Slides
Page 1
CONFIDENTIAL & PROPRIETARY Full Year 2025 Results Call Corporate Update & Financial Results February 26, 2026
Page 2
Forward -looking statements 2 This presentation contains forward-looking statements, including statements regarding, among other things, future results, performance or achievements, expectations regarding pipeline development, anticipated approval and commercialization of navenibart, pharmaceutical research and development, such as drug discovery, preclinical and clinical development activities and related timelines, expected HAE portfolio revenue growth and addressable market, anticipated benefits, performance, and competitive positioning of, and market size for, navenibart and BCX17725, potential best-in-class or first-in-class positioning of product candidates, intellectual property runway for our products and product candidates, and BioCryst’s plans, objectives, expectations, intentions, growth strategies and other statements that are not historical facts. 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 as described herein, including meeting the expected timelines; ongoing and future preclinical and clinical development of product candidates may take longer than expected and may not have positive results; the outcome of preclinical testing and early clinical trials may not be predictive of the success of later clinical trials, and interim results of a clinical trial do not necessarily predict final results; BioCryst may not be able to enroll the required number of subjects in planned clinical trials of product candidates; BioCryst may not advance human clinical trials with product candidates as expected; the FDA or other applicable regulatory agency may require additional studies beyond the studies planned for products and product candidates, may not provide regulatory clearances which may result in delay of planned clinical trials, may not review regulatory filings on our expected timeline, may impose certain restrictions, warnings, or other requirements on products and product candidates, may impose a clinical hold with respect to product candidates, or may withhold, delay or withdraw market approval for products and product candidates; the sustainability of profitability and positive cash flow may not meet management’s expectations; statements and projections regarding financial guidance and goals and the attainment of such goals may differ from actual results based on market factors and BioCryst’s ability to execute its operational and budget plans; and actual financial results may not be consistent with expectations, including that revenue, operating expenses and cash usage may not be within management’s expected ranges. 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 (the “SEC”), 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.
Page 3
3 This presentation includes non-GAAP financial measures that differ from measures calculated in accordance with generally accepted accounting principles in the United States of America (“GAAP”), including financial measures labeled as “non-GAAP.” We believe providing these non-GAAP measures, which show our results with these items adjusted, is valuable and useful since they allow management and investors to better understand the company’s financial performance in the absence of certain non-cash items such as stock-based compensation and certain special events and allow investors to more accurately understand our current and past period results and more easily compare them to future results. These non-GAAP measures also correspond with the way we expect investors and financial analysts to compare our results. Our non-GAAP measures should be considered only as supplements to, and not as substitutes for or in isolation from, our other measures of financial information prepared in accordance with GAAP, such as GAAP revenue or operating income. Our references to non-GAAP operating profit (loss) and non-GAAP ORLADEYO revenue constitute non-GAAP financial measures. These non-GAAP financial measures are calculated using our GAAP results, adjusted to show the results without including, as applicable, revenues and expenses associated with our European ORLADEYO business, license revenue related to the license of intellectual property to Neopharmed Gentili S.p.A., transaction-related costs, non-cash stock-based compensation expense, and workforce reduction expenses. A reconciliation between each