Core Insights - BioCryst Pharmaceuticals announced new data on ORLADEYO (berotralstat) for hereditary angioedema (HAE) treatment to be presented at the EAACI meeting in Glasgow from June 13 to 16, 2025 [1][2][3] Group 1: Presentation Details - Four abstracts will be presented at the EAACI meeting, including a pooled analysis of the APeX-2 and APeX-J trials focusing on the impact of berotralstat on quality of life for HAE patients [1] - A focus group study reported positive perceptions from patients following berotralstat treatment [2] - Interim results from the APeX-P study indicated that berotralstat use reduced the number of HAE attacks requiring treatment in pediatric patients [3] Group 2: Product Information - ORLADEYO (berotralstat) is the first oral therapy specifically designed to prevent HAE attacks in patients aged 12 years and older, functioning as a plasma kallikrein inhibitor [4][5] - The recommended dosage is one capsule per day, with caution advised for dosages higher than 150 mg due to potential QT prolongation [6][7] - The safety and effectiveness of ORLADEYO in pediatric patients under 12 years have not been established, and there is limited data on its use during pregnancy [9][10] Group 3: Company Overview - BioCryst Pharmaceuticals is a global biotechnology company focused on improving the lives of individuals with hereditary angioedema and other rare diseases, utilizing structure-guided drug design for developing therapeutics [11]

Core Insights - New data presented at the 14th C1-inhibitor Deficiency & Angioedema Workshop supports the use of berotralstat (ORLADEYO) for hereditary angioedema (HAE) across all age groups, demonstrating significant efficacy and safety [1][2] Group 1: Efficacy and Safety of Berotralstat - The APeX-P trial showed a reduction in the percentage of days with angioedema symptoms among young children, with a drop from a mean of 11% to 4% over 12 weeks, sustained up to 48 weeks [4] - In the Italian Expanded Access Program, median Angioedema Control Test scores improved from 11.5 to 16.0 over six months, indicating better disease control [6] - The Berolife study reported a decrease in the median monthly attack rate from 2.25 at baseline to 0.55 after six months of treatment in adolescents [10][11] Group 2: Patient Demographics and Treatment Cohorts - The APeX-P study involved children aged 2 to 11 years, with a median age of 8 years and a mean of 18 missed school days annually due to HAE prior to treatment [4] - The Italian Expanded Access Program included 22 patients receiving berotralstat free of charge, with 15 transitioning to commercially available ORLADEYO upon program closure [5][7] Group 3: Treatment Administration and Mechanism - ORLADEYO is the first oral therapy specifically designed to prevent HAE attacks in patients aged 12 years and older, administered as one capsule daily [12] - The treatment works by decreasing the activity of plasma kallikrein, which is involved in the pathophysiology of HAE [12]

Group 1 - BioCryst Pharmaceuticals (BCRX) closed at $10.59, with a 26.2% gain over the past four weeks, and a mean price target of $16.50 indicating a 55.8% upside potential [1] - The average of 12 short-term price targets ranges from a low of $11 to a high of $30, with a standard deviation of $5.32, suggesting variability in analyst estimates [2] - Analysts show strong agreement on BCRX's ability to report better earnings than previously predicted, which supports the potential for stock upside [4][11] Group 2 - The Zacks Consensus Estimate for the current year has increased by 366.9% over the past month, with six estimates revised higher 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 [13] - While consensus price targets may not be reliable for predicting stock gains, they can indicate potential price movement direction [13]

Core Insights - BioCryst Pharmaceuticals announced significant real-world evidence demonstrating the effectiveness of ORLADEYO (berotralstat) in reducing hereditary angioedema (HAE) attack rates in adolescents and individuals with severe HAE over an 18-month follow-up period [1][3][7] Group 1: Study Findings - The study presented at the ISPOR 2025 conference showed that patients experienced significantly lower HAE attack rates while on ORLADEYO, with a reduction from a baseline of 7.78-8.23 attacks/month to 1.24-1.90 attacks/month [10] - Specifically, patients had 6.25 fewer attacks/month at 12 months and 6.43 fewer attacks/month at 18 months after starting ORLADEYO [10] - Adolescents aged 12-17 years also showed significant reductions, with 1.56 fewer attacks/month at 12 months and 1.85 fewer attacks/month at 18 months [10] Group 2: Patient Population - The findings were based on a retrospective pre-post study involving 56 U.S. patients with C1-inhibitor deficiency and 99 U.S. adolescents with HAE [5][6] - The results indicate that ORLADEYO is effective across different age groups and severity levels of HAE, providing confidence to both physicians and patients [3][7] Group 3: Product Information - ORLADEYO is the first and only oral therapy specifically designed for the prophylaxis of HAE attacks in patients aged 12 years and older, functioning as a plasma kallikrein inhibitor [8][9] - The therapy is administered as a once-daily capsule, which works by decreasing the activity of plasma kallikrein to prevent HAE attacks [8]

