Ribitol

📌 今日要点速览 🧬 SION — SION-719 囊性纤维化 IIa 期试验失败，汗液氯化物仅降 -1.0 mmol/L（p=0.7），股价暴跌约 90% 至 。 竞争威胁解除， 当日 530.83 💊 TENAX — 口服左西孟旦（TNX-103）III 期 LEVEL 试验未达主要终点，股价 -84% 至 $2，计划寻求 FDA Type C 会议 🤝 JAZZ — 以 亿首付 5 亿里程碑收购 Actio Biosciences，获得 KCNT1 癫痫管线 ABS-1230 📊 BBIO — Q2 营收 亿（ 2.224 亿，三个管线 PDUFA 日期确定（11/27、5/8/27、待定） 🎯 COGT — Q2 财报，bezuclastinib GIST PDUFA 11/30，AdvSM NDA 已提交（6/30），现金 $8.659 亿 🧠 BHVN — Q2 财报，BHV-1300 Graves' 病关键试验启动，MoDE/TRAP 平台多管线推进 💰 APGE — Q2 净亏 万， 收购待完成（135.11/股，约 $109 亿），APEX IIb 期阳性 🔬 SANA — Q2 现金 $1.605 亿，SC451 IND 年内申报，SG293 首次人体数据年内读出 🎯 ORKA — Q2 净亏 $4120 万，EVERLAST-A Week 28 数据 Q3 末读出，III 期计划 2027 H1 🧬 VERA — Q2 净亏 $1.095 亿，IgAN ORIGIN 3 最终分析 Q3，sBLA Q4 申报 💊 OLMA — Q2 净亏 $6320 万，palazestrant III 期推进中，OP-3136 I 期进行 📊 CATX — Q2 财报，VMT-α-NET III 期 site activation 目标年底，现金 $2.37 亿 📊 板块总览 指标 数值 XBI 收盘价 $158.03 日涨跌幅 +$0.66 (+0.42%) ▲ 日内区间 158.55 成交量 ~745 万股 XBI 周一微涨 0.42%，连续第六个交易日收阳。板块内部呈现"财报驱动分化"格局：Q2 财报密集发布带动多只中市值股波动，同时 SION 和 TENAX 两只临床失败股暴跌形成鲜明对比。JAZZ 收购 Actio 标志着私有生物科技 M&A 浪潮延续。 💡 市场影响分析：8/10 是 XBI 财报季最密集的交易日之一，12 家成分股同日发布 Q2 业绩或重大事件。SION 临床失败释放的资金和注意力部分流向 VRTX（+7%）和其他 CF 领域，板块整体保持稳健但个股分化加剧。 新闻总表 公司 事件 影响 SION SION-719 IIa 期 CF 试验失败 ▲▼ 股价 -90%+ TENAX TNX-103 III 期 LEVEL 试验失败 ▼ 股价 -84% JAZZ 收购 Actio Biosciences 500M M&A BBIO Q2 营收 $243.7M (+120%) 财报 COGT Q2 财报 + bezuclastinib PDUFA 11/30 财报+催化 VERA Q2 财报 + ORIGIN 3 最终分析 Q3 财报+催化 OLMA Q2 财报 + palazestrant III 期推进 财报 BHVN Q2 财报 + BHV-1300 关键试验启动 财报+管线 SANA Q2 财报 + SC451/SG293 IND 即将 财报+管线 APGE Q2 财报 + AbbVie 收购待完成 财报+M&A ORKA Q2 财报 + III 期 2027 H1 财报+催化 CATX Q2 财报 + III 期 site activation 财报+管线 逐家公司详情 🧬 Sionna Therapeutics (SION) — SION-719 IIa 期 CF 试验失败，股价崩塌 Sionna 公布 SION-719（下一代 CFTR 补充剂）IIa 期临床试验结果，主要终点汗液氯化物仅降低 -1.0 mmol/L，统计学上无意义（p=0.7）。该药物与 Vertex 的 Trikafta 联合使用时未显示出有意义的附加效果。 试验设计：SION-719 联合 Trikafta 治疗 CF 患者，评估汗液氯化物改善 结果：-1.0 mmol/L（远低于有临床意义的阈值），p=0.7 市场反应：股价暴跌约 90% 至 $4.19 对 VRTX 影响：竞争威胁解除，Vertex 当日 +7% 至 $530.83 分析师评论：结果"出人意料"，此前市场预期 SION-719 有潜力成为 Trikafta 的补充疗法 💡 市场影响分析：SION 的失败是 Vertex 在 CF 领域垄断地位的又一确认。Vertex 目前控制全球 CF 市场约 90% 份额，SION-719 是少数几个有潜力挑战其地位的在研药物之一。试验失败后，Vertex 的竞争护城河进一步加深。对 SION 而言，公司尚有其他 CF 管线（SION-619、SION-919），但核心资产受挫将严重影响融资能力。 💊 Tenax Therapeutics (TENAX) — 口服左西孟旦 III 期失败 Tenax 公布 TNX-103（口服左西孟旦）III 期 LEVEL 试验结果，未达到主要终点。该试验评估口服左西孟旦治疗肺动脉高压合并右心衰竭患者的疗效。 试验设计：口服左西孟旦 vs 安慰剂，主要终点为临床恶化时间 结果：未达到主要终点 市场反应：股价暴跌 -84% 至约 $2 后续计划：计划寻求 FDA Type C 会议讨论后续路径 背景：左西孟旦为钙增敏剂，静脉制剂已在海外获批用于急性心衰 💡 市场影响分析：TENAX 是典型的微市值生物科技股（失败前市值约 $5000 万），临床失败后几乎面临存亡危机。口服左西孟旦是公司唯一临床阶段资产，III 期失败意味着需要重新评估整个开发策略。这也提醒投资者微市值单资产公司的极端风险。 🤝 Jazz Pharmaceuticals (JAZZ) — $13 亿收购 Actio Biosciences Jazz 宣布收购私有生物科技公司 Actio Biosciences，交易对价包括 $8.2 亿 首付 + 最高 $5 亿 里程碑，总交易价值可达 $13 亿。 核心资产：ABS-1230，靶向 KCNT1 通道的罕见癫痫药物 适应症：KCNT1 基因突变相关的罕见癫痫（包括 EIIEE 和 MMPSI） 当前阶段：临床前/早期临床 战略意义：扩展 Jazz 神经科学管线，补充现有睡眠/癫痫产品组合 行业背景：2026 年私有生物科技 M&A 浪潮加速，多家大型药企通过收购补充管线 💡 市场影响分析：JAZZ 以神经科学为核心，此次收购进一步深化在罕见癫痫领域的布局。KCNT1 癫痫是高度未满足需求的适应症，目前无获批的靶向疗法。$8.2 亿首付对于早期资产而言偏高，反映 Jazz 对罕见神经科学资产的强烈需求。M&A 浪潮对 XBI 成分股中的中市值公司估值有正面溢出效应。 📊 BridgeBio Pharma (BBIO) — Q2 营收暴增 120%，三管线齐发 BridgeBio 公布 2026 年 Q2 财报，营收 $2.437 亿，同比增长 +120%。 Attruby 单药：Q2 收入 $2.224 亿，ATTR 淀粉样变性市场持续放量 BBP-418：PDUFA 目标日 11/27（LMNA 心肌病） Encaleret：PDUFA 目标日 5/8/2027（ADH1 低钙血症） 第三管线：第三个 PDUFA 待确定 现金：融资后约 $17 亿，跑道至 2028 年后 战略：从单产品向多产品商业化转型 💡 市场影响分析：BBIO 是 2026 年生物科技商业化成功的标杆案例。Attruby 单药季度收入突破 亿，验证了 淀粉样变性市场的巨大潜力。三个 日期确定意味着 年将迎来密集催化。17 亿现金为持续研发和商业化提供保障，公司正从"单一产品生物科技"向"多产品生物制药公司"转型。 🎯 Cogent Biosciences (COGT) — Q2 财报 + bezuclastinib 双适应症推进 Cogent 公布 Q2 财报，同时更新 bezuclastinib 管线进展。 Q2 财报：具体数据待补充 Bezuclastinib GIST：PDUFA 目标日 11/30（胃肠道间质瘤） AdvSM NDA：已提交（6/30），系统性肥大细胞增多症 现金（pro forma）：$8.659 亿 管线策略：bezuclastinib 双适应症（GIST + AdvSM）同步推进 💡 市场影响分析：COGT 的 bezuclastinib 是 KIT D816V 抑制剂，同时瞄准 GIST 和 AdvSM 两个适应症。GIST PDUFA 11/30 是近期关键催化，AdvSM NDA 已提交意味着双适应症商业化在即。$8.659 亿 pro forma 现金为公司提供了充足的商业化资金。 🧬 Vera Therapeutics (VERA) — Q2 财报 + IgAN 关键催化临近 Vera 公布 Q2 财报，更新 atacicept 管线进展。 Q2 净亏损：$1.095 亿 现金：$4.992 亿 ORIGIN 3 最终分析：预计 Q3 2026 读出 sBLA 申报：预计 Q4 2026 Atacicept：靶向 BAFF/APRIL 的融合蛋白，IgA 肾病适应症 商业化准备：加速布局 💡 市场影响分析：VERA 的 atacicept 是 IgA 肾病领域的有力竞争者。ORIGIN 3 最终分析 Q3 读出是公司最大催化事件，阳性结果将支撑 sBLA 申报。IgA 肾病赛道竞争激烈（Travere、Calliditas 等），atacicept 的差异化定位至关重要。$4.992 亿现金为商业化启动提供保障。 💊 Olema Pharmaceuticals (OLMA) — Q2 财报 + palazestrant III 期推进 Olema 公布 Q2 财报，更新选择性雌激素受体降解剂（SERD）管线进展。 Q2 净亏损：$6320 万 现金：$4.611 亿 Palazestrant：III 期临床试验推进中（ER+ HER2- 乳腺癌） OP-3136：I 期临床试验进行中（下一代 SERD） 管线策略：palazestrant 单药 + 联合方案双轨推进 💡 市场影响分析：OLMA 的 palazestrant 是乳腺癌 SERD 赛道的后来者，面临 elacestrant（Stemline）和 camizestrant（AstraZeneca）的竞争。III 期数据读出将决定其市场地位。$4.611 亿现金为完成 III 期提供保障，但商业化竞争将是更大挑战。 🧠 Biohaven (BHVN) — Q2 财报 + BHV-1300 关键试验启动 Biohaven 公布 Q2 财报，更新多管线进展。 Q2 财报：具体数据待补充 BHV-1300：Graves' 病关键试验已启动 MoDE（分子降解引擎）平台：多管线推进 TRAP（靶向受体衰减蛋白）平台：多管线推进 管线布局：神经科学 + 免疫/炎症双轮驱动 💡 市场影响分析：BHVN 从偏头痛起家后正快速扩展至自免领域。BHV-1300 是 MoDE 平台的首个关键试验，Graves' 病适应症进入后期开发是平台验证的重要里程碑。MoDE/TRAP 双平台策略如果成功，将打开多个适应症的大门。 🔬 Sana Biotechnology (SANA) — Q2 财报 + IND 申报临近 Sana 公布 Q2 财报，更新细胞疗法管线进展。 Q2 现金：$1.605 亿 SC451：IND 申报预计年内提交（1 型糖尿病） SG293：首次人体数据预计年内读出（CD19 CAR-T） UP421：NEJM 随访数据更新 技术平台：fusogen 平台驱动细胞疗法创新 💡 市场影响分析：SANA 的 fusogen 平台是细胞疗法领域的创新方向，SC451 和 SG293 的 IND/临床数据将是关键验证点。$1.605 亿现金相对紧张，IND 申报和临床数据读出将决定融资能力。UP421 在 NEJM 的数据更新有助提升市场关注度。 💰 Apogee Pharmaceuticals (APGE) — Q2 财报 + AbbVie 收购待完成 Apogee 公布 Q2 财报，AbbVie 收购交易正在进行中。 Q2 净亏损：$8590 万 现金：$13 亿 AbbVie 收购：$135.11/股，总交易价值约 $109 亿，预计近期完成 APEX IIb 期：Part B 阳性结果（肥胖适应症） 战略：收购完成后将成为 AbbVie 肥胖管线核心 💡 市场影响分析：APGE 被 AbbVie 以 109 亿收购是 2026 年肥胖赛道最大 M&A 之一。