Financial Data and Key Metrics Changes - For Q4 2024, total revenue was $131.5 million, with Orladeo contributing $124.2 million [39] - Full year 2024 total revenue reached $450.7 million, with Orladeo accounting for $437.7 million [39] - Non-GAAP operating profit was $62.9 million, a significant increase from a non-GAAP operating loss of $48.1 million in 2023 [40] - Cash at the end of 2024 was approximately $343 million, with net cash utilization for Q4 at $8.4 million [41] Business Line Data and Key Metrics Changes - Orladeo revenue grew by 34% year-over-year, maintaining the same number of new patients as in its first year [9] - For Q4, $17.2 million (13.9%) of Orladeo's revenue came from ex-US markets, while for the full year, it was $51.7 million (11.8%) [39] - The company raised Orladeo revenue guidance for 2025 to between $535 million and $550 million [14] Market Data and Key Metrics Changes - 97% of surveyed US HAE treaters are considering prescribing Orladeo, with 59% extremely likely to prescribe it to more patients [17] - The company noted a significant increase in the percentage of Medicare patients able to afford co-payments due to the Inflation Reduction Act [13] Company Strategy and Development Direction - The company aims to file an NDA for Orladeo for pediatric use in children aged 2 to 11 this year [21] - BCX1775 for Netherton syndrome and Avoralstat for diabetic macular edema are advancing in clinical trials, with expectations for meaningful data readouts this year [21][28] - Long-term revenue guidance includes a three-year CAGR of 20%, targeting over $750 million by 2027 and $1 billion by 2029 [44] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the execution and performance across the company, highlighting a strong position for future growth [8] - The management team noted that the execution in 2024 has set the stage for positive and sustainable cash flow and EPS in the second half of 2025 [43] - There is optimism regarding the potential for BCX1775 to address an unmet need in Netherton syndrome, which is currently underdiagnosed [27] Other Important Information - The company is seeing strong commercial demand for RAPIVAB due to the prevalence of flu, despite some uncertainty around future government funding [42] - The pediatric oral granule formulation of Orladeo has shown promising results in trials, with high tolerability and retention rates [35][114] Q&A Session Summary Question: Can you narrow down when data from Netherton patients is expected? - Management indicated that data will be available later this year, with a focus on skin healing and KLK5 activity [51][52] Question: What is the current paid patient proportion for Orladeo? - The company reported a 73.5% paid rate across all patients, with about 20% of patients on Medicare [120] Question: How does the pediatric study's tolerability compare to adults? - The pediatric oral granules have shown good tolerability and compliance, similar to adult experiences [98] Question: What is the expected timeline for dosing Netherton syndrome patients? - Dosing for Netherton syndrome patients is expected to begin around mid-year, with data on skin activity anticipated by year-end [83] Question: What percentage of KLK5 suppression is needed for clinical benefit? - The company is looking for nearly complete suppression of KLK5 activity, although the exact percentage required for clinical benefit is not yet known [128]

