Transthyretin Amyloid Cardiomyopathy

- In this exploratory substudy, treatment with acoramidis was associated with possible cardiac structural and functional improvement compared with placebo, with potential cardiac amyloid regression - The data demonstrate that targeting near-complete transthyretin (TTR) stabilization with acoramidis may enable cardiac remodeling and functional recovery in patients with ATTR-CM - These results are the first prospective, longitudinal evaluation of cardiac structure and function by CMR imaging in a double-blind, placebo-controlled, interventional study in ATTR-CM - The findings from this study build upon positive results from BridgeBio’s global ATTRibute-CM Phase 3 trial, wherein the primary endpoint was met (Win Ratio of 1.8) with a high statistical significance (p<0.0001); the substudy data are consistent with the cardiovascular clinical benefits observed with acoramidis - Acoramidis was well-tolerated, with no safety signals of potential clinical concern observed - BridgeBio’s New Drug Application (NDA) has been accepted by the U.S. Food and Drug Administration (FDA) with a PDUFA action date of November 29, 2024; a Marketing Authorization Application (MAA) for acoramidis has been accepted by the European Medicines Agency (EMA), with an expected decision in 2025 PALO ALTO, Calif., April 07, 2024 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, today presented results from the exploratory CMR imaging substudy of ATTRibute-CM, its Phase 3 trial of acoramidis in ATTR-CM. These data were presented at the American College of Cardiology (ACC) Annual Scientific Sessions & Expo in a moderated poster session by Yousef Razvi, M.D. of University College London. ATTRibute-CM was designed to study the efficacy and safety of acoramidis, an investigational, next-generation, orally-administered, highly potent, small molecule stabilizer of TTR. Based on the positive results from ATTRibute-CM, BridgeBio submitted an NDA to the U.S. FDA, which has been accepted with a PDUFA action date of November 29, 2024, and an MAA to the European Medicines Agency, with a decision expected in 2025. “CMR is the reference non-invasive imaging method to evaluate cardiac structure, function, and amyloid burden in patients with ATTR-CM. The results shown in the imaging substudy are indicative of potential improvement of cardiac structure and function in patients with ATTR-CM, consistent with the clinical outcomes with acoramidis treatment observed in the ATTRibute-CM Phase 3 clinical trial. The impact on cardiac amyloid load highlights the potential that acoramidis could lead to cardiac amyloid regression in a proportion of patients with ATTR-CM, which we have not observed in such a controlled clinical trial to date,” said Marianna Fontana, M.D., Ph.D., Professor of Cardiology and Honorary Consultant Cardiologist at the National Amyloidosis Centre, Division of Medicine, University College London, and Principal Investigator of the Substudy. CMR imaging was performed at baseline before the first dose in 35 participants or within three months after the first dose in 17 participants (range, 14-105 days); subsequent CMR imaging was performed at months 12, 24, and 30 in those individuals who were available to undergo imaging, which was influenced by survival. Images were read centrally at the National Amyloidosis Centre in a fashion blinded to other clinical data. Findings included: Key CMR imaging parameters measuring cardiac structure, including mean indexed left ventricular mass, were found to be stable or have a trend towards improvement on acoramidis vs. deteriorated on placebo over 30 monthsTreatment with acoramidis preserved or was associated with a trend towards improvement in measures of cardiac function including left ventricular ejection fraction and stroke volume compared to initial measures, and relative to placebo In July 2023, BridgeBio announced positive results from ATTRibute-CM, reporting a highly statistically significant result, demonstrated by a Win Ratio of 1.8 (p<0.0001) on the primary endpoint (a hierarchical analysis prioritizing in order: all-cause mortality, then frequency of cardiovascular hospitalization, then change from baseline in N-terminal prohormone of brain natriuretic peptide, then change from baseline in 6-minute walk distance). Acoramidis was well-tolerated, with no safety signals of potential clinical concern identified. BridgeBio has also presented analyses from ATTRibute-CM at the European Society of Cardiology Congress 2023 and at the American Heart Association Scientific Sessions 2023. In February 2024, BridgeBio shared positive results of a single-arm Phase 3 study of acoramidis in Japanese patients with ATTR-CM, including no mortality observed in the trial at 30 months. About BridgeBio Pharma, Inc.BridgeBio Pharma Inc. (BridgeBio) is a commercial-stage biopharmaceutical company founded to discover, create, test and deliver transformative medicines to treat patients who suffer from genetic diseases and cancers with clear genetic drivers. BridgeBio’s pipeline of development programs ranges from early science to advanced clinical trials. BridgeBio was founded in 2015 and its team of experienced drug