Beam Therapeutics Unveils New Safety, Efficacy Data From Sickle Cell Disease Candidate, Stock Gains
Beam Therapeutics Unveils New Safety, Efficacy Data From Sickle Cell Disease Candidate, Stock Gains
No VOCs were reported post-engraftment.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).
在星期六，beam therapeutics公司（纳斯达克：BEAM）公布了其BEACON 1/2期试验中关于BEAm-101在严重血管闭塞危机（VOCs）患者中安全性和有效性的新数据。
The data were featured at the American Society of Hematology (ASH) Annual Meeting and Exposition.
这些数据在美国血液学会（ASH）年会上展示。
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.
与beam之前公布的数据一致，七名接受试验性基础编辑疗法BEAm-101治疗的患者的更新数据展示了胎儿血红蛋白（HbF）的显著和持久增加，以及镰状血红蛋白（HbS）的减少、白细胞和血小板的快速植入以及溶血指标的正常化或改善。
No VOCs were reported post-engraftment.
植入后没有报告出现血管闭塞危机。
Key highlights include the following:
关键亮点包括：
- 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.
- 保护性胎儿血红蛋白（HbF）迅速且持续增加：所有患者的内源性HbF水平均超过60%，相应的镰状血红蛋白（HbS）降低到40%以下且稳定。在消除输血后观察到了HbF的全细胞分布。
- 稳健且持续的总血红蛋白（Hb）水平：在去除输注的血液后，患者的总血红蛋白水平迅速增加，贫血得到改善。
- 溶血指标正常化：包括间接胆红素、结合蛋白、乳酸脱氢酶和网织红细胞在内的溶血关键指标在所有患者接受BEAm-101治疗后均已正常或改善。
- 安全性剖面与布斯尔芬和自体造血干细胞移植一致。
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.
在11月，该公司表示有一名患者因呼吸衰竭去世，可能与布斯尔芬的预处理相关，此事件发生在BEAm-101输注四个月后。然而，研究者认为此死亡与BEAm-101无关。
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.
上周日，beam therapeutics宣布其工程化电芯抗体逃逸(ESCAPE)预处理平台的新数据，该数据在美国血液学会年会和博览会上展示。
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.
ESCAPE包含两个研究药物产品，BEAm-103和BEAm-104。该公司打算推进BEAm-103和BEAm-104在镰形细胞病和β-地中海贫血的开发。
In the preclinical study, administration of the BEAM-104 edited cells to antibody-conditioned animals led to long-term engraftment.
在临床前研究中，向抗体预处理动物施用BEAm-104编辑的电芯导致长期嵌合。
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.
使用BEAm-103单克隆抗体的剂量导致野生型红细胞被编辑电芯迅速且几乎完全取代，从而早期诱导出具有治疗相关水平的HbF。
BEAM-103 dosing was well tolerated, with no need for transfusions, antibiotics, or additional supportive care.
BEAm-103给药耐受良好，不需要输血、抗生素或额外的支持性护理。
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."
威廉·布莱尔写道："虽然患者的死亡是令人遗憾的，但我们认为这一事件突显了需要更少毒性的预处理期权，我们认为beam在这方面处于领先地位。"
Price Action: At last check on Monday, BEAM stock was up 0.52% at $26.89.
价格动态：最后一次检查时，beam therapeutics股票上涨了0.52%，报$26.89。
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译文内容由第三方软件翻译。
