[Executive Summary / Highlights](index=1&type=section&id=Executive%20Summary%20%2F%20Highlights) [Q2 2024 Performance Highlights](index=1&type=section&id=Q2%202024%20Performance%20Highlights) BioCryst achieved significant financial and operational progress in Q2 2024, with ORLADEYO net revenue growing 34% year-over-year, achieving GAAP operating profit, and raising full-year ORLADEYO revenue guidance - BioCryst demonstrated strong performance in the first half of 2024, primarily driven by ORLADEYO's market success, as the company progresses towards its profitability goals[2](index=2&type=chunk) Key Financial Metrics | Metric | Q2 2024 | Change (YoY) | | :--- | :--- | :--- | | ORLADEYO Net Revenue | $108.3 Million | +34% | | GAAP Operating Profit | $8.8 Million | Switched from Loss to Profit | | Non-GAAP Operating Profit | $21.9 Million | Switched from Loss to Profit | [ORLADEYO Performance and Guidance](index=1&type=section&id=ORLADEYO%20Performance%20%26%20Guidance) ORLADEYO demonstrated strong performance in Q2 2024 with net revenue growing 34% year-over-year, leading the company to raise its full-year revenue guidance to $420-$435 million, reflecting sustained market demand and improved patient services ORLADEYO Revenue and Full-Year Guidance | Metric | Q2 2024 | Change (YoY) | | :--- | :--- | :--- | | ORLADEYO Net Revenue | $108.3 Million | +34% | **2024 Full-Year ORLADEYO Revenue Guidance:** | Metric | New Guidance | Old Guidance | | :--- | :--- | :--- | | Global Net Revenue | $420-$435 Million | $390-$400 Million | - ORLADEYO's revenue growth and increased full-year guidance reflect strong market demand, favorable patient outcomes, and the company's ongoing efforts to improve patient services and market access[4](index=4&type=chunk) [ORLADEYO Commercial Update](index=1&type=section&id=ORLADEYO%20Commercial%20Update) [Q2 2024 ORLADEYO Net Revenue](index=1&type=section&id=Q2%202024%20ORLADEYO%20Net%20Revenue) ORLADEYO achieved net revenue of **$108.3 million** in Q2 2024, a 34% year-over-year increase, primarily driven by operational improvements and enhanced patient payor mix ORLADEYO Net Revenue Comparison | Metric | Q2 2024 | Q2 2023 | Change (YoY) | | :--- | :--- | :--- | :--- | | ORLADEYO Net Revenue | $108.3 Million | $81.0 Million | +33.7% | - Operational improvements drove revenue above expectations, with the overall proportion of paying patients increasing by **3%** from the end of 2023 to **74.4%**[2](index=2&type=chunk) [Operational Improvements and Patient Metrics](index=1&type=section&id=Operational%20Improvements%20%26%20Patient%20Metrics) ORLADEYO maintains high new patient initiation and strong retention rates, with over 60% of paying patients continuing treatment for at least one year, and market research indicates significantly increased physician prescribing intent - New patient initiations remained consistent with the prior two quarters, matching new patient demand from the first three quarters post-launch in 2021[3](index=3&type=chunk) - Patient retention remains strong, with over **60%** of paying patients continuing treatment for at least one year[3](index=3&type=chunk) - Recent market research indicates that **52%** of allergists/immunologists are 'extremely likely' to prescribe ORLADEYO for more patients, up from **29%** in early 2023[3](index=3&type=chunk) [International Expansion](index=1&type=section&id=International%20Expansion) ORLADEYO has secured additional approvals and reimbursement authorizations in Europe and Latin America, now commercialized in over 20 countries globally, with ex-U.S. revenue growing 51% year-over-year and accounting for 11% of global net revenue - ORLADEYO has received additional approvals and reimbursement authorizations in Europe and Latin America, and is now commercialized in over **20** countries worldwide[3](index=3&type=chunk) International ORLADEYO Performance | Metric | Q2 2024 | | :--- | :--- | | Ex-U.S. ORLADEYO Revenue Growth (YoY) | +51% | | Share of Global ORLADEYO Net Revenue | 11% | [Market Differentiator and Peak Sales Potential](index=1&type=section&id=Market%20Differentiator%20%26%20Peak%20Sales%20Potential) ORLADEYO, as a once-daily oral medication, demonstrates a significant 91% reduction in HAE attacks, a differentiated advantage positioning it for **$1 billion** peak sales - ORLADEYO demonstrated a **91%** reduction in HAE attacks in long-term clinical trials, with