discoverers, developers, and innovators are committed to applying advances in genetic medicine to help patients as quickly as possible. For more information visit bridgebio.com and follow us on LinkedIn and Twitter. BridgeBio Pharma, Inc. Forward-Looking StatementsThis press release contains forward-looking statements. Statements in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended (the Securities Act), and Section 21E of the Securities Exchange Act of 1934, as amended (the Exchange Act), which are usually identified by the use of words such as “anticipates,” “believes,” “estimates,” “expects,” “intends,” “may,” “plans,” “projects,” “seeks,” “should,” “continue,” “will,” and variations of such words or similar expressions. We intend these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Exchange Act. These forward-looking statements, including statements relating to the clinical and therapeutic potential of our programs and product candidates, including our clinical development program for acoramidis for patients with transthyretin amyloid cardiomyopathy, the timing and success of our clinical development programs, the progress of our ongoing and planned clinical trials of acoramidis for patients with transthyretin amyloid cardiomyopathy, including the expectations of receiving a PDUFA action from the FDA on November 29, 2024 and receiving a decision from the EMA on the MAA for acoramidis in 2025, the expected intellectual property protection of acoramidis, our planned interactions with regulatory authorities, the statements regarding the potential clinical benefits or of potential benefits for ATTR-CM patients in the quotes of Dr. Fontana, and the timing of these events, reflect our current views about our plans, intentions, expectations and strategies, which are based on the information currently available to us and on assumptions we have made. Although we believe that our plans, intentions, expectations, and strategies as reflected in or suggested by those forward-looking statements are reasonable, we can give no assurance that the plans, intentions, expectations, or strategies will be attained or achieved. Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a number of risks, uncertainties and assumptions, including, but not limited to, initial and ongoing data from our clinical trials not being indicative of final data, the design and success of ongoing and planned clinical trials, difficulties with enrollment in our clinical trials, adverse events that may be encountered in our clinical trials, the FDA or other regulatory agencies not agreeing with our regulatory approval strategies, components of our filings, such as clinical trial designs, conduct and methodologies, or the sufficiency of data submitted, potential adverse impacts due to the global COVID-19 pandemic such as delays in regulatory review, manufacturing and supply chain interruptions, adverse effects on healthcare systems and disruption of the global economy, the impacts of current macroeconomic and geopolitical events, including changing conditions from the COVID-19 pandemic, hostilities in the Middle East and Ukraine, increasing rates of inflation and rising interest rates, on our overall business operations and expectations, as well as those risks set forth in the Risk Factors section of our Annual Report on Form 10-K for the year ended December 31, 2023 and our other filings with the U.S. Securities and Exchange Commission. Moreover, we operate in a very competitive and rapidly changing environment in which new risks emerge from time to time. These forward-looking statements are based upon the current expectations and beliefs of our management as of the date of this press release, and are subject to certain risks and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. Except as required by applicable law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise. BridgeBio Media Contact:Vikram Balicontact@bridgebio.com (650)-789-8220

Bayer will be letting go of 90 employees at its Whippany, New Jersey site, the company revealed this week in a Worker Adjustment and Retraining Notification (WARN). Bayer opened its U.S. headquarters on the 94-acre campus in Whippany back in 2013. The headquarters consolidated Bayer HealthCare’s operations that had previously been spread across Morristown, Montville, and Wayne, New Jersey, as well as Tarrytown, New York, into one campus. The layoff news comes just a few months after the drugmaker announced a significant workforce reduction at its German sites. At the time, the company also unveiled a new global operating model, "Dynamic Shared Ownership," which has the aim of “improving agility and operational efficiency by reducing hierarchies, eliminating bureaucracy, and expediting decision-making processes.” Earlier this month, Bayer secured exclusive rights to commercialize BridgeBio's promising heart disease drug, acoramidis, in Europe, as the drug awaits approval from regulators. Acoramidis, an experimental treatment for the rare and deadly heart condition ATTR-CM, could significantly impact heart failure outcomes. Bayer's deal with BridgeBio includes payments up to $310 million, covering initial, milestone payments, plus royalties.

