If you like what you have just read and want to receive at least 4 exclusive stock tips every week focused on Pharma, Biotech and Healthcare, then join me at my marketplace channel, Haggerston BioHealth . Invest alongside the model portfolio or simply access the investment bank-grade financial models and research. I hope to see you there.Back in November 2023, I published a note on BioCryst (NASDAQ: BCRX ) to Seeking Alpha, assigning its stock a "Sell" rating. At the time, shares were priced at $5.5, and in ...

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].

Investors clearly weren't in a forgiving mood on Monday when it came to BioCryst Pharmaceuticals (BCRX -9.99%) stock. After the commercial-stage biotech published its fourth-quarter and full-year 2024 results, they traded its shares down by an even 10% on the day. That fall was far steeper than the 0.5% slip of the benchmark S&P 500 (^GSPC -0.50%).A mixed final quarter of 2024For the quarter, BioCryst managed to increase its revenue by 41% to $131.5 million. The company also narrowed its generally accepted ...

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 ...

Financial Data and Key Metrics Changes - For Q4 2024, total revenue was $131.5 million, with Orladeo contributing $124.2 million [39] - Full year 2024 total revenue reached $450.7 million, with Orladeo accounting for $437.7 million [39] - Non-GAAP operating profit was $62.9 million, a significant increase from a non-GAAP operating loss of $48.1 million in 2023 [40] - Cash at the end of 2024 was approximately $343 million, with net cash utilization for Q4 at $8.4 million [41] Business Line Data and Key Metrics Changes - Orladeo revenue grew by 34% year-over-year, maintaining the same number of new patients as in its first year [9] - For Q4, $17.2 million (13.9%) of Orladeo's revenue came from ex-US markets, while for the full year, it was $51.7 million (11.8%) [39] - The company raised Orladeo revenue guidance for 2025 to between $535 million and $550 million [14] Market Data and Key Metrics Changes - 97% of surveyed US HAE treaters are considering prescribing Orladeo, with 59% extremely likely to prescribe it to more patients [17] - The company noted a significant increase in the percentage of Medicare patients able to afford co-payments due to the Inflation Reduction Act [13] Company Strategy and Development Direction - The company aims to file an NDA for Orladeo for pediatric use in children aged 2 to 11 this year [21] - BCX1775 for Netherton syndrome and Avoralstat for diabetic macular edema are advancing in clinical trials, with expectations for meaningful data readouts this year [21][28] - Long-term revenue guidance includes a three-year CAGR of 20%, targeting over $750 million by 2027 and $1 billion by 2029 [44] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the execution and performance across the company, highlighting a strong position for future growth [8] - The management team noted that the execution in 2024 has set the stage for positive and sustainable cash flow and EPS in the second half of 2025 [43] - There is optimism regarding the potential for BCX1775 to address an unmet need in Netherton syndrome, which is currently underdiagnosed [27] Other Important Information - The company is seeing strong commercial demand for RAPIVAB due to the prevalence of flu, despite some uncertainty around future government funding [42] - The pediatric oral granule formulation of Orladeo has shown promising results in trials, with high tolerability and retention rates [35][114] Q&A Session Summary Question: Can you narrow down when data from Netherton patients is expected? - Management indicated that data will be available later this year, with a focus on skin healing and KLK5 activity [51][52] Question: What is the current paid patient proportion for Orladeo? - The company reported a 73.5% paid rate across all patients, with about 20% of patients on Medicare [120] Question: How does the pediatric study's tolerability compare to adults? - The pediatric oral granules have shown good tolerability and compliance, similar to adult experiences [98] Question: What is the expected timeline for dosing Netherton syndrome patients? - Dosing for Netherton syndrome patients is expected to begin around mid-year, with data on skin activity anticipated by year-end [83] Question: What percentage of KLK5 suppression is needed for clinical benefit? - The company is looking for nearly complete suppression of KLK5 activity, although the exact percentage required for clinical benefit is not yet known [128]

Financial Data and Key Metrics Changes - For the full year 2024, total revenue was $450.7 million, with Orlodeo contributing $437.7 million [29] - The company achieved a non-GAAP operating profit of $62.9 million, a significant increase from a non-GAAP operating loss of $48.1 million in 2023 [29] - Cash at the end of the year was approximately $343 million, with net cash utilization for the quarter at $8.4 million [30] Business Line Data and Key Metrics Changes - Orlodeo revenue grew by 34% year-over-year, reflecting strong execution by the commercial team [6][10] - In Q4, Orlodeo generated $124.2 million, with $17.2 million (13.9%) coming from ex-U.S. sales [29] - The company raised its Orlodeo revenue guidance for 2025 to between $535 million and $550 million [11] Market Data and Key Metrics Changes - The company noted a higher percentage of Medicare patients on Orlodeo can afford their co-payments due to the Inflation Reduction Act [10] - The paid patient rate across the entire patient base was 73.5%, with expectations to increase further [44][94] - The company expects to see continued strong demand in early 2025, consistent with the momentum from 2024 [10] Company Strategy and Development Direction - The company aims to file an NDA for Orlodeo for pediatric patients aged 2 to 11 this year [16][26] - The pipeline includes advancing BCX17725 for Netherton syndrome and ivorlstat for diabetic macular edema, with clinical data expected later this year [7][24] - The long-term revenue guidance includes a three-year CAGR of 20%, targeting over $750 million by 2027 and $1 billion by 2029 [30][31] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the execution and performance, stating that the momentum is expected to carry into 2025 and beyond [9] - The management highlighted the importance of the upcoming clinical data and the potential for BCX17725 to address an unmet need in Netherton syndrome [19][20] - The company anticipates positive and sustainable quarterly cash flow and EPS in the second half of 2025 [30] Other Important Information - The company is presenting real-world evidence at the Quad AI Conference, showcasing the effectiveness of Orlodeo in reducing attack rates for HAE patients [12] - The pediatric trial for Orlodeo showed that granules were safe and well-tolerated, with a high retention rate among participants [26][88] Q&A Session Summary Question: Timing for data from Netherton syndrome patients - Management indicated that data will be available later this year, with a focus on skin healing and KLK5 activity [35][38] Question: Proportion of paid patients and guidance for Orlodeo - The paid patient rate was 73.5% overall, with expectations for Medicare patients to increase significantly due to affordability improvements [44][94] Question: Tolerability of pediatric formulation compared to adults - The pediatric formulation showed good tolerability and compliance, similar to adult experiences [74] Question: International demand for Orlodeo - Demand in Europe, Canada, and Japan is growing, with confidence in Orlodeo's effectiveness leading to increased prescriptions [80][81] Question: Historical reauthorization rates - Approximately half of patients go through reauthorization in Q1, with improvements noted in the process over the years [104]

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 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 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]