Accessible version US Biopharmaceuticals 2026 Year Ahead: What to expect from our coverage universe in 2026 Price Objective Change Biopharma favorable despite lingering uncertainties Despite its challenges, Biopharma ended 2025 on a high note (NBI +32%, DRG +21% vs. SPX +17%)—with, in our view, reasons for optimism heading into 2026. Admittedly, the macro concerns weighing on 1H25 sentiment (inflation, tariffs) remain overhangs. That said, 1) sector-specific worries, mostly around drug pricing, eased; 2) lo ...

Summary of BridgeBio Pharma FY Conference Call (December 03, 2025) Company Overview - **Company**: BridgeBio Pharma (NasdaqGS:BBIO) - **Key Products**: Attruby, Encaleret, BBP-418, Infegratinib - **Focus**: Rare diseases, particularly skeletal dysplasia and muscular dystrophies Key Accomplishments in 2025 - **Attruby Launch**: Successful launch with a run rate of approximately $440 million in revenue, targeting ATTR-CM patients [6][5][8] - **Phase Three Data**: Positive results from two phase three trials, indicating potential curative effects for Encaleret and BBP-418 [6][7] - **Sales Potential**: Estimated peak sales potential of $6 billion for the current pipeline, with $4 billion attributed to Attruby and $1 billion each for Encaleret and BBP-418 [8][9] Market Insights - **ATTR-CM Market**: Estimated market size of $15-20 billion, with BridgeBio projecting Attruby to achieve at least $4 billion in sales [12][11] - **Market Share**: Currently holds over 20% market share in treatment-naive patients [12] - **Competitive Landscape**: Concerns regarding the entry of Tafamidis generics, but BridgeBio emphasizes Attruby's unique clinical data and efficacy [14][19] Clinical Data Highlights - **Attruby Efficacy**: Demonstrated a 50% reduction in cardiovascular hospitalization and a 42% reduction in mortality at 30 months [16] - **Encaleret for ADH1**: Potential billion-dollar market opportunity with a patient population of approximately 12,000 in the U.S. and 25,000 in Europe [22][23] - **BBP-418 for Limb-Girdle Muscular Dystrophy**: First data set showing statistically significant improvement in clinical endpoints, with a market opportunity of about 7,000 patients in the U.S. and Europe [31][34] Future Opportunities - **Infegratinib**: First oral therapeutic option for achondroplasia, targeting both MAPK and STAT1 pathways, with promising phase two data showing significant improvements in height Z-scores [37][39] - **Phase Three Trial**: Anticipated readout in Q1 2026, with a focus on annualized height velocity and proportionality as key endpoints [48][50] Risks and Considerations - **Efficacy Concerns**: Potential variability in treatment effects across different age groups in the phase three trial [59][62] - **Market Competition**: Need to differentiate from existing therapies, particularly in terms of convenience and efficacy [71][72] Conclusion - **Strategic Focus**: BridgeBio is positioned for significant growth in the rare disease market, with a strong pipeline and a commitment to innovative therapies that address unmet medical needs. The upcoming phase three results for Infegratinib will be critical in validating its market potential and competitive positioning.

