BridgeBio Pharma Seeks Partner To Develop Gene Therapy For Inherited Condition
BridgeBio Pharma Seeks Partner To Develop Gene Therapy For Inherited Condition
On Tuesday, BridgeBio Pharma, Inc. (NASDAQ:BBIO) released topline results from the Phase 1/2 open-label ADventure study of BBP-631, an investigational adeno-associated virus (AAV) 5 gene therapy, for congenital adrenal hyperplasia (CAH).
週二，BridgeBio Pharma, Inc.（納斯達克股票代碼：BBIO）公佈了針對先天性腎上腺增生（CAH）的研究性腺相關病毒（AAV）5基因療法 BBP-631 的1/2期開放標籤研究的主要結果。
CAH is a rare, inherited disorder that affects the adrenal glands and causes a hormone imbalance. The adrenal glands are on top of the kidneys and produce hormones that help the body function.
CAH 是一種罕見的遺傳性疾病，會影響腎上腺並導致激素失衡。腎上腺位於腎臟頂部，產生有助於身體機能的激素。
Also Read: Why Is BridgeBio Pharma Stock Trading Higher On Friday?
另請閱讀：爲什麼週五BridgeBio Pharma的股票交易價格上漲？
To date, key results from the study include:
迄今爲止，該研究的主要結果包括：
- Increased endogenous cortisol production was achieved in all patients at higher doses.
- At the two highest dose levels, a maximum change from the baseline post-ACTH stimulation test of 4.7 μg/dL and 6.6 μg/dL was observed, respectively, with cortisol levels as high as 11 μg/dL achieved.
- Substantial and durable increases in 11-deoxycortisol, the product of 21-hydroxylase, and reductions in 17-hydroxyprogesterone (17-OHP), the substrate of 21-hydroxylase, provide compelling evidence of durable BBP-631 transgene activity. At the highest dose levels, sustained 11-deoxycortisol averaged a 55-fold increase from baseline, with a maximum of 99-fold increase. These represent an average maximum of 23-fold the upper limit of normal.
- Robust reduction in 17-hydroxyprogesterone, with most patients reaching a reduction of ≥50%, with a max reduction of 95%.
- 在較高劑量下，所有患者的內源性皮質醇產量均有所增加。
- 在兩個最高劑量水平下，觀察到與ACTH刺激後的基線測試相比的最大變化分別爲4.7 μg/dL和6.6 μg/dL，皮質醇水平高達11 μg/dL。
- 21-羥化酶的產物 11-脫氧皮質醇的大幅持續增加，以及21-羥化酶的底物17-羥孕酮（17-OHP）的減少爲持久的 BBP-631 轉基因活性提供了令人信服的證據。在最高劑量水平下，持續的11-脫氧皮質醇平均比基線增加55倍，最大增加99倍。這表示平均最大值是正常上限的23倍。
- 17-羥孕酮大幅降低，大多數患者降低 ≥ 50%，最大降幅爲 95%。
BBP-631 has been well tolerated, with only mild to moderate treatment-emergent adverse events (TEAEs), and no treatment-related SAEs have been reported.
BBP-631 耐受性良好，僅出現輕度至中度治療緊急不良事件 (TEAE)，沒有報告與治療相關的急性不良事件。
"While the data to date are not yet transformational, the study showed for the first time that people living with CAH can indeed make their own cortisol, and that gene therapy can be safely administered in this patient population..." said Neil Kumar, CEO and Founder of BridgeBio.
BridgeBio首席執行官兼創始人尼爾·庫馬爾說：「儘管迄今爲止的數據尚未帶來變革，但該研究首次表明，CAH患者確實可以自己製造皮質醇，並且可以安全地對這些患者群體進行基因療法...」
BridgeBio also said it will no longer be pursuing development of BBP-631 for CAH and is seeking partnership opportunities to support future development of BBP-631 or next-generation gene therapies for the treatment of CAH.
BridgeBio還表示，它將不再尋求用於CAH的 BBP-631 的開發，並正在尋求合作機會，以支持用於治療CAH的 BBP-631 或下一代基因療法的未來開發。
Price Action: BBIO stock is down 3.07% at $29.07 at the last check on Wednesday.
價格走勢：在週三的最後一次支票中，BBIO股價下跌3.07％，至29.07美元。
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譯文內容由第三人軟體翻譯。