BioCryst Pharmaceuticals (BCRX) Conference Call Summary Company Overview - BioCryst Pharmaceuticals is a commercial stage and profitable biotech company focused on developing medicines for rare diseases, particularly hereditary angioedema (HAE) [3][4] - The company's commercial product, Orlodeo, is a once-daily oral pill for HAE patients, which has been on the market for about five years and is projected to generate peak sales of $1 billion [4][6] Financial Performance - BioCryst raised its sales guidance for Orlodeo to $580 to $600 million for the current year [5][27] - The company is in a strong cash position and expects to be profitable on a full-year basis moving forward [6] Product Success and Market Dynamics - Orlodeo's success is attributed to its efficacy and convenience, with approximately 60% of patients experiencing significant efficacy [9][10] - The product has seen strong demand, with about half of its business coming from patients switching from injectable therapies [10][11] - Market research indicates that patients require compelling reasons to switch therapies, which has contributed to Orlodeo's steady growth [22][23] Pipeline Developments - BioCryst has two additional programs in clinical trials: one for Netherton syndrome (Phase 1) and another for diabetic macular edema (DME) [5][6] - The company anticipates data from both programs by the end of the year [50][66] Netherton Syndrome - Netherton syndrome is a severe rare disease with no current treatment options, affecting skin turnover and leading to significant health risks [50][51] - BioCryst's product, BCX 17725, aims to replace the missing protein that regulates skin turnover [51][52] - The estimated patient population for Netherton syndrome in the U.S. is between 3,000 to 5,000 [55] Diabetic Macular Edema (DME) - Avorostat, initially developed for HAE, is being repurposed for DME using a suprachoroidal delivery method [60][61] - The company is set to begin patient enrollment for the DME trial in Australia [62] Market Research and Competitive Landscape - BioCryst employs comprehensive market research, including Monte Carlo simulations, to accurately forecast sales and understand market dynamics [18][19] - The company maintains a competitive edge in the oral prophylactic space against injectable therapies, with Orlodeo and Tekxaro being market leaders [20][21] Future Outlook - The company aims to achieve $800 million in U.S. sales by adding approximately 200 new patients annually and achieving an 85% reimbursement rate [30][32] - The pediatric formulation of Orlodeo is expected to significantly impact the market, with an estimated 500 affected children in the U.S. [38][39] - BioCryst plans to continue building its cash balance and has already paid off $75 million of its debt early this quarter [68] Conclusion - BioCryst Pharmaceuticals is positioned for continued growth with a strong commercial product, a promising pipeline, and a solid financial outlook, making it a notable player in the biotech sector focused on rare diseases [69]

