Tafamidis

iStock, Jackie Niam While Alnylam’s recent reduced revenue guidance spooked investors, analysts remain broadly bullish on the biotech’s commercial prospects. Alnylam was flying high one year ago. The RNAi pioneer’s share price was rising ever closer to $500 apiece amid the rapid growth of its transthyretin-mediated amyloidosis (ATTR) franchise thanks to a March 2025 FDA nod and the progress of late-phase programs. Today, the stock has fallen to around $250. The company’s share price collapsed when management lowered full-year guidance in July. Sales of TTR silencer Amvuttra for the second quarter fell short of analyst expectations and Alnylam reduced its full-year guidance by about $200 million. The news raised doubts about the RNAi product’s prospects in a market served by small-molecule TTR stabilizers from BridgeBio Pharma and Pfizer. Earnings Alnylam absorbs $10.8B blowback after cutting full-year guidance for TTR franchise Alnylam’s revenue cut “may create a near term credibility overhang and could “raise questions on peak sales potential, even if there’s tailwinds in the competitive landscape,” according to Stifel. July 31, 2026 · 2 min read · Tristan Manalac Read more Guggenheim Securities analysts set out the concerns raised by the revised guidance in a July 31 note to investors. The cut, which happened despite Amvuttra being the “sole, differentiated modality” in ATTR cardiomyopathy (ATTR-CM), suggested that the commercial opportunity is smaller than expected, the drug is mispriced or TTR stabilizers are becoming the preferred modality, the analysts said. Alnylam’s stock has yet to recover. However, while the Guggenheim team was “on the sidelines on the stock” even before the guidance cut, other analysts have retained faith in a comeback. TD Cowen assigned a $482 price target to Alnylam back in November 2025, when the stock traded above $430. The analysts reiterated the target last month, even as Alnylam traded below $250. The debates driving Alnylam’s stock BMO Capital Markets analysts, who began covering Alnylam in August with a $318 price target, set out three key debates about the biotech’s prospects in an Aug. 18 note to investors. Alnylam bulls and bears are split on multiple questions, the analysts said—about the size of the ATTR market, whether the company’s next-generation RNAi candidate nucresiran creates value and the likelihood of gene silencing becoming a major drug modality. The BMO team sided with the bulls on each point, arguing that Alnylam cut its guidance because the population of patients that Amvuttra served most urgently—those for whom stabilizers hadn’t worked—"exhausted sooner than expected.” Through that perspective, the guidance cut changed the shape of the sales ramp, not the size of the opportunity. “The path to $10 billion+ in franchise sales was never through the second-line,” the analysts wrote, “it always ran through front-line starts and expansion of the diagnosed population.” cardiovascular disease ‘Worse than expected’ Wainua data muddies water for TTR silencers AstraZeneca and Ionis Pharmaceuticals’ data drop divulging more details from its highly watched Phase 3 transthyretin silencer trial “further confuses the picture” for Wainua when combined with the standard of care for ATTR-CM, analysts say. August 28, 2026 · 2 min read · Gabrielle Masson Read more As the analysts acknowledge, there is another way to view the guidance cut. An Alnylam bear may argue that cutting sales guidance two quarters into the ATTR-CM launch says something about the size of the pool. Another skeptical reading is that, having burned through the second-line market, Alnylam faces the harder challenge of competing with Pfizer’s Vyndamax and BridgeBio’s Attruby for first-line patients. Alnylam is running Phase 3 trials of nucresiran in ATTR-CM and ATTR with polyneuropathy (ATTR-PN)—the indication for which Amvuttra was first approved, back in 2022. The late-stage failure of AstraZeneca and Ionis’ Wainua in ATTR-CM in July raised questions about nucresiran, but after the dust settled, analysts became more comfortable with Alnylam’s prospects. In a Sept. 3 note, Stifel analysts said endpoint selection and patient enrollment drove Wainua’s failure. That view, which was echoed by BMO analysts, suggests Alnylam “is positioned for success with their event-driven study that is enriched for earlier-stage patients,” the Stifel team said. The broadening RNAi pipeline Beyond ATTR, Alnylam is applying its technology