135.11/股的收购价较公告前有显著溢价。APEX IIb 期 Part B 阳性结果进一步验证了 APGE 的差异化 GLP-1/GIP 双激动剂在肥胖领域的潜力。收购完成后，AbbVie 将在肥胖赛道与 Lilly/Novo 竞争。 🎯 Oruka Therapeutics (ORKA) — Q2 财报 + III 期 2027 H1 启动 Oruka 公布 Q2 财报，更新银屑病管线进展。 Q2 净亏损：$4120 万 现金：$11 亿 EVERLAST-A：Week 28 数据预计 Q3 末 读出（银屑病） EVERLAST-B：已入组完成 III 期计划：2027 H1 启动 ORKA-004：TL1A 靶向，Q4 进入临床 💡 市场影响分析：ORKA 的银屑病长效抗体方案如果成功，将挑战现有 IL-23 抑制剂的市场地位。EVERLAST-A Week 28 数据是近期关键催化。$11 亿现金为 III 期开发提供充足跑道。TL1A 靶向的 ORKA-004 进入临床标志着公司向炎症性肠病扩展。 📊 Caribou Biosciences (CATX) — Q2 财报 + III 期 site activation 年底 Caribou 公布 Q2 财报，更新 CAR-T 细胞疗法管线进展。 Q2 财报：具体数据待补充 VMT-α-NET：III 期 site activation 目标年底（神经内分泌肿瘤） 现金：$2.37 亿 技术平台：CRISPR 基因编辑驱动下一代 CAR-T 管线策略：同种异体 CAR-T 差异化定位 💡 市场影响分析：CATX 的 VMT-α-NET 是同种异体 CAR-T 在神经内分泌肿瘤领域的探索，III 期 site activation 年底完成是重要里程碑。$2.37 亿现金需要精打细算，III 期启动后的融资压力值得关注。同种异体 CAR-T 领域竞争激烈（Allogene、Cellectis 等），VMT-α-NET 的差异化定位至关重要。 📅 催化剂日历 日期 公司 事件 8/13 ANNX Q2 财报预期发布 8/17 BMY Iberdomide PDUFA（首个 CELMoD） 8/22 CAPR Deramiocel PDUFA（DMD，高风险） 8/27 GILD BIC/LEN HIV 治疗 PDUFA 8/28 CYTK ESC ACACIA-HCM 完整结果 Q3 末 ORKA EVERLAST-A Week 28 数据 Q3 VERA ORIGIN 3 最终分析 9/30 SRRK Apitegromab PDUFA 11/1 AGIO Mitapivat SCD sNDA PDUFA 11/27 BBIO BBP-418 PDUFA（LMNA 心肌病） 11/30 COGT Bezuclastinib GIST PDUFA Q4 VERA sBLA 申报 Q4 ORKA ORKA-004 TL1A 进入临床 年内 SANA SC451 IND 申报 年内 SANA SG293 首次人体数据 2027 H1 ORKA III 期启动 行业动态（非 XBI 成分股） 🔬 Silence Therapeutics (SLN) — Divesiran II 期 SANRECO 试验阳性 Silence Therapeutics（非 XBI 成分股）公布 divesiran（siRNA 靶向 POLRIMA）II 期 SANRECO 试验阳性结果。 主要终点：88% vs 19%（治疗 vs 安慰剂），p<0.001 适应症：真性红细胞增多症（PV） 机制：siRNA 靶向 POLRIMA，抑制异常红细胞生成 市场反应：股价 +39% 意义：验证 siRNA 在血液疾病中的治疗潜力 ⚠️ FDA 拒绝 ITM-11 放射性药物 FDA 拒绝 ITM Isotope Technologies Munich 的放射性药物 ITM-11（Lu-177 标记放射性配体），原因是生产质量问题。 ITM-11 定位为 Novartis Pluvicto 的竞争对手 适应症：胃肠胰神经内分泌肿瘤（GEP-NET） 拒绝原因：CMC（化学制造控制）问题，非疗效/安全性 行业影响：放射性药物赛道生产壁垒凸显 ⚠️ 免责声明：本日数据来源于公开市场信息，不构成任何投资建议。

- $243.7 million in total second quarter revenues, primarily comprised of $222.4 million of U.S. Attruby® net product revenue, with growth led by the treatment-naïve segment as physicians increasingly start and keep patients on Attruby - Attruby is the first ATTR-CM therapy associated with direct kidney protection, with post-hoc analyses published in Circulation: Heart Failure showing a pro with ACE inhibitors, ARBs, and SGLT2s including an early, reversible eGFR dip, an improved chronic eGFR slope relative to placebo, and a 13.7% reduction in urinary albumin-to-creatinine ratio through Month 30; the magnitude of the acute eGFR dip was positively associated with greater early cardiovascular benefit; BridgeBio will explore the potential for Attruby to treat other orphan kidney indications - Real-world evidence continues to differentiate Attruby from tafamidis, with an independent propensity score-matched analysis of 286 patient pairs from the TriNetX network published in JSCAI associating acoramidis with a 37% reduction in composite cardiovascular events (p=0.002) and a 34% reduction in hospitalizations (p=0.002) at six months; further independent RWE using electronic health records are expected, and we are confident Attruby will consistently demonstrate clinical superiority over tafamidis to the benefit of patients and healthcare delivery systems for which heart failure remains a top concern - All three planned NDAs are now submitted to the FDA: BBP-418 for LGMD2I/R9 was accepted with Priority Review (PDUFA November 27, 2026); encaleret for ADH1 was accepted with Priority Review (PDUFA May 8, 2027), with no advisory committee planned for either; oral infigratinib for achondroplasia has been submitted, with U.S. launch expected mid-2027 ezstandalone.cmd.push(function () { ezstandalone.showAds(131); }); - Diagnosis and awareness continue to accelerate ahead of the launches: in ADH, more than 2,200 unique patients are now identified under the dedicated ICD-10 code, at approximately 70 new diagnoses per month; in LGMD2I/R9, BridgeBio is investing in awareness and multidisciplinary care at MDA Care Center Network sites, where we expect 85% of target physicians to be familiar with the BBP-418 pro data by launch - The oral encaleret and oral infigratinib franchises continue to expand beyond their first indications: RECLAIM-HP in chronic hypoparathyroidism has begun screening patients with topline data anticipated in late 2027 or early 2028, CALIBRATE-PEDS in pediatric ADH1 has completed enrollment in its first cohort, and a Phase 2 update in hypochondroplasia is expected in the second half of 2026 - $720.2 million in cash, cash equivalents, and marketable securities as of June 30, 2026, which does not include the $1 billion preferred equity financing that closed on July 1, 2026 - BridgeBio will host a Commercial Day in New York City on October 8, 2026, to discuss commercial readiness and launch strategy across its three upcoming launches PALO ALTO, Calif., Aug. 10, 2026 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a commercial-stage, multi-product biopharmaceutical company focused on developing medicines for genetic conditions, announced today its financial results for the second quarter ended June 30, 2026, and provided an update on Attruby’s commercial progress. Pipeline Overview: Program Status Next expected milestone Acoramidis for ATTR-CM Approved in U.S., E.U., Japan, Switzerland, Brazil, and U.K. New data to be shared at ESC 2026 BBP-418 for LGMD2I/R9 