Financial Data and Key Metrics Changes - For the full year 2024, total revenue was $450.7 million, with Orlodeo contributing $437.7 million [29] - The company achieved a non-GAAP operating profit of $62.9 million, a significant increase from a non-GAAP operating loss of $48.1 million in 2023 [29] - Cash at the end of the year was approximately $343 million, with net cash utilization for the quarter at $8.4 million [30] Business Line Data and Key Metrics Changes - Orlodeo revenue grew by 34% year-over-year, reflecting strong execution by the commercial team [6][10] - In Q4, Orlodeo generated $124.2 million, with $17.2 million (13.9%) coming from ex-U.S. sales [29] - The company raised its Orlodeo revenue guidance for 2025 to between $535 million and $550 million [11] Market Data and Key Metrics Changes - The company noted a higher percentage of Medicare patients on Orlodeo can afford their co-payments due to the Inflation Reduction Act [10] - The paid patient rate across the entire patient base was 73.5%, with expectations to increase further [44][94] - The company expects to see continued strong demand in early 2025, consistent with the momentum from 2024 [10] Company Strategy and Development Direction - The company aims to file an NDA for Orlodeo for pediatric patients aged 2 to 11 this year [16][26] - The pipeline includes advancing BCX17725 for Netherton syndrome and ivorlstat for diabetic macular edema, with clinical data expected later this year [7][24] - The long-term revenue guidance includes a three-year CAGR of 20%, targeting over $750 million by 2027 and $1 billion by 2029 [30][31] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the execution and performance, stating that the momentum is expected to carry into 2025 and beyond [9] - The management highlighted the importance of the upcoming clinical data and the potential for BCX17725 to address an unmet need in Netherton syndrome [19][20] - The company anticipates positive and sustainable quarterly cash flow and EPS in the second half of 2025 [30] Other Important Information - The company is presenting real-world evidence at the Quad AI Conference, showcasing the effectiveness of Orlodeo in reducing attack rates for HAE patients [12] - The pediatric trial for Orlodeo showed that granules were safe and well-tolerated, with a high retention rate among participants [26][88] Q&A Session Summary Question: Timing for data from Netherton syndrome patients - Management indicated that data will be available later this year, with a focus on skin healing and KLK5 activity [35][38] Question: Proportion of paid patients and guidance for Orlodeo - The paid patient rate was 73.5% overall, with expectations for Medicare patients to increase significantly due to affordability improvements [44][94] Question: Tolerability of pediatric formulation compared to adults - The pediatric formulation showed good tolerability and compliance, similar to adult experiences [74] Question: International demand for Orlodeo - Demand in Europe, Canada, and Japan is growing, with confidence in Orlodeo's effectiveness leading to increased prescriptions [80][81] Question: Historical reauthorization rates - Approximately half of patients go through reauthorization in Q1, with improvements noted in the process over the years [104]

Financial Performance - ORLADEYO® (berotralstat) net revenue for full year 2024 reached $437 million, a 34% year-over-year increase[8] - The company expects to approach quarterly earnings per share profitability in the second half of fiscal year 2025[4] Guidance and Forecasts - The company provided guidance for full year 2025 ORLADEYO net revenue and total revenue[4] - Full year 2025 operating expenses guidance was also provided[4] Corporate Events and Announcements - BioCryst will present at the 43rd Annual J.P. Morgan Healthcare Conference on January 14, 2025[4] - Preliminary unaudited results for Q4 and full year 2024 were announced on January 10, 2025[4]