Financial Performance - Revenues for Q1 2025 were $145,534, a 57% increase compared to $92,761 in Q1 2024[22] - Net income for Q1 2025 was $32, compared to a net loss of $35,379 in Q1 2024[22] - Total revenues for the three months ended March 31, 2025, were $145,534,000, a 57% increase from $92,761,000 in the same period of 2024[37] - Net product sales for ORLADEYO amounted to $143,787,000 for the three months ended March 31, 2025, compared to $89,272,000 in the prior year, reflecting a 61% growth[37] - The Company recorded a segment net income of $32,000 for Q1 2025, a significant recovery from a net loss of $35,379,000 in Q1 2024[178] - Total revenues for the three months ended March 31, 2025, were $145.5 million, up from $92.8 million for the same period in 2024, driven by a $45.4 million increase in ORLADEYO net revenue[212] Expenses - Research and development expenses decreased to $37,270 from $46,493 in Q1 2024, a reduction of approximately 20%[22] - Selling, general and administrative expenses increased to $82,469 from $59,491, a rise of approximately 38%[22] - The Company incurred $82,469,000 in selling, general and administrative expenses for Q1 2025, up from $59,491,000 in Q1 2024[178] - Stock-based compensation expense increased from $13,652,000 in Q1 2024 to $21,368,000 in Q1 2025, an increase of about 56.7%[152] - Selling, general and administrative expenses for Q1 2025 were $82.5 million, up from $59.5 million in Q1 2024, reflecting increased commercial expenses and stock-based compensation[217] Assets and Liabilities - Total current assets decreased to $409,977 from $421,968 as of December 31, 2024[21] - Total liabilities decreased to $931,974 from $966,354 as of December 31, 2024[21] - Cash and cash equivalents increased to $105,190 from $104,713 as of December 31, 2024[21] - The Company reported a total stockholders' deficit of $451,927 as of March 31, 2025, improved from $475,934 as of December 31, 2024[21] - The Company had investments with a total estimated fair value of $210,450,000 as of March 31, 2025, down from $236,460,000 at December 31, 2024[110] Revenue Recognition and Receivables - Trade receivables are primarily due from specialty pharmacy customers in the U.S. and third-party distributors, with standard payment terms of 30 to 90 days[58] - The Company recognizes revenue from license fees and royalties when the earnings process is complete, with royalties based on licensees' net sales of covered products[48][50] - The Company evaluates trade receivables for estimated losses and provides reserves based on current economic conditions and historical collection experience[60] - The Company recorded approximately 84% and 87% of total net revenues from sales of ORLADEYO through a single specialty pharmacy for the three months ended March 31, 2025 and 2024, respectively[94] Inventory and Production - The Company’s inventory primarily consists of ORLADEYO and peramivir, valued at the lower of cost or estimated net realizable value[61] - Total inventory as of March 31, 2025, was $31,970,000, slightly up from $31,274,000 at December 31, 2024[115] - The Company expenses costs related to production of pre-launch inventory as research and development expense until regulatory approval is received, after which inventory costs are capitalized[64] - The Company has significant judgment in determining the need for valuation reserves for inventory, which includes excess, obsolete, or unmarketable inventory[63] Debt and Financing - The Company has entered into royalty purchase agreements with RPI and OMERS, totaling $275,000,000 in cash for future royalty payments[116] - As of March 31, 2025, the carrying value of royalty financing obligations was approximately $500,918, with effective interest rates of 21.6% for the 2020 RPI Agreement and 10.0% for the OMERS Agreement[127] - The Company had total borrowings of $300,000 under the Pharmakon Loan Agreement as of March 31, 2025, with interest expense of $9,153 for the quarter[138] - The Pharmakon Loan Agreement allows for quarterly interest-only payments until the maturity date of April 17, 2028, with an effective interest rate of 12.31% for the three months ended March 31, 2025[134] - The Company incurred debt fees and issuance costs of $11,147 associated with the Tranche A Loan, which are being amortized over the term of the loan[141] Clinical Development and Regulatory - The FDA has cleared the investigational new drug application for BCX17725, enabling clinical trials to enroll patients in the United States[209] - The company expects to submit a new drug application to the FDA in 2025 to expand the ORLADEYO label to children aged 2 to 11[206] - The ongoing APeX-P clinical trial is assessing an oral granule formulation of ORLADEYO in pediatric patients aged 2 to 11[189] - The Company anticipates initial clinical data from the avoralstat program targeted by the end of 2025[210] Market and Competitive Landscape - The Company is seeking to defend its intellectual property rights against Annora Pharma's ANDA filing for a generic version of ORLADEYO, which could impact future revenues[179] - ORLADEYO is projected to reach a global peak of $1 billion in annual net revenues, with approximately 80% expected from the United States[190] - The percentage of U.S. HAE patients preferring an oral prophylaxis therapy increased to 70% in 2025, up from 50% in 2023[207]