PDUFA date set for November 27, 2026 with Priority Review Launch upon FDA approval Encaleret for ADH1 PDUFA date set for May 8, 2027 with Priority Review; MAA submitted to EMA Launch upon FDA approval Oral infigratinib for achondroplasia NDA submitted to FDA FDA sets PDUFA date Oral Encaleret for chronic hypoparathyroidism First investigational sites activated for RECLAIM-HP, Phase 3 study First participant dosed in Q3 2026 Oral infigratinib for hypochondroplasia ACCEL 2/3 enrollment ongoing Phase 2 clinical trial update in 2H 2026 Depleter for ATTR-CM Development candidate nomination Submit IND to the FDA in 2027 “I'm excited by the growing body of evidence continuing to demonstrate Attruby is the drug of choice for all ATTR-CM patients, and particularly for those who are treatment-naïve, including the first-ever demonstration of early, sustained kidney-protective effects in ATTR-CM alongside the cardiac benefit we've established. Furthermore, this was the quarter all three of our pipeline programs, BBP-418, encaleret, and infigratinib, moved from data into active regulatory review, with our first PDUFA date now set for November 27, 2026, which is a level of strategic execution and discipline I'm proud of. Finally, with the $1 billion preferred equity financing we completed, we have a balance sheet sized to run all three launches at full strength, without diverting resources from the development engine that produced them,” said Neil Kumar, Ph.D., Co-Founder and CEO of BridgeBio. Commercial Updates: The second quarter total revenues, net totaled $243.7 million, comprised of $222.4 million of U.S. Attruby net product revenue, $15.4 million from royalty revenue, and $5.8 million in license and services revenue. “We continue to see strong growth this quarter for Attruby with our first-line share climbing again,” said Matt Outten, Chief Commercial Officer of BridgeBio. “What comes next will continue to shape BridgeBio’s next chapter as we prepare for three potential approvals in three different diseases, all with best-in-class potential, each backed by the same commercial engine that made Attruby a success. We look forward to continuing to deliver for patients and addressing the gaps within the treatment paradigm for rare disease.” Pipeline Updates: Attruby (acoramidis) – First and only near-complete (≥90%) transthyretin (TTR) stabilizer for treatment of transthyretin amyloid cardiomyopathy (ATTR-CM): New post-hoc analyses published in Circulation: Heart Failure showed acoramidis was associated with a rapid, reversible estimated glomerular filtration rate (eGFR) dip alongside a placebo-corrected 15.5% reduction in urinary albumin-to-creatinine ratio (UACR) by Day 28, followed by a sustained improvement in chronic eGFR slope (+2.47 mL/min/1.73m²/year) and a 13.7% UACR reduction through Month 30. This pro that of direct-acting kidney medicines such as ACE inhibitors, ARBs, and SGLT2 inhibitors, and has not previously been observed with any approved ATTR-CM therapy. Participants with larger eGFR dips had a 58% lower risk of death or cardiovascular hospitalization in year one, suggesting the kidney effect may contribute to acoramidis' early cardiovascular benefit. New data from ATTRibute-CM presented in two late-breaking oral presentations at Heart Failure 2026 further demonstrated acoramidis’ differentiated clinical profile. The first showed a reduction in the risk of outpatient worsening heart failure by 41% versus placebo with separation of curves seen within 30 days and sustained through Month 30. The second showed a significant reduction in serum transthyretin variability, which is associated with lower mortality. Real-world evidence continues to demonstrate that Attruby is differentiated from other therapies in the speed and strength of benefit. An independent propensity score-matched analysis of 286 patient pairs from the TriNetX network, presented at SCAI 2026 Scientific Sessions and published in JSCAI , associated acoramidis with a 37% reduction in composite cardiovascular events (HR 0.63; p=0.002) and a 34% reduction in hospitalizations (HR 0.67; p=0.002) at six months, with effects deepening at nine months and significant reductions across heart failure exacerbation, arrhythmia, and acute kidney injury. A separate propensity score-weighted analysis observed a 43% reduction in outpatient diuretic intensification (HR 0.57; p=0.021) versus tafamidis. A third independent study conducted in EPIC COSMOS and to be published later in 2026 validated the comparative effectiveness of acoramidis over tafamidis. These three real-world datasets converge on consistent, statistically significant benefit in contemporary patients on modern background therapy, reinforcing that Attruby's stabilization advantage is showing up in outcomes that matter to physicians, patients, and payers. BridgeBio initiated ASCEND-ATTR, a Phase 4 study using cardiac MRI and echocardiography to characterize the long-term effects of acoramidis on disease reversal as measured by cardiac structure, function, and amyloid burden over 36 months. This builds on the evidence of regression observed in ATTRibute-CM and the open-label extension. Additional data will be shared in two oral presentations and six moderated posters at the European Society of Cardiology (ESC) Congress 2026. BBP-418 – Glycosylation substrate for limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9): BridgeBio