Summary of BioCryst Pharmaceuticals (BCRX) Conference Call Company Overview - **Company**: BioCryst Pharmaceuticals (BCRX) - **Event**: 2025 Conference on May 14, 2025 - **Speakers**: John Stonehouse (CEO), Charlie Geier (Chief Commercial Officer) Key Industry Insights - **Impact of Executive Orders**: The recent executive order regarding most favored nation status may affect BioCryst, but details are still unclear. The company is monitoring the situation closely [3][4][5] - **Tariff Position**: BioCryst feels well-positioned regarding tariffs due to low cost of goods for Orlodea and a redundant supply chain in both Europe and the US [8] Financial Performance - **Q1 Results**: BioCryst reported a 51% year-over-year growth in Q1, driven by strong demand and an increase in the paid rate for Orlodea [13][14] - **Paid Rate Increase**: The paid rate for patients has jumped to 84%, with Medicare segment reaching 89%, attributed to the IRA making drugs more affordable [15] - **Sales Guidance**: The company is confident in achieving at least $800 million in US sales, with a global sales target of $1 billion by 2029 [27][29] Product Insights - **Orlodea**: The product has shown strong market demand and patient retention due to its efficacy and convenience. Patients prefer Orlodea for its oral administration and low attack rates [18][19][20][22] - **Market Stickiness**: The stickiness of the market is attributed to patient satisfaction with Orlodea, making it difficult for new competitors to gain traction [20] Pipeline Developments - **BCX1775**: The company is advancing its first biologic for Netherton Syndrome, aiming to control KLK activity and improve skin turnover [36][38] - **Avorostat**: This drug is being explored for diabetic macular edema (DME), with a focus on its potential to address unmet needs in patients who do not respond to VEGF therapies [68][70] Clinical Trials and Future Outlook - **Clinical Trials**: BioCryst is conducting studies for both BCX1775 and Avorostat, with expectations for data by the end of the year. The company is optimistic about the potential for accelerated pathways to market if results are favorable [61][66][78] - **Market Potential**: The company believes that the market for Netherton Syndrome could grow significantly if a targeted therapy is introduced, similar to the growth seen in other rare diseases [56] Conclusion - BioCryst Pharmaceuticals is positioned for strong growth with its current product offerings and pipeline developments. The company is actively monitoring regulatory changes and market dynamics while focusing on delivering effective therapies for rare diseases.

Core Viewpoint - The FDA has accepted BioCryst Pharmaceuticals' New Drug Application for ORLADEYO, which, if approved, would be the first targeted oral prophylactic therapy for pediatric patients with hereditary angioedema (HAE) under the age of 12, with a PDUFA target action date of September 12, 2025 [1][5]. Group 1: FDA Approval and Application - The FDA granted Priority Review for the NDA of ORLADEYO for pediatric patients aged 2 to 11 years [1]. - ORLADEYO is already approved for patients aged 12 years and older and is commercially available in over 30 countries [6]. Group 2: Clinical Trial Data - The NDA is based on positive interim data from the APeX-P clinical trial, which is the largest trial evaluating prophylactic therapy for HAE in the specified age group [3]. - Interim results indicated that ORLADEYO was well tolerated, showing a consistent safety profile and early, sustained reductions in monthly attack rates [3]. Group 3: Market Potential and Company Strategy - The company aims to provide a more convenient therapeutic option for young children with HAE, responding to demand from patients, caregivers, and physicians [2]. - BioCryst has also filed for a line extension application with the European Medicines Agency and plans additional regulatory filings in Japan and Canada [5].

