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（納斯達克：BEAM）公佈了其BEACON 1/2期試驗中關於BEAm-101在嚴重血管閉塞危機（VOCs）患者中安全性和有效性的新數據。
The data were featured at the American Society of Hematology (ASH) Annual Meeting and Exposition.
這些數據在美國血液學會（ASH）年會上展示。
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.
與beam之前公佈的數據一致，七名接受試驗性基礎編輯療法BEAm-101治療的患者的更新數據展示了胎兒血紅蛋白（HbF）的顯著和持久增加，以及鐮狀血紅蛋白（HbS）的減少、白細胞和血小板的快速植入以及溶血指標的正常化或改善。
No VOCs were reported post-engraftment.
植入後沒有報告出現血管閉塞危機。
Key highlights include the following:
關鍵亮點包括：
- 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.
- 保護性胎兒血紅蛋白（HbF）迅速且持續增加：所有患者的內源性HbF水平均超過60%，相應的鐮狀血紅蛋白（HbS）降低到40%以下且穩定。在消除輸血後觀察到了HbF的全細胞分佈。
- 穩健且持續的總血紅蛋白（Hb）水平：在去除輸注的血液後，患者的總血紅蛋白水平迅速增加，貧血得到改善。
- 溶血指標正常化：包括間接膽紅素、結合蛋白、乳酸脫氫酶和網織紅細胞在內的溶血關鍵指標在所有患者接受BEAm-101治療後均已正常或改善。
- 安全性剖面與布斯爾芬和自體造血幹細胞移植一致。
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，該公司表示有一名患者因呼吸衰竭去世，可能與布斯爾芬的預處理相關，此事件發生在BEAm-101輸注四個月後。然而，研究者認爲此死亡與BEAm-101無關。
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.
上週日，beam therapeutics宣佈其工程化電芯抗體逃逸(ESCAPE)預處理平台的新數據，該數據在美國血液學會年會和博覽會上展示。
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包含兩個研究藥物產品，BEAm-103和BEAm-104。該公司打算推進BEAm-103和BEAm-104在鐮形細胞病和β-地中海貧血的開發。
In the preclinical study, administration of the BEAM-104 edited cells to antibody-conditioned animals led to long-term engraftment.
在臨床前研究中，向抗體預處理動物施用BEAm-104編輯的電芯導致長期嵌合。
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單克隆抗體的劑量導致野生型紅細胞被編輯電芯迅速且幾乎完全取代，從而早期誘導出具有治療相關水平的HbF。
BEAM-103 dosing was well tolerated, with no need for transfusions, antibiotics, or additional supportive care.
BEAm-103給藥耐受良好，不需要輸血、抗生素或額外的支持性護理。
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·布萊爾寫道："雖然患者的死亡是令人遺憾的，但我們認爲這一事件突顯了需要更少毒性的預處理期權，我們認爲beam在這方面處於領先地位。"
Price Action: At last check on Monday, BEAM stock was up 0.52% at $26.89.
價格動態：最後一次檢查時，beam therapeutics股票上漲了0.52%，報$26.89。
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譯文內容由第三人軟體翻譯。
