BridgeBio Pharma (NasdaqGS:BBIO) 2025 Conference November 18, 2025 11:00 AM ET Company ParticipantsMatt Outten - Chief Commercial OfficerThomas Trimarchi - CFOConference Call ParticipantsAndrew Tsai - Senior Biotech AnalystAndrew TsaiWelcome to Day Two of our London Healthcare Conference. I'm Andrew Tsai, Senior Biotech Analyst at Jefferies, and it's my pleasure to have the BridgeBio team with me. To my direct right, Thomas Trimarchi, CFO, and to his right, Matt Outten, Chief Operating Officer.Matt OuttenCo ...

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]

Core Insights - Alnylam Pharmaceuticals reported strong third-quarter results, driven by its leading drug Amvuttra, which significantly exceeded sales expectations and demonstrated robust growth in patient demand [3][10]. Financial Performance - Amvuttra generated $685 million in sales during the third quarter, surpassing the expected $622 million and more than doubling from $259 million in the same period last year [3]. - Total sales across all products reached $851 million, reflecting a 103% increase and beating forecasts of $791 million [10]. - The company anticipates total product sales for the year to be between $2.95 billion and $3.05 billion, marking a 10% increase at the midpoint compared to previous projections [10]. Product and Market Position - Amvuttra is the only approved treatment for transthyretin amyloidosis in patients with both polyneuropathy and cardiomyopathy, with only 20% of patients diagnosed [7]. - The drug competes with offerings from Pfizer, BridgeBio, and Ionis Pharmaceuticals, but Alnylam's targeted approach has positioned it favorably in the market [5][6]. Growth Drivers - The company identified 170 health systems that prescribe approximately 80% of TTR medicines, ensuring that 90% of patients can access Amvuttra treatment within a 10-mile radius [8]. - A significant portion of U.S. patients are receiving insurance coverage for Amvuttra, further supporting its market penetration [8]. Future Outlook - Alnylam is developing a next-generation drug, nucresiran, which aims to further reduce problematic TTR protein levels and could be administered every six months [11]. - The company expects to launch nucresiran for cardiomyopathy patients around 2030, depending on clinical trial outcomes [12].

Financial Data and Key Metrics Changes - Total revenues for Q3 2025 were $120.7 million, consisting of $108.1 million from Attruby net product revenue, $4.3 million in royalty revenue, and $8.3 million in license and services revenue, compared to $2.7 million in the same period last year, indicating a significant increase in total revenues driven primarily by Attruby sales [25][26][27] - Operating expenses for Q3 2025 were $259.3 million, up from $193.9 million in the same period last year, with a notable increase in SG&A expenses [26][27] Business Line Data and Key Metrics Changes - Attruby generated $108.1 million in net product sales in Q3 2025, with 5,259 unique patient prescriptions delivered to 1,355 unique healthcare providers, reflecting strong commercial execution [11][25] - The company aims for a market share of 30-35% by volume in the ATTR-CM market, with continued momentum in both scientific and commercial aspects of the Attruby franchise [6][11] Market Data and Key Metrics Changes - The ATTR-CM market continues to expand, with growth coming from all segments, and an increase in prescribing from both returning and new physicians [18][20] - The company has seen a steady rise in first-time prescribers adopting Attruby, indicating growing prescriber confidence and sustained demand [20][21] Company Strategy and Development Direction - The company is focused on launching new products from its late-stage pipeline, including Encalirate and BBP-418, which have shown promising phase III results [4][22] - The strategy includes leveraging the experience gained from the Attruby launch to inform future product launches and expanding disease state education initiatives [22][23] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in achieving a market share of over 30% for Attruby, supported by strong clinical data and ongoing market expansion [6][11] - The company anticipates continued growth in diagnosis rates for ATTR-CM, with expectations to reach approximately 250,000 diagnosed patients in the U.S. [39] Other Important Information - The company ended Q3 2025 with a strong cash position of $645.9 million, providing significant cash runway for future investments [27] - The company is actively building infrastructure for global commercialization to ensure access to its therapies worldwide [24] Q&A Session Summary Question: What is the percentage of new patient share for Attruby? - Management estimates that the naive share is well in the 20s, with double-digit growth in overall prescriptions quarter-on-quarter [30][31] Question: Can you elaborate on ATTR-CM diagnosis rates? - There has been robust growth in diagnosis rates, with ongoing excitement and education in the field, leading to increased identification of new patients [39] Question: Thoughts on Pfizer's 28-day free trial program? - Management views it positively as it encourages competition and emphasizes the importance of access programs [44][46] Question: How does the ex-US opportunity compare to the US? - The ex-US opportunity is promising, with Bayer successfully commercializing in Europe, although pricing dynamics differ [50][52] Question: Discussions with payers regarding formulary positioning of Attruby? - Management has not had discussions about formulary positioning with payers but remains focused on ensuring Attruby's availability [54][56] Question: What is driving the growth in unique prescribers and prescriptions per prescriber? - Growth is driven by both increased penetration within existing accounts and expansion into new centers, with a focus on educating physicians [58][60] Question: What are the differentiating elements for Infigratinib in achondroplasia? - Infigratinib is expected to be more efficacious, safer, and more convenient due to its oral route of administration [84][86]