this high level of attack control being its primary market differentiator[4](index=4&type=chunk) - The trajectory for ORLADEYO, as a once-daily oral medication, to achieve **$1 billion** in peak sales is becoming increasingly clear[4](index=4&type=chunk) [Rare Disease Pipeline Update](index=1&type=section&id=Rare%20Disease%20Pipeline%20Update) [ORLADEYO Label Expansion (Pediatric)](index=2&type=section&id=ORLADEYO%20Label%20Expansion%20(Pediatric)) The company plans to submit regulatory applications in 2025 to expand ORLADEYO's label for an oral granule formulation for children aged two and older, making it the first oral prophylactic therapy for pediatric HAE - The company plans to submit regulatory applications in **2025** to expand ORLADEYO's label for an oral granule formulation for children aged two and older[6](index=6&type=chunk) - ORLADEYO is set to become the first oral prophylactic therapy for pediatric HAE[6](index=6&type=chunk) [BCX10013 (Factor D Inhibitor) Update](index=2&type=section&id=BCX10013%20(Factor%20D%20Inhibitor)%20Update) The company completed clinical evaluation of oral Factor D inhibitor BCX10013; despite being safe and well-tolerated, observed clinical activity was lower than other market therapies, leading to plans to discontinue its development - The oral Factor D inhibitor BCX10013 was safe and well-tolerated across all studied doses[6](index=6&type=chunk) - Observed clinical activity levels were lower than other therapies on the market, and potential partners declined further investment[6](index=6&type=chunk) - BioCryst plans to discontinue the development of BCX10013[6](index=6&type=chunk) [BCX17725 (KLK-5 Inhibitor for Netherton Syndrome)](index=2&type=section&id=BCX17725%20(KLK-5%20Inhibitor%20for%20Netherton%20Syndrome)) The company expects to advance BCX17725, a KLK-5 inhibitor for Netherton syndrome, into clinical stages by the end of 2024; Netherton syndrome is a severe, rare, lifelong genetic disorder with no approved treatments - The company expects to advance BCX17725, a KLK-5 inhibitor for Netherton syndrome, into clinical stages by the end of **2024**[6](index=6&type=chunk) - Netherton syndrome is a severe, rare, lifelong genetic disorder affecting the skin, hair, and immune system, with no currently approved treatments[7](index=7&type=chunk) [Avoralstat (Plasma Kallikrein Inhibitor for DME)](index=2&type=section&id=Avoralstat%20(Plasma%20Kallikrein%20Inhibitor%20for%20DME)) The company plans to advance Avoralstat, a plasma kallikrein inhibitor, into clinical trials for diabetic macular edema (DME) patients in 2025, aiming to reduce vascular leakage and edema with potentially less frequent injections by targeting the kallikrein-kinin pathway - The company plans to advance Avoralstat, a plasma kallikrein inhibitor, into clinical trials for diabetic macular edema (DME) patients in **2025**[7](index=7&type=chunk) - Avoralstat targets the kallikrein-kinin system on retinal vascular endothelial cells, aiming to reduce vascular leakage and edema, and potentially achieve long-acting exposure and less frequent injections via suprachoroidal administration[8](index=8&type=chunk) [Second Quarter 2024 Financial Results](index=2&type=section&id=Second%20Quarter%202024%20Financial%20Results) [Total Revenues](index=2&type=section&id=Total%20Revenues) Total revenues reached **$109.3 million** in Q2 2024, a **32.5%** increase from Q2 2023, primarily driven by strong growth in ORLADEYO net revenue Total Revenues Overview | Metric | Q2 2024 | Q2 2023 | Change (YoY) | | :--- | :--- | :--- | :--- | | Total Revenues | $109.3 Million | $82.5 Million | +32.5% | | ORLADEYO Net Revenue | $108.3 Million | $81.0 Million | +33.7% | [Operating Expenses](index=2&type=section&id=Operating%20Expenses) Total operating expenses in Q2 2024 were **$100.6 million**, a **2.5%** year-over-year decrease, with R&D expenses falling due to project terminations and SG&A increasing due to commercial expansion Operating Expenses Overview | Metric | Q2 2024 | Q2 2023 | Change (YoY) | | :--- | :--- | :--- | :--- | | Total Operating Expenses | $100.6 Million | $103.2 Million | -2.5% | | Operating Expenses Excluding Non-Cash Stock-Based Compensation | $87.4 Million | $90.4 Million | -3.3% | | Non-Cash Stock-Based Compensation | $13.2 Million | - | - | [Research and Development Expenses](index=2&type=section&id=Research%20and%20Development) R&D expenses