non-GAAP financial measure and its respective closest equivalent GAAP financial measure is provided in the tables in the appendix. We also provide our non-GAAP operating expense outlook for full year 2026, which refers to our expected GAAP operating expense, excluding stock- based compensation, restructuring and transaction-related costs. We have not provided a reconciliation against the comparable forward-looking GAAP measure because we are unable to predict with reasonable certainty the full amount of stock-based compensation expense or restructuring or transaction-related costs for the full year 2026 without unreasonable effort. Stock-based compensation expense is uncertain and depends on various factors, including our future hiring and retention needs, as well as the future fair market value of our common stock, which is difficult to predict and subject to change. In addition, we are unable to predict with reasonable certainty the full amount of restructuring and transaction-related costs as the related costs are dependent on various factors that have not yet or have only recently occurred. The actual amount of stock-based compensation, restructuring and transaction-related costs for the full year 2026 could have a material impact on GAAP reported results for the guidance period. Non-GAAP Financial Measures
Page 4
AGENDA 4 Corporate update Charlie Gayer President and Chief Executive Officer Pipeline update Dr. Bill Sheridan Chief Development Officer Financial update Babar Ghias Chief Financial Officer Q&A
Page 5
BioCryst: delivering sustainable growth in rare disease 5 Steady growth with high cash flow visibility • Sustainable $1B peak revenue opportunity for ORLADEYO • >80% contribution margin 1 • IP runway into 20402 Maximizing potential of rare disease pipeline • Netherton syndrome: high unmet need and potential for best-in-class therapy • Targeted rare disease- focused discovery • Externalizing non-core assets Via strategic business development • Focus on de-risked late- stage rare disease assets • Near-term value creation • Leveraging existing operating infrastructure 1. Contribution margin defined as revenue minus direct costs (COGS + S&M) 2. Pediatric extension through May 2040; composition of matter patent Value creation through three key strategic growth pillars Commercial Product Internal R&D External Opportunities
Page 6
Our pipeline 6Navenibart, BCX17725, avoralstat, and STAR-0310 are investigational and have not been deemed safe and effective by the FDA. ASSET PROGRAM PRE -CLINICAL PHASE 1 PHASE 2 PHASE 3/PIVOTAL APPROVED / COMMERCIAL CORE PROGRAMS ORLADEYO ® (berotralstat) Oral Plasma Kallikrein Inhibitor Hereditary Angioedema (HAE) ORLADEYO ® (berotralstat) Oral Plasma Kallikrein Inhibitor in Pediatrics Hereditary Angioedema (HAE) Navenibart Monoclonal Antibody Plasma Kallikrein Inhibitor Hereditary Angioedema (HAE) BCX17725 Protein Therapeutic Netherton Syndrome Undisclosed Rare Diseases NON-CORE PROGRAMS RAPIVAB ® (peramivir injection) Infectious Diseases Avoralstat Ocular Plasma Kallikrein Inhibitor Diabetic Macular Edema (DME) STAR-0310 Monoclonal Antibody OX40 Antagonist Atopic Dermatitis
Page 7
ORLADEYO: the first and only approved targeted oral for HAE prophylaxis 7 • ORLADEYO (berotralstat) is a plasma kallikrein inhibitor indicated for prophylaxis to prevent attacks of HAE in patients ages 2+ • Discovered in BioCryst labs • Approved in US Dec 2020; now in 6th year of launch • Pediatric formulation (pellets) approved Dec 12, 2025 • IP through May 20401 >1,600 patients on therapy 2 >3,500 patients have tried since launch >1,500 unique prescribers (figures reflect ORLADEYO metrics in US market) 1. Pediatric extension through May 2040; composition of matter patent 2. All patients including Quick Start, paid, and patient assistance program (PAP) At year -end 2025:
Page 8
ORLADEYO offers proven long -term attack control, demonstrated by strong real -world efficacy data 8 Great efficacy + differentiated convenience Strong retention over time (% of patients persistent at various time points)3 ORLADEYO efficacy is consistent with ~80 -90% reduction in attack rate provided by injectable therapies 86% 76% 61% 3 months 6 months 12 months Leads to 1. BL: baseline 2. Data from APeX-2 open label extension study; 150mg, n=21 completers; Kiani-Alikhan S, et al. J Allergy Clin Immunol Pract. 2024;12(3):733-743.e10. 3. Zuraw BL, et al. Allergy Asthma Proc. 2025 May 1;46(3):209-217. Persistence is defined as having no gap in treatment ≥45 days after the treatment initiation date. 2.8 1.1 0.9 1 0.6 0.6 1 0.6 0.7 0.3 0 0.5 1 1.5 2 2.5 3 BL¹ 1 3 6 9 12 15 18 21 24 HAE monthly attack rate Months of treatment with ORLADEYO²
