Tafamidis

- 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

—Supports BioCryst’s evolution toward a more disciplined R&D model centered on external innovation— RESEARCH TRIANGLE PARK, N.C., Oct. 05, 2026 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced the formation of a Scientific Advisory Board (SAB) comprising seven accomplished scientific and industry leaders with deep expertise across rare disease research and drug development. The SAB will provide independent scientific and strategic guidance as the Company advances its research and development strategy and builds a high value novel drug pipeline. “The formation of BioCryst’s Scientific Advisory Board marks an important step in the evolution of our new R&D model,” said Dr. Sandeep Menon, BioCryst’s Chief Research and Development Officer. “External innovation is increasingly central to how we build our pipeline, and guidance from leading scientists and physicians will sharpen our thinking and strengthen the diligence behind each program we evaluate and advance. The Scientific Advisory Board will work closely with me and our Chief Scientific Officer, Dr. David Jenkins, as we shape and execute BioCryst’s R&D strategy.” “Building on our success in developing and commercializing medicines for hereditary angioedema, we are bringing the same rigor to the next generation of BioCryst medicines,” said Charlie Gayer, Chief Executive Officer. “With the expertise of these highly experienced scientific advisors, BioCryst will be better positioned to evaluate emerging opportunities and translate their potential into meaningful benefits for patients.” The SAB will provide strategic guidance across BioCryst’s research and development activities, including pipeline expansion, external innovation opportunities, translational science and clinical development. The board will also offer insights on regulatory strategy and help strengthen connections with leading scientific, medical and patient communities worldwide. "Few companies have demonstrated BioCryst’s ability to translate rigorous science into rare disease medicines that reach patients, and that track record is what makes the Company’s evolution so compelling," said Dr. Anthony Manning, Chair of the Scientific Advisory Board. "The formation of this Board is an important step for BioCryst, and it is a privilege to chair a group with this depth of experience across rare disease drug discovery, translational science and clinical development. I am confident the Company’s decisions about which science to pursue and how to advance it will benefit from the collective judgment of its members." Scientific Advisory Board Members: Anthony (Tony) Manning, PhD, Chair, is Principal at Manning Bio Worldwide, LLC, advising biotechnology companies and academic institutions following a 30-year career in pharmaceutical drug discovery and development. As Chief Scientific Officer at Momenta Pharmaceuticals (now part of Johnson & Johnson), he built a pipeline of first-in-class therapeutics for rare autoantibody-driven diseases, including nipocalimab (IMAAVY), now FDA-approved for generalized myasthenia gravis and warm autoimmune hemolytic anemia, with additional indications in development. Earlier, he led drug discovery efforts at Roche Pharmaceuticals and Biogen Idec, contributing to the approvals of Actemra and Rituxan, and to the development of litifilimab, now in Phase 3 for lupus. Dr. Christine Bulawa, PhD, received a PhD from the University of Wisconsin-Madison and completed postdoctoral studies at Massachusetts Institute of Technology (MIT). As part of her 20+ years in rare disease drug discovery, she was the first employee of FoldRx Pharmaceuticals and instrumental in the preclinical development of tafamidis, the first pharmacological treatment for transthyretin amyloidosis. As a senior leader in Pfizer’s Rare Disease Research Unit, Dr. Bulawa built a pipeline of innovative programs based on rigorously validated targets, spanning portfolio entry to clinical development, with a focus on small molecule and genetic therapies to treat degenerative neurological, neuromuscular and protein aggregation diseases. Dr. Eric A. Hughes, MD, PhD, serves as Executive Vice President, Global Research & Development and Chief Medical Officer at Teva Pharmaceuticals. A physician-scientist who earned his MD and PhD from Yale University, Dr. Hughes brings nearly 20 years of biopharmaceutical industry experience and is helping advance Teva’s transformation into a leading innovative biopharmaceutical company, enabled by a world-class generics business. Prior to Teva, he served in leadership roles at Vertex, Novartis and Bristol Myers Squibb. Dr. Stephanie Noviello, MD, MPH, is Chief Medical Officer at ILiAD Biotechnologies, Inc. She is a clinical development leader with extensive experience across infectious diseases, virology, immunology, rare diseases and skeletal diseases at Novartis, Motif BioSciences, Bristol-Myers Squibb and