believes BBP-418 is positioned to become the first approved therapy for individuals living with LGMD2I/R9, addressing a significant unmet need in this disease and potentially representing the first approval of a therapy for any form of LGMD. On May 27, 2026, the FDA accepted the Company's New Drug Application (NDA) for BBP-418 and granted Priority Review, assigning a Prescription Drug User Fee Act (PDUFA) target action date of November 27, 2026. No advisory committee meeting is currently planned. FORTIFY, the Phase 3 clinical trial of BBP-418, successfully met all pre-specified primary and secondary endpoints of its 12-month interim analysis, supporting its potential as a disease-modifying therapy. The topline results can be found here . Additional positive results demonstrating the rapid and consistent treatment effect and favorable safety pro BBP-418 were presented in March at the 2026 MDA Clinical and Scientific Conference in a late-breaking oral presentation. 1 At the 19th International Congress on Neuromuscular Diseases in July 2026, BridgeBio presented interim FORTIFY data showing favorable patient-reported outcomes for BBP-418-treated individuals compared to placebo, demonstrating that the improvements observed on functional outcomes and biomarkers translate to how patients feel and function. BridgeBio invested $100,000 to strengthen multidisciplinary LGMD care through a Muscular Dystrophy Association (MDA) Care Advance Grant supporting initiatives at Stanford Health Care and the University of Minnesota and engaged the LGMD2I/R9 community at the 2026 European LGMD2I/R9 Conference and the 2026 Iowa Wellstone Dystroglycanopathy Patient & Family Conference. BridgeBio's neuromuscular U.S. field teams are hired, trained, and deployed across medical, commercial, and market access, engaging in scientific exchange, disease state education, and account profiling as appropriate in the pre-approval setting. Promotional activity will commence only upon FDA approval, consistent with regulatory requirements. Based on the FORTIFY interim analysis results, BridgeBio is also engaging regulatory agencies to identify an expedited path to approval for BBP-418 in Europe. The Company intends to initiate clinical studies of BBP-418 in LGMD2I/R9 for individuals less than 12 years of age in the first half of 2027, and in LGMD2M/R13 and LGMD2U/R20 in the near future. Encaleret – Calcium-sensing receptor (CaSR) antagonist for autosomal dominant hypocalcemia type 1 (ADH1) and chronic hypoparathyroidism: BridgeBio believes encaleret is positioned to become the first approved therapy specifically indicated for individuals living with ADH1, in both the U.S. and the EU. The FDA re-considered its review designation of the Company's NDA filing for encaleret in ADH1 and has granted Priority Review, with a PDUFA target action date of May 8, 2027. No advisory committee meeting is currently planned. BridgeBio submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for the use of encaleret in ADH1. Diagnosis of ADH1 in the U.S. continues to accelerate, with more than 2,200 unique patients identified under the dedicated ICD-10 code for ADH (E20.810) from its introduction in October 2023 through June 2026, a rate of approximately 70 new diagnoses per month. CALIBRATE-PEDS, the registrational Phase 2/3 study of encaleret in pediatric ADH1, completed enrollment in the first of four cohorts (adolescents 12 to 17 years of age). RECLAIM-HP, the Phase 3 study of encaleret in chronic hypoparathyroidism, has activated its first investigational sites and screening has initiated. Chronic hypoparathyroidism affects approximately 200,000 patients in the U.S. and EU, and represents a substantial expansion of the encaleret opportunity beyond ADH1. Oral infigratinib – FGFR3 inhibitor for achondroplasia and hypochondroplasia: BridgeBio believes oral infigratinib is positioned to become the first approved oral therapy and a potential best-in-class option for children living with achondroplasia and hypochondroplasia. The Company submitted an NDA to the FDA for oral infigratinib in achondroplasia and is on track to submit an MAA to the EMA in the fourth quarter of 2026. BridgeBio anticipates a U.S. launch in mid-2027 and an EU approval in the second half of 2027. Oral infigratinib has received Breakthrough Therapy, Fast Track, and Rare Pediatric Disease designations from the FDA, and Orphan Drug designation from both the FDA and the EMA. Results from PROPEL 3, the Phase 3 trial of oral infigratinib in achondroplasia, were published in the New England Journal of Medicine 2 and simultaneously presented at the International Congress of Children's Bone Health 2026. Oral infigratinib is the only achondroplasia program with Phase 3 results published in the New England Journal of Medicine. In these results 3 , oral infigratinib significantly improved arm span Z-score versus placebo (LS mean +0.37 SD; p<0.0001) at 52 weeks, the first statistically significant placebo-controlled arm span improvement reported in an achondroplasia trial. PROPEL 3 achieved its pre-specified primary efficacy endpoint of change from baseline in absolute height velocity at Week 52 (p<0.0001), and oral infigratinib showed the first statistically significant improvement in body proportionality against placebo in achondroplasia in children 3 to younger than 8 years old in a