Page 9
ORLADEYO: highly achievable path to $1B peak revenue 9 • ~150 net patient adds per year (adult + pediatric) • Paid rate improvement toward 85% at YE 2029 vs. 81% at YE 2025 2 • Modest annual price increases • Contribution from ex-US geographies $563M 2025¹ 2026 2029 $625-645M $1B Peak 1. Non-GAAP revenue excluding EU for FY 2025 2. Paid rate calculation does not include patients on Quick Start program Key drivers to peak
Page 10
Navenibart : a long -acting plasma kallikrein inhibitor for HAE prophylaxis 10 • Potential to be the first therapy for HAE prophylaxis with dosing every 3 or 6 months • Obtained through acquisition of Astria Therapeutics in January 2026 • Currently in pivotal Phase 3 studies • IP through 2042 Trusted mechanism & modality Monoclonal antibody inhibitor of plasma kallikrein Compelling efficacy data High affinity and potency with fast onset delivers rapid, effective attack prevention Infrequent dosing schedule YTE modification for extended half-life Pain-free subcutaneous administration Citrate-free, high-concentration formulation, delivered via autoinjector Program is on track to support regulatory filing by end of 2027
Page 11
Navenibart : potential for strong efficacy with optimal dosing profile 11 Q3M/Q6M, 3/6-month dosing NOTE: Efficacy data presented are derived from different clinical trials conducted at different times by different sponsors, with differences in trial design and patient populations. As a result, cross-trial comparisons cannot be made, and no head-to-head clinical trials have been conducted. ANDEMBRY: US Prescribing Information (2025). TAKHZYRO; US Prescribing Information (2025). Dawnzera; US Prescribing Information (2025). Navenibart data from: Long-Term, Sustained, Robust Hereditary Angioedema Attack Suppression with Navenibart Administered Every 3 and 6 Months: ALPHA-SOLAR Interim Results; poster being presented at the 2026 American Academy of Allergy, Asthma & Immunology (AAAAI) Annual Meeting, February 27–March 2, 2026. 30% 40% 50% 60% 70% 80% 90% 100% 0 4 8 12 16 20 24 28 Mean attack rate reduction Dosing interval (weeks) Navenibart (Q6M) 92% mean attack reduction 13.3 months mean follow up n=17 90% mean attack reduction 11.9 months mean follow up n=12 Navenibart (Q3M) (Q4W) (Q8W)
Page 12
BioCryst positioned to offer the leading oral and injectable 12 Navenibart Prefer an oral therapy Prefer an injectable therapy HAE patients receiving prophylaxis primarily fall into two categories: Oral preference Has steadily increased since 2023 Consistent growth in ORLADEYO demand reflects this dynamic Injectable preference Many patients are satisfied on existing injectables, although there is a clear unmet need Remaining unmet needs: • Q3M or longer dosing • Administration improvements • Reduced injection site pain Source: BioCryst Quarterly Market Research Surveys (2023- 2025) Combined portfolio to reach distinct, durable, and growing segments, covering full spectrum of patient preference Navenibart’s profile maps precisely to the remaining unmet needs in the injectable market Opportunity to offer the most patient -friendly options, optimally serving the HAE patient community
Page 13
Navenibart opportunity : 5,000+ patients Significant addressable opportunity in HAE 13 Estimated US patients on prophylaxis Source: BioCryst Internal Market Research Study (Conducted Jun 2025), 2018-2023 administrative claims data 1. Other includes Cinryze, androgens, acute used as prophy, and deucrictibant 7,600 8,400 9,100 9,700 2025 2026 2027 2028 2029 2030 2031 2032 2033 2034 Other¹ Injectable prophy ORLADEYO ORLADEYO predicted share: 2,000+ stable patients The durability of the ORLADEYO patient base is driven by strong, injectable-like efficacy and the convenience of oral administration
Page 14
BioCryst HAE franchise expected to generate double digit revenue growth into the next decade 14 $1B+ cash balance by 2029 1. Implied projections for navenibart based on Wall Street analyst research 2. Non-GAAP operating expenses expected to grow at a mid-single digit CAGR 3. Non-GAAP revenue excluding EU for FY 2025 Significant revenue growth Operating leverage & synergies with Astria Strong profitability and cash flow generation Enhanced financial capacity for future BD 2025E ex-EU³ 2029E 2033E $563M $1B+ ~15% CAGR~15% CAGR $1.8B+ HAE portfolio revenue 1 Illustrative operating expenses2