Core Insights - BridgeBio Pharma reported a total revenue of $120.7 million for Q3 2025, driven primarily by $108.1 million in U.S. Attruby net product revenue, alongside $4.3 million from royalty revenue and $8.3 million in license and services revenue [1][3][10] - The company has seen significant commercial progress, with 5,259 unique patient prescriptions written by 1,355 unique prescribers as of October 25, 2025, indicating strong adoption of Attruby since its FDA approval in November 2024 [1][2][4] - BridgeBio plans to file New Drug Applications (NDAs) for BBP-418 and encaleret in the first half of 2026, aiming for full approval of these therapies [1][5][8] Commercial Performance - Attruby's launch has been characterized by strong physician adoption and continued growth across all market segments, reflecting its differentiated clinical profile [4][6] - The company reported a significant increase in total revenues, with a $118 million rise compared to the same period last year, primarily due to the commercial success of Attruby [10][11] Pipeline Developments - Positive interim analysis results from the FORTIFY Phase 3 study of BBP-418 showed a 1.8x increase in glycosylated αDG from baseline at 3 months, with sustained improvements at 12 months [1][8] - The CALIBRATE Phase 3 study of encaleret for ADH1 met its primary endpoint, with 76% of participants achieving target calcium levels at Week 24 [1][8] - The PROPEL 3 study of infigratinib for achondroplasia is fully enrolled, with topline results expected in early 2026 [1][8] Financial Overview - As of September 30, 2025, the company had $645.9 million in cash, cash equivalents, and marketable securities, positioning it well to support the commercialization of Attruby and advance its late-stage pipeline [1][9] - Operating costs for Q3 2025 were $265.9 million, reflecting increased investments in support of Attruby's commercial launch [14][15] Key Metrics - The net loss attributable to common stockholders for Q3 2025 was $182.7 million, compared to $162.0 million for the same period in the prior year, with a net loss per share of $0.95 [22][23] - Total revenues for the nine months ended September 30, 2025, were $347.9 million, up from $216.0 million in the prior year [11][12]

Core Insights - The article highlights stocks reaching new 52-week highs, indicating market recognition of strong fundamentals and potential catalysts for growth [1][2]. Company Summaries Cogent Biosciences Inc. (COGT) - Cogent Biosciences plans to file its first New Drug Application for Bezuclastinib by the end of 2025, targeting non-advanced systemic mastocytosis [3]. - A phase III trial comparing Bezuclastinib with Sutent is ongoing, with results expected in the second half of 2025 [4]. - The stock reached a 52-week high of $16.99, up from $7.25 when last featured [5]. Assembly Biosciences Inc. (ASMB) - Assembly Biosciences is advancing four key development programs, with ABI-5366 expected to enter phase 2 studies in mid-2026 [6]. - Interim data for ABI-1179 is anticipated this fall, while ABI-6250 is in a phase 1a trial [7]. - The stock hit a 52-week high of $28, up from $14.53 when last featured [8]. Compass Therapeutics Inc. (CMPX) - Compass Therapeutics is conducting a phase 2/3 study of Tovecimig for advanced biliary tract cancer, with analyses of secondary endpoints expected in Q1 2026 [9][10]. - The stock reached a 52-week high of $4.39, up from $2.91 when last featured [11]. NewAmsterdam Pharma Company N.V. (NAMS) - NewAmsterdam Pharma is developing Obicetrapib as a cholesterol-lowering therapy, with positive data from the BROADWAY trial [12][13]. - The company has completed two additional phase III trials and submitted marketing applications to the EMA [16]. - The stock reached a 52-week high of $39.76, up from $21.56 when last featured [17]. Mineralys Therapeutics Inc. (MLYS) - Mineralys is developing Lorundrostat for uncontrolled hypertension, with a pivotal phase III trial achieving its primary endpoint [19]. - A phase II trial for overweight participants with OSA is ongoing, with topline results expected in 1H 2026 [20]. - The stock hit a 52-week high of $43.88, up from $10.34 when last featured [20]. Kymera Therapeutics Inc. (KYMR) - Kymera is set to report data from its phase I trial of KT-621 this quarter, with phase 2b studies planned for late 2025 and early 2026 [21][22]. - The stock reached a 52-week high of $60, up from $40 when last featured [22]. Insmed Inc. (INSM) - Insmed has two approved drugs and is conducting a phase 3 trial of Arikayce, with topline results expected in 1H 2026 [24][25]. - The stock hit a high of $166.54, up from $76.54 when last featured [26]. Adaptive Biotechnologies Corp. (ADPT) - Adaptive Biotechnologies expects MRD revenue between $190 million and $200 million for 2025, up from $145.5 million in 2024 [28][29]. - The stock reached a 52-week high of $15.94, up from $9.80 when last featured [29]. BridgeBio Pharma Inc. (BBIO) - BridgeBio has upcoming topline results from the FORTIFY and CALIBRATE studies expected in Fall 2025 [30]. - The stock reached a 3-year high of $56.24, up from $25.10 when last featured [31]. Tarsus Pharmaceuticals Inc. (TARS) - Tarsus reported strong sales for Xdemvy, with Q2 2025 sales of $102.7 million, compared to $40.8 million in Q2 2024 [32]. - The stock hit an all-time high of $70.15, up from $25.01 when last featured [34]. Palvella Therapeutics Inc. (PVLA) - Palvella's QTORIN is under development for various skin diseases, with a phase 2 trial expected to report data in mid-December 2025 [35][36]. - The stock reached a 52-week high of $76.76, up from $25 when last featured [36]. Merus N.V. (MRUS) - Merus agreed to be acquired by Genmab for $97 per share, with the deal expected to close in early Q1 2026 [37]. - The stock was at $39.71 when last featured [39]. Nephros Inc. (NEPH) - Nephros reported net revenue of $4.4 million for Q2 2025, marking its third consecutive quarter of profitability [40][41]. - The stock hit a 52-week high of $5.98, up from $2.93 when last featured [42].