Financial Data and Key Metrics Changes - The company reported quarterly revenue of $145.5 million, with $134 million coming from Orlodayo, marking a significant increase from previous quarters [6][27] - Annual revenue guidance for Orlodayo has been raised to between $580 million and $600 million, reflecting a growth of 33% to 37% over the previous year [7][28] - Operating profit for Q1 2025 was $21.2 million, with net income slightly positive, and cash at the end of the quarter was $317 million [28][30] Business Line Data and Key Metrics Changes - The U.S. commercial team has successfully transitioned patients from free drug to paid status, achieving an 84% paid rate among commercial insurance patients, up from 73.5% [12][27] - The paid patient rate increased by 10 percentage points in Q1, with two-thirds of this improvement attributed to the Inflation Reduction Act [11][57] - The gross to net ratio improved, with expectations to maintain an average of around 15% for the full year [14][28] Market Data and Key Metrics Changes - The demand for Orlodayo has remained strong, with new prescriptions slightly exceeding the best quarter from the previous year [9][62] - The company anticipates capturing more revenue opportunities as the patient base continues to grow, aiming for $1 billion in global revenue by 2029 [13][62] Company Strategy and Development Direction - The company is focused on advancing its pipeline and has submitted a pediatric NDA for Orlodayo, aiming to introduce an oral granule formulation for patients aged 2 to 11 [16][30] - The company is also progressing with clinical trials for new treatments targeting Netherton syndrome and diabetic macular edema, with initial data expected by the end of the year [17][26] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the company's financial strength and ability to achieve profitability this year, one year earlier than previously planned [7][30] - The management highlighted the importance of sustainable revenue growth and the ability to pay down debt while investing in the pipeline [8][30] Other Important Information - The company paid down $75 million of its debt in April, reducing outstanding debt to $249 million, which is expected to save approximately $23 million in interest payments over the life of the debt [28][30] - The company is well-positioned in a competitive market, with a focus on providing effective treatments for underserved patient populations [30] Q&A Session Summary Question: What is the potential for pediatric patients on TAKHZYRO? - Management estimates around 500 pediatric patients on TAKHZYRO in the U.S., with at least 200 potentially appropriate for prophylactic therapy [34][35] Question: What factors contributed to the increase in paid patients? - The increase in paid patients was primarily due to improved Medicare coverage and better execution in the commercial segment [46][57] Question: What benchmarks are being looked for in the DME program? - The company is looking for a clear reduction in retinal edema and thickness as a benchmark for moving forward with the DME program [92][93] Question: How does the company plan to handle the pediatric launch? - The company aims to minimize the lag in deployment upon approval and is preparing to promote the pediatric formulation immediately [100][101] Question: What is the expected dosing range for the new treatment? - The initial dosing for the new treatment will be around six milligrams, with adjustments based on patient response [105][106]

Financial Data and Key Metrics Changes - The company reported quarterly revenue of $145.5 million, with $134 million attributed to Orlodayo, marking a significant increase from the previous year [6][28] - Annual revenue guidance for Orlodayo has been raised to between $580 million and $600 million, reflecting a growth of 33% to 37% over last year [6][29] - Operating profit for Q1 2025 was $21.2 million, with net income reported as slightly positive [29] - Cash at the end of the quarter stood at $317 million, and the company reduced its outstanding debt by $75 million, bringing it down to $249 million [28][30] Business Line Data and Key Metrics Changes - The U.S. commercial team successfully transitioned patients from free drug to paid status, achieving a 10 percentage point increase in the paid patient rate, now at 84% [10][12] - The paid rate among Medicare patients improved significantly, with 89% now receiving paid therapy [93] - The gross to net ratio improved, with expectations to maintain it around 15% for the full year [14][51] Market Data and Key Metrics Changes - The company noted a strong demand for Orlodayo, with new prescriptions slightly exceeding the best quarter from the previous year [8][10] - The percentage of U.S. patients who strongly prefer oral prophylaxis increased to 70%, up from 51% in 2023 [10] - The company anticipates reaching $1 billion in global revenue by 2029, with a more profitable path than previously expected [13][64] Company Strategy and Development Direction - The company is focusing on expanding its pipeline, with significant milestones achieved, including the submission of a pediatric NDA for Orlodayo [16] - The company aims to capture more revenue from Orlodayo as the patient base continues to grow, with a long-term goal of 85% paid therapy [12][13] - The company is committed to disciplined capital allocation to create sustainable long-term revenue growth while reducing debt [30][31] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the sustainability of the paid rate improvement, viewing 84% as a new floor for future performance [118] - The company is optimistic about the impact of the Inflation Reduction Act on patient affordability and access to Orlodayo [11][57] - Management highlighted the importance of real-world evidence in convincing payers of the drug's efficacy, which has improved payer conversations [94] Other Important Information - The company is preparing for the launch of a pediatric formulation of Orlodayo, with expectations for rapid deployment following approval [106] - The company is also advancing its clinical pipeline, with a focus on rare diseases and the potential for transformative treatments [16][18] Q&A Session Summary Question: What is the potential impact of the pediatric launch on Orlodayo? - Management expects the pediatric launch to be a significant tailwind, with around 500 pediatric patients in the U.S. potentially appropriate for prophylactic therapy [35][36] Question: Can you elaborate on the improvements in getting patients onto paid drug? - The team made investments in the reauthorization process and improved their execution, leading to a higher paid rate [46][47] Question: What benchmarks are being set for the DME program? - The company is looking for a clear reduction in retinal edema as a benchmark for moving forward with the DME program [41][97] Question: How sustainable is the paid rate improvement? - Management believes the paid rate improvement is sustainable, with 84% being a new baseline for future performance [118] Question: What is the expected timeline for the pediatric formulation launch? - The company aims to minimize the lag time between approval and market availability, similar to previous launches [106][107]

First Quarter 2025 Results Call Corporate Update & Financial Results May 5, 2025 CONFIDENTIAL & PROPRIETARY Forward-looking statements President and Chief Executive Officer ORLADEYO® update Charlie Gayer Chief Commercial Officer BioCryst's presentation contains forward-looking statements, including, but not limited to, statements regarding future results and forward-looking financial information, company performance, achievements, future market share or size, and expectations regarding pipeline development. ...