Part I. Financial Information [Financial Statements](index=8&type=section&id=Item%201.%20Financial%20Statements) The unaudited consolidated financial statements for Q3 and nine months ended September 30, 2024, present revenues, net losses, and key balance sheet figures, highlighting ORLADEYO sales and significant liabilities [Condensed Consolidated Balance Sheets](index=8&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) The balance sheet as of September 30, 2024, shows total assets of **$491.3 million**, with **$349.4 million** in cash, and total liabilities of **$959.8 million**, leading to a stockholders' deficit Condensed Consolidated Balance Sheet Highlights (in thousands) | Account | Sep 30, 2024 | Dec 31, 2023 | | :--- | :--- | :--- | | **Assets** | | | | Cash, cash equivalents, and investments | $349,439 | $388,987 | | Total current assets | $427,125 | $495,966 | | Total assets | $491,254 | $516,960 | | **Liabilities & Stockholders' Deficit** | | | | Total current liabilities | $153,567 | $149,988 | | Royalty financing obligations (non-current) | $481,775 | $508,034 | | Secured term loan | $314,333 | $303,231 | | Total liabilities | $959,817 | $972,488 | | Total stockholders' deficit | $(468,563) | $(455,528) | [Condensed Consolidated Statements of Comprehensive Loss](index=9&type=section&id=Condensed%20Consolidated%20Statements%20of%20Comprehensive%20Loss) For Q3 and nine months ended September 30, 2024, revenues increased to **$117.1 million** and **$319.2 million**, respectively, with net losses narrowing to **$36.1 million** and **$62.1 million** Financial Performance Summary (in thousands, except per share data) | Metric | Three Months Ended Sep 30, 2024 | Three Months Ended Sep 30, 2023 | Nine Months Ended Sep 30, 2024 | Nine Months Ended Sep 30, 2023 | | :--- | :--- | :--- | :--- | :--- | | Revenues | $117,085 | $86,742 | $319,178 | $238,011 | | Total operating expenses | $98,663 | $98,663 | $317,213 | $299,050 | | Income (loss) from operations | $(11,921) | $(11,921) | $1,965 | $(61,039) | | Net loss | $(36,149) | $(36,149) | $(62,086) | $(164,808) | | Net loss per share | $(0.19) | $(0.19) | $(0.30) | $(0.87) | [Condensed Consolidated Statements of Cash Flows](index=10&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) For the nine months ended September 30, 2024, net cash used in operating activities significantly improved to **$46.8 million**, with **$34.8 million** provided by investing activities Cash Flow Summary (in thousands) | Cash Flow Activity | Nine Months Ended Sep 30, 2024 | Nine Months Ended Sep 30, 2023 | | :--- | :--- | :--- | | Net cash used in operating activities | $(46,807) | $(86,223) | | Net cash provided by (used in) investing activities | $34,796 | $(103,025) | | Net cash (used in) provided by financing activities | $(1,740) | $35,658 | | **Decrease in cash, cash equivalents and restricted cash** | **$(13,383)** | **$(153,750)** | [Notes to Condensed Consolidated Financial Statements](index=13&type=section&id=Notes%20to%20Condensed%20Consolidated%20Financial%20Statements) The notes detail significant accounting policies, revenue recognition for ORLADEYO, and key disclosures on royalty financing obligations, the Pharmakon Loan Agreement, and workforce reduction costs - The company's primary revenue source is **ORLADEYO**, with sales recognized upon delivery to customers, and net revenue recorded after estimating variable considerations like government rebates, chargebacks, and co-payment assistance[51](index=51&type=chunk)[53](index=53&type=chunk) - The company has significant long-term liabilities from royalty financing agreements with RPI and OMERS, totaling **$514.8 million** as of September 30, 2024, for rights to future royalties on ORLADEYO and BCX10013 sales[129](index=129&type=chunk)[131](index=131&type=chunk)[143](index=143&type=chunk) - In April 2023, the company entered into a **$450 million** loan agreement with Pharmakon, drawing an initial **$300 million** tranche to repay previous debt, with the loan bearing interest at **SOFR + 7.00%** and maturing in **April 2028**[145](index=145&type=chunk) - In January 2024, the company implemented a workforce reduction, incurring **$3.4 million** in severance and related costs recognized in 2023, with an additional **$1.3 million** in one-time termination benefits recognized in Q1 2024[199](index=199&type=chunk)[200](index=200&type=chunk)[201](index=201&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=39&type=section&id=Item%202.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses the company's rare disease focus, led by ORLADEYO, pipeline developments including BCX10013 discontinuation, and financial performance, affirming sufficient liquidity for the next 12 months [Overview and Recent Developments](index=39&type=section&id=Overview%20and%20Recent%20Developments) BioCryst, a rare disease biotech, highlights ORLADEYO's market expansion, pipeline advancements for BCX17725 and avoralstat, discontinuation of BCX10013, and a new **$69 million** HHS contract for RAPIVAB - The company anticipates the global commercial market for ORLADEYO could reach a peak of **$1 billion** in annual net revenues, with approximately **80%** expected from the United States[209](index=209&type=chunk) - Development of **BCX10013**, an oral Factor D inhibitor, will be discontinued due to observed clinical activity being less than other therapies on the market[214](index=214&type=chunk)[229](index=229&type=chunk) - In