Summary of BridgeBio Pharma FY Conference Call Company Overview - **Company**: BridgeBio Pharma (NasdaqGS:BBIO) - **Key Product**: Attruby, a treatment for ATTR-CM (transthyretin amyloid cardiomyopathy) - **Market Context**: The company has seen a stock price increase of approximately 160% year-to-date, outperforming the XBI index which increased by 30% [1] Core Insights and Arguments Attruby Launch and Market Share - **Launch Success**: The launch of Attruby is described as going extremely well, with the company achieving a market share in the first-line treatment setting now well into the 20% range, with a long-term goal of 30-40% [2][3] - **Clinical Data**: Attruby is noted for having near-complete clinical data on its label, including a 50% reduction in hospitalization at 30 months and a 42% reduction in all-cause mortality and hospitalization [3] - **Market Access Initiatives**: The company has implemented a 28-day free trial program and offers extensive support through patient access liaisons and reimbursement managers, facilitating easier access to Attruby [4] Q4 Expectations and Market Dynamics - **Q4 Performance**: The company anticipates a strong performance in Q4, despite having three fewer selling weeks compared to other quarters. The discontinuation of Pfizer's Vyndaqel is expected to be more beneficial in 2026 rather than Q4 [6][8] - **Switching Patients**: The company aims to convert patients from Vyndaqel to Attruby, particularly in accounts where Vyndaqel was previously used [9][10] Competitive Landscape - **Competitor Actions**: Pfizer's introduction of a similar free trial program for Vyndamax is acknowledged, but the company believes that clinical differentiation will maintain its competitive edge [11][12] - **Diagnosis Rates**: The diagnosis of ATTR-CM is still considered underdiagnosed, with significant room for growth. The number of PYP scans has grown at a CAGR of about 30% since 2019 [13][14] Future Market Opportunities - **Generic Competition**: The potential entry of generics for Vyndaqel in 2028 is discussed, but the company emphasizes that Attruby's clinical differentiation will sustain its market position [16][18] - **Achondroplasia Market**: The company is preparing for the launch of infigratinib for achondroplasia, positioning it as a potential first oral treatment targeting the FGFR3 mutation [19][21] Pricing Strategy - **Pricing Considerations**: The company plans to price new products in accordance with their opportunity size, with potential pricing for rare diseases ranging from $400,000 to $900,000 [38][39] Additional Important Points - **Regulatory Filings**: The company expects to file NDAs for Encaleret and BBP-418 in the first half of next year, with launches anticipated in Q4 2026 and Q1 2027 [36] - **Safety and Efficacy**: The company aims to maintain low rates of hyperphosphatemia in patients treated with infigratinib, with a target of no more than 20% experiencing low-grade hyperphosphatemia [28] This summary encapsulates the key points discussed during the conference call, highlighting BridgeBio Pharma's strategic positioning, market dynamics, and future outlook.

BridgeBio Pharma (BBIO), a mid-cap biotech company, has investors thrilled with its commercial engine scaling following the approval of Acoramidis (brand name Attruby), a therapy for a serious heart condition called transthyretin amyloid cardiomyopathy (ATTR-CM). Additionally, the company is getting ready to introduce potentially three new products in 2026, pending approval. While BBIO stock is up more than 150% year to date, outpacing the S&P 500 Index ($SPX) gain of 15%, Wall Street sees more upside and ...