to big markets including hypertension and Alzheimer’s disease. BMO analysts compared gene silencing today to where monoclonal antibodies were in the late 1990s. Antibody developers ignored doubters and established a new, major modality. If gene silencing biotechs do the same, “Alnylam will own the success” more than any other company, BMO analysts said. A Phase 3 cardiovascular outcomes trial of blood pressure prospect zilebesiran “tests whether RNAi can be delivered to a population-scale chronic indication,” BMO analysts said. A successful readout “could unlock a significant commercial opportunity and highlight the value of Alnylam’s ex-ATTR franchise,” the analysts added in a Sept. 17 note. Still, skepticism abounds. Stifel analysts, also writing on Sept. 17, said they “remain cautious on the safety pro continue to question whether the modest [blood pressure] reductions observed in [Phase 2] are sufficient to drive a clinically meaningful [cardiovascular outcomes trial] benefit.” Alnylam is betting that consistent, modest blood pressure reductions can cut the risk of cardiovascular events. Earnings BridgeBio continues ATTR ascent as Alnylam’s Amvuttra falls short of expectations BridgeBio Pharma’s transthyretin amyloidosis medicine Attruby is off to a strong launch, while Alnylam lowered full-year sales guidance for competitor Amvuttra. August 12, 2026 · 2 min read · Tristan Manalac Read more If that bet pays off, Alnylam could cement its position at the forefront of RNAi therapeutics. ADARx Pharmaceuticals’ $446.3 million IPO in September and the 2025 deal between Novartis and China’s Argo Biopharmaceutical illustrate the growing and increasingly global nature of the sector. But Alnylam was the pioneer, and its market cap is several-fold larger than its closest biotech rivals. Having turned its first-mover advantage into a $3.3 billion cash cushion, Alnylam is considering striking deals for drug candidates and technologies for delivering therapies to new tissues, Stifel analysts said. Alnylam is looking at areas that are complementary to what it is already working on from a commercial and R&D perspective, the analysts added. Alnylam also has opportunities to reignite enthusiasm for its internal pipeline. This year, the biotech is set to publish Phase 2 data on a bleeding disorder drug candidate, as well as Phase 1 results on Huntington’s disease and obesity programs. Alnylam is aiming to report five or more key readouts in 2027 and in 2028. .responsive-container { display: flex; flex-wrap: wrap; border: 1px solid #ededed; font-family: Helvetica, Arial, Sans-Serif; padding: 20px 20px 10px 20px; } .column-left { flex: 1; max-width: 45%; padding: 20px 20px 10px 20px; } .column-right { flex: 2; padding: 20px 20px 10px 20px; } @media (max-width: 768px) { .column-left, .column-right { max-width: 100%; flex: 100%; padding: 10px 0; } } Subscribe to BioPharm Executive! Market insights, trending business and policy stories for biopharma leaders hbspt.forms.create({ region: "na1", portalId: "4413123", formId: "7c139b3c-ff0b-44e0-bffe-f449801dca59", onFormSubmitted: function($form, data) { window.dataLayer = window.dataLayer || []; window.dataLayer.push({ 'event': 'GTMevent', 'event_name': 'newsletter_sign_up' }); } });

- In an independent, propensity score-matched analysis of 2,684 individuals living with ATTR-CM from Epic COSMOS, the largest U.S. electronic health record database, acoramidis was associated with a statistically significant 18% lower risk of a composite of death, all-cause hospitalization, new-onset atrial fibrillation, and diuretic intensification versus tafamidis (HR 0.82; 95% CI 0.73-0.93; p<0.01) - Early separation of the composite curves was noted within one month in this contemporary cohort of patients from 2025, with consistent results across unadjusted, matched, and biomarker-adjusted analyses - Acoramidis was associated with significant reductions in hospitalization, among the outcomes that matter most to people living with ATTR-CM, with a 27% lower risk of cardiovascular hospitalization, 27% lower risk of heart failure hospitalization, and 25% lower risk of all-cause hospitalization as compared to tafamidis (all p<0.001) - These findings replicate the earlier Wright et al. Komodo claims analysis published in Cardiology and Therapy , in which acoramidis was associated with a 34% reduction in clinical worsening events versus tafamidis (p=0.046), making this the second real-world comparative effectiveness analysis, and