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—
— 在 400 mg bid 或 500 mg bid 的劑量口服 BCX9930 之後，100% 的治療天真患者和 83% 的 C5 反應不足的患者沒有輸血—
—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—
—未接受治療的患者的平均血紅蛋白從8.3 g/dL增加到11.8 g/dL，C5反應不足患者的平均血紅蛋白從8.9 g/dL增加到12.2 g/dL，這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的關鍵試驗和腎臟補體介導疾病的概念驗證試驗預計將於2021年下半年開始—
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.
北卡羅來納州三角研究園，2021 年 3 月 22 日（GLOBE NEWSWIRE）— BioCryst 製藥有限公司 （納斯達克：BCRX）今天宣佈，在一項正在進行的劑量範圍試驗中，其口服D因子抑制劑 BCX9930 顯著增加了血紅蛋白並減少了輸血，該試驗針對的是未接受過治療（以前沒有使用C5抑制劑進行治療）陣發性夜間血紅蛋白尿症（PNH）患者以及對C5抑制劑反應不足的PNH患者。在試驗中，BCX9930 是安全的，總體耐受性良好。
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.
基於這些結果以及最近與美國和歐洲監管機構的互動，該公司計劃在2021年下半年直接進入PNH的關鍵試驗和腎臟補體介導疾病的概念驗證試驗。
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.
試驗中的PNH患者在以400 mg bid或500 mg的出價給藥後，關鍵的實驗室生物標誌物也有所減少，例如網織紅細胞數量、乳酸脫氫酶（LDH）（天真治療的患者）和C3選擇化百分比（C5反應不足的患者）。
“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.
意大利阿韋利諾聖朱塞佩·莫斯卡蒂醫院醫學博士、博士安東尼奧·裏西塔諾說：“在這項使用口服藥物的試驗中，輸血量顯著減少，血紅蛋白的增加解決了患者和醫生未得到滿足的需求——一種通過控制血管內和血管外溶血可以最大限度地提高血液學益處的PNH療法，” 也是該審判的首席調查員。
“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.
他補充說：“這些結果使我感到非常鼓舞，這些結果可能會使近端抑制劑，尤其是這種口服抗因子D藥物，成爲改變PNH以及可能改變其他替代途徑介導疾病的治療模式的藥物。”
譯文內容由第三人軟體翻譯。