Uniqure's Hemophilia B Gene Therapy Unlikely To Be Cause Of Liver Cancer Reported In One Patient
Uniqure's Hemophilia B Gene Therapy Unlikely To Be Cause Of Liver Cancer Reported In One Patient
Uniqure NV (NASDAQ: QURE) has reported results of a comprehensive investigation into the case of hepatocellular carcinoma (HCC) diagnosed in one patient in the HOPE-B pivotal trial of etranacogene dezaparvovec, its hemophilia B gene therapy candidate.
What Happened: The trial was put on clinical last year in December, following the submission of a safety report in mid-December relating to a possibly related severe adverse event associated with a preliminary diagnosis of hepatocellular carcinoma in one patient.
The results of the investigation found that it is implausible the HCC was caused by etranacogene dezaparvovec.
Multiple analyses conducted by an independent laboratory and reviewed by external experts in the field show that AAV vector integration in the patient's tissue sample was extremely rare and accounted for 0.027% of the cells in the sample.
Why It Matters: The whole-genome sequencing of the tumor confirmed that the patient had several genetic mutations characteristic of HCC and independent of vector integration.
Finally, gene expression analysis of the tumor and adjacent tissue suggested a precancerous state in the liver consistent with several risk factors that predispose this patient to HCC.
The external lab analyzed more than 220,000 cells from the tissue sample and identified 60 cells with random integration events that have no known association with the development of HCC," stated Ricardo Dolmetsch, uniQure president of research and development.
The company has shared the data with the FDA and is prepared to have further communications regarding the clinical hold status in the second quarter of 2021.
All patients in uniQure's hemophilia B gene therapy program, including the 54 patients in HOPE-B, have now had abdominal ultrasounds performed one year after dosing. Each will continue to be monitored by their care teams.
No other cases of HCC have been reported in uniQure clinical trials conducted in more than 100 patients in hemophilia B and other indications, with some patients dosed more than ten years ago.
Price Action: QURE shares are trading 9.47% higher at $32.01 in the premarket session on the last check Monday.
Uniqure NV (NASDAQ: QURE) has reported results of a comprehensive investigation into the case of hepatocellular carcinoma (HCC) diagnosed in one patient in the HOPE-B pivotal trial of etranacogene dezaparvovec, its hemophilia B gene therapy candidate.
UnQure NV(納斯達克：Qure)有報告了一項全面調查的結果在血友病B基因治療候選藥物etranacogene dezparvovec的HOPE-B關鍵試驗中，一名患者被診斷為肝細胞癌(HCC)。
What Happened: The trial was put on clinical last year in December, following the submission of a safety report in mid-December relating to a possibly related severe adverse event associated with a preliminary diagnosis of hepatocellular carcinoma in one patient.
發生的事情：這個試驗於去年12月進行臨牀試驗。在12月中旬提交了一份安全報告後，該報告涉及一名患者初步診斷為肝細胞癌可能相關的嚴重不良事件。
The results of the investigation found that it is implausible the HCC was caused by etranacogene dezaparvovec.
調查結果發現，肝細胞癌不可能是由四疊紀去扎帕沃韋克引起的。
Multiple analyses conducted by an independent laboratory and reviewed by external experts in the field show that AAV vector integration in the patient's tissue sample was extremely rare and accounted for 0.027% of the cells in the sample.
由獨立實驗室進行並經該領域外部專家審查的多項分析表明，患者組織樣本中的AAV載體整合極為罕見，佔樣本細胞的0.027%。
Why It Matters: The whole-genome sequencing of the tumor confirmed that the patient had several genetic mutations characteristic of HCC and independent of vector integration.
重要的原因：腫瘤的全基因組測序證實，患者有幾個肝癌特有的基因突變，與載體整合無關。
Finally, gene expression analysis of the tumor and adjacent tissue suggested a precancerous state in the liver consistent with several risk factors that predispose this patient to HCC.
最後，腫瘤和鄰近組織的基因表達分析表明，肝臟處於癌前狀態，這與該患者易患肝癌的幾個危險因素一致。
The external lab analyzed more than 220,000 cells from the tissue sample and identified 60 cells with random integration events that have no known association with the development of HCC," stated Ricardo Dolmetsch, uniQure president of research and development.
外部實驗室分析了組織樣本中的22萬多個細胞，鑑定出60個存在隨機整合事件的細胞，這些細胞與HCC的發展沒有已知的聯繫，“uniQure研究和開發總裁裏卡多·多爾梅奇(Ricardo Dolmetsch)説。
The company has shared the data with the FDA and is prepared to have further communications regarding the clinical hold status in the second quarter of 2021.
該公司已經與FDA分享了數據，並準備在2021年第二季度就臨牀持有狀態進行進一步溝通。
All patients in uniQure's hemophilia B gene therapy program, including the 54 patients in HOPE-B, have now had abdominal ultrasounds performed one year after dosing. Each will continue to be monitored by their care teams.
UnQure血友病B基因治療計劃中的所有患者，包括HOPE-B中的54名患者，現在都在服藥一年後接受了腹部超聲波檢查。每名患者都將繼續接受其護理團隊的監測。
No other cases of HCC have been reported in uniQure clinical trials conducted in more than 100 patients in hemophilia B and other indications, with some patients dosed more than ten years ago.
在對100多名血友病B患者和其他適應症患者進行的UnQure臨牀試驗中，沒有其他肝癌病例的報告，有些患者在十多年前就服用了藥物。
Price Action: QURE shares are trading 9.47% higher at $32.01 in the premarket session on the last check Monday.
價格行動：在週一的最後一次檢查中，Qure股價在盤前交易時段上漲9.47%，至32.01美元。
譯文內容由第三人軟體翻譯。
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