侏儒症的治疗，正迎来被全面颠覆的时刻。 作为最常见的侏儒症类型，软骨发育不全（ACH）过去很长时间无特效疗法，直到2021年，"孤儿药之王"BioMarin研 发的Vosoritide，作为首个获批药物，才开启了药物对症治疗时代。凭借独家优势，其2025年销售额高达9.27亿美元。 但这种垄断格局，即将被打破，多款在研新药正从作用机制到给药方式，向Vosoritide发起挑战。Ascendis的TransCon- CNP通过包裹技术延长了药物作用时间，实现了周剂给药，目前正处于FDA审批阶段；而BridgeBio的口服药 Infigratinib，则从作用机制到用药体验实现全面突破。 Infigratinib靶向的FGFR3（成纤维细胞生长因子受体3）正是驱动ACH疾病发生的关键靶点，2月12日，BridgeBio公布 Infigratinib在ACH中取得的首个具有统计学显著改善意义的3期顶线结果，公司计划下半年向FDA提交新药申请。 获批后的长期扩展临床数据显示，Vosoritide的生长促进效应可持续至少7年。 尽管目前全球仅有5000名婴幼儿使用该药，但罕见病药物的高定价模式，叠加长期持续给药的治疗需 ...

分组1 - BridgeBio Pharma reported a quarterly loss of $1 per share, which was worse than the Zacks Consensus Estimate of a loss of $0.75, and compared to a loss of $1.31 per share a year ago, indicating a surprise of -34.05% [1] - The company posted revenues of $154.18 million for the quarter ended December 2025, surpassing the Zacks Consensus Estimate by 2.77%, and showing a significant increase from year-ago revenues of $5.88 million [2] - Over the last four quarters, BridgeBio Pharma has surpassed consensus revenue estimates four times, but has only exceeded consensus EPS estimates once [2] 分组2 - The stock has underperformed the market, losing about 11.2% since the beginning of the year, while the S&P 500 has declined by only 0.1% [3] - The current consensus EPS estimate for the coming quarter is -$0.64 on revenues of $173.51 million, and for the current fiscal year, it is -$1.79 on revenues of $900.31 million [7] - The Zacks Industry Rank for Medical - Generic Drugs is currently in the bottom 34% of over 250 Zacks industries, indicating potential challenges for stocks in this sector [8]

Chief Commercial Officer Matt Houghton said Attruby’s growth has been strongest in first-line patients and attributed momentum to the “exceptional data” for the therapy and the company’s field execution. He emphasized that the company has historically provided a quarterly “new patient start” figure, but said BridgeBio will stop reporting that metric going forward because competitors do not disclose comparable data and continued disclosure could create a competitive disadvantage.BridgeBio reiterated that Att ...

BridgeBio (NasdaqGS:BBIO) Q4 2025 Earnings call February 24, 2026 04:30 PM ET Company ParticipantsAdi Jayaraman - Senior Managing DirectorAndrew Tsai - Managing DirectorChinmay Shukla - SVP of Strategic FinanceJustin To - CEO of Skeletal DysplasiasMatt Outten - Chief Commercial OfficerNeil Kumar - CEOTom Trimarchi - President and CFOConference Call ParticipantsBiren Amin - Managing Director and Investment Analyst in Life SciencesEliana Merle - Senior Biotech Equity Research AnalystMani Foroohar - Senior Man ...

BridgeBio (NasdaqGS:BBIO) Q4 2025 Earnings call February 24, 2026 04:30 PM ET Company ParticipantsAdi Jayaraman - Senior Managing DirectorAndrew Tsai - Managing DirectorChinmay Shukla - SVP of Strategic FinanceJustin To - CEO of Skeletal DysplasiasMatthew Outten - Chief Commercial OfficerNeil Kumar - CEOTom Trimarchi - President and CFOConference Call ParticipantsBiren Amin - Managing Director and Investment Analyst in Life SciencesEliana Merle - Senior Biotech Equity Research AnalystMani Foroohar - Senior ...

BridgeBio (NasdaqGS:BBIO) Q4 2025 Earnings call February 24, 2026 04:30 PM ET Speaker9Good afternoon. I'll be your conference operator today. All lines have been placed on mute to prevent any background noise. After the company's remarks, there will be a question and answer session. If you would like to ask a question, press star followed by 1 on your telephone keypad. If you'd like to withdraw your question, press the pound key. Thank you. Before we begin, I'd like to remind everyone that today's call may ...