pre-specified exploratory analysis. Oral infigratinib remains the only therapy to demonstrate efficacy measures beyond annualized height velocity and height Z-score in a placebo-controlled setting at 52 weeks. The topline results can be found here . U.S. commercial and medical affairs leadership are in place, with the field medical team fully onboarded and regional sales directors building field teams aligned to the diagnosed and treatable population living with achondroplasia. The Company has initiated development of the achondroplasia program from birth to under 3 years of age and is actively enrolling participants. The Company is enrolling participants in the observational run-in study for the Phase 3 trial in hypochondroplasia. An update of the Phase 2 program in hypochondroplasia is expected in the second half of 2026. _______________ 1 2 3 Corporate Updates: On July 1, 2026, BridgeBio closed $1 billion in preferred equity led by Sixth Street with participation from HealthCare Royalty, an affiliate of KKR. The financing enables BridgeBio to fund three potential upcoming launches while continuing to invest in indication expansion across the pipeline. Financial Updates: Cash, Cash Equivalents and Marketable Securities Cash, cash equivalents and marketable securities totaled $720.2 million and $587.5 million as of June 30, 2026 and December 31, 2025, respectively. Total Revenues, Net Three Months Ended June 30, Six Months Ended June 30, 2026 2025 2026 2025 (in thousands) Net product revenue $ 222,440 $ 71,501 $ 403,036 $ 108,240 License and services revenue 5,804 37,440 10,223 117,130 Royalty revenue 15,432 1,624 24,932 1,828 Total revenues, net $ 243,676 $ 110,565 $ 438,191 $ 227,198 Total revenues, net for the three months ended June 30, 2026 were $243.7 million compared to $110.6 million for the same period in 2025. The $133.1 million increase was primarily driven by a $150.9 million increase in net product revenue from Attruby, and a $13.8 million increase in royalty revenue primarily earned from net product sales of BEYONTTRA in the EU and Japan. These increases were partially offset by a decrease in license and services revenue primarily due to recognition of $30.0 million of regulatory milestone-related revenue during the three months ended June 30, 2025. Total revenues, net for the six months ended June 30, 2026 were $438.2 million compared to $227.2 million for the same period in 2025. The $211.0 million increase was primarily driven by a $294.8 million increase in net product revenue from Attruby, and a $23.1 million increase in royalty revenue primarily earned from net product sales of BEYONTTRA in the EU and Japan. These increases were partially offset by a decrease in license and services revenue primarily due to recognition of $105.0 million of regulatory milestone-related revenues during the six months ended June 30, 2025. Total Operating Costs and Expenses Three Months Ended June 30, Six Months Ended June 30, 2026 2025 2026 2025 (in thousands) Total cost of revenues $ 15,046 $ 3,653 $ 24,985 $ 6,292 Research and development 149,448 111,231 276,084 222,662 Selling, general and administrative 186,261 129,154 350,157 235,519 Restructuring, impairment, and related charges — 805 — 1,375 Total operating costs and expenses $ 350,755 $ 244,843 $ 651,226 $ 465,848 Total operating costs and expenses for the three months ended June 30, 2026 were $350.8 million, compared to $244.8 million for the same period in 2025. The $106.0 million increase was primarily driven by a $57.1 million increase in selling, general and administrative (SG&A) expenses, reflecting continued investment in both the ongoing commercialization of Attruby and the pre-commercial activities for BridgeBio's Phase 3 product candidates, a $38.2 million increase in research and development (R&D) expenses to support the development of late-stage product candidates, and an $11.4 million increase in total cost of revenues primarily due to higher sales volume of Attruby. Total operating costs and expenses for the six months ended June 30, 2026 were $651.2 million, compared to $465.8 million for the same period in 2025. The $185.4 million increase was primarily driven by a $114.6 million increase in SG&A expenses, reflecting continued investment in both the ongoing commercialization of Attruby and the pre-commercial activities for BridgeBio's Phase 3 product candidates, an $18.7 million increase in total cost of revenues primarily due to higher sales volume of Attruby, and a $53.4 million increase in R&D expenses to support headcount growth and the development of late-stage product candidates. Stock-based compensation expenses included in operating costs and expenses for the three months ended June 30, 2026 were $44.5 million, of which $28.4 million, $15.4 million, and $0.7 million were included in SG&A expenses, R&D expenses, and cost of goods sold, respectively. Stock-based compensation expenses included in operating costs and expenses for the same period in 2025 were $37.3 million, of which $23.2 million, $14.0 million, and $0.1 million were included in SG&A expenses, R&D expenses, and cost of goods sold, respectively. Stock-based compensation expenses included in operating costs and expenses for the six months ended June 30, 2026 were $77.9 million, of which $48.6 million, $27.6 million, and $1.7 million were included in SG&A expenses, R&D