Core Insights - BioCryst Pharmaceuticals announced the departure of Dr. Helen Thackray, the chief research and development officer, effective September 1, 2025, transitioning to an advisory role until the end of the year [1][2] - Dr. Thackray joined BioCryst in 2019 as a board member and became the chief research and development officer in 2021, being a finalist for the CEO succession process [2] - The CEO of BioCryst, Jon Stonehouse, expressed gratitude for Dr. Thackray's contributions, including the launch of a new protein therapeutics platform and advancements in various clinical programs [3] Company Overview - BioCryst Pharmaceuticals is a global biotechnology company focused on improving the lives of individuals with hereditary angioedema and other rare diseases [5] - The company utilizes structure-guided drug design to develop innovative oral small-molecule and protein therapeutics targeting challenging diseases [5] - BioCryst has commercialized ORLADEYO (berotralstat), the first oral, once-daily plasma kallikrein inhibitor, and is advancing a pipeline of therapies [5]

Financial Performance & Guidance - BioCryst's Q2 2025 was strong, driven by increased ORLADEYO demand[14] - ORLADEYO revenue is expected to meet the previous guidance range of $580-600 million for FY25, even excluding Q4 2025 EU revenue[16] - The company anticipates achieving full-year operating profit in 2026 and positive net income & cash flows in 2026+[58] - Cash, cash equivalents, restricted cash & investments totaled $287 million as of June 30, 2025[63] - The company paid down $50 million of its senior credit facility in July 2025[62] ORLADEYO & Market Dynamics - Market research indicates a growing patient preference for oral administration of HAE long-term prophylaxis (LTP) since 2023[17] - Monte Carlo simulation projects ORLADEYO reaching a steady state of over 2,000 patients in the US by 2028[25] - ORLADEYO revenue advances to royalty-free tier (>$550M)[61] - The blended royalty rate for ORLADEYO is expected to decline to approximately 4% at peak sales once OMERS reaches its cap[67] Pipeline Development - The PDUFA target date for the pediatric NDA for ORLADEYO is December 12, 2025[32] - An IND has been cleared by the FDA for BCX17725, a targeted KLK5 inhibitor for treating Netherton syndrome (NS)[37]

Core Insights - Clearside Biomedical has successfully completed an End-of-Phase 2 meeting with the FDA, leading to alignment on the Phase 3 program design for CLS-AX in wet AMD [1][5] - The company aims for a flexible three-to-six-month dosing label for CLS-AX, which is expected to be commercially attractive if approved [2][5] - Significant progress has been made by Clearside's partners in advancing their programs, including approvals and ongoing clinical trials [2][5] Recent Highlights - BioCryst Pharmaceuticals has received authorization to initiate its first clinical trial in Australia for avoralstat, targeting diabetic macular edema [5] - Arctic Vision's NDA for ARCATUS has been accepted for review in China, with approvals already granted in Australia and Singapore [5] - Over 15 presentations on suprachoroidal drug delivery were made at major ophthalmic medical meetings this year, showcasing advancements in retinal disease treatments [1][5] Financial Results - License and other revenue for Q1 2025 was $2.3 million, a significant increase from $0.2 million in Q1 2024, primarily due to license fees from partners [8][11] - R&D expenses decreased to $4.5 million in Q1 2025 from $5.6 million in Q1 2024, attributed to lower clinical trial costs [8][11] - The net loss for Q1 2025 was $8.2 million, or $0.11 per share, compared to a net loss of $11.8 million, or $0.17 per share, in Q1 2024 [8][11] Company Overview - Clearside Biomedical focuses on revolutionizing the delivery of therapies to the back of the eye through the suprachoroidal space, aiming to improve patient outcomes [6] - The company's lead program, CLS-AX, is in development for the treatment of wet AMD, with plans for a Phase 3 program underway [6] - Clearside has developed its first product, XIPERE, which is commercially available in the U.S. through a partner [6][9]

—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 open for BCX17725 for Netherton syndrome— RESEARCH TRIANGLE PARK, N.C., May 05, 2025 (GLOBE NEWS ...