decreased to **$37.6 million** in Q2 2024, a **26.6%** year-over-year reduction, primarily due to the termination of BCX10013 and BCX9930 programs, partially offset by increased investment in other pipeline projects and ORLADEYO pediatric trials R&D Expenses Comparison | Metric | Q2 2024 | Q2 2023 | Change (YoY) | | :--- | :--- | :--- | :--- | | R&D Expenses | $37.6 Million | $51.2 Million | -26.6% | - The decrease in R&D expenses was primarily attributable to reduced spending and termination of the BCX10013 and BCX9930 programs[9](index=9&type=chunk) [Selling, General and Administrative Expenses](index=2&type=section&id=Selling%2C%20General%20and%20Administrative) SG&A expenses increased to **$61.2 million** in Q2 2024, a **20.0%** year-over-year rise, mainly due to higher commercial expenses supporting revenue growth, new launch regions, international expansion, and increased resources for accounting and IT functions SG&A Expenses Comparison | Metric | Q2 2024 | Q2 2023 | Change (YoY) | | :--- | :--- | :--- | :--- | | Selling, General and Administrative Expenses | $61.2 Million | $51.0 Million | +20.0% | - The increase in expenses was primarily due to higher commercial expenses supporting revenue growth, new launch regions, international business expansion, and increased resources for accounting and IT functions[10](index=10&type=chunk) [Operating Profit/Loss (GAAP and Non-GAAP)](index=2&type=section&id=Operating%20Profit%2FLoss%20(GAAP%20%26%20Non-GAAP)) The company achieved a GAAP operating profit of **$8.8 million** in Q2 2024, a significant improvement from a **$20.7 million** GAAP operating loss in the prior year, with non-GAAP operating profit reaching **$21.9 million** after adjusting for non-cash stock-based compensation Operating Profit/Loss Comparison | Metric | Q2 2024 | Q2 2023 | | :--- | :--- | :--- | | GAAP Operating Profit (Loss) | $8.8 Million | ($20.7 Million) | | Non-GAAP Operating Profit (Loss) | $21.9 Million | ($7.9 Million) | [Interest Expense and Net Loss](index=3&type=section&id=Interest%20Expense%20%26%20Net%20Loss) Interest expense in Q2 2024 was **$24.7 million**, a **14.5%** year-over-year decrease, while net loss significantly narrowed to **$12.7 million** (or **$0.06** per share) from **$75.3 million** (or **$0.40** per share) in the prior year, which included a one-time debt extinguishment charge Interest Expense and Net Loss Comparison | Metric | Q2 2024 | Q2 2023 | Change (YoY) | | :--- | :--- | :--- | :--- | | Interest Expense | $24.7 Million | $28.9 Million | -14.5% | | Net Loss | $12.7 Million | $75.3 Million | -83.1% | | Basic and Diluted Net Loss Per Share | $0.06 | $0.40 | -85% | | Q2 2023 One-Time Debt Extinguishment Charge | - | $29.0 Million | - | | Q2 2023 Non-GAAP Net Loss Per Share | - | $0.24 | - | [Cash Position](index=3&type=section&id=Cash%20Position) As of June 30, 2024, total cash, cash equivalents, restricted cash, and investments amounted to **$338.1 million**, with cash used in operating activities for Q2 2024 being **$0.2 million** Cash and Investments | Metric | June 30, 2024 | June 30, 2023 | | :--- | :--- | :--- | | Cash, Cash Equivalents, Restricted Cash, and Investments | $338.1 Million | $415.7 Million | **Q2 2024 Cash Used in Operating Activities:** | Metric | Amount | | :--- | :--- | | Cash Used in Operating Activities | $0.2 Million | [Explanation of Non-GAAP Pro Forma Financial Measures](index=3&type=section&id=Non-GAAP%20Pro%20Forma%20Financial%20Measures%20Explanation) The company provides non-GAAP pro forma financial measures to help investors better understand financial performance adjusted for one-time events, such as the Athyrium debt extinguishment loss and non-cash stock-based compensation expenses, facilitating comparison with future results - Non-GAAP measures are intended to help the company and investors better understand financial performance adjusted for one-time events, such as the Athyrium debt extinguishment loss and non-cash stock-based compensation expenses[13](index=13&type=chunk)[14](index=14&type=chunk) - These non-GAAP measures should be considered supplementary to, and not as a substitute for or in isolation from, GAAP financial information[13](index=13&type=chunk) [Financial Outlook for 2024](index=3&type=section&id=Financial%20Outlook%20for%202024) [ORLADEYO Revenue Guidance](index=3&type=section&id=ORLADEYO%20Revenue%20Guidance) Based on ORLADEYO's operational