Summary of BridgeBio Pharma Conference Call Company Overview - **Company**: BridgeBio Pharma (NasdaqGS:BBIO) - **Conference Date**: November 18, 2025 - **Key Speakers**: Thomas Trimarchi (CFO), Matt Outten (Chief Operating Officer) Core Company Insights - **Attruby Performance**: The major commercial product, Attruby, is experiencing continued growth across patient segments and prescribers, with significant share growth in frontline settings [6][8] - **Pipeline Developments**: Two successful phase three readouts for limb-girdle type 2I and ADH1 programs are expected to lead to NDA submissions early next year, with plans for ex-US filings [7][8] - **Future Expectations**: Anticipation of three approved products by this time next year, indicating a strong growth trajectory for the company [8] Market Potential and Sales Projections - **Attruby Market Share**: The peak sales potential for Attruby is projected at 30-40% market share in a growing market, with a strong start since its launch [10][11] - **Real-World Data**: The company is gathering real-world data to support Attruby's differentiation, including a 50% reduction in hospitalizations, which is the best seen to date [11][12] - **Sales Growth Drivers**: Continued growth is expected due to increasing patient diagnoses and education efforts, with a consistent upward trend in new patient additions [14][20] Competitive Landscape - **First-Line Treatment Share**: In the U.S., Attruby's first-line share is around 20%, while in Germany, Bayer has achieved over 50% share due to different reimbursement dynamics [21][22] - **Market Dynamics**: The presence of multiple approved products in the ATTR-CM space is expected to accelerate patient identification and market growth [14][20] Clinical Trials and Studies - **Upcoming Studies**: The company is conducting various studies, including a cardiac imaging study and a primary prevention study for Attruby, with results expected in the coming years [33][34] - **Limb-Girdle Muscular Dystrophy (LGMD2I)**: The interim data shows significant improvements in patient outcomes, with plans for a deeper analysis at the MDA conference in 2026 [43][45] - **ADH1 Program**: The phase three study for ADH1 has shown that 76% of patients could stop taking calcium supplements, indicating a strong market opportunity [50][51] Product Differentiation - **Achondroplasia Treatment**: The upcoming treatment targets FGFR3 directly, which is expected to provide significant benefits over existing therapies that are injectables [58][59] - **Oral Administration Advantage**: The oral small molecule formulation for achondroplasia is anticipated to be a game-changer compared to current injectable therapies, addressing patient compliance issues [59][60] Financial Guidance and Market Strategy - **Potential for Guidance**: The company is considering providing financial guidance to help investors better understand future expectations [35][39] - **Market Expansion Plans**: BridgeBio is building out its ex-US capabilities and plans to market its drugs independently in various regions, alongside partnerships [41][42] Conclusion - BridgeBio Pharma is positioned for significant growth with a strong pipeline and market strategies. The company is focused on expanding its product offerings and enhancing patient access while navigating competitive dynamics in the biotech landscape.

Core Insights - Acoramidis has shown significant clinical benefits in reducing all-cause mortality in patients with variant ATTR-CM, particularly in the V142I subpopulation, with a reported 69% reduction in mortality through Month 42 [1][4][5] - The study results highlight the importance of early diagnosis and treatment for patients with the V142I variant, who have historically faced challenges in accessing care [2][6] - Acoramidis is a near-complete transthyretin stabilizer, approved by the U.S. FDA and other regulatory agencies for treating ATTR-CM [7][9] Efficacy and Clinical Outcomes - The ATTRibute-CM study demonstrated a 59% risk reduction in all-cause mortality in the overall variant population at Month 42, with a statistically significant 69% reduction in the V142I population [4][5] - Functional capacity improvements were noted, including an 87-meter increase in 6-minute walk distance and a 20-point improvement in Kansas City Cardiomyopathy Questionnaire scores through Month 30 [5] - Acoramidis consistently improved clinical outcomes, functional status, quality of life, and NT-proBNP levels across various patient subgroups, regardless of atrial fibrillation status [6][7] Regulatory and Market Position - Acoramidis is marketed as Attruby® in the U.S. and BEYONTTRA® in Europe, with indications for reducing cardiovascular death and related hospitalizations in ATTR-CM patients [7][9] - The ongoing open-label extension study aims to provide further insights into the long-term benefits and durability of acoramidis treatment [6][7] Disparities in Treatment and Outcomes - The study highlighted demographic disparities in treatment initiation and outcomes, particularly among different racial and gender groups, emphasizing the need for equitable care in ATTR-CM [6][7] - Geographic disparities in ATTR-CM prevalence were noted, suggesting a correlation with access to specialized care centers [6][7]