expenses, and cost of goods sold, respectively. Stock-based compensation expenses included in operating costs and expenses for the same period in 2025 were $66.7 million, of which $41.2 million, $25.3 million, and $0.2 million were included in SG&A expenses, R&D expenses, and cost of goods sold, respectively. Total Other Expense, Net Total other expense, net for the three and six months ended June 30, 2026, was $(48.8) million and $(109.4) million, respectively, compared to $(47.4) million and $(112.6) million, respectively, for the same periods in 2025. The increase in total other expense, net of $1.4 million for the three months ended June 30, 2026, compared to the same period in 2025 was primarily driven by a $15.3 million increase in noncash interest expense related to deferred royalty obligations, and was partially offset by a $13.8 million decrease in net loss from equity method investments. The decrease in total other expense, net of $3.2 million for the six months ended June 30, 2026, compared to the same period in 2025 was primarily driven by a $21.2 million decrease in loss on extinguishment of debt recognized in 2025, and an $11.0 million decrease in net loss from equity method investments. These decreases were partially offset by a $31.2 million increase in noncash interest expense related to deferred royalty obligations. Net Loss Attributable to Common Stockholders of BridgeBio and Net Loss per Share For the three and six months ended June 30, 2026, the Company recorded a net loss attributable to common stockholders of BridgeBio of $152.2 million and $316.3 million, respectively, compared to $181.9 million and $349.3 million, respectively, for the same periods in 2025. For the three and six months ended June 30, 2026, the Company reported a net loss per share of $0.78 and $1.62, respectively, compared to $0.95 and $1.84, respectively, for the same periods in 2025. BRIDGEBIO PHARMA, INC. Condensed Consolidated Statements of Operations (in thousands, except share and per share amounts) Three Months Ended June 30, Six Months Ended June 30, 2026 2025 2026 2025 (Unaudited) (Unaudited) Revenues: Net product revenue $ 222,440 $ 71,501 $ 403,036 $ 108,240 License and services revenue 5,804 37,440 10,223 117,130 Royalty revenue 15,432 1,624 24,932 1,828 Total revenues, net 243,676 110,565 438,191 227,198 Operating costs and expenses: Cost of revenues: Cost of goods sold 10,528 2,848 18,260 4,882 Cost of license, services, and royalty revenue 4,518 805 6,725 1,410 Total cost of revenues 15,046 3,653 24,985 6,292 Research and development 149,448 111,231 276,084 222,662 Selling, general and administrative 186,261 129,154 350,157 235,519 Restructuring, impairment, and related charges — 805 — 1,375 Total operating costs and expenses 350,755 244,843 651,226 465,848 Loss from operations (107,079 ) (134,278 ) (213,035 ) (238,650 ) Other income (expense), net: Interest income 6,659 3,898 12,905 9,283 Interest expense (13,278 ) (11,607 ) (26,220 ) (29,728 ) Noncash interest expense on deferred royalty obligations (1) (41,345 ) (26,030 ) (81,218 ) (50,050 ) Loss on extinguishment of debt — — — (21,155 ) Net loss from equity method investments (6,435 ) (20,189 ) (24,718 ) (35,745 ) Other income, net 5,587 6,548 9,840 14,779 Total other expense, net (48,812 ) (47,380 ) (109,411 ) (112,616 ) Loss before income taxes (155,891 ) (181,658 ) (322,446 ) (351,266 ) Provision for income taxes — 2,100 — 2,100 Net loss (155,891 ) (183,758 ) (322,446 ) (353,366 ) Net loss attributable to redeemable convertible noncontrolling interests and noncontrolling interests 3,675 1,855 6,187 4,041 Net loss attributable to common stockholders of BridgeBio $ (152,216 ) $ (181,903 ) $ (316,259 ) $ (349,325 ) Net loss per share attributable to common stockholders of BridgeBio, basic and diluted $ (0.78 ) $ (0.95 ) $ (1.62 ) $ (1.84 ) Weighted-average shares used in computing net loss per share attributable to common stockholders of BridgeBio, basic and diluted 195,785,884 190,517,215 195,290,642 190,332,261 (1) Including related party amounts of $(5,575) and $(10,936), respectively, for the three and six months ended June 30, 2026. Three Months Ended June 30, Six Months Ended June 30, Stock-based Compensation 2026 2025 2026 2025 (Unaudited) (Unaudited) Cost of goods sold $ 685 $ 126 $ 1,655 $ 217 Research and development 15,439 14,000 27,583 25,255 Selling, general and administrative 28,398 23,213 48,647 41,211 Restructuring, impairment and related charges — — — 46 Total stock-based compensation $ 44,522 $ 37,339 $ 77,885 $ 66,729 BRIDGEBIO PHARMA, INC. Condensed Consolidated Balance Sheets (In thousands) June 30, 2026 December 31, 2025 (Unaudited) (1) Assets Cash, cash equivalents and marketable securities $ 720,160 $ 587,482 Accounts receivable, net 254,484 139,444 Inventories 52,842 26,753 Prepaid expenses and other current assets 65,652 44,070 Equity method investments 55,095 79,972 Property and equipment, net 5,226 5,366 Operating lease right-of-use assets 17,079 8,149 Intangible assets, net 26,641 28,077 Other assets 20,120 16,712 Total assets $ 1,217,299 $ 936,025 Liabilities, Redeemable Convertible Noncontrolling Interests and Stockholders' Deficit Accounts payable $ 27,202 $ 36,228 Accrued and other current liabilities (2) 306,630 238,361 Operating lease liabilities 18,288 10,003 Deferred revenue 15,970 20,270 2027 Notes, net 547,955 547,015 2029 