Financial Performance - ORLADEYO® (berotralstat) net revenue for full year 2024 reached $437 million, a 34% year-over-year increase[8] - The company expects to approach quarterly earnings per share profitability in the second half of fiscal year 2025[4] Guidance and Forecasts - The company provided guidance for full year 2025 ORLADEYO net revenue and total revenue[4] - Full year 2025 operating expenses guidance was also provided[4] Corporate Events and Announcements - BioCryst will present at the 43rd Annual J.P. Morgan Healthcare Conference on January 14, 2025[4] - Preliminary unaudited results for Q4 and full year 2024 were announced on January 10, 2025[4]

Core Insights - BioCryst Pharmaceuticals reported a strong financial performance for Q4 2024, with ORLADEYO net revenue reaching $124.2 million, a 36.6% increase year-over-year, and total revenues of $131.5 million, up 40.8% year-over-year [1][10] - The company has raised its FY 2025 ORLADEYO net revenue guidance to between $535 million and $550 million, reflecting strong demand and positive early signs from Medicare patients [1][23] - BioCryst aims to achieve positive EPS and cash flow by the second half of 2025, building on a non-GAAP operating profit of $62.9 million in 2024 [1][27] Financial Performance - For FY 2024, total revenues were $450.7 million, a 36.0% increase from $331.4 million in FY 2023, primarily driven by ORLADEYO's performance [17] - The GAAP operating loss for FY 2024 was $2.5 million, significantly improved from a loss of $103.7 million in FY 2023 [21] - The net loss for FY 2024 was $88.9 million, or $0.43 per share, compared to a net loss of $226.5 million, or $1.18 per share, in FY 2023 [22] Product Development and Pipeline - BioCryst plans to expand the ORLADEYO label to children aged 2 to 11, with a New Drug Application (NDA) submission expected this year [2][6] - Initial clinical data from BCX17725 for Netherton syndrome and avoralstat for diabetic macular edema is anticipated by the end of 2025 [1][5] - The company is advancing its pipeline with a focus on rare diseases, aiming to provide differentiated therapies [5][6] Market and Commercial Strategy - The company noted that 73.5% of U.S. patients were on paid ORLADEYO products by the end of Q4 2024, an increase from 71.4% at the end of 2023 [7] - A market survey indicated that 97% of HAE treaters are considering prescribing ORLADEYO, with a significant increase in the likelihood of current prescribers to recommend it [7] - BioCryst has received final reimbursement for ORLADEYO in Portugal and is now reimbursed in all major Western European countries, except the Netherlands, which is expected in H1 2025 [7][8] Cost Management and Profitability Outlook - R&D expenses for Q4 2024 decreased to $49.4 million from $70.1 million in Q4 2023, while SG&A expenses increased to $80.5 million, reflecting increased commercial investment [11][12] - The company expects to maintain a revenue growth rate significantly outpacing operating expense growth, projecting a CAGR of around 20% for revenue over the next three years [26] - BioCryst anticipates achieving meaningful and sustainable profitability, with hundreds of millions in cash added to the balance sheet [9][26]

Core Insights - BioCryst Pharmaceuticals announced positive interim results from the APeX-P clinical trial for ORLADEYO, showing significant reductions in HAE attack rates in pediatric patients aged 2 to <12 years [1][2][7] - The trial is the largest to date evaluating a prophylactic therapy for HAE in this age group, with data to be presented at the 2025 AAAAI/WAO Joint Congress [2][17] - ORLADEYO demonstrated safety and tolerability, with no new safety signals identified, and resulted in early and sustained reductions in monthly attack rates [7][18] Clinical Trial Results - APeX-P included a 12-week standard-of-care treatment period followed by an open-label ORLADEYO treatment period lasting up to 144 weeks, with interim data from 17 participants who completed at least 48 weeks of treatment [7][14] - The median monthly attack rate decreased from 0.96 attacks/month during the standard-of-care period to 0 attacks/month after one month of ORLADEYO treatment, maintaining a median of 0 through month 12 [7][14] - Participants with ≥5 baseline attacks/month experienced the largest reductions, with 6.20 fewer attacks/month at 12 months [14] Real-World Evidence - New real-world evidence presented at the congress showed statistically significant HAE attack rate reductions in patients with C1-INH deficiency and normal C1-INH levels after starting ORLADEYO [5][14] - Patient-reported outcomes indicated high satisfaction with ORLADEYO, with many patients willing to switch from other long-term prophylaxis therapies [12][23] - The study highlighted that 83% of participants experienced symptom onset before six years of age, and 90% were diagnosed within the same timeframe [7] Patient Insights - Patients reported a willingness to switch long-term prophylaxis therapies, with factors such as treatment burden and administration preference influencing their decisions [12][23] - Most participants reported less frequent and less severe attacks after starting ORLADEYO, with high satisfaction rates regarding their treatment [12][23] - The study included insights from U.S. patients who had been treated with ORLADEYO, showing a mean age of 43.2 years and a mean of 13.4 years since diagnosis [23]