improvements and strong market demand in the first half of 2024, the company raised its full-year 2024 global ORLADEYO net revenue guidance to **$420-$435 million** 2024 Full-Year ORLADEYO Revenue Guidance | Metric | New Guidance | Old Guidance | | :--- | :--- | :--- | | Global Net Revenue | $420-$435 Million | $390-$400 Million | [Operating Expense Outlook](index=3&type=section&id=Operating%20Expense%20Outlook) The company maintains its previous operating expense outlook, projecting full-year 2024 operating expenses between **$365-$375 million**, consistent with full-year 2023, excluding non-cash stock-based compensation and one-time severance costs 2024 Full-Year Operating Expense Outlook | Metric | Amount | | :--- | :--- | | Operating Expenses | $365-$375 Million | **Notes:** * Consistent with full-year 2023 * Excludes non-cash stock-based compensation expenses * Excludes one-time severance costs related to workforce reduction implemented in Q1 2024 [Long-term Financial Milestones](index=3&type=section&id=Long-term%20Financial%20Milestones) The company anticipates achieving full-year operating profit (excluding non-cash stock-based compensation) in 2024, nearing quarterly positive EPS and cash flow in H2 2025, and achieving full-year EPS profitability and positive cash flow in 2026 without additional financing - The company expects to achieve full-year operating profit (excluding non-cash stock-based compensation) in **2024**[16](index=16&type=chunk) - Anticipates nearing quarterly positive earnings per share (EPS) and positive cash flow (excluding non-cash stock-based compensation) in the second half of **2025**[16](index=16&type=chunk) - Projects achieving full-year EPS profitability and positive cash flow in **2026**[16](index=16&type=chunk) - The company is confident in achieving these financial milestones without raising additional capital and does not intend to draw on the additional **$150 million** of debt available from Pharmakon[16](index=16&type=chunk) [Company Information](index=3&type=section&id=Company%20Information) [About BioCryst Pharmaceuticals](index=4&type=section&id=About%20BioCryst%20Pharmaceuticals) BioCryst Pharmaceuticals is a global biotechnology company focused on developing oral small molecule and protein therapies for complement-mediated and other rare diseases through structure-guided drug design, having successfully commercialized ORLADEYO - BioCryst Pharmaceuticals is a global biotechnology company dedicated to improving the lives of patients with complement-mediated and other rare diseases[18](index=18&type=chunk) - The company leverages its expertise in structure-guided drug design to develop best-in-class or first-in-class oral small molecule and protein therapies[18](index=18&type=chunk) - Has commercialized **ORLADEYO** (berotralstat), the first once-daily oral plasma kallikrein inhibitor[18](index=18&type=chunk) [Forward-Looking Statements](index=4&type=section&id=Forward-Looking%20Statements) This press release contains forward-looking statements involving known and unknown risks, uncertainties, and other factors that could cause actual results to differ materially from expectations; investors should refer to the company's SEC filings for important factors that may affect forward-looking statements - This press release contains forward-looking statements involving known and unknown risks, uncertainties, and other factors that could cause BioCryst's actual results, performance, or achievements to differ materially from those expressed or implied by such forward-looking statements[19](index=19&type=chunk) - Investors should not place undue reliance on these forward-looking statements and should refer to the company's periodic filings with the U.S. Securities and Exchange Commission (such as 10-K, 10-Q, and 8-K reports) for important factors that may affect actual results[19](index=19&type=chunk) [Consolidated Financial Summary](index=4&type=section&id=Consolidated%20Financial%20Summary) [Statements of Operations (Unaudited)](index=4&type=section&id=Statements%20of%20Operations%20(Unaudited)) This section provides BioCryst Pharmaceuticals, Inc.'s unaudited consolidated statements of operations, detailing key financial data such as revenues, expenses, operating profit/loss, and net loss for Q2 and the first six months of 2024 and 2023 Consolidated Statements of Operations (Unaudited) | Metric (in Thousands) | Q2 