Notes, net 741,918 740,890 2031 Notes, net 565,528 564,565 2033 Notes, net 620,087 — Deferred royalty obligations, net (3) 879,374 855,030 Other long-term liabilities 240 244 Redeemable convertible noncontrolling interests (1,018 ) (570 ) Total BridgeBio stockholders' deficit (2,515,215 ) (2,086,610 ) Noncontrolling interests 10,340 10,599 Total liabilities, redeemable convertible noncontrolling interests and stockholders' deficit $ 1,217,299 $ 936,025 (1) The condensed consolidated balance sheet as of December 31, 2025 is derived from the audited consolidated financial statements as of that date. (2) Including related party amounts of $5,626 and $2,003 as of June 30, 2026 and December 31, 2025, respectively. (3) Including related party amounts of $206,208 and $204,650 as of June 30, 2026 and December 31, 2025, respectively. BRIDGEBIO PHARMA, INC. Condensed Consolidated Statements of Cash Flows (In thousands) Six Months Ended June 30, 2026 2025 (Unaudited) Operating activities: Net loss $ (322,446 ) $ (353,366 ) Adjustments to reconcile net loss to net cash used in operating activities: Stock-based compensation 77,314 63,123 Net loss from equity method investments 24,718 35,745 Noncash interest expense on deferred royalty obligations (1) 81,218 50,050 Change in fair value of the embedded derivative associated with the deferred royalty obligation (5,730 ) (5,499 ) Amortization of debt discount and issuance costs 3,745 3,056 Depreciation and amortization 2,239 2,601 Noncash lease expense 3,094 2,230 Loss on extinguishment of debt — 21,155 Dividend from investment in equity securities — (2,302 ) Other noncash adjustments, net 1,502 35 Changes in operating assets and liabilities: Accounts receivable, net (115,040 ) (72,146 ) Inventories (28,166 ) (16,582 ) Prepaid expenses and other current assets (20,227 ) (22,745 ) Other assets (3,283 ) (174 ) Accounts payable (9,026 ) 16,516 Accrued compensation and benefits (25,755 ) (15,637 ) Accrued research and development liabilities 23,032 (2,977 ) Operating lease liabilities (3,674 ) (3,117 ) Deferred revenue (4,300 ) (6,491 ) Other liabilities (2) 52,405 26,609 Net cash used in operating activities (268,380 ) (279,916 ) Investing activities: Purchases of marketable securities (63,937 ) (7,908 ) Maturities of marketable securities 39,600 — Payment for intangible assets — (6,095 ) Purchases of property and equipment (512 ) (594 ) Net cash used in investing activities (24,849 ) (14,597 ) Financing activities: Proceeds from issuance of 2033 Notes 632,500 — Issuance costs and discounts associated with 2033 Notes (13,227 ) — Proceeds from issuance of 2031 Notes — 575,000 Issuance costs and discounts associated with 2031 Notes — (12,034 ) Proceeds from royalty obligation under Royalty Purchase Agreement (as described in Note 9) — 300,000 Issuance costs associated with royalty obligation under Royalty Purchase Agreement — (2,012 ) Repayment of term loans — (459,000 ) Repayments of deferred royalty obligations (3) (33,082 ) (2,005 ) Repurchase of common stock (210,003 ) (48,276 ) Repurchase of RSU shares to satisfy tax withholding (8,341 ) (3,771 ) Proceeds from stock option exercises, net of repurchases 27,812 9,680 Proceeds from common stock issuances under ESPP 5,466 3,237 Transactions with noncontrolling interests — 1,550 Net cash provided by financing activities 401,125 362,369 Net increase in cash, cash equivalents, and restricted cash 107,896 67,856 Cash, cash equivalents, and restricted cash at beginning of period 572,140 683,244 Cash, cash equivalents, and restricted cash at end of period $ 680,036 $ 751,100 (1) Including a related party amount of $10,936 for the six months ended June 30, 2026. (2) Including a related party amount of $5,626 for the six months ended June 30, 2026. (3) Including a related party amount of $(5,784) for the six months ended June 30, 2026. Six Months Ended June 30, 2026 2025 (Unaudited) Supplemental Disclosure of Cash Flow Information: Cash paid for interest $ 20,316 $ 23,271 Supplemental Disclosures of Noncash Investing and Financing Information: Transfers to noncontrolling interests $ (5,464 ) $ (1,640 ) Recognized intangible asset recorded to “Accrued and other current liabilities” $ — $ 2,400 Deferred and unpaid issuance costs recorded to “Accrued and other current liabilities” $ 1,355 $ 998 Reconciliation of Cash, Cash Equivalents and Restricted Cash: Cash and cash equivalents $ 677,911 $ 748,953 Restricted cash — Included in “Prepaid expenses and other current assets” 549 449 Restricted cash — Included in “Other assets” 1,576 1,698 Total cash, cash equivalents and restricted cash at end of periods shown on the condensed consolidated statements of cash flows $ 680,036 $ 751,100 Webcast Information BridgeBio will host a conference call and webcast to discuss second quarter financial results today, August 10, 2026, at 4:30 pm ET. This event can be accessed at or by visiting the “Events & Presentations” page within the Investors section of the BridgeBio website at . A replay of the webcast will be available on the BridgeBio website for 30 days following the event. About Attruby ® (acoramidis) INDICATION Attruby is a transthyretin stabilizer indicated for the treatment of the cardiomyopathy of wild-type or variant transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular death and cardiovascular-related hospitalization. IMPORTANT SAFETY