文章核心观点 - 公司的主要产品acoramidis在ATTR-CM患者中显示出显著的疗效,包括降低死亡率、住院率和改善生活质量等 [5][6] - 公司正在积极推进多个III期临床试验的全面入组,包括acoramidis、encaleret、BBP-418和infigratinib等,预计2025年将有多个产品获得关键性试验结果 [3][4][6] - 公司通过最近的融资活动,现金储备充足,为后续产品商业化和管线推进提供了有力支持 [7] 财务表现 - 公司Q2营收为2.168百万美元,较上年同期增长32% [8][9][10] - 运营成本和费用同比增加30百万美元,主要用于支持商业化准备和管线推进 [11][12][13] - 其他收益大幅增加,主要来自于子公司脱离合并的收益 [18][19][20] - 归属于普通股东的净亏损同比大幅收窄 [21][22] 管线进展 Acoramidis (ATTR-CM) - 在国际淀粉样蛋白研讨会和欧洲心力衰竭学会大会上公布了多项积极的临床数据分析结果 [5][6] - 包括显著降低复合终点事件风险、改善生命质量等 [5][6] - 公司计划今年内启动预防性试验ACT-EARLY [6] Encaleret (ADH1) - III期CALIBRATE试验正在入组,预计2025年公布关键结果 [4] BBP-418 (LGMD2I/R9) - III期FORTIFY试验入组进度超预期,计划进行中期分析 [4][6] - 获得罕见儿童疾病指定,有望获得优先审评券 [6] Infigratinib (Achondroplasia/Hypochondroplasia) - II期PROPEL 2试验Cohort 5数据显示显著提高年身高速度和改善身材比例 [6] - 获得快速通道和罕见儿童疾病指定,有望获得优先审评券 [6] - 启动观察性前导试验ACCEL,为III期试验铺平道路 [6]

PALO ALTO, Calif., July 23, 2024 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (NASDAQ:BBIO) ("BridgeBio" or the "Company"), a commercial-stage biopharmaceutical company focused on genetic diseases, announced that Thomas Trimarchi, Ph.D., has been appointed President and Chief Operating Officer (COO) of the company. Dr. Trimarchi will assume his new responsibilities effective immediately and will continue to report directly to CEO and founder, Neil Kumar, Ph.D. In this newly created position, Dr. Trimarchi wil ...

- BridgeBio has surpassed its interim analysis enrollment target for its Phase 3 FORTIFY study of BBP-418 in individuals living with LGMD2I/R9, with top-line results from the interim analysis expected in 2025 - Recent Type C interactions with U.S. Food and Drug Administration (FDA) focused on the validated glycosylated alpha-dystroglycan (αDG) bioassay and our interim analysis plans reinforce BridgeBio's belief that there is potential to pursue Accelerated Approval for BBP-418 - Rare Pediatric Disease Desig ...

NEW YORK, June 13, 2024 (GLOBE NEWSWIRE) -- Pomerantz LLP is investigating claims on behalf of investors of BridgeBio Pharma, Inc. (“BridgeBio” or the “Company”) (NASDAQ: BBIO). Such investors are advised to contact Danielle Peyton at newaction@pomlaw.com or 646-581-9980, ext. 7980. The investigation concerns whether BridgeBio and certain of its officers and/or directors have engaged in securities fraud or other unlawful business practices. [Click here for information about joining the class action] On June ...

NEW YORK, June 05, 2024 (GLOBE NEWSWIRE) -- Pomerantz LLP is investigating claims on behalf of investors of BridgeBio Pharma, Inc. (“BridgeBio” or the “Company”) (NASDAQ: BBIO). Such investors are advised to contact Danielle Peyton at newaction@pomlaw.com or 646-581-9980, ext. 7980. The investigation concerns whether BridgeBio and certain of its officers and/or directors have engaged in securities fraud or other unlawful business practices. [Click here for information about joining the class action] On June ...

- In Cohort 5 of PROPEL 2 (0.25 mg/kg/day), oral treatment with infigratinib resulted in a statistically significant and sustained increase in annualized height velocity (AHV), with a mean change from baseline of +2.51cm/yr at Month 12, and +2.50 cm/yr at Month 18 (p=0.0015) - At Month 18, there was a statistically significant improvement in body proportionality (p-value of 0.001). The mean upper to lower body segment ratio was 1.88 at Month 18, as compared to 2.02 at baseline - Infigratinib continues to be ...

- Acoramidis treatment resulted in increased serum transthyretin (TTR) levels by Day 28 that were sustained and were correlated with a reduced risk of all-cause mortality (ACM), cardiovascular mortality (CVM), and cardiovascular-related hospitalization (CVH) in ATTR-CM participants through month 30 - Acoramidis treatment resulted in a significant improvement in the composite endpoint of CVM and CVH in ATTR-CM participants, with benefit evident as early as Month 3 - In ATTRibute-CM, participants with at leas ...

- Acoramidis treatment resulted in increased serum transthyretin (TTR) levels by Day 28 that were sustained and were correlated with a reduced risk of all-cause mortality (ACM), cardiovascular mortality (CVM), and cardiovascular-related hospitalization (CVH) in ATTR-CM participants through month 30 - Acoramidis treatment resulted in a significant improvement in the composite endpoint of CVM and CVH in ATTR-CM participants, with benefit evident as early as Month 3 - In ATTRibute-CM, participants with at leas ...

- BridgeBio to host investor call on Wednesday, May 29, 2024 at 5:30 pm ET, with presentations from Mathew Maurer, M.D. of Columbia University Irving Medical Center, U.S. and Ahmad Masri, M.D., M.S. of Oregon Health & Science University, U.S. PALO ALTO, Calif., May 24, 2024 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, announced today that 12 oral and moderated poster presentatio ...

- BridgeBio to host investor call on Wednesday, May 29, 2024 at 5:30 pm ET, with presentations from Mathew Maurer, M.D. of Columbia University Irving Medical Center, U.S. and Ahmad Masri, M.D., M.S. of Oregon Health & Science University, U.S. PALO ALTO, Calif., May 24, 2024 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, announced today that 12 oral and moderated poster presentatio ...