Core Viewpoint - BioCryst Pharmaceuticals has received a recommendation from Infarmed in Portugal for ORLADEYO® (berotralstat) to be used for the routine prevention of recurrent attacks of hereditary angioedema (HAE) in patients aged 12 years and older, marking a significant advancement in treatment options for HAE patients in Portugal [1][2]. Group 1: Product Information - ORLADEYO® (berotralstat) is the first and only oral therapy specifically designed to prevent attacks of hereditary angioedema in patients aged 12 years and older, with a recommended dosage of one capsule per day [3]. - The mechanism of action for ORLADEYO involves decreasing the activity of plasma kallikrein, which is responsible for HAE attacks [3]. - The product is indicated for prophylaxis to prevent attacks of hereditary angioedema in both adult and pediatric patients aged 12 years and older [4]. Group 2: Market Access and Regulatory Status - The Infarmed recommendation follows the European Commission's marketing authorization of ORLADEYO in April 2021, and the product is currently licensed in 44 countries [2]. - The approval in Portugal enhances access to modern prophylaxis for HAE patients, providing more options for healthcare providers and potentially improving the quality of life for patients [2]. Group 3: Safety and Usage Information - The safety and effectiveness of ORLADEYO for treating acute HAE attacks have not been established, and it is not recommended for such use [5]. - Dosages higher than 150 mg once daily are not recommended due to the risk of QT prolongation, with common adverse reactions including abdominal pain, vomiting, diarrhea, back pain, and gastroesophageal reflux disease [6]. - A reduced dosage of 110 mg is advised for patients with moderate or severe hepatic impairment [6].

Core Insights - BioCryst Pharmaceuticals, Inc. announced the presentation of five abstracts on ORLADEYO® (berotralstat) for hereditary angioedema (HAE) at the 2025 AAAAI/WAO joint congress [1][2] - A late-breaking abstract will present results from the APeX-P trial focusing on pediatric patients aged 2 to <12 years [2] - The congress is scheduled for February 28 to March 3, 2025, in San Diego [2] Company Overview - BioCryst Pharmaceuticals is a biotechnology company dedicated to improving the lives of individuals with hereditary angioedema and other rare diseases [11] - The company specializes in structure-guided drug design to develop innovative small-molecule and protein therapeutics [11] - ORLADEYO® is the first oral, once-daily plasma kallikrein inhibitor approved for the prophylactic treatment of HAE in patients aged 12 years and older [3][4] Product Information - ORLADEYO® (berotralstat) is indicated for the prevention of HAE attacks in adults and pediatric patients aged 12 years and older [4] - The drug works by inhibiting plasma kallikrein, thus preventing HAE attacks with a once-daily dosage [3] - The safety and effectiveness of ORLADEYO in pediatric patients under 12 years have not been established [10] Clinical Data Presentation - The five abstracts to be presented include: - HAE attack rates in pediatric patients aged 2 to <12 years [8] - Real-world attack rates before and after berotralstat initiation among patients with C1-inhibitor deficiency [8] - Real-world attack rates for patients without C1-inhibitor deficiency [8] - Patient willingness to change long-term prophylaxis based on disease burden and treatment effectiveness [8] - Patient-reported impacts of berotralstat on attack frequency and severity [8]

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].

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 ...