Financial Performance - Attruby generated $362.4 million in U.S. net product revenues in 2025, reflecting increasing patient adoption and prescriber utilization [44]. - Beyonttra contributed $105 million in license and services revenue in 2025 following approvals in Europe and Japan [47]. - BridgeBio has obtained FDA approval for three products and has treated over 8,500 patients with its approved medicines [21]. - The company received gross cash proceeds of $500.0 million in December 2024 following FDA approval of Attruby on November 22, 2024 [184]. Market Potential - The total global addressable market for ATTR therapeutic interventions could exceed $20.0 billion, with diagnosed ATTR-CM patients in the U.S. growing from fewer than 5,000 in 2019 to more than 50,000 in 2025 [48]. - The estimated global prevalence of ATTRwt-CM and ATTRv-CM is over 400,000 and 40,000 patients, respectively, indicating significant underdiagnosis in the population [53]. - The market opportunity for achondroplasia and other FGFR-driven skeletal dysplasias is estimated to exceed $5.0 billion globally [82]. - The total global market opportunity for hypochondroplasia is expected to approach that of achondroplasia, driven by growing awareness and clinical trials [120]. Clinical Development - Positive Phase 3 results for infigratinib in achondroplasia, BBP-418 in limb-girdle muscular dystrophy type 2I/R9, and encaleret in autosomal dominant hypocalcemia type 1 are expected to lead to NDA submissions in 2026 [42]. - The Phase 3 PROPEL 3 study for low-dose infigratinib showed a statistically significant improvement in annualized height velocity of +2.10 cm/year compared to placebo [79]. - Enrollment in the Phase 2 portion of the ACCEL 2/3 trial for hypochondroplasia has been completed, with proof-of-concept results expected in the second half of 2026 [122]. - The company plans to initiate a registrational Phase 3 study for encaleret in pediatric ADH1 in Q1 2026 [99]. Regulatory and Compliance - The company is subject to extensive government regulations regarding the research, development, and marketing of drug products, which require significant time and financial resources [186]. - The FDA regulates drugs and biologics under the Federal Food, Drug, and Cosmetic Act, requiring substantial data to demonstrate quality, safety, and efficacy before marketing [188]. - The FDA targets ten months for the initial review of a new molecular entity NDA or original BLA, and six months for priority review applications [203]. - Manufacturers must comply with ongoing FDA regulations post-approval, including monitoring adverse experiences and adhering to advertising standards [219]. Competitive Landscape - Acoramidis (Attruby/Beyonttra) competes with Pfizer's Vyndaqel and Alnylam's vutrisiran for the treatment of ATTR-CM [137]. - The company faces competition from established pharmaceutical and biotechnology companies, which may have greater resources [135][136]. Partnerships and Agreements - Bayer License Agreement granted an exclusive license for acoramidis with an upfront payment of $135.0 million and potential milestone payments up to $600.0 million through 2026 [156]. - Eidos received an upfront payment of $25.0 million from Alexion for the license to commercialize Beyonttra in Japan, with a regulatory milestone payment of $30.0 million recognized in June 2025 [160]. - QED entered into a license agreement with Kyowa Kirin for infigratinib, receiving an upfront payment of $100.0 million and potential milestone payments up to $81.4 million [168]. Product Development and Innovation - The decentralized hub-and-spoke model allows multiple programs to advance concurrently, enhancing the probability of success over time [33]. - BBP-812 has the potential to become the first disease-modifying therapy for Canavan disease, having received multiple designations from the FDA and EMA [124]. - BridgeBio has initiated a next-generation depleter program for ATTR-CM, which could expand the addressable patient population by removing existing amyloid deposits [127].

$154.2 million in total fourth quarter revenues, net, and $502.1 million in full year revenues, net, primarily comprised of net product revenue of $146.0 million and $362.4 million, respectivelyBridgeBio reported three positive Phase 3 trial readouts in just over three months, a demonstration of its unique model for sustainable drug development as described in a recent peer-reviewed manuscriptAttruby continues to demonstrate clinical differentiation as a first-choice therapy in ATTR-CM with the greatest TTR ...

BridgeBio Pharma, Inc. (NASDAQ: BBIO) is a biopharmaceutical company focused on developing treatments for genetic conditions. The company is set to release its quarterly earnings on February 24, 2026. Wall Street anticipates an earnings per share (EPS) of -$0.75 and revenue of around $149.1 million. BBIO will also host a conference call to discuss these results and provide business updates.BBIO's financial metrics reveal some challenges. The company has a negative price-to-earnings (P/E) ratio of -16.23, in ...

PALO ALTO, Calif., Feb. 23, 2026 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a biopharmaceutical company focused on developing medicines for genetic conditions, today announced that members of its management team will participate in the following healthcare investor conferences: TD Cowen 46th Annual Healthcare Conference, Boston, MA: Fireside Chat on Monday, March 2 at 3:50 pm ESTLeerink Partners Global Healthcare Conference, Miami, FL: Fireside Chat on Tuesday, ...