglobin (HbS) below 40% that was durable. A pancellular distribution of HbF was observed after the elimination of transfused blood.
- Robust and Sustained Total Hemoglobin (Hb) Levels: Total hemoglobin levels increased rapidly with a resolution of anemia in patients after the elimination of the transfused blood.
- Normalization of Hemolysis Markers: Key markers of hemolysis, including indirect bilirubin, haptoglobin, lactate dehydrogenase and reticulocytes, normalized or improved in all patients following BEAM-101 treatment.
- Safety Profile Consistent with Busulfan and Autologous Hematopoietic Stem Cell Transplantation.
In November, the company said that one patient died due to respiratory failure likely related to busulfan conditioning four months after BEAM-101 infusion. However, the investigator determined that the death was unrelated to BEAM-101.
On Sunday, Beam Therapeutics announced new data for its Engineered Stem Cell Antibody Evasion (ESCAPE) conditioning platform, which was presented at the American Society of Hematology Annual Meeting and Exposition.
ESCAPE comprises two investigational drug products, BEAM-103 and BEAM-104. The company intends to advance BEAM-103 and BEAM-104 for development in sickle cell disease and beta-thalassemia.
In the preclinical study, administration of the BEAM-104 edited cells to antibody-conditioned animals led to long-term engraftment.
Dosing with the BEAM-103 mAb led to rapid and near complete replacement of wild-type erythroid cells by edited cells, leading to early induction of therapeutically relevant levels of HbF.
BEAM-103 dosing was well tolerated, with no need for transfusions, antibiotics, or additional supportive care.
William Blair writes, "While the patient death is unfortunate, we see the event as highlighting the need for less-toxic preconditioning options, and we think Beam is leading the space in this regard."
Price Action: At last check on Monday, BEAM stock was up 0.52% at $26.89.
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