EXHIBIT 99.1 BioCryst Reports First Quarter 2025 Financial Results and Provides Business Update —Q1 2025 ORLADEYO net revenue of $134.2 million (+51 percent y-o-y)— —Full year 2025 ORLADEYO revenue guidance increased to $580 million to $600 million— —Company now expects to be profitable for full year 2025, a year ahead of schedule— —Company pays down $75 million of debt; saves approximately $23.5 million over life of loan— —NDA submitted for ORLADEYO oral granules for children with HAE aged 2-11— —U.S. IND ...

Core Insights - BioCryst Pharmaceuticals reported a strong Q1 2025 performance with ORLADEYO net revenue of $134.2 million, reflecting a 51% year-over-year increase [1][6] - The company has raised its full-year 2025 revenue guidance for ORLADEYO to between $580 million and $600 million, up from previous estimates [1][15] - BioCryst anticipates achieving profitability for the full year 2025, a year earlier than previously expected [1][16] Financial Performance - Total revenues for Q1 2025 reached $145.5 million, a 56.8% increase from $92.8 million in Q1 2024 [8] - Research and development expenses decreased to $37.3 million, down 19.8% from $46.5 million in Q1 2024 [9] - Selling, general and administrative expenses rose to $82.5 million, a 38.7% increase compared to $59.5 million in Q1 2024 [10] - Operating income for Q1 2025 was $21.2 million, compared to an operating loss of $14.5 million in Q1 2024 [11] Debt Management - The company paid down $75 million of its debt early in Q2 2025, which is expected to save approximately $23.5 million in interest over the loan's life [1][14] Product Development - BioCryst submitted a new drug application (NDA) for ORLADEYO oral granules for children aged 2-11 with hereditary angioedema (HAE) [1][5] - The company is progressing with clinical trials for BCX17725 for Netherton syndrome and avoralstat for diabetic macular edema (DME) [7][11] Market Dynamics - The percentage of ORLADEYO patients on paid drug increased to approximately 84%, up from 73.5% at the end of 2024, indicating strong market demand [6][4] - A recent survey indicated that 70% of U.S. HAE patients prefer oral prophylaxis therapy, up from 50% in 2023 [6]

Core Insights - BioCryst Pharmaceuticals has appointed Steve Frank, a leader in healthcare investment banking, to its board of directors, enhancing its strategic capabilities in the biopharma sector [1][3]. Company Overview - BioCryst Pharmaceuticals is a global biotechnology company focused on developing innovative therapies for rare diseases, leveraging expertise in structure-guided drug design [6]. - The company has commercialized ORLADEYO (berotralstat), the first oral, once-daily plasma kallikrein inhibitor, and is advancing a pipeline of small-molecule and protein therapies [6]. Leadership and Expertise - Steve Frank has extensive experience in healthcare investment banking, previously serving as chairman of global healthcare investment banking at J.P. Morgan and leading Bear Stearns' healthcare investment banking before its acquisition [2]. - His background includes directing State Farm's multi-billion-dollar life science equity portfolio in the 1980s and 1990s, showcasing his investment acumen [2]. Strategic Positioning - BioCryst is positioned for growth with a commercial product nearing $1 billion in peak sales and a strong cash position that allows independence from capital markets [4]. - The company is focused on accelerating towards profitability while advancing its pipeline of potential best-in-class medicines [4].