2024 | Q2 2023 | Six Months Ended 2024 | Six Months Ended 2023 | | :--- | :--- | :--- | :--- | :--- | | ORLADEYO Revenue | 108,288 | 81,009 | 197,155 | 149,423 | | Total Revenues | 109,332 | 82,491 | 202,093 | 151,269 | | Research and Development Expenses | 37,623 | 51,247 | 84,116 | 99,635 | | Selling, General and Administrative Expenses | 61,214 | 50,997 | 120,578 | 98,864 | | Total Operating Expenses | 100,571 | 103,194 | 207,820 | 200,387 | | Operating Profit (Loss) | 8,761 | (20,703) | (5,727) | (49,118) | | Interest Expense | (24,733) | (28,915) | (49,239) | (56,311) | | Loss on Debt Extinguishment | — | (29,019) | — | (29,019) | | Net Loss | (12,674) | (75,326) | (48,053) | (128,659) | | Basic and Diluted Net Loss Per Share | (0.06) | (0.40) | (0.23) | (0.68) | [Balance Sheet Data](index=5&type=section&id=Balance%20Sheet%20Data) This section presents BioCryst Pharmaceuticals, Inc.'s balance sheet data as of June 30, 2024 (unaudited) and December 31, 2023 (audited), including cash, accounts receivable, total assets, liabilities, and stockholders' equity Consolidated Balance Sheet Data (in Thousands) | Metric (in Thousands) | June 30, 2024 (Unaudited) | December 31, 2023 (Note 1) | | :--- | :--- | :--- | | Cash, Cash Equivalents, Restricted Cash, and Investments | $338,100 | $388,987 | | Restricted Cash | 1,804 | 1,804 | | Accounts Receivable | 56,950 | 56,950 | | Total Assets | 516,960 | 516,960 | | Secured Term Loan | 303,231 | 303,231 | | Royalty Financing Obligation | 531,599 | 531,599 | | Accumulated Deficit | (1,729,212) | (1,681,159) | | Stockholders' Deficit | (455,528) | (455,528) | | Shares of Common Stock Outstanding | 205,771 | 205,771 | [Reconciliation of Adjusted Net Loss and EPS](index=5&type=section&id=Reconciliation%20of%20Adjusted%20Net%20Loss%20and%20EPS) This section provides a reconciliation of GAAP net loss to adjusted net loss and GAAP basic and diluted net loss per share to adjusted basic and diluted net loss per share, primarily adjusting for one-time items like R&D restructuring charges and the Athyrium term loan extinguishment loss Reconciliation of GAAP Net Loss to Adjusted Net Loss and GAAP Basic and Diluted Net Loss Per Share to Adjusted Basic and Diluted Net Loss Per Share (in Thousands) | Metric (in Thousands) | Q2 2024 | Q2 2023 | Six Months Ended 2024 | Six Months Ended 2023 | | :--- | :--- | :--- | :--- | :--- | | GAAP Net Loss | (12,674) | (75,326) | (48,053) | (128,659) | | Less: One-Time R&D Restructuring Charges | — | — | (1,264) | — | | Less: One-Time Loss on Athyrium Term Loan Extinguishment | — | (29,019) | — | (29,019) | | Adjusted Net Loss | (12,674) | (46,307) | (46,789) | (99,640) | | GAAP Basic and Diluted Net Loss Per Share | (0.06) | (0.40) | (0.23) | (0.68) | | Adjusted Basic and Diluted Net Loss Per Share | (0.06) | (0.24) | (0.23) | (0.53) | [Reconciliation of Adjusted Operating Income (Loss)](index=6&type=section&id=Reconciliation%20of%20Adjusted%20Income%20(Loss)%20from%20Operations) This section provides a reconciliation of GAAP operating profit (loss) to adjusted operating profit (loss), primarily adjusted by excluding stock-based compensation expenses to offer a clearer view of operational performance Reconciliation of GAAP Operating Income (Loss) to Adjusted Operating Income (Loss) (in Thousands) | Metric (in Thousands) | Q2 2024 | Q2 2023 | Six Months Ended 2024 | Six Months Ended 2023 | | :--- | :--- | :--- | :--- | :--- | | GAAP Operating Profit (Loss) | 8,761 | (20,703) | (5,727) | (49,118) | | Less: Stock-Based Compensation Expense | (13,173) | (12,841) | (26,825) | (26,848) | | Adjusted Operating Profit (Loss) | 21,934 | (7,862) | 21,098 | (22,270) |

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 March 31, 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 ...

BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX) Q1 2024 Earnings Call Transcript May 6, 2024 8:30 AM ET Company Participants John Bluth - IR Jon Stonehouse - CEO Charlie Gayer - CCO Helen Thackray - Chief R&D Officer Anthony Doyle - CFO Conference Call Participants Maury Raycroft - Jefferies Tazeen Ahmad - Bank of America Serge Belanger - Needham & Company Liisa Bayko - Evercore ISI Stacy Ku - TD Cowen Gena Wang - Barclays Francois Brisebois - Oppenheimer Jon Wolleben - JMP Securities Chris Raymond - Piper San ...

First Quarter 2024 Results Call Corporate Update & Financial Results May 6, 2024 ...

