Core Viewpoint - BioCryst Pharmaceuticals (BCRX) has shown a significant price increase of 48.9% over the past four weeks, with a mean price target of $16.25 indicating a potential upside of 61.2% from the current price of $10.08 [1] Price Targets and Analyst Estimates - The mean estimate consists of 12 short-term price targets with a standard deviation of $5.66, indicating variability among analysts; the lowest estimate is $8 (20.6% decline), while the highest is $30 (197.6% increase) [2] - Analysts' price targets can often mislead investors, as empirical research shows that they rarely indicate the actual price direction of a stock [7] - A low standard deviation in price targets suggests a high degree of agreement among analysts regarding the stock's price movement, which can serve as a starting point for further research [9] Earnings Estimates and Analyst Consensus - There is growing optimism among analysts regarding BCRX's earnings prospects, as indicated by a strong consensus in revising EPS estimates higher, which correlates with potential stock price increases [11] - The Zacks Consensus Estimate for the current year has increased by 341.6% over the past month, with five estimates rising and no negative revisions [12] - BCRX holds a Zacks Rank 2 (Buy), placing it in the top 20% of over 4,000 ranked stocks based on earnings estimates, suggesting a strong potential upside [13]

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]

Core Insights - BioCryst Pharmaceuticals reported first-quarter sales of $145.50 million, a 52.7% increase year over year, surpassing the consensus estimate of $126.87 million [1] - The growth was primarily driven by Orladeyo's net revenue of $134.2 million, which increased by $45.3 million year over year, compared to $88.9 million in the same quarter of the previous year [1] Financial Performance - The net income for the first quarter of 2025 was $32 thousand, or $0.00 per share, a significant improvement from a net loss of $35.4 million, or $0.17 per share, in the first quarter of 2024 [2] - Operating income for the first quarter of 2025 was $21.2 million, compared to an operating loss of $14.5 million for the same period in 2024 [3] - Non-GAAP operating income, excluding stock-based compensation, was $42.6 million for the first quarter of 2025, compared to a non-GAAP operating loss of $0.8 million in the first quarter of 2024 [3] Guidance and Outlook - BioCryst raised its 2025 revenue outlook for Orladeyo to between $580 million and $600 million, up from the previous estimate of $535 million to $550 million [4] - The company now anticipates full-year 2025 operating expenses to be between $440 million and $450 million, an increase from the prior guidance of $425 million to $435 million [4] - The expectation for achieving net income and positive cash flows has been accelerated to 2025, previously anticipated for 2026 [5] - Positive cash flow is defined as the improvement in cash and equivalents from year-end 2024 to year-end 2025, excluding a $75 million prepayment made in April 2025 [5] Market Reaction - Following the announcement, BCRX stock closed up by 23.50% to $11.03 [5]

Group 1 - The article emphasizes that investing is a learning process, where failures serve as tuition and successes contribute to lessons learned [1] - The author has approximately five years of focused research on various stocks, particularly in the healthcare sector [1] Group 2 - There is no current stock or derivative position in any mentioned companies, but there is a potential for initiating a long position within the next 72 hours [2] - The article reflects the author's personal opinions and is not influenced by compensation from any company mentioned [2]

BioCryst Pharmaceuticals (BCRX) Q1 2025 Earnings Call May 05, 2025 08:30 AM ET Company Participants John Bluth - Investor RelationsJon Stonehouse - Chairman, CEO, President & Interim CFOCharles Gayer - Chief Commercial OfficerHelen Thackray - Chief Research & Development OfficerSteve Seedhouse - Biotechnology Equity ResearchGena Huidong Wang - Managing Director, Biotech Equity ResearchLaura Chico - Managing Director, Equity ResearchLiisa Bayko - Managing DirectorJon Wolleben - Managing Director Conference C ...

Core Insights - BioCryst Pharmaceuticals (BCRX) reported break-even quarterly earnings per share, surpassing the Zacks Consensus Estimate of a loss of $0.07, and showing improvement from a loss of $0.17 per share a year ago, resulting in an earnings surprise of 100% [1] - The company posted revenues of $145.53 million for the quarter ended March 2025, exceeding the Zacks Consensus Estimate by 14.97%, and up from $92.76 million year-over-year [2] - BioCryst shares have increased approximately 18.8% since the beginning of the year, contrasting with a -3.3% decline in the S&P 500 [3] Earnings Outlook - The future performance of BioCryst's stock will largely depend on management's commentary during the earnings call and the company's earnings outlook [4] - Current consensus EPS estimate for the upcoming quarter is -$0.03 on revenues of $137.38 million, and for the current fiscal year, it is -$0.09 on revenues of $565.22 million [7] Industry Context - The Medical - Drugs industry, to which BioCryst belongs, is currently ranked in the top 25% of over 250 Zacks industries, indicating a favorable outlook compared to the bottom 50% [8] - Another company in the same industry, ProPhase Labs, Inc. (PRPH), is expected to report a quarterly loss of $0.18 per share, reflecting a year-over-year change of -157.1% [9]

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 ...