the first in electronic health records, to favor acoramidis, supporting the reproducibility of the benefit - ATTRibute-CM data also presented at HFSA underscore the importance of hospitalization reduction: mortality risk approximately doubled after a single cardiovascular hospitalization and quadrupled after two or more, making hospitalization prevention a central treatment goal in ATTR-CM - Together, these data have the potential to inform the use of acoramidis as the stabilizer of choice, both in newly diagnosed patients and in those on other disease-modifying agents PALO ALTO, Calif., Oct. 11, 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 that new, independent and contemporary real-world evidence as well as Phase 3 ATTRibute-CM sub-analyses of acoramidis in transthyretin amyloid cardiomyopathy (ATTR-CM) were presented at the Heart Failure Society of America (HFSA) Annual Scientific Meeting (ASM) 2026 in Phoenix, Arizona. Acoramidis is the only selective small molecule, orally administered, near-complete (≥90%) transthyretin (TTR) stabilizer. "In a prior real-world claims analysis, acoramidis was associated with fewer diuretic intensification events, suggesting the possibility of greater clinical stability on acoramidis, but this obviously needed to be interpreted with caution. This new, larger, electronic health record study of nearly 2,700 individuals with ATTR-CM with longer follow-up and more granular data is better able to address the question, and shows a similar signal, with a significant difference in rates of cardiovascular or heart failure hospitalizations favoring acoramidis compared to tafamidis, with approximately one quarter lower risk of events," said David Lanfear, M.D., M.S., Chief Scientific Officer, Vice President of Research at Henry Ford Health, U.S. "While electronic health record studies have well-recognized limitations, these data allow us to see how these therapies perform outside of a clinical trial, across a diverse and contemporary patient population. Moreover, there appears to be some consistency of findings across data sources and endpoints, supporting validity. Additional investigations to replicate and extend these findings are warranted." In a retrospective analysis conducted and presented by Dr. Lanfear, individuals with ATTR-CM newly initiating acoramidis had lower rates of adverse cardiac outcomes than individuals newly initiating tafamidis. The study leveraged data from Epic COSMOS, the largest U.S. electronic health record database, with over 300 million patients. This dataset includes detailed, anonymized clinical records captured in the course of routine patient care, such as laboratory values and medication records. After matching, 910 acoramidis-treated and 1,774 tafamidis-treated individuals were included, with similar baseline characteristics across treatment groups. Key findings included: The composite endpoint of death, all-cause hospitalization, new-onset atrial fibrillation, or diuretic intensification was significantly lower with acoramidis across all three analyses: unadjusted (HR 0.82; 95% CI 0.72-0.90; p<0.001), primary (18% lower risk; HR 0.82; 95% CI 0.73-0.93; p<0.01), and a sensitivity analysis adjusting for a combined standardized NT-proBNP and BNP variable where available (HR 0.84; 95% CI 0.72-0.97; p=0.02) Acoramidis-treated individuals had statistically significantly lower rates of hospitalization than tafamidis-treated individuals, including a 27% lower risk of cardiovascular hospitalization (HR 0.73; 95% CI 0.62-0.86; p<0.001), a 27% lower risk of heart failure hospitalization (HR 0.73; 95% CI 0.62-0.87; p<0.001), and a 25% lower risk of all-cause hospitalization (HR 0.75; 95% CI 0.64-0.88; p<0.001) Diuretic intensification, an early marker of disease progression, was 17% lower with acoramidis (HR 0.83; 95% CI 0.71-0.95; p<0.01). Diuretic intensification was defined as use of parenteral loop diuretics, initiation or dose-equivalent escalation of oral loop diuretics, or use of a thiazide-like diuretic Differences in death (HR 0.85; 95% CI 0.62-1.20; p=0.34) and new-onset atrial fibrillation (HR 0.88; 95% CI 0.68-1.13; p=0.31) numerically favored acoramidis, consistent with the composite, but did not reach statistical significance, reflecting lower event counts and limited duration of follow-up for these longer-term endpoints. These endpoints will be reassessed as the cohort matures Kaplan-Meier time-to-event curves for the composite endpoint showed early and sustained