EXHIBIT 99.1 BioCryst Reports First Quarter 2024 Financial Results and Provides Business Update —Q1 2024 ORLADEYO net revenue grows 30 percent y-o-y to $88.9 million— —Full-year 2024 ORLADEYO revenue guidance adjusted to $390-$400 million (top end of prior guidance range) — —Pipeline programs, including BCX17725 for Netherton syndrome and avoralstat for DME, advancing on schedule into clinical trials— RESEARCH TRIANGLE PARK, N.C., May 06, 2024 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) ...

BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX) Q4 2023 Earnings Conference Call February 26, 2024 8:30 AM ET Company Participants John Bluth - Chief Communications Officer Jon Stonehouse - Chief Executive Officer Ryan Arnold - Chief Medical Officer Charlie Gayer - Chief Commercial Officer Anthony Doyle - Chief Financial Officer Conference Call Participants Tazeen Ahmad - Bank of America Brian Abrahams - RBC Capital Markets Jessica Fye - JP Morgan Liisa Bayko - Evercore ISI Stacy Ku - TD Cowen Serge Belanger - ...

Table of Contents Title of each class Trading Symbol(s) Name of each exchange on which registered Common Stock, $0.01 par value BCRX Nasdaq Global Select Market Emerging growth company o UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 Form 10-K x ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2023 o TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition pe ...

Part I. Financial Information [Item 1. Financial Statements](index=8&type=section&id=Item%201.%20Financial%20Statements) The unaudited condensed consolidated financial statements for Q3 2023 reflect increased revenues, narrower net loss, and balance sheet changes [Condensed Consolidated Balance Sheets](index=8&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) Condensed Consolidated Balance Sheets (in thousands) | Account | Sep 30, 2023 | Dec 31, 2022 | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $150,905 | $304,767 | | Investments | $246,685 | $119,543 | | Total current assets | $502,466 | $516,500 | | Total assets | $522,924 | $550,000 | | **Liabilities and Stockholders' Deficit** | | | | Total current liabilities | $90,763 | $105,514 | | Royalty financing obligations | $535,186 | $501,655 | | Secured term loans | $297,995 | $231,624 | | Total stockholders' deficit | ($410,986) | ($294,597) | | Total liabilities and stockholders' deficit | $522,924 | $550,000 | [Condensed Consolidated Statements of Comprehensive Loss](index=9&type=section&id=Condensed%20Consolidated%20Statements%20of%20Comprehensive%20Loss) Condensed Consolidated Statements of Comprehensive Loss (in thousands, except per share data) | Metric | Three Months Ended Sep 30, 2023 | Three Months Ended Sep 30, 2022 | Nine Months Ended Sep 30, 2023 | Nine Months Ended Sep 30, 2022 | | :--- | :--- | :--- | :--- | :--- | | Revenues | $86,742 | $75,827 | $238,011 | $191,282 | | Total operating expenses | $98,663 | $93,272 | $299,050 | $293,406 | | Loss from operations | ($11,921) | ($17,445) | ($61,039) | ($102,124) | | Net loss | ($36,149) | ($42,520) | ($164,808) | ($175,575) | | Basic and diluted net loss per common share | ($0.19) | ($0.23) | ($0.87) | ($0.95) | [Condensed Consolidated Statements of Cash Flows](index=10&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) Condensed Consolidated Statements of Cash Flows (in thousands) | Cash Flow Activity | Nine Months Ended Sep 30, 2023 | Nine Months Ended Sep 30, 2022 | | :--- | :--- | :--- | | Net cash used in operating activities | ($86,223) | ($138,001) | | Net cash used in investing activities | ($103,025) | ($205,453) | | Net cash provided by financing activities | $35,658 | $83,828 | | Decrease in cash, cash equivalents and restricted cash | ($153,750) | ($259,391) | | Cash, cash equivalents and restricted cash at end of period | $152,489 | $248,343 | [Notes to Condensed Consolidated Financial Statements](index=13&type=section&id=Notes%20to%20Condensed%20Consolidated%20Financial%20Statements) - The company believes its financial resources as of September 30, 2023, are **sufficient to fund operations for at least the next 12 months**, despite expecting 2023 expenses to exceed revenues[43](index=43&type=chunk) Revenue Breakdown (in thousands) | Revenue Source | Three Months Ended Sep 30, 2023 | Three Months Ended Sep 30, 2022 | Nine Months Ended Sep 30, 2023 | Nine Months Ended Sep 30, 2022 | | :--- | :--- | :--- | :--- | :--- | | ORLADEYO (U.S.) | $75,268 | $60,106 | $208,934 | $162,743 | | ORLADEYO (Outside U.S.) | $10,416 | $5,866 | $26,173 | $18,155 | | **Total ORLADEYO** | **$85,684** | **$65,972** | **$235,107** | **$180,898** | | Other revenues | $1,058 | $9,855 | $2,904 | $10,384 | | **Total revenues** | **$86,742** | **$75,827** | **$238,011** | **$191,282** | - On April 17, 2023, the company entered into a new **$450 million Loan Agreement** with Pharmakon, drawing an initial **$300 million**; proceeds