Core Insights - BioCryst Pharmaceuticals announced positive interim results from the APeX-P clinical trial for ORLADEYO, showing significant reductions in hereditary angioedema (HAE) attack rates in pediatric patients aged 2 to <12 years [1][2][6] - The company plans to submit a New Drug Application to the FDA within the year, addressing a significant unmet need for children with HAE [2][3] Clinical Trial Results - APeX-P is the largest trial evaluating prophylactic therapy for HAE in young patients, demonstrating that ORLADEYO is safe and well-tolerated, with early and sustained reductions in monthly attack rates [2][3] - In the standard-of-care period, the median monthly attack rate was 0.96 attacks/month, which dropped to 0 after one month of ORLADEYO treatment, maintaining a median of 0 through month 12 [6][11] - 83% of participants experienced symptom onset before six years of age, with 90% diagnosed within the same timeframe [6] Real-World Evidence - New real-world evidence presented at the 2025 AAAAI/WAO Joint Congress indicates statistically significant reductions in HAE attack rates for patients with C1-INH deficiency and normal C1-INH levels after starting ORLADEYO [4][11] - Patients with ≥5 baseline attacks/month experienced a reduction of 6.20 attacks/month at 12 months and 6.37 attacks/month at 18 months [11] - Among patients with 0 attacks/month at baseline, 70% maintained this low attack rate at 12 months [11] Patient-Reported Outcomes - Patient-reported outcomes indicate a willingness to switch long-term prophylaxis (LTP) and improved treatment satisfaction after initiating ORLADEYO [9][10] - Participants reported less frequent and severe attacks after switching to ORLADEYO, with high satisfaction rates regarding their treatment [21][22] Future Directions - BioCryst is committed to addressing the needs of HAE patients and advancing its pipeline of therapies, with ORLADEYO being the first oral therapy specifically designed for HAE prevention [15][23]

Core Viewpoint - The market anticipates BioCryst Pharmaceuticals (BCRX) will report a year-over-year increase in earnings driven by higher revenues when it releases its results for the quarter ended December 2024 [1] Financial Expectations - The upcoming earnings report is expected to be released on February 24, 2025, with a consensus estimate of a quarterly loss of $0.06 per share, reflecting a year-over-year change of +78.6% [3] - Revenues are projected to be $130.8 million, which is a 40% increase from the same quarter last year [3] Estimate Revisions - The consensus EPS estimate has remained unchanged over the last 30 days, indicating that analysts have not significantly altered their initial estimates during this period [4] - The Most Accurate Estimate for BioCryst is lower than the Zacks Consensus Estimate, resulting in an Earnings ESP of -7.14%, suggesting a bearish outlook from analysts [10] Earnings Surprise Prediction - The Zacks Earnings ESP model indicates that a positive or negative reading can predict the deviation of actual earnings from the consensus estimate, but its predictive power is more significant for positive readings [7] - A positive Earnings ESP combined with a strong Zacks Rank (1-3) has historically led to a positive surprise nearly 70% of the time [8] Historical Performance - In the last reported quarter, BioCryst was expected to post a loss of $0.07 per share and did so, resulting in no surprise [12] - Over the last four quarters, the company has beaten consensus EPS estimates two times [13] Industry Comparison - Another player in the Zacks Medical - Drugs industry, Ardelyx (ARDX), is expected to post earnings of $0.02 per share for the same quarter, indicating a year-over-year change of +116.7% and revenues expected to be $111.19 million, up 223.6% from the previous year [17] - Ardelyx has an Earnings ESP of 48.49%, suggesting a higher likelihood of beating the consensus EPS estimate [18]