Page 15
1. Based on healthcare claims analysis Image: https://www.nethertonsyndrome.com/about-nethertons.php BCX17725: a targeted KLK5 inhibitor for Netherton syndrome (NS) What is Netherton syndrome? • A severe, rare, genetic disorder with widespread skin involvement and systemic complications • Causes premature separation of skin layers, severe inflammation, and infection risk • Diagnosed US population of ~1,6001 with potential to grow to 3,000-5,000 with greater diagnosis and treatment • No approved targeted therapies Aligned with core rare disease focus • High unmet need • Potential for market expansion over time due to misdiagnosis or underdiagnosis • Prescriber landscape comparable to HAE’s: addressable by small sales team • In-house analytics expertise primed to aid diagnosis and market development Clear biology • Validated target and known cause of disease: SPINK5 gene variant causes KLK5 overactivity • BCX17725 is a systemically-administered KLK5 inhibitor Potential for BioCryst to be first mover • BCX17725 could be the first-in-class targeted systemic therapy Why Netherton syndrome? 15
Page 16
16 BCX17725 KLK7 Skin barrier dysfunction Inflammatory cascade Atopy PAR2 Downstream consequences of KLK5 activation Degrades epithelial junctions • KLK5 initiates the pathologic protease cascade (KLK7, KLK14) and inflammation (via PAR2) in the skin • BCX17725 designed to stop KLK5 overactivity at the top of the pathway KLK5 KLK14 BCX17725 targets KLK5, the key player in Netherton syndrome Petrova E and Hovnanian A. Exp Opin Orphan Drugs (2020) 8(11): 455-487 PAR2: protease-activated receptor 2 KLK = kallikrein
Page 17
17 Initial healthcare claims analysis indicates a diagnosed US prevalence of ~1,600 Netherton Syndrome Population Assumptions Ongoing analysis leveraging NLP models, EMR data, and rare disease analogs suggest potential for US diagnosed population to increase to greater than 3,000 0 5 10 15 20 25 Fabry HAE PNH C3G Pompe Est. Prevalence (000’s) Before Targeted Therapies After Targeted Therapies +214% +67% +200% +67% +70% HAE, hereditary angioedema; PNH, paroxysmal nocturnal hemoglobinuria; C3G, Complement 3 glomerulopathy; NLP, natural language processing; EMR, electronic medial records Sources: Cantor Analysis 2024; Internal Analysis Change in US Prevalence After Launch of Targeted Therapy Targeted therapies drive diagnosis of rare diseases
Page 18
Part 3: NS patients N = 1-3 anticipated MAD SAD Multiple Dose Multiple Dose Parts 1 & 2: Healthy volunteers Part 4: NS patients N = up to 12 12 weeks of treatment, open-label, safety and efficacy 4 weeks of treatment, open-label, safety and PK This study is designed to help us understand: 1. Preliminary safety 2. Systemic exposure 3. Distribution into skin 4. Early efficacy signals 5. Dosing for pivotal study BCX17725 Phase 1 study design 18 Data from Part 4 expected by YE 2026 Goal of study: inform and plan for pivotal study in 2027 Completed Completed SAD, single ascending dose; MAD, multiple ascending dose
Page 19
Finance summary 19 (Figures in millions) 1. Non-GAAP ORLADEYO revenue excludes revenues associated with the European ORLADEYO business which was sold to Neopharmed Gentili S.p.A. on October 1, 2025. 2. Non-GAAP operating profit excludes revenues and expenses associated with the European ORLADEYO business, license revenue related to the license of intellectual property to Neopharmed Gentili S.p.A., stock-based compensation, transaction-related costs, and workforce reduction expense. 3. Non-GAAP operating expenses exclude stock-based compensation, restructuring, and transaction-related costs. 4. Unaudited, pro forma figures reflect items as of Dec 31, 2025, with adjustments related to the close of the Astria acquisition. Cash and investments includes BioCryst and Astria cash, cash equivalents, restricted cash, and short-term investments as of December 31, 2025 plus net proceeds from the Blackstone loan facility, less cash consideration paid for the Astria acquisition and estimated deal related fees. Excludes any adjustments for severance and related costs. 5. Senior credit facility reflects the Blackstone loan drawn on January 23, 2026. 6. Shares outstanding reflect the issuance of approximately 37M shares as consideration paid in the Astria acquisition. 2025 RESULTS1, 2 FY 2025 FY 2024 FY 2025 Non-GAAP FY 2024 Non-GAAP ORLADEYO revenue $602 $438 $563 $395 Operating profit (loss) $341 $(3) $214 $72 OTHER ITEMS4, 5, 6 PRO FORMA DEC 31, 2025 DEC 31, 2025 Cash and investments $279 $338 Senior credit facility (Blackstone loan at SOFR + 4.5%) $400 $0 Basic shares outstanding 250 213 FY 2026 GUIDANCE3 AS OF FEB 26, 2026 JAN 12, 2026 ORLADEYO revenue Unchanged $625-645 Total revenue Unchanged $635-660 Non-GAAP operating expenses Unchanged $450-470
