BioCryst's Oral Factor D Inhibitor, BCX9930, Advancing to Pivotal Trials in PNH Following Successful Proof of Concept Trial
BioCryst's Oral Factor D Inhibitor, BCX9930, Advancing to Pivotal Trials in PNH Following Successful Proof of Concept Trial
—Following doses of 400 mg bid or 500 mg bid of oral BCX9930, 100 percent of treatment-naïve patients and 83 percent of C5 inadequate response patients were transfusion-free—
-服用400毫克BID或500毫克BID口服BCX9930后，100%的治疗幼稚患者和83%的C5反应不足的患者无需输血-
—Mean hemoglobin increased from 8.3 g/dL to 11.8 g/dL in treatment-naïve patients and from 8.9 g/dL to 12.2 g/dL in C5 inadequate response patients, demonstrating control of hemolysis—
-平均血红蛋白在未接受治疗的患者中从8.3 g/dL增加到11.8 g/dL，在C5反应不足的患者中从8.9 g/dL增加到12.2g/dL，表明溶血得到了控制-
—Pivotal trials in PNH and proof of concept trials in renal complement-mediated disease expected to begin in 2H 2021 —
-PNH的关键试验和肾脏补体介导的疾病的概念验证试验预计将于2021年下半年开始-
RESEARCH TRIANGLE PARK, N.C., March 22, 2021 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX) today announced that its oral Factor D inhibitor, BCX9930, significantly increased hemoglobin and reduced transfusions in an ongoing dose-ranging trial in treatment-naïve (no prior treatment with C5 inhibitors) paroxysmal nocturnal hemoglobinuria (PNH) patients, and in PNH patients with an inadequate response to C5 inhibitors. BCX9930 was safe and generally well-tolerated in the trial.
北卡罗来纳州研究三角公园，2021年3月22日(环球通讯社)BioCryst制药公司(BioCryst PharmPharmticals，Inc.)(纳斯达克：BCRX)今天宣布，其口服D因子抑制剂BCX9930在一项正在进行的剂量范围试验中显著增加血红蛋白并减少输血，该试验用于治疗幼稚(事先未使用C5抑制剂治疗)阵发性睡眠性血红蛋白尿(PNH)患者以及对C5抑制剂反应不足的PNH患者。BCX9930在试验中是安全的，总体耐受性良好。
Based on these results, and recent interactions with U.S. and European regulators, the company plans to advance directly into pivotal trials in PNH and proof of concept trials in renal complement-mediated diseases in the second half of 2021.
基于这些结果，以及最近与美国和欧洲监管机构的互动，该公司计划在2021年下半年直接进入PNH的关键试验和肾脏补体介导的疾病的概念验证试验。
PNH patients in the trial also experienced reductions in key laboratory biomarkers, such as reticulocyte count, lactate dehydrogenase (LDH) (treatment-naïve patients) and percentage of C3 opsonization (patients with inadequate C5 response) following dosing at 400 mg bid or 500 mg bid.
试验中的PNH患者在服用400毫克BID或500毫克BID后，还经历了关键实验室生物标记物的减少，如网织红细胞计数、乳酸脱氢酶(LDH)(未接受治疗的患者)和C3调理百分比(C5反应不充分的患者)。
“The significant reduction in transfusions and increases in hemoglobin seen in this trial with an oral medicine address an unmet need for patients and physicians -- a PNH therapy that can maximize hematological benefit through the control of both intravascular and extravascular hemolysis,” said Antonio Risitano, M.D., Ph.D., San Giuseppe Moscati Hospital, Avellino, Italy, and principal investigator of the trial.
安东尼奥·里西塔诺(Antonio Risitano)、医学博士、博士、该试验的主要研究员安东尼奥·里西塔诺(Antonio Risitano)说：“在口服药物的试验中，输血的显著减少和血红蛋白的增加解决了患者和医生尚未得到满足的需求--这是一种PNH疗法，可以通过控制血管内和血管外溶血来最大限度地提高血液学效益。”安东尼奥·里西塔诺是意大利阿维利诺的圣朱塞佩·莫斯卡蒂医院(San Giuseppe Moscati Hospital)的医学博士和该试验的主要研究员。
“I am very encouraged by these results which may position proximal inhibitors, and in particular this oral anti-Factor D agent, as medications changing the treatment paradigm of PNH, and possibly of other alternative pathway mediated diseases,” he added.
他补充说：“我对这些结果感到非常鼓舞，这些结果可能会定位近端抑制剂，特别是这种口服抗D因子药物，因为药物改变了PNH的治疗模式，可能还改变了其他替代途径介导的疾病的治疗模式。”
译文内容由第三方软件翻译。