Financial Performance - Revenues for 2024 reached $450.7 million, a 36% increase from $331.4 million in 2023[459]. - Total operating expenses for 2024 were $453.3 million, compared to $435.1 million in 2023, reflecting a slight increase of 4%[459]. - The net loss for 2024 was $88.9 million, significantly improved from a net loss of $226.5 million in 2023[459]. - The balance of accumulated deficit as of December 31, 2024, was $1.77 billion, an increase from $1.68 billion in 2023[474]. - The company reported a loss before provision for income taxes of $86,954,000 in 2024, a decrease from $226,229,000 in 2023 and $244,383,000 in 2022[620]. - The company's effective tax rate for 2024 resulted in an income tax expense of $1,927,000, significantly lower than $310,000 in 2023 and $2,733,000 in 2022[620]. Cash and Assets - Cash and cash equivalents decreased to $104.7 million in 2024 from $110.6 million in 2023, a decline of approximately 5%[462]. - Total assets decreased to $490.4 million in 2024 from $517.0 million in 2023, a reduction of about 5%[457]. - The total investments maturing in one year or less decreased to $216,137,000 in 2024 from $278,344,000 in 2023, a decline of approximately 22%[545]. - As of December 31, 2024, the total assets measured at fair value were $236,460,000, a decrease from $278,344,000 in 2023, representing a decline of approximately 15%[543]. Expenses - Research and development expenses for 2024 were $174.6 million, down from $216.6 million in 2023, a decrease of 19%[459]. - Selling, general and administrative expenses increased to $266.1 million in 2024, up from $213.9 million in 2023, an increase of 24%[459]. - The Company recorded a depreciation expense of $1,246,000 for the year ended December 31, 2024, compared to $1,655,000 in 2023[551]. - Operating lease expense for the year ended December 31, 2024 was $2,301, an increase from $2,018 in 2023 and $1,578 in 2022[591]. Revenue Sources - Product sales, net for 2024 reached $442.7 million, up from $324.7 million in 2023, reflecting a 36% growth[473]. - ORLADEYO revenues in the U.S. reached $386.0 million in 2024, up 34% from $288.4 million in 2023[539]. - The company recorded total ORLADEYO revenues of $437.7 million in 2024, compared to $326.0 million in 2023, reflecting a 34% year-over-year growth[539]. - Collaborative and other revenues for 2024 were $8.0 million, an increase from $6.7 million in 2023[473]. Debt and Financing - The company reported an effective interest rate of 13.14% on the $300.0 million borrowing under the Pharmakon Loan Agreement for the year ended December 31, 2024[448]. - As of December 31, 2024, total borrowings under the Pharmakon Loan Agreement amounted to $323.7 million, including $10.041 million capitalized as Pharmakon PIK Interest Payments[578]. - The Company had total borrowings of $300 million under the Pharmakon Loan Agreement as of December 31, 2023, with interest expense of $27.326 million for that year[579]. - The effective interest rate on the Athyrium Term Loans was 13.71% for the year ended December 31, 2023, compared to 12.87% for the previous year[586]. Strategic Focus - The company is focused on the commercialization of ORLADEYO® (berotralstat) and anticipates potential government stockpiling orders for its products[14]. - The company is implementing strategic plans for its business and product candidates, focusing on best-in-class and first-in-class medicines[14]. - The company continues to evaluate opportunities for funding future operations, including out-licensing and government funding[469]. Risks and Compliance - The company relies heavily on third parties for product development and commercialization, which could materially impact its financial condition if these relationships fail[20]. - The company faces intense competition, which could reduce demand for its products if it fails to compete effectively[22]. - The company is subject to various laws and regulations, and non-compliance could result in substantial penalties and harm to its reputation[22]. - The company has recorded a valuation allowance against substantially all potential tax assets due to uncertainties in utilizing deferred tax assets[520]. Stock and Compensation - The Company’s stock-based compensation expense for 2024 was $65.4 million, compared to $55.6 million in 2023[474]. - The weighted average grant date fair value of restricted stock unit awards granted during 2024 was $7.32, up from $6.71 in 2023[607]. - Total stock-based compensation expense for the year ended December 31, 2024 was $65,413, up from $55,615 in 2023 and $44,701 in 2022[601]. - The total intrinsic value of stock option awards exercised under the Incentive Plan was $3,601 for the year ended December 31, 2023, compared to $21,150 in 2022[606].

Fourth Quarter 2024 Results Call Corporate Update & Financial Results February 24, 2025 CONFIDENTIAL & PROPRIETARY Forward-looking statements BioCryst's presentation contains forward-looking statements, including, but not limited to, statements regarding future results and forward-looking financial information, company performance, achievements, future market share or size, and expectations regarding pipeline development. These statements are subject to known and unknown risks, uncertainties and other facto ...