Page 20
ORLADEYO direct revenue has reached zero -royalty tier 20 (Figures in millions) Note: At Peak royalty estimates are for illustrative purposes only. 1. 2026E and At Peak figures illustrate royalties net of royalties on European sales because Neopharmed Gentili S.p.A remits license revenue to BioCryst for these amounts. 2. Assumes the OMERS royalty has reached its cap. 3. Direct sales include the United States, key European markets and other markets where ORLADEYO is sold directly or through distributors. 4. Royalties on sales in the US, Europe, and markets where ORLADEYO is sold directly are zero over $550M but royalties on sales in Japan may increase. Partner Rate Tiers (Direct3) Cumulative Cap Royalty Pharma $0-350M: $350M-550M: Over $550M: 9.5% 4.5% None None OMERS $0-350M: $350M-550M: Over $550M: 10% 3% None 1.55x ($233M) • Total royalty burden has reached a soft cap now that revenues are over the $550M tier4 • Overall royalties expected to remain relatively flat until the OMERS royalty hits its cumulative payback cap (expected in 2029), at which point the total amount will fall approximately in half. Royalty termsIllustrative Royalty Dynamic (Total net royalties paid and payable1) $74 $86 $85 $85 $43 2024 2025 2026E At Peak At Peak post- OMERS² Mid-$80s Mid-$40s Mid-$80s
Page 21
Appendix: Non -GAAP reconciliations 21 U.S. GAAP European ORLADEYO Business 1 Other Non- G AAP Adjustm ents2 Non-G AAP U.S. GAAP European ORLADEYO Business 1 Other Non- G AAP Adjustm ents2 Non-G AAP Revenues: Revenues: ORLADEYO: ORLADEYO: U.S. $ 548,779 $ - $ - $ 548,779 U.S. $ 385,961 $ - $ - $ 385,961 Outside of U.S. 53,060 38,658 - 14,402 Outside of U.S. 51,699 43,130 - 8,569 Total ORLA DEYO 601,839 38,658 - 563,181 Total ORLA DEYO 437,660 43,130 - 394,530 License revenue 243,980 - 243,271 709 License revenue - - - - Other revenues 29,018 - - 29,018 Other revenues 13,052 - - 13,052 Total revenues 874,837 38,658 243,271 592,908 Total revenues 450,712 43,130 - 407,582 Ex penses: Ex penses: Cost of product sales 19,075 2,465 9 16,601 Cost of product sales 12,269 2,879 - 9,390 Research and development (ex cluding stock-based com pensation) 136,616 1,539 2,040 133,037 Research and development (ex cluding stock-based com pensation) 143,353 2,723 1,201 139,429 Sales and marketing (ex cluding stock-based com pensation) 177,085 30,495 2,502 144,088 Sales and marketing (ex cluding stock-based com pensation) 152,166 35,252 - 116,914 G eneral and administrative (ex cluding stock-based com pensation) 116,006 8,089 22,912 85,005 G eneral and administrative (ex cluding stock-based com pensation) 80,054 9,999 63 69,992 Stock-based com pensation 85,066 - 85,066 - Stock-based com pensation 65,413 - 65,413 - Total operating ex penses 533,848 42,588 112,529 378,731 Total operating ex penses 453,255 50,853 66,677 335,725 Incom e (loss) from operations $ 340,989 $ (3,930)$ 130,742 $ 214,177 Incom e (loss) from operations $ (2,543)$ (7,723)$ (66,677)$ 71,857 $ 243,271 Workforce reduction ex pense $ 1,264 Transaction-related costs $ 21,149 Stock-based com pensation $ 65,413 Workforce reduction ex pense $ 6,314 Stock-based com pensation $ 85,066 Tw elv e Months E nded December 31, 2024 1 Represents revenues and ex penses associated with our European ORLADEY O business which was sold to Neopharm ed G entili S.p.A. on October 1, 2025. 2 Reflects the following non-G AAP adjustm ents for the twelve m onths ended Decem ber 31, 2024: Reconciliation of Non-GAAP Income (Loss) From Operations (in thousands) Tw elv e Months E nded December 31, 2025 1 Represents revenues and ex penses associated with our European ORLADEY O business which was sold to Neopharm ed G entili S.p.A. on October 1, 2025. 2 Reflects the following non-G AAP adjustm ents for the twelve m onths ended Decem ber 31, 2025: License revenue related to the license of intellectual property to Neopharmed G entili S.p.A.