were used to repay the existing **$241.8 million Athyrium Credit Agreement**[133](index=133&type=chunk)[223](index=223&type=chunk) - Upon repayment of the Athyrium Term Loans, the company incurred a **one-time loss on extinguishment of debt of $29.0 million** for the nine months ended September 30, 2023, which included prepayment fees and the write-off of unamortized financing costs[150](index=150&type=chunk) - Subsequent to the quarter end, on October 23, 2023, Baker Bros. Advisors LP exercised all remaining pre-funded warrants, resulting in the issuance of **14,997 common shares**; **no warrants remain outstanding**[182](index=182&type=chunk) - On November 3, 2023, the company entered into a license agreement with Clearside Biomedical, Inc. to develop avoralstat for diabetic macular edema, involving a **$5 million upfront fee** and potential future milestones and royalties[182](index=182&type=chunk)[183](index=183&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=37&type=section&id=Item%202.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses rare disease strategy, ORLADEYO commercialization, financial results, and pipeline advancements [Overview and Recent Developments](index=37&type=section&id=Overview%20and%20Recent%20Developments) - The company's strategy focuses on developing and commercializing **first-in-class or best-in-class oral therapeutics** for rare diseases, with a key product being **ORLADEYO for hereditary angioedema (HAE)**[186](index=186&type=chunk)[187](index=187&type=chunk) - BioCryst anticipates the global commercial market for ORLADEYO has the potential to reach a peak of **$1 billion in annual net revenues**, with **70-80% expected from the U.S.**[188](index=188&type=chunk) - The company is advancing its complement program, including **BCX10013 for renal complement-mediated diseases**, and has introduced **several new pipeline candidates**[190](index=190&type=chunk)[192](index=192&type=chunk) - **Oral C5 Inhibitor:** For generalized myasthenia gravis (gMG), with clinical trials expected to begin in 2025 - **Bifunctional Complement Inhibitor:** An anti-C2 monoclonal antibody for complex renal diseases, with clinical trials expected in 2025 - **Oral C2 Inhibitor:** For autoimmune hemolytic anemias, with a lead molecule selection expected in 2025 - **BCX17725:** A KLK5 inhibitor for Netherton syndrome, with clinical trials expected to begin in 2024 - **Avoralstat:** Licensed with Clearside's microinjector for diabetic macular edema (DME)[200](index=200&type=chunk)[201](index=201&type=chunk)[202](index=202&type=chunk)[203](index=203&type=chunk)[204](index=204&type=chunk) [Results of Operations](index=41&type=section&id=Results%20of%20Operations) Comparison of Results of Operations (in millions) | Metric | Q3 2023 | Q3 2022 | Change | 9M 2023 | 9M 2022 | Change | | :--- | :--- | :--- | :--- | :--- | :--- | :--- | | Total Revenues | $86.7 | $75.8 | +14.4% | $238.0 | $191.3 | +24.4% | | R&D Expenses | $46.9 | $52.7 | -11.0% | $146.5 | $180.1 | -18.7% | | SG&A Expenses | $50.6 | $36.9 | +37.1% | $149.5 | $109.2 | +36.9% | | Interest Expense | $27.3 | $24.8 | +10.1% | $83.7 | $72.6 | +15.3% | - The increase in Q3 and 9-month 2023 revenue was primarily driven by higher ORLADEYO net revenue, which grew by **$19.7 million and $54.2 million** respectively, partially offset by a decrease in other revenues due to a 2022 RAPIVAB stockpiling sale to HHS[207](index=207&type=chunk)[214](index=214&type=chunk) - **R&D expenses decreased** in 2023 due to reduced investment following the discontinuation of the BCX9930 (Factor D) and BCX9250 (FOP) programs in late 2022[209](index=209&type=chunk)[216](index=216&type=chunk) - **SG&A expenses increased significantly** due to investment in expanding the U.S. commercial team for ORLADEYO and growing international operations[210](index=210&type=chunk)[217](index=217&type=chunk) [Liquidity and Capital Resources](index=43&type=section&id=Liquidity%20and%20Capital%20Resources) - As of September 30, 2023, the company had principal liquidity sources of approximately **$150.9 million in cash and cash equivalents** and **$246.7 million in available-for-sale investments**[226](index=226&type=chunk) - On April 17, 2023, the company entered into a new **$450 million loan agreement** with Pharmakon, drawing an initial **$300 million** to repay its previous Athyrium credit facility and for general corporate purposes; **three additional tranches of $50 million each** are available at the company's option through September 30, 2024[223](index=223&type=chunk) - Management believes that based on current revenue and expense expectations, the company's financial resources are **sufficient to fund operations for at least the next 12 months**[230](index=230&type=chunk) [Quantitative and Qualitative Disclosures About Market Risk](index=50&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) The