Group 1: BioCryst Pharmaceuticals, Inc. (BCRX) - BioCryst has a Zacks Rank 1 and the Zacks Consensus Estimate for its current year earnings increased by 285.7% over the last 60 days [1] - The company's shares gained 15% over the last three months, while the S&P 500 declined by 7.2% [1] - BioCryst possesses a Momentum Score of A [1] Group 2: WidePoint Corporation (WYY) - WidePoint has a Zacks Rank 1 and the Zacks Consensus Estimate for its current year earnings increased by 108.3% over the last 60 days [2] - The company's shares gained 35.7% over the last three months, compared to the S&P 500's decline of 7.2% [2] - WidePoint possesses a Momentum Score of B [2] Group 3: Rambus Inc. (RMBS) - Rambus has a Zacks Rank 1 and the Zacks Consensus Estimate for its current year earnings increased by 3.9% over the last 60 days [3] - The company's shares gained 11.6% over the past month, while the S&P 500 advanced by 6.9% [3] - Rambus possesses a Momentum Score of B [3]

Group 1 - Chain Bridge Bancorp, Inc. (CBNA) has seen a 9.5% increase in the Zacks Consensus Estimate for its current year earnings over the last 60 days [1] - Fresenius Medical Care AG (FMS) has experienced a 4.5% increase in the Zacks Consensus Estimate for its current year earnings over the last 60 days [1] - Brenntag SE (BNTGY) has seen a 14.1% increase in the Zacks Consensus Estimate for its next year earnings over the last 60 days [2] - WidePoint Corporation (WYY) has experienced a significant 108.3% increase in the Zacks Consensus Estimate for its current year earnings over the last 60 days [2] - BioCryst Pharmaceuticals, Inc. (BCRX) has seen an impressive 285.7% increase in the Zacks Consensus Estimate for its current year earnings over the last 60 days [3]

Core Viewpoint - BioCryst Pharmaceuticals (BCRX) is positioned as a strong investment opportunity due to its improving earnings outlook and analysts' increasing earnings estimates [1][2]. Earnings Estimates - Analysts are optimistic about BioCryst's earnings prospects, leading to higher estimates that are expected to positively impact the stock price [2]. - The current quarter's earnings estimate is projected at $0.02 per share, reflecting a significant increase of +133.33% from the previous year [5]. - For the full year, the earnings estimate is expected to be $0.13 per share, representing a change of +130.95% from the prior year [6]. Estimate Revisions - Over the last 30 days, the Zacks Consensus Estimate for BioCryst has increased by 158.18%, with three estimates moving higher and no negative revisions [5]. - The consensus estimate for the current year has seen a remarkable increase of 341.56% during the same period [7]. Zacks Rank - BioCryst currently holds a Zacks Rank 2 (Buy), indicating strong agreement among analysts in revising earnings estimates upward [8]. - Stocks with Zacks Rank 1 (Strong Buy) and 2 (Buy) have historically outperformed the S&P 500 [8]. Stock Performance - BioCryst shares have appreciated by 48.9% over the past four weeks, suggesting strong investor confidence in the company's earnings growth prospects [9].

Core Viewpoint - Momentum investing focuses on following a stock's recent price trends, aiming to buy high and sell higher, with the expectation that established trends will continue [1] Company Summary: BioCryst Pharmaceuticals (BCRX) - BCRX currently holds a Momentum Style Score of A, indicating strong momentum characteristics [2] - The company has a Zacks Rank of 2 (Buy), suggesting it is positioned for potential outperformance in the market [3] - Over the past week, BCRX shares increased by 6.44%, significantly outperforming the Zacks Medical - Drugs industry, which rose by 0.39% [5] - In a longer timeframe, BCRX's monthly price change is 48.89%, compared to the industry's 6.21% [5] - Over the last quarter, BCRX shares rose by 14.81%, and they have increased by 97.65% over the past year, while the S&P 500 saw movements of -6.34% and 9.94%, respectively [6] - The average 20-day trading volume for BCRX is 5,448,160 shares, indicating strong trading activity [7] Earnings Outlook - In the past two months, 6 earnings estimates for BCRX have been revised upwards, with no downward revisions, leading to an increase in the consensus estimate from -$0.07 to $0.13 [9] - For the next fiscal year, 6 estimates have also moved upwards without any downward revisions [9] Conclusion - Considering the strong momentum indicators and positive earnings outlook, BCRX is identified as a 2 (Buy) stock with a Momentum Score of A, making it a potential candidate for near-term investment [11]