September 2024, the company was awarded a contract by HHS for the procurement of RAPIVAB (peramivir injection) for the Strategic National Stockpile, worth up to **$69 million** over five years, with the first order of **$13.9 million** executed[230](index=230&type=chunk)[231](index=231&type=chunk) [Results of Operations](index=42&type=section&id=Results%20of%20Operations) Q3 2024 revenues increased to **$117.1 million**, driven by ORLADEYO sales, while R&D expenses decreased and SG&A expenses rose to support commercial growth Q3 2024 vs Q3 2023 Performance (in millions) | Metric | Q3 2024 | Q3 2023 | Change | | :--- | :--- | :--- | :--- | | Total Revenues | $117.1 | $86.7 | +$30.4 | | R&D Expenses | $41.1 | $46.9 | -$5.8 | | SG&A Expenses | $65.1 | $50.6 | +$14.5 | | Interest Expense | $24.8 | $27.3 | -$2.5 | Nine Months 2024 vs 2023 Performance (in millions) | Metric | 9M 2024 | 9M 2023 | Change | | :--- | :--- | :--- | :--- | | Total Revenues | $319.2 | $238.0 | +$81.2 | | R&D Expenses | $125.2 | $146.5 | -$21.3 | | SG&A Expenses | $185.7 | $149.5 | +$36.2 | | Interest Expense | $74.1 | $83.7 | -$9.6 | [Liquidity and Capital Resources](index=44&type=section&id=Liquidity%20and%20Capital%20Resources) The company's liquidity, primarily from cash and investments totaling **$349.4 million**, is deemed sufficient for the next 12 months, supported by reduced operating cash burn and no additional debt draws - As of September 30, 2024, the company's principal sources of liquidity were approximately **$96.8 million** in cash and cash equivalents and **$252.6 million** in available-for-sale investments[253](index=253&type=chunk) - The company believes its financial resources are sufficient to fund operations for at least the **next 12 months**, having no immediate plans to access capital markets and having not drawn down additional available debt[266](index=266&type=chunk) - Net cash used in operating activities for the nine months ended September 30, 2024, was **$46.8 million**, a significant reduction from the **$86.2 million** used in the same period in 2023[254](index=254&type=chunk)[255](index=255&type=chunk) [Quantitative and Qualitative Disclosures About Market Risk](index=52&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) The company faces interest rate risk from its variable-rate Pharmakon Loan Agreement and foreign currency risk from international sales, but does not currently use hedging instruments - The company is subject to **interest rate risk** from its variable-rate **Pharmakon Loan Agreement**, where borrowings accrue interest based on **SOFR**[303](index=303&type=chunk) - **Foreign currency risk** arises from European sales denominated in **Euros** and **British Pounds**, and Japanese royalties in **Yen**, though the company does not currently engage in foreign currency hedging[307](index=307&type=chunk)[308](index=308&type=chunk) [Controls and Procedures](index=53&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded that disclosure controls and procedures were effective as of September 30, 2024, with a previously reported material weakness now remediated - The CEO and CFO concluded that as of September 30, 2024, the company's disclosure controls and procedures are **effective**[311](index=311&type=chunk) - The material weakness that was reported in the previous quarter has been **remediated**[312](index=312&type=chunk) Part II. Other Information [Risk Factors](index=54&type=section&id=Item%201A.%20Risk%20Factors) The company faces significant risks including a history of losses, drug development uncertainties, reliance on third parties, commercialization challenges, substantial indebtedness, and intellectual property concerns - The company has a **history of losses** and may need to **raise additional capital** in the future, which may not be available on acceptable terms[315](index=315&type=chunk)[318](index=318&type=chunk) - Success is heavily dependent on managing the product pipeline, advancing candidates through **uncertain clinical trials**, and receiving **regulatory approvals**, with failures possible at any stage[321](index=321&type=chunk)[323](index=323&type=chunk) - The company **relies heavily on third parties** for manufacturing and distribution, and any failure by these vendors could significantly delay development and commercialization[368](index=368&type=chunk)[369](index=369&type=chunk) - **Significant indebtedness** under the Pharmakon Loan Agreement includes **restrictive covenants** that limit operational flexibility and could lead to **default** if breached[437](index=437&type=chunk)[441](index=441&type=chunk)[443](index=443&type=chunk) [Other Information](index=82&type=section&id=Item%205.%20Other%20Information) No director or officer adopted or terminated a Rule 10b5-1 or non-Rule 10b5-1 trading arrangement during Q3 2024 - **No director or officer** adopted or terminated a **Rule 10b5-1** or **non-Rule 10b5-1** trading arrangement during the three months ended September 30, 2024[516](index=516&type=chunk) [Exhibits](index=83&type=section&id=Item%206.%20Exhibits) This section lists exhibits filed with the Form 10-Q, including the Equity Award Retirement Policy, CEO/CFO certifications, and Inline XBRL financial statements - Key exhibits filed include the BioCryst Pharmaceuticals, Inc. **Equity Award Retirement Policy**, **CEO/CFO certifications** under Sarbanes-Oxley Sections 302 and 906, and **XBRL data files**[518](index=518&type=chunk)

BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX) Q3 2024 Earnings Conference Call November 4, 2024 8:30 AM ET Company Participants John Bluth - Chief Communications Officer Jon Stonehouse - CEO Charlie Gayer - Chief Commercial Officer Anthony Doyle - CFO Helen Thackray - Chief R&D Officer Jinky Rosselli - Chief Data and Insights Officer Conference Call Participants Nick Lenard - JPMorgan Tazeen Ahmad - Bank of America Brian Abrahams - RBC Capital Markets Maury Raycroft - Jefferies Stacy Ku - TD Cowen Liisa Bayk ...

| --- | --- | --- | |-------------------------------------------------------|-------|-------| | | | | | | | | | | | | | Third Quarter 2024 Results Call | | | | Corporate Update & Financial Results November 4, 2024 | | | | CONFIDENTIAL & PROPRIETARY | | | Forward-looking statements BioCryst's presentation contains forward-looking statements, including statements regarding future results, unaudited and forward-looking financial information and company performance, achievements, or future market share. These s ...

EXHIBIT 99.1 BioCryst Reports Third Quarter 2024 Financial Results and Provides Business Update — Q3 2024 ORLADEYO net revenue of $116.3 million (+35.7 percent y-o-y) — — Full-year 2024 ORLADEYO revenue guidance adjusted to $430-$435 million (top end of prior guidance range) — — Company introduces full-year 2024 total product revenue guidance of $443-$448 million based on additional 2024 revenue from RAPIVAB — — BCX17725 for Netherton syndrome advances into the clinic — — Company generates GAAP operating pr ...

Table of Contents Title of each class Trading Symbol(s) Name of each exchange on which registered Common Stock BCRX Nasdaq Global Select Market UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q x QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2024 or o TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from ______________________ to ...

| --- | --- | --- | |----------------------------------------------------------------|-------|-------| | | | | | | | | | | | | | Second Quarter 2024 Results Call | | | | Co rpo ra te U pd a te & F ina nc ia l R esults August 5, 2024 | | | | CONFID ENTIAL & PRO PRIETA RY | | | Forward-looking statements BioCryst's presentation contains forward-looking statements, including statements regarding future results, unaudited and forward-looking financial information and company performance or achievements. These s ...

BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX) Q2 2024 Earnings Call Transcript August 5, 2024 8:30 AM ET Company Participants John Bluth - Chief Communications Officer Jon Stonehouse - CEO Charlie Gayer - Chief Commercial Officer Anthony Doyle - CFO Helen Thackray - Chief R&D Officer Conference Call Participants Tazeen Ahmad - Bank of America Brian Abrahams - RBC Capital Markets Chris Raymond - Piper Sandler Gena Wang - Barclays Maury Raycroft - Jefferies Stacy Ku - TD Cowen Jon Wolleben - JMP Securities Ser ...