BioCryst Pharmaceuticals(BCRX) - 2024 Q2 - Quarterly Results

Executive Summary / Highlights Q2 2024 Performance Highlights 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] 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 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] ORLADEYO Commercial Update Q2 2024 ORLADEYO Net Revenue 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] Operational Improvements and Patient Metrics 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] - Patient retention remains strong, with over 60% of paying patients continuing treatment for at least one year[3] - 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] International Expansion 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] 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 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] - The trajectory for ORLADEYO, as a once-daily oral medication, to achieve $1 billion in peak sales is becoming increasingly clear[4] Rare Disease Pipeline Update ORLADEYO Label Expansion (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] - ORLADEYO is set to become the first oral prophylactic therapy for pediatric HAE[6] BCX10013 (Factor D Inhibitor) Update 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] - Observed clinical activity levels were lower than other therapies on the market, and potential partners declined further investment[6] - BioCryst plans to discontinue the development of BCX10013[6] BCX17725 (KLK-5 Inhibitor for Netherton Syndrome) 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] - Netherton syndrome is a severe, rare, lifelong genetic disorder affecting the skin, hair, and immune system, with no currently approved treatments[7] Avoralstat (Plasma Kallikrein Inhibitor for DME) 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] - 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] Second Quarter 2024 Financial Results Total Revenues 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 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 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] Selling, General and Administrative Expenses 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] Operating Profit/Loss (GAAP and Non-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 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 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 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][14] - These non-GAAP measures should be considered supplementary to, and not as a substitute for or in isolation from, GAAP financial information[13] Financial Outlook for 2024 ORLADEYO Revenue Guidance 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 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 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] - 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] - Projects achieving full-year EPS profitability and positive cash flow in 2026[16] - 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] Company Information About BioCryst Pharmaceuticals 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] - 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] - Has commercialized ORLADEYO (berotralstat), the first once-daily oral plasma kallikrein inhibitor[18] Forward-Looking Statements 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] - 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] Consolidated Financial Summary Statements of Operations (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 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 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) 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) |