separation between treatment groups over follow-up (p=0.0014) The consistency of the treatment effect across unadjusted, matched, and biomarker-adjusted analyses and across multiple clinical outcomes supports the robustness of the observed difference These findings in electronic health record data replicate and expand on the results of the first peer-reviewed, claims-based real-world comparative effectiveness study of TTR stabilizers in ATTR-CM, in which acoramidis was associated with a 34% reduction in a composite of clinical worsening events (diuretic intensification, heart failure hospitalization, and mortality) versus tafamidis (p=0.046), supporting the reproducibility of the observed benefit across independent data sources and data types. As with all observational studies, there are inherent limitations. Although propensity-score matching balanced patient baseline characteristics and results remained consistent across both unweighted and sensitivity analyses, the possibility of residual confounding cannot be fully excluded. Follow-up was relatively short due to recent approval of acoramidis, which limited the number of events available to assess less frequent outcomes, such as mortality. These factors would not be expected to affect one treatment group differently than the other, and the consistency of results across multiple endpoints and in multiple studies supports the robustness of the findings. As data continue to mature, additional analyses with longer follow-up periods will be shared. The full results of this analysis are currently under peer-review by a journal. Three additional analyses presented at HFSA ASM 2026 continued to reinforce the acoramidis narrative, reaffirming the importance of reducing cardiovascular-related hospitalizations (CVH) in ATTR-CM, the differentiated cardiorenal impact of acoramidis, and the benefit of acoramidis within variant ATTR-CM patients: Cardiovascular-related hospitalizations associated with higher risk of mortality: Association Between the Burden of CVH and All-Cause Mortality (ACM) in Transthyretin Amyloid Cardiomyopathy: Insights from ATTRibute-CM , presented by Quan Bui, M.D. of UC San Diego Health, U.S. In this post hoc analysis of ATTRibute-CM (pooled treatment groups; N=611), fewer than half of participants who experienced two or more cardiovascular-related hospitalizations were alive at 30 months. Survival fell from 86.7% in participants with no CVH events to 68.4% with one and 47.7% with two or more (log-rank p<0.0001) Mortality risk approximately doubled after a single hospitalization and quadrupled after two or more, establishing CVH burden as a marker of subsequent mortality risk and reinforcing hospitalization prevention as a clinically meaningful treatment goal in ATTR-CM Acoramidis reduced the annualized frequency of CVH by 50% through Month 30 versus placebo (p < 0.0001), with Kaplan-Meier curves for time to first CVH separating from Month 3 Unique cardiorenal impact of acoramidis: Early eGFR Dip Following Acoramidis Initiation is Associated With Reduced Risk of Mortality and Recurrent Cardiovascular-Related Hospitalizations in ATTR-CM, presented by Ahmad Masri, M.D., M.S. of Oregon Health & Science University, U.S. In the ATTRibute-CM study, acoramidis-treated participants with a greater Day 28 eGFR dip following treatment initiation were associated with a significantly reduced risk of mortality or recurrent CVH versus placebo, with no clinically concerning changes observed in laboratory parameters or blood pressure These findings provide additional context for the reported association between greater early eGFR decline and improved first CVH outcomes with acoramidis, and build upon the 42% reduction in recurrent CVH/ACM previously demonstrated with acoramidis over placebo in ATTRibute-CM These results build upon analyses previously published in Circulation: Heart Failure; details on results can be found here Acoramidis benefit in p.Val142Ile variant patients: Acoramidis Preserves Functional Capacity and Quality of Life in p.Val142Ile Variant Transthyretin Amyloid Cardiomyopathy: Results from ATTRibute-CM, presented by Lily Stern, M.D. of Cedars-Sinai Heart Institute, U.S. In this post hoc analysis of 35 ATTRibute-CM participants with the p.Val142Ile variant (acoramidis, n = 23; placebo, n = 12), acoramidis significantly attenuated 30-month declines in heart failure-related health status and quality of life versus placebo: differences of 23.3 points in KCCQ-OS score (p = 0.005), 25.8 points in EQ VAS score (p = 0.003), and 0.26 in EQ-5D-5L index score (p = 0.018), each several-fold above published thresholds for clinically meaningful change. Decline in 6-minute walk distance was numerically smaller with acoramidis (LSM difference: 67.2 m; p = 0.136) These findings complement the previously reported 69% reduction in the risk of all-cause mortality or first cardiovascular-related hospitalization with acoramidis through Month 30 in this subgroup In addition to the two rapid-fire oral presentations, one oral presentation, and one poster presentation highlighted, there was one additional rapid-fire oral presentation and five additional poster presentations along with four encore poster presentations shared at HFSA ASM 2026, which included: Ratio of Serum Transthyretin to NT-proBNP is a Novel Prognostic Measure for Transthyretin Amyloid Cardiomyopathy: Insights from ATTRibute-CM , presented by James L. Januzzi, M.D. of Massachusetts General Hospital, U.S. and simultaneously published in JACC: Heart Failure In ATTR-CM, early acoramidis-mediated sTTR increases were associated with later NT-proBNP decreases. The Day 28 sTTR to Month 12 NT-proBNP ratio, integrating early pharmacodynamic response with downstream cardiac stress, is a prognostic biomarker of clinical risk in ATTR-CM Falls and Fractures Drive Increased Hospitalizations and Costs in Patients with Transthyretin Amyloid Cardiomyopathy Compared with Controls , presented by Nitasha Sarswat, M.D. of University of Chicago, U.S. ATTR-CM was associated with increased falls and fall-related consequences, including fractures, bleeding after a fall, discontinuation of oral anticoagulant use, and related hospitalizations and costs, compared with matched heart failure (HF) and non-HF controls, consistent with substantial disease burden beyond cardiac manifestations and with higher healthcare resource utilization Evaluating the Long-Term Effects of Acoramidis on Cardiac Function, Structure, and Amyloid Burden in Transthyretin Amyloid Cardiomyopathy: ASCEND-ATTR , presented by Ahmad Masri, M.D., M.S. of Oregon Health & Science University, U.S. ASCEND-ATTR will determine whether long-term transthyretin stabilization is associated with sustained improvement in myocardial structure, function, and amyloid burden. By integrating serial CMR and echocardiography, the study aims to provide mechanistic insights into cardiac remodeling during treatment and help define imaging biomarkers of therapeutic response Real-World Burden of Transthyretin Amyloid Cardiomyopathy Disease and Outcomes in Patients on Tafamidis Prior to Initiating Acoramidi s, presented by Richard Wright, M.D. of Pacific Heart Institute, U.S. Nearly half of patients receiving tafamidis exhibited markers of ATTR-CM clinical worsening within the 6 months preceding acoramidis initiation. This burden appears higher than previously reported real-world disease progression rates after tafamidis initiation and warrants further study. Future research should evaluate drivers of switching therapy and outcomes after transitioning to acoramidis Acoramidis Reduces Risk of Cardiovascular-related Mortality and Hospitalization in Women with Transthyretin Amyloid Cardiomyopathy, presented by Margot Davis, M.D. of University of British Columbia, Vancouver, Canada Among women enrolled in ATTRibute-CM, who entered the trial older and with a more advanced clinical phenotype than men, acoramidis reduced the risk of cardiovascular mortality or first cardiovascular-related hospitalization through Month 30 and attenuated the rise in NT-proBNP versus placebo. Treatment effects in women aligned with the overall population, with no significant treatment-by-sex interaction. This may represent the first reported analysis from a phase 3 ATTR-CM trial to show a nominally statistically significant treatment effect for this endpoint among women. While the small number of women enrolled limits precision, these findings support the efficacy of acoramidis in women and underscore the need for adequately powered, sex-informed evidence in ATTR-CM Effect of Acoramidis on Heart Failure-Related Health Status Before and After Cardiovascular-Related Hospitalization: Insights from ATTRibute-CM , presented by Ahmad Masri, M.D., M.S. of Oregon Health & Science University, U.S. Worsening KCCQ-OS precedes CVH and may serve as an early indicator of an adverse outcome in patients with ATTR-CM. Following a CVH event, acoramidis was associated with recovery of health status versus continued decline observed with placebo, suggesting a beneficial effect on post-CVH health status trajectory Acoramidis is approved as Attruby® by the U.S. FDA and is approved as BEYONTTRA® by the European Medicines Agency (EMA), Japanese Pharmaceuticals and Medical Devices Agency, Swissmedic, the Swiss Agency for Therapeutic Products, the UK Medicines and Healthcare Products Regulatory Agency, and the Brazilian Health Regulatory Agency (ANVISA) with all labels specifying near-complete stabilization of TTR. Additional data on the benefit of Attruby for individuals with ATTR-CM is planned for future medical meetings. 