INFORMATION Adverse Reactions Diarrhea (11.6% vs 7.6%) and upper abdominal pain (5.5% vs 1.4%) were reported in patients treated with Attruby versus placebo, respectively. The majority of these adverse reactions were mild and resolved without drug discontinuation. Discontinuation rates due to adverse events were similar between patients treated with Attruby versus placebo (9.3% and 8.5%, respectively). About BridgeBio Pharma, Inc. BridgeBio exists to develop transformative medicines for genetic conditions. Millions of people worldwide living with genetic conditions lack treatment options, often because drug development for small patient populations can be commercially challenging. We aim to bridge the gap between advancements in genetic science and meaningful medicines for underserved patient populations. Our decentralized, hub-and-spoke model is designed for speed, precision, and scalability. Autonomous and empowered teams focus on individual conditions, while a central hub provides the clinical, regulatory, and commercial capabilities needed to bring innovation to market. For more information, visit bridgebio.com and follow us on LinkedIn , X , Facebook , Instagram , YouTube , and TikTok . BridgeBio Pharma, Inc. Forward-Looking Statements This 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,” “continues,” “estimates,” “expects,” “hopes,” “intends,” “may,” “plans,” “projects,” “remains,” “seeks,” “should,” “will,” and variations of such words or similar expressions. BridgeBio intends 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 express and implied statements relating to the continued commercial success and market potential of Attruby/Beyonttra (acoramidis); the Company’s expectations regarding timing of regulatory submissions and target action dates, approvals and commercial launches in the U.S. and Europe, including for BBP-418 in LGMD2I/R9, encaleret in ADH1, and infigratinib in achondroplasia; the Company’s expectations regarding the timing and outcome of pre-commercial activities, including activities designed to support three potential launches; the timing of the Company’s clinical trials, milestones and expected updates for its various programs and pipeline; the safety and the potential benefits of the Company’s product and product candidates; the Company’s anticipated presentations of data; and the Company’s belief that its recent financing will enable the Company to fund its potential launches and expansion across its pipeline. Such statements reflect the Company’s current views about the Company’s plans, intentions, expectations and strategies, which are based on the information currently available to it and on assumptions the Company has made. Although the Company believes that its plans, intentions, expectations and strategies as reflected in or suggested by those forward-looking statements are reasonable, the Company 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 the Company’s preclinical studies and clinical trials not being indicative of final data, the potential size of the target patient populations the Company’s product candidates are designed to treat not being as large as anticipated, the design and success of ongoing and planned clinical trials, future regulatory filings, approvals and/or sales, despite having ongoing and future interactions with the FDA or other regulatory agencies to discuss potential paths to registration for the Company’s product candidates, the FDA or such other regulatory agencies not agreeing with the Company’s regulatory approval strategies, components of the Company’s filings, such as clinical trial designs, conduct and methodologies, or the sufficiency of data submitted, the Company’s pre-commercial activities, commercial launches or operational execution not occurring on anticipated timelines or not supporting planned launches as expected, real-world experience with Attruby/Beyonttra not being consistent with observed biochemical differentiation or not translating into differentiated clinical, commercial or market outcomes, the continuing success of the Company’s collaborations, the Company’s ability to obtain additional funding, including through less dilutive sources of capital than equity financings, potential volatility in the Company’s share price, the Company’s share repurchase program being modified, suspended or discontinued, or share repurchases not delivering the anticipated benefits or proving to be a more attractive use of capital than other alternatives, the impacts of current macroeconomic and geopolitical events, including changing conditions from hostilities in Ukraine and the Middle East, increasing rates of inflation and changing interest rates, on business operations and expectations, as well as those risks set forth in the Risk Factors section of the Company’s most recent Quarterly Report on Form 10-Q and Annual Report on Form 10-K and the Company’s other filings with the U.S. Securities and Exchange Commission. Moreover, the Company operates 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 the Company’s 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, BridgeBio assumes no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise. BridgeBio Media Contact: Bubba Murarka, Executive Vice President contact@bridgebio.com (650)-789-8220 BridgeBio Investor Contact: Kristen Kelleher, Director of Investor Relations ir@bridgebio.com