Core Insights - BioCryst Pharmaceuticals will present five abstracts on ORLADEYO (berotralstat) at the 2025 AAAAI/WAO joint congress, focusing on its use for hereditary angioedema (HAE) [1][2] Group 1: Product Information - ORLADEYO is the first and only oral therapy specifically designed to prevent HAE attacks in patients aged 12 years and older, administered as one capsule daily [3][4] - The drug functions as a plasma kallikrein inhibitor, reducing the activity of plasma kallikrein to prevent HAE attacks [3] Group 2: Clinical Trials and Research - A late-breaking abstract will present results from the APeX-P trial, which evaluates ORLADEYO in pediatric patients aged 2 to under 12 years with HAE [2] - The five abstracts will cover various aspects, including real-world attack rates before and after berotralstat initiation and patient-reported impacts on attack frequency and severity [8] Group 3: Safety and Efficacy - The safety and effectiveness of ORLADEYO for treating acute HAE attacks have not been established, and it is not recommended for such use [5][10] - Common adverse reactions include abdominal pain, vomiting, diarrhea, back pain, and gastroesophageal reflux disease, occurring in 10% or more of patients [6] Group 4: Company Overview - BioCryst Pharmaceuticals is a global biotechnology company focused on developing therapies for hereditary angioedema and other rare diseases, leveraging expertise in structure-guided drug design [11]

Core Viewpoint - BioCryst Pharmaceuticals, Inc. will report its fourth quarter 2024 financial results on February 24, 2025, and will host a conference call to discuss these results and provide a corporate update [1]. Company Overview - BioCryst Pharmaceuticals is a global biotechnology company focused on improving the lives of individuals with complement-mediated and other rare diseases [3]. - The company utilizes structure-guided drug design to develop first-in-class or best-in-class small-molecule and protein therapeutics aimed at challenging diseases [3]. - BioCryst has commercialized ORLADEYO® (berotralstat), which is the first oral, once-daily plasma kallikrein inhibitor, and is advancing a pipeline of small-molecule and protein therapies [3].

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]

Core Insights - BioCryst Pharmaceuticals expects ORLADEYO net revenue to be between $515 million and $535 million in 2025, with total revenue (including RAPIVAB) projected between $540 million and $560 million [1][5] - The company achieved operating profit in 2024 (excluding stock-based compensation) and anticipates approaching quarterly EPS profitability and positive cash flow in the second half of 2025 [1][8] - A new drug application for ORLADEYO granules aimed at children up to age 12 is planned for submission in 2025, addressing a significant unmet medical need [1][2] - The pipeline is advancing with clinical programs for BCX17725 (Netherton syndrome) and avoralstat (diabetic macular edema) expected to progress in 2025 [1][2] Revenue Performance - Preliminary, unaudited ORLADEYO net revenue for Q4 2024 was $123.5 million, reflecting a 36% year-over-year increase, while full-year 2024 net revenue reached $437 million, up 34% year-over-year [3][4] - Total revenue for Q4 2024 was $130.8 million, a 40% increase year-over-year, with full-year total revenue at $450 million, marking a 36% growth year-over-year [5] Operating Expenses - The company forecasts full-year 2025 GAAP operating expenses to be between $485 million and $495 million, which includes an estimated $60 million for stock-based compensation [6] - Operating expenses, excluding stock-based compensation, are projected to be between $425 million and $435 million, reflecting additional investments for the pediatric product launch and ongoing clinical activities [6] Profitability Outlook - Revenue growth in 2024 significantly outpaced operating expense growth, and this trend is expected to continue [7] - The company anticipates an annual compound annual growth rate (CAGR) for revenue of around 20% over the next three years, compared to a projected CAGR of approximately 5% for operating expenses [8] - BioCryst expects to achieve quarterly positive EPS and positive cash flow in the second half of 2025, with full-year profitability anticipated in 2026 [8]

RESEARCH TRIANGLE PARK, N.C., Jan. 07, 2025 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX) today announced that the company will present at the 43rd Annual J.P. Morgan Healthcare Conference in San Francisco on Tuesday, January 14, 2025, at 6:00 p.m. ET. Link to the live audio webcast and replay of the presentation may be accessed in the Investors & Media section of BioCryst's website at www.biocryst.com. About BioCryst PharmaceuticalsBioCryst Pharmaceuticals is a global biotechnology compa ...

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 ...

BioCryst Pharmaceuticals (BCRX) came out with a quarterly loss of $0.07 per share in line with the Zacks Consensus Estimate. This compares to loss of $0.19 per share a year ago. These figures are adjusted for non-recurring items.A quarter ago, it was expected that this drugmaker would post a loss of $0.19 per share when it actually produced a loss of $0.06, delivering a surprise of 68.42%.Over the last four quarters, the company has surpassed consensus EPS estimates two times.BioCryst, which belongs to the ...

| --- | --- | --- | |-------------------------------------------------------|-------|-------| | | | | | | | | | | | | | 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 ...