药渡数据-「全球新药批准月报」真实/可溯源/紧贴业内需求 012024年2月全球/中国新药批准动态及重磅药物解析 022024年2月全球/中国新药申报上市动态及重磅药物解析Part 1全球/中国新药批准动态01全球/中国新药2月批准概览2024年2月全球新药批准共计26个，其中中国新药批准共计8个。本月全球批准新药中，血液和淋巴系统及肿瘤领域占比均为19%，涉及多发性骨髓瘤，胆道癌及非小细胞肺癌等；其次是免疫和消化系统，适应症包括溃疡性结肠炎及短肠综合征等。本月中国批准新药中，血液和淋巴系统占比最大，达25%，适应症涉及阵发性睡眠性血红蛋白尿症，获得性血友病A等。02全球/中国新药2月批准详情全球/中国NDA批准详情（部分）全球/中国BLA批准详情全球/中国扩展适应症批准详情（部分）全球/中国新剂型批准详情（部分）全球/中国新复方批准详情（部分）03重磅批准品种分析Crovalimab（可伐利单抗）可伐利单抗是罗氏公司研发的一种经过工程化改造的新一代C5抑制剂。其主要作用是阻断补体C5裂解为C5a和C5b，从而有望有效地抑制补体的活化过程。这一药物采用了等电点、新生儿Fc受体（FcRn）以及pH依赖性亲和性技术，这使得它不仅能够与C5迅速而高效地结合，从而增强内皮细胞对与C5结合的可伐利单抗的吸收效果，同时还通过新生儿Fc受体介导的机制实现可伐利单抗的高效再循环。阵发性睡眠性血红蛋白尿症（PNH）是一种罕见且危及生命的血液疾病，全球约2万名患者。在未接受补体抑制剂治疗的情况下，患者的五年内死亡的风险达35%。患者临床表现包括红细胞会被补体系统破坏，导致贫血、疲劳、血栓和肾脏相关等并发症。派圣凯®在中国的获批，是其在全球所有国家中的首次获批，这也是罗氏集团第一次在中国市场实现一款创新物的全球首发。03重磅药物解析2024年3月可能获批药物预测预计2024年3月FDA可能批准药物（部分）Part 22024年2月全球/中国新药申报上市动态01全球/中国新药申报上市情况概览2024年2月全球新药NDA/BLA项目共计49个，其中中国NDA/BLA项目24个；特殊资格认定申报项目共计47个，其中中国申报项目8个。本月海外NDA/BLA申报项目主要集中肿瘤36%、血液和淋巴系统16%及神经系统12%等领域，中国NDA/BLA申报项目主要集中肿瘤25%及感染13%等领域。本月全球特殊资格认定申报项目共计47个（中国8个），主要集中肿瘤占比为32%、血液和淋巴系统24%及神经系统15%，细分适应症包括杜氏肌营养不良，非细胞肺癌及多发性骨髓瘤。02全球新药注册申报情况2024年2月全球NDA申报情况（部分）2024年2月全球BLA申报情况（部分）2024年2月全球特殊资格认定申报情况（部分）03中国新药注册申报情况2024年2月中国NDA申报情况（部分）2024年2月中国BLA申报情况（部分）2024年2月中国特殊资格认定申报情况（部分）04重磅申报品种分析AcoramidisAcoramidis是BridgeBio Pharma研发的一种新型可口服的高效转甲状腺素蛋白（TTR）小分子稳定剂，旨在有效稳定四聚体转甲状腺素蛋白 (TTR)，从一开始阻止导致TTR淀粉样变性 (ATTR) 的一系列分子事件。转甲状腺素蛋白淀粉样变心肌病（ATTR-CM）是由于不稳定的转甲状腺素蛋白(TTR)四聚体解离成单体后错误折叠为淀粉样物质,淀粉样物质沉积于心肌间质所致，ATTR-CM是一种限制性心肌病，会导致心力衰竭，通常伴有射血分数保留。Ø30个月时分析的主要终点，主要分析以分层方式比较了四种结果：全因死亡率、心血管相关住院、N 端 B 型利钠肽原 (NT-proBNP) 相对于基线的变化以及步行6分钟距离（6MWD）。Ø研究共入组 632 名患者，主要分层终点分析具有高度显著的统计学差异，主要终点的Win Ratio为1.8（95% CI 1.4-2.2；p<0.0001）。咨询、合作请联系作者小D有话说为方便读者阅读及保存，我们将周报原文整理成了PDF版本，如需获取全文，可点击最上方蓝字，在药渡Daily公众号后台回复“2月批准月报”，即可下载。