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 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 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, or the negative of these terms or other comparable terminology are intended to identify forward-looking statements, though not all forward-looking statements necessarily contain these identifying words. 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 include statements regarding the potential clinical significance and therapeutic implications of the real-world and ATTRibute-CM data regarding acoramidis, including the potential for the findings to inform treatment selection for newly diagnosed patients and patients currently receiving other disease-modifying therapies and to support switching individuals to acoramidis; the potential reproducibility, durability and broader clinical significance of observed benefits associated with acoramidis across independent data sources, including with respect to cardiovascular and heart failure hospitalizations, clinical stability and other outcomes; the potential clinical and therapeutic implications of the ATTRibute-CM analyses, including with respect to cardiovascular hospitalization, cardiorenal outcomes, biomarkers, health status and outcomes in patient subgroups; the design, conduct and anticipated findings of ASCEND-ATTR, including whether long-term TTR stabilization is associated with sustained improvement in myocardial structure, function and amyloid burden and the potential to identify imaging biomarkers of therapeutic response; and BridgeBio’s plans and expectations regarding additional analyses, longer-term follow-up, future research, publications and presentations of data regarding acoramidis. 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, the risk that results from retrospective, observational, real-world evidence studies, including studies based on electronic health records or administrative claims data, may be subject to confounding, selection bias, residual bias, incomplete or inaccurate data, limited follow-up or other limitations and may not be predictive of future clinical outcomes or treatment effects; that observed associations and differences between acoramidis and tafamidis may not be replicated in additional analyses, independent studies or longer-term data or may not translate into improved long-term clinical outcomes; that results from post hoc analyses, subgroup analyses or other analyses may not be predictive of future clinical outcomes or treatment effects and may not be replicated in additional analyses or studies; that observed effects on cardiovascular hospitalizations, cardiorenal outcomes, biomarkers, health status or outcomes in patient subgroups may not be replicated or translate into meaningful long-term clinical benefit; that the findings may not meaningfully inform treatment selection or support switching patients to acoramidis; that mechanistic or prognostic interpretations of observed data, including potential relationships among TTR stabilization, cardiac remodeling, cardiorenal effects, biomarkers and clinical outcomes, may not be borne out by further analyses or additional data; that ASCEND-ATTR may not demonstrate sustained improvement in myocardial structure, function or amyloid burden or identify useful imaging biomarkers of therapeutic response; that additional analyses, longer-term follow-up or future research may yield results that differ from or do not confirm the findings described in this press release; that plans or timing for future analyses, medical meeting presentations or scientific publications may change; 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: Kaitlyn Reilly, Director, Communications contact@bridgebio.com (650) 789-8220 BridgeBio Investor Contact: Kristen Kelleher, Director, Investor Relations ir@bridgebio.com