Core Insights - Acoramidis has shown significant clinical benefits in reducing all-cause mortality in patients with variant ATTR-CM, particularly in the V142I subpopulation, with a reported 69% reduction in mortality through Month 30 and Month 42 [1][3][4] - The study highlights the importance of early diagnosis and treatment for patients with the V142I variant, who have historically faced challenges in accessing care [2] - The findings reflect advancements in precision medicine and equity in cardiovascular care, emphasizing the need for continued research and development in this area [2] Summary by Sections Clinical Study Results - The ATTRibute-CM study demonstrated a 59% risk reduction in all-cause mortality (ACM) in the overall variant population at Month 42 compared to placebo [3] - In the V142I subpopulation, there was a 69% risk reduction in ACM through Month 30 and Month 42 [3][4] - Acoramidis also showed improvements in functional capacity, with a least-squares mean difference of 87 meters in the 6-minute walk test and a 20-point difference in the Kansas City Cardiomyopathy Questionnaire score through Month 30 [4] Patient Population Insights - The V142I variant affects 3-4% of the U.S. Black population, highlighting a significant unmet need for effective treatments in this demographic [1] - The study's results are particularly meaningful for patients with variant ATTR-CM, who have limited access to early diagnosis and treatment options [2] Regulatory and Market Information - Acoramidis is approved as Attruby® by the U.S. FDA and as BEYONTTRA® by the European Medicines Agency and other regulatory bodies, indicating its recognized efficacy in stabilizing transthyretin [6][8] - Future data on the benefits of Attruby for ATTR-CM patients is anticipated to be presented at upcoming medical meetings [7] Safety and Adverse Reactions - Adverse reactions reported with Attruby included diarrhea (11.6% vs 7.6% for placebo) and upper abdominal pain (5.5% vs 1.4% for placebo), with most being mild and resolving without discontinuation [9]

New York, NY, Nov. 07, 2025 (GLOBE NEWSWIRE) -- Following a challenge submitted by Pfizer, Inc., BBB National Programs’ National Advertising Division will refer BridgeBio Pharma Inc. to the relevant government agencies for review and possible enforcement action for failure to participate in the National Advertising Division (NAD) inquiry. Pfizer and BridgeBio sell prescription medication to treat transthyretin amyloid cardiomyopathy, a progressive and fatal disease caused by the buildup of a protein called ...

Core Insights - BridgeBio Pharma, Inc. is set to present ten moderated digital posters at the American Heart Association (AHA) Scientific Sessions 2025, highlighting its focus on genetic diseases [1] Group 1: Research Presentations - The presentations will include findings on Acoramidis and its effects on all-cause mortality in patients with the p.V142I (V122I) variant of ATTR-CM, with a specific session on November 8 at 3:15 pm CT [2] - Acoramidis has been shown to reduce all-cause mortality and first cardiovascular hospitalization in patients with variant transthyretin amyloid cardiomyopathy, with results presented on November 8 at 9:15 am CT [2] - Additional findings indicate that Acoramidis lowers NT-proBNP levels in a larger proportion of study participants compared to placebo, independent of atrial fibrillation status [2] Group 2: Demographic and Geographic Insights - A presentation will address demographic disparities in Tafamidis treatment and clinical outcomes across the United States, scheduled for November 8 at 12:15 pm CT [3] - Geographic disparities in the prevalence of transthyretin amyloid cardiomyopathy among U.S. veterans will also be discussed on November 8 at 3:15 pm CT [3] - Insights from the ATTRibute-CM study will reveal associations between serum transthyretin levels at day 28 and cardiovascular outcomes, presented on November 9 at 3:15 pm CT [3] Group 3: Clinical Outcomes and Safety Information - Acoramidis has shown improvement in clinical outcomes, function, quality of life, and NT-proBNP levels in patients with transthyretin amyloid cardiomyopathy, regardless of atrial fibrillation status at baseline [3] - The drug has been associated with a reduction in the risk of all-cause mortality and cardiovascular-related hospitalization in participants with early-stage heart failure, presented on November 8 at 3:15 pm CT [3] - Important safety information indicates that adverse reactions such as diarrhea and upper abdominal pain were reported, with similar discontinuation rates between Acoramidis and placebo [5]

Group 1 - BridgeBio Pharma, Inc. is a commercial-stage biotechnology company focused on delivering Acoramidis for the treatment of ATTR-CM, marketed as Attruby in the US and Beyonttra in the EU and Japan [1] - Beyonttra is commercialized by Bayer, indicating a partnership that may enhance market reach and distribution capabilities for the drug [1]