company faces interest rate and foreign currency risks, with no material impact from inflation on results - The company is subject to **interest rate risk** from its Pharmakon Loan Agreement, which bears interest at a **variable rate** based on the three-month SOFR plus a margin; as of September 30, 2023, the **effective interest rate was 13.24%**[267](index=267&type=chunk) - **Foreign currency risk** exists due to commercial sales in Europe (Euros, British Pound) and royalties from Japan (Japanese yen), as well as operations in other regions; the company **does not currently engage in foreign currency hedging**[272](index=272&type=chunk)[273](index=273&type=chunk) [Controls and Procedures](index=51&type=section&id=Item%204.%20Controls%20and%20Procedures) Disclosure controls were ineffective due to material weaknesses in internal financial reporting from staffing issues; remediation is underway - The CEO and CFO concluded that disclosure controls and procedures were **not effective** as of September 30, 2023, due to **material weaknesses in internal control over financial reporting**[276](index=276&type=chunk) - The material weaknesses stem from **staffing issues and insufficient resources** in the accounting function, leading to **inadequate review** of manual journal entries and general ledger account reconciliations[276](index=276&type=chunk)[277](index=277&type=chunk) - Planned remediation includes **hiring more accounting personnel**, **engaging a third-party specialist**, and **updating policies, procedures, and training** related to internal controls[278](index=278&type=chunk) - Despite the material weaknesses, management believes the condensed consolidated financial statements in the report are **fairly presented in all material respects in conformity with U.S. GAAP**[280](index=280&type=chunk) Part II. Other Information [Risk Factors](index=53&type=section&id=Item%201A.%20Risk%20Factors) The company outlines significant business, financial, development, regulatory, and market risks impacting its operations - **Business Risks:** The company has a **history of losses** and may **need to raise additional capital**; its success depends on **advancing its pipeline**, particularly **BCX10013**, and the **commercial success of ORLADEYO**, which faces **intense competition**[290](index=290&type=chunk)[292](index=292&type=chunk)[296](index=296&type=chunk)[339](index=339&type=chunk) - **Financial & Control Risks:** The company has identified **material weaknesses in its internal control over financial reporting**, which could **harm investor confidence and the stock price**; the business is also subject to **significant indebtedness** under the Pharmakon Loan Agreement, which contains **restrictive covenants**[414](index=414&type=chunk)[389](index=389&type=chunk) - **Development & Regulatory Risks:** Drug development is **complex, lengthy, and expensive**, with a **high risk of failure**; the company **relies heavily on third parties** for manufacturing and clinical trials, and obtaining and maintaining regulatory approvals is **uncertain and subject to extensive requirements**[298](index=298&type=chunk)[305](index=305&type=chunk)[312](index=312&type=chunk) - **International & Market Risks:** International expansion exposes the company to **varied legal, political, and financial risks**, including **foreign currency fluctuations** and **complex reimbursement environments**; **unpredictable market and economic conditions**, such as **inflation, rising interest rates, and geopolitical conflicts**, could adversely affect operations and access to capital[396](index=396&type=chunk)[398](index=398&type=chunk)[431](index=431&type=chunk) [Other Information](index=80&type=section&id=Item%205.%20Other%20Information) During Q3 2023, no director or officer adopted or terminated Rule 10b5-1 or non-Rule 10b5-1 trading arrangements - During the three months ended September 30, 2023, **no director or officer adopted or terminated a Rule 10b5-1 trading arrangement or a non-Rule 10b5-1 trading arrangement**[437](index=437&type=chunk) [Exhibits](index=81&type=section&id=Item%206.%20Exhibits) This section provides a comprehensive list of all exhibits filed with the Form 10-Q

BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX) Q3 2023 Earnings Conference Call November 2, 2023 8:30 AM ET Company Participants John Bluth - Investor Relations Jon Stonehouse - Chief Executive Officer Charlie Gayer - Chief Commercial Officer Jinky Rosselli - Chief Data & Insights Officer Anthony Doyle - Chief Financial Officer Conference Call Participants Tazeen Ahmad - Bank of America Stacy Ku - Cowen & Co. Liisa Bayko - Evercore ISI Serge Belanger - Needham & Company Operator Good morning and welcome to th ...

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, 2023 or o TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from ______________________ to ...