Schering-Plough Research Institute. Dr. Shashi Ramaiah, DVM, PhD, DACVP, DABT, is a board-certified Veterinary Pathologist and Toxicologist who spent 18 years at Pfizer, most recently as Vice President and Global Head of Discovery, Investigative and Translational Sciences. He is Founder and CEO of SPECCRO, a drug development advisory firm that helps biotechnology companies and investors de-risk programs and portfolios, drawing on more than 40 senior industry experts and extensive engagement with national and international scientific consortia. Dr. Art Taveras, PhD, is a seasoned biopharmaceutical executive with over 35 years of industry experience, specializing in leading R&D divisions across major public pharmaceutical corporations and biotech firms including Biogen Idec, Transform Therapeutics, X4 Pharmaceuticals, CoMET Therapeutics, ShangPharma ChemPartner, Alantos Pharmaceuticals and Schering-Plough. His strategic expertise as President, CEO, CSO and R&D Leader has advanced robust pipelines and key global R&D partnerships, culminating in over 175 patent application filings, 150 publications, 60 clinical trials, 6 regulatory agency approvals and 5 therapeutic platform technologies across commercial and clinical-stage assets for diseases in neurology, oncology, immunology, metabolism and rare disease. Dr. Ajay Yekkirala, PhD, is an experienced serial entrepreneur who co-founded Superluminal Medicines at RA Capital and built a strong investor syndicate including NVIDIA, Eli Lilly, Insight Partners, Catalio, BVF and Perceptive Advisors. As Chief Scientific Officer, he directs the AI/ML platform build and all pipeline R&D. Prior to Superluminal, Dr. Yekkirala founded Blue Therapeutics to develop non-addictive painkillers. He received his PhD from the University of Minnesota and completed postdoctoral training at Harvard Medical School. He has authored several papers published in high-impact, peer-reviewed journals and advised various committees at the NIH, including EPPIC-NET at NINDS and Medication Development Research Committee at NIDA. About BioCryst Pharmaceuticals BioCryst is a global biotechnology company focused on developing and commercializing medicines for hereditary angioedema (HAE) and other rare diseases, driven by its deep commitment to improving the lives of people living with these conditions. BioCryst has commercialized ORLADEYO® (berotralstat), the first oral, once-daily plasma kallikrein inhibitor and is advancing a pipeline of potential first-in-class or best-in-class therapeutics for rare diseases. For more information, please visit or follow us on LinkedIn . Forward-Looking Statements This press release contains forward-looking statements, including statements regarding future results, performance, or achievements, expectations regarding BioCryst’s strategic shift to prioritize external innovation, expectations regarding pipeline development, the anticipated role and potential benefits of BioCryst’s Scientific Advisory Board, and statements related to BioCryst’s future operations, prospects and pipeline programs. These statements involve known and unknown risks, uncertainties and other factors which may cause BioCryst’s actual results, performance, or achievements to be materially different from any future results, performance, or achievements expressed or implied by the forward-looking statements. These statements reflect our current views with respect to future events and are based on assumptions and are subject to risks and uncertainties. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Some of the factors that could affect the forward-looking statements contained herein include: BioCryst’s ability to successfully progress its pipeline development plans; guidance and recommendations provided by the Scientific Advisory Board may not improve BioCryst’s evaluation or advancement of potential programs or result in the anticipated benefits; the commercial viability of BioCryst’s future rare disease therapies; ongoing and future development of product candidates may take longer than expected and may not have positive results; product candidates, if approved, may not achieve market acceptance; the results of BioCryst’s partnerships with third parties may not meet BioCryst’s current expectations; and BioCryst’s ability to realize the contemplated benefits of its new R&D strategy and model and successfully evaluate and execute external opportunities. This list is not exclusive. To see a more comprehensive list of risks, please refer to the documents BioCryst files periodically with the Securities and Exchange Commission, specifically BioCryst’s most recent Annual Report on Form 10-K, Quarterly Reports on Form 10-Q, and Current Reports on Form 8-K, which identify important factors that could cause actual results to differ materially from those contained in BioCryst’s projections and forward-looking statements. Contact: Investors: investorrelations@biocryst.com Media: media@biocryst.com