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Second Quarter 2026 Results Call Corporate Update & Financial Results August 5 , 2026 bio_cryst 2 wa
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Forward -looking statements 2 This presentation contains forward-looking statements, including statements regarding, among other things, future results, performance or achievements, such as expected full year 2026 revenue and operating expenses; expectations regarding pipeline development; anticipated approval and commercialization of navenibart; pharmaceutical research and development, such as 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; potential future milestone payments or royalties; the scalability of our commercial infrastructure; our ability to successfully execute future product launches; expectations related to future cash flow and profitability; 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; BioCryst’s ability to successfully transition to its new sole source specialty pharmacy for ORLADEYO shipments to patients and to continue to successfully commercialize ORLADEYO, including the successful launch of the ORLADEYO oral pellet formulation; uncertainties related to BioCryst’s ability to successfully execute its plan to close the Birmingham research facility and wind-down its internal discovery programs, including the timing and costs of such action; 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 results of BioCryst’s partnerships with third parties may not meet BioCryst’s current expectations, including that our partners may fail to reach performance milestones or achieve certain royalty thresholds under our license agreements; 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, 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.
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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 certain items adjusted, is valuable and useful since they can provide greater transparency into the financial results of core, ongoing operations and improve comparability across reporting periods. 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. A reconciliation between each second quarter non-GAAP financial measure and its respective closest equivalent GAAP financial measure is provided in the tables in the appendix. In addition, we provide non-GAAP ORLADEYO revenue for the full year 2025. This refers to our GAAP ORLADEYO revenue of $602M for the full year 2025, adjusted to exclude $39M in revenue associated with our European ORLADEYO business. 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
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AGENDA 4 Corporate update Charlie Gayer President and Chief Executive Officer Pipeline update Dr. Sandeep Menon Chief Research and Development Officer Financial update Babar Ghias Chief Financial Officer Q&A
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Our pipeline 5Navenibart, BCX17725, 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 NON-CORE PROGRAMS RAPIVAB ® (peramivir injection) Infectious Diseases STAR-0310 Monoclonal Antibody OX40 Antagonist Atopic Dermatitis
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ORLADEYO: the first and only approved targeted oral for HAE prophylaxis 6 • 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:
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ORLADEYO offers proven long -term attack control, demonstrated by strong real -world efficacy data 7 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 4 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. 4. TAKHZYRO, ANDEMBRY, and DAWNZERA US Prescribing Information (January 2025, June 2025, and August 2025, respectively) 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
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Navenibart: a long -acting plasma kallikrein inhibitor for HAE prophylaxis 8 • 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 study • 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 on track for top line data in Q3 2027
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Navenibart pivotal program design 9 Top-line data for both Q3M and Q6M doses on track for Q3 2027 600 mg Q3M 600 mg, then 300mg Q3M 600 mg Q6M (1 dose) Placebo R 600mg Q3M 300mg Q3M 600 mg Q6M PRIMARY EFFICACY AT 12 MONTHS Study 301: 6 months Blinded, placebo-controlled ADULTS n = 1351 3:3:3:2 randomization Study 302 Part 1: 6 months Blinded Study 302 Part 2: Open-label after all reach end of 302 Part 1 1. 135 target enrollment; 157 actual enrollment including adolescents
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Navenibart: potential for strong efficacy with optimal dosing profile 10 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; 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)
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BioCryst positioned to offer the leading oral and injectable 11 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
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Navenibart opportunity : 5,000+ patients Significant addressable opportunity in HAE 12 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
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BioCryst HAE franchise expected to generate double digit revenue growth into the next decade 13 $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 capacity for business development, deleveraging, or share buybacks 2025 ex-EU³ 2029E 2033E $563M $1B+ ~15% CAGR~15% CAGR $1.8B+ HAE portfolio revenue 1 Illustrative operating expenses2
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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? 14
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15 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
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16 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
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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. Dose selection BCX17725 Phase 1 study design 17 Data from Part 4 expected by YE 2026 Proof of concept to enable next stage of development Completed Completed SAD, single ascending dose; MAD, multiple ascending dose
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Finance summary 18 (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, transaction-related costs, and stock-based compensation. 3. Non-GAAP operating expenses exclude stock-based compensation, restructuring, and transaction-related costs. 4. Cash and investments includes cash equivalents, restricted cash, and short-term investments as of June 30, 2026. RESULTS1, 2 GAAP NON-GAAP Q2 2026 Q2 2025 Q2 2026 Q2 2025 ORLADEYO revenue $158 $157 $158 $144 Operating profit $98 $30 $113 $61 OTHER ITEMS4, JUN 30, 2026 Cash and investments $354 Senior credit facility (Blackstone loan at SOFR + 4.5%) $400 FY 2026 GUIDANCE3 AS OF AUG 5, 2026 MAY 6, 2026 ORLADEYO revenue Unchanged $625-645 Total revenue $690-715 $635-660 Non-GAAP operating expenses $420-440 $450-470
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ORLADEYO direct revenue has reached zero -royalty tier 19 (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
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Appendix: Non -GAAP reconciliations 20 Reconciliation of Non-GAAP Income From Operations (in thousands) U.S. GAAP Non-G AAP Adjustm ents Non-G AAP U.S. GAAP Non-G AAP Adjustm ents1 Non-G AAP Revenues: Revenues: ORLA DEYO 158,202 - 158,202 ORLA DEYO 156,837 13,310 143,527 License and other revenues 60,048 - 60,048 License and other revenues 6,516 - 6,516 Total revenues 218,250 - 218,250 Total revenues 163,353 13,310 150,043 Ex penses: Ex penses: Cost of product sales - ORLADEY O 3,737 - 3,737 Cost of product sales - ORLADEY O 2,388 1,080 1,308 Cost of product sales - peram ivir 103 - 103 Cost of product sales - peram ivir 410 - 410 Research and developm ent (ex cluding stock-based com pensation) 46,496 - 46,496 Research and developm ent (ex cluding stock-based com pensation) 34,059 1,286 32,773 Sales and m arketing (ex cluding stock-based com pensation) 33,947 - 33,947 Sales and m arketing (ex cluding stock-based com pensation) 45,589 12,165 33,424 G eneral and adm inistrative (ex cluding stock-based com pensation) 20,810 - 20,810 G eneral and adm inistrative (ex cluding stock-based com pensation) 29,817 8,577 21,240 Stock-based com pensation 14,692 14,692 - Stock-based com pensation 21,304 21,304 - Total operating ex penses 119,785 14,692 105,093 Total operating ex penses 133,567 44,412 89,155 Incom e (loss) from operations $ 98,465 $ (14,692)$ 113,157 Incom e from operations $ 29,786 $ (31,102)$ 60,888 $ 13,310 $ 1,080 $ 1,286 $ 12,165 $ 2,689 $ 5,888 $ 21,304 Three Months Ended June 30, 2025Three Months Ended June 30, 2026 1 Reflects the following non-G AAP adjustm ents for the three m onths ended June 30, 2025: 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: ORLADEYO revenue Cost of product sales - ORLADEY O Research and developm ent (ex cluding stock-based com pensation) Sales and m arketing (ex cluding stock-based com pensation) G eneral and adm inistrative (ex cluding stock-based com pensation) Transaction-related costs associated with the sale of our European ORLADEY O to Neopharm ed Gentili S.p.A. Stock-based com pensation