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

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

Core Insights - BioCryst Pharmaceuticals will present five abstracts on ORLADEYO (berotralstat) at the 2025 AAAAI/WAO joint congress, focusing on its use for hereditary angioedema (HAE) [1][2] Group 1: Product Information - ORLADEYO is the first and only oral therapy specifically designed to prevent HAE attacks in patients aged 12 years and older, administered as one capsule daily [3][4] - The drug functions as a plasma kallikrein inhibitor, reducing the activity of plasma kallikrein to prevent HAE attacks [3] Group 2: Clinical Trials and Research - A late-breaking abstract will present results from the APeX-P trial, which evaluates ORLADEYO in pediatric patients aged 2 to under 12 years with HAE [2] - The five abstracts will cover various aspects, including real-world attack rates before and after berotralstat initiation and patient-reported impacts on attack frequency and severity [8] Group 3: Safety and Efficacy - The safety and effectiveness of ORLADEYO for treating acute HAE attacks have not been established, and it is not recommended for such use [5][10] - Common adverse reactions include abdominal pain, vomiting, diarrhea, back pain, and gastroesophageal reflux disease, occurring in 10% or more of patients [6] Group 4: Company Overview - BioCryst Pharmaceuticals is a global biotechnology company focused on developing therapies for hereditary angioedema and other rare diseases, leveraging expertise in structure-guided drug design [11]

Core Viewpoint - BioCryst Pharmaceuticals, Inc. will report its fourth quarter 2024 financial results on February 24, 2025, and will host a conference call to discuss these results and provide a corporate update [1]. Company Overview - BioCryst Pharmaceuticals is a global biotechnology company focused on improving the lives of individuals with complement-mediated and other rare diseases [3]. - The company utilizes structure-guided drug design to develop first-in-class or best-in-class small-molecule and protein therapeutics aimed at challenging diseases [3]. - BioCryst has commercialized ORLADEYO® (berotralstat), which is the first oral, once-daily plasma kallikrein inhibitor, and is advancing a pipeline of small-molecule and protein therapies [3].

Core Insights - BioCryst Pharmaceuticals expects ORLADEYO net revenue to be between $515 million and $535 million in 2025, with total revenue (including RAPIVAB) projected between $540 million and $560 million [1][5] - The company achieved operating profit in 2024 (excluding stock-based compensation) and anticipates approaching quarterly EPS profitability and positive cash flow in the second half of 2025 [1][8] - A new drug application for ORLADEYO granules aimed at children up to age 12 is planned for submission in 2025, addressing a significant unmet medical need [1][2] - The pipeline is advancing with clinical programs for BCX17725 (Netherton syndrome) and avoralstat (diabetic macular edema) expected to progress in 2025 [1][2] Revenue Performance - Preliminary, unaudited ORLADEYO net revenue for Q4 2024 was $123.5 million, reflecting a 36% year-over-year increase, while full-year 2024 net revenue reached $437 million, up 34% year-over-year [3][4] - Total revenue for Q4 2024 was $130.8 million, a 40% increase year-over-year, with full-year total revenue at $450 million, marking a 36% growth year-over-year [5] Operating Expenses - The company forecasts full-year 2025 GAAP operating expenses to be between $485 million and $495 million, which includes an estimated $60 million for stock-based compensation [6] - Operating expenses, excluding stock-based compensation, are projected to be between $425 million and $435 million, reflecting additional investments for the pediatric product launch and ongoing clinical activities [6] Profitability Outlook - Revenue growth in 2024 significantly outpaced operating expense growth, and this trend is expected to continue [7] - The company anticipates an annual compound annual growth rate (CAGR) for revenue of around 20% over the next three years, compared to a projected CAGR of approximately 5% for operating expenses [8] - BioCryst expects to achieve quarterly positive EPS and positive cash flow in the second half of 2025, with full-year profitability anticipated in 2026 [8]