Berotralstat Hydrochloride

–Excluding European ORLADEYO revenue for the full year 2025, preliminary 2025 ORLADEYO net revenue was $563 million (+43 percent y-o-y on a comparable basis)– –ORLADEYO net revenue expected to be between $625 million and $645 million in 2026– –BioCryst expects continued non-GAAP profitability in 2026 even after expected close of the proposed acquisition of Astria Therapeutics in Q1 2026– RESEARCH TRIANGLE PARK, N.C., Jan. 12, 2026 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced preliminary, unaudited ORLADEYO (berotralstat) net revenue for the fourth quarter and full year 2025. The company also provided guidance for ORLADEYO net revenue, total revenue, and operating expenses for full year 2026. “2025 was a transformative year for BioCryst. Continued strong demand for ORLADEYO and outstanding patient outcomes have solidified its position as the leading oral, once-daily prophylaxis treatment for HAE. The successful sale of our European ORLADEYO business further strengthened our financial position, and the proposed acquisition of Astria marks an exciting step toward expanding our impact for HAE patients. With our advancing pipeline, flexibility to continue pursuing value-creating business development opportunities, and dedication of our exceptional team, we are confident in our ability to deliver innovative treatments for rare disease patients and drive sustainable growth well into the future,” said Charlie Gayer, President and Chief Executive Officer. Preliminary Fourth Quarter and Full Year 2025 ORLADEYO Revenue, 2026 ORLADEYO and Total Revenue Outlook, and Cash Position at Year-End 2025 Preliminary, unaudited ORLADEYO net revenue in the fourth quarter of 2025 was $151 million (+22 percent y-o-y; +36% y-o-y on a comparable basis, excluding European ORLADEYO revenue for the fourth quarter of 2024). Preliminary, unaudited ORLADEYO net revenue for full year 2025 was $601 million (+37 percent y-o-y), beating the company’s prior guidance range of $590 million to $600 million. Excluding European ORLADEYO revenue for the full year 2025, preliminary, unaudited ORLADEYO net revenue was $563 million (+43 percent y-o-y on a comparable basis). The company expects full year 2026 global net ORLADEYO revenue to be between $625 million and $645 million, and expects full year 2026 total revenue, including RAPIVAB® (peramivir injection), to be between $635 million and $660 million. Preliminary, unaudited cash, cash equivalents, restricted cash & investments as of December 31, 2025, were $338 million. Operating Expense Outlook The company expects full year 2026 non-GAAP operating expenses, excluding stock-based compensation, restructuring, and transaction-related costs, to be between $380 million and $390 million. This does not include operating expenses from Astria Therapeutics after the expected close of the acquisition in Q1 2026. Upon the expected close of the Astria acquisition, the company expects additional non-GAAP operating expenses in 2026 to be between $70 million and $80 million to support the ongoing Phase 3 enrollment of navenibart and commercial readiness manufacturing activities. These costs are expected to trend down over the next two years as the trial is completed and the full impact of operational synergies is realized. Full Year 2026 Financial Guidance Item As of January 12, 2026 ORLADEYO revenue $625 million to $645 million Total revenue $635 million to $660 million Non-GAAP operating expense $380 million to $390 million Non-GAAP operating expense including Astria acquisition $450 million to $470 million Presentation at 44th Annual J.P. Morgan Healthcare Conference The company will present today at 1:30 p.m. ET at the 44th Annual J.P. Morgan Healthcare Conference in San Francisco. A link to the live audio webcast and replay of the presentation may be accessed in the Investors section of BioCryst’s website at . About BioCryst Pharmaceuticals BioCryst is a global biotechnology company focused on developing and commercializing medicines for hereditary angioedema (“HAE”) and other rare diseases, driven by its deep commitment to improving the lives of people living with these conditions. BioCryst has commercialized ORLADEYO ® (berotralstat), the first oral, once-daily plasma kallikrein inhibitor, and is advancing a pipeline of potential first-in-class or best-in-class oral small-molecule and injectable protein therapeutics for a range of rare diseases. For more information, please visit or follow us on LinkedIn . Non-GAAP Financial Measures The information furnished in this release includes non-GAAP financial measures that differ from measures calculated in accordance with generally accepted accounting principles in the United States of America (“GAAP”), including financial measures labeled as “non-GAAP.” We believe providing these non-GAAP measures, which show our results with these items adjusted, is valuable and useful since they allow management and investors to better understand the company’s financial performance in the absence of certain non-cash items such as stock-based compensation and certain special events and allow investors to more accurately understand our current and past period results and more easily compare them to future results. These non-GAAP measures also correspond with the way we expect investors and financial analysts to compare our results. Our non-GAAP measures should be considered only as supplements to, and not as substitutes for or in isolation from, our other measures of financial information prepared in accordance with GAAP, such as GAAP revenue or operating income. Our references to the “non-GAAP” financial measure of preliminary, unaudited 2025 ORLADEYO revenue, excluding European ORLADEYO revenue for the full year 2025, constitutes a non-GAAP financial measure. It refers to our preliminary GAAP results, adjusted to show the results without including $38 million of European ORLADEYO revenue for the nine months ended September 30, 2025. Our measure of 36% y-o-y growth on a comparable basis for preliminary, unaudited ORLADEYO net revenue in the fourth quarter of 2025 was calculated using the non-GAAP financial measure of Q4 2024 ORLADEYO net revenue, adjusted to exclude $13 million of European ORLADEYO revenue for the three months ended December 31, 2024. Our measure of 43% y-o-y growth on a comparable basis for preliminary, unaudited ORLADEYO net revenue for the full year 2025 was calculated using the non-GAAP financial measure of full year 2024 ORLADEYO net revenue, adjusted to exclude $43 million of European ORLADEYO revenue for the twelve months ended December 31, 2024. Our reference to expected non-GAAP profitability excludes stock-based compensation, restructuring and transaction-related costs. We also provide our non-GAAP operating expense outlook for full year 2026, which refers to our expected GAAP operating expense, excluding stock-based compensation, restructuring and transaction-related costs. We have not provided a reconciliation against the comparable forward-looking GAAP measure because we are unable to predict with reasonable certainty the full amount of stock-based compensation expense or restructuring and transaction-related costs for the full year 2026 without unreasonable effort. Stock-based compensation expense is uncertain and depends on various factors, including our future hiring and retention needs, as well as the future fair market value of our common stock, which is difficult to predict and subject to change. In addition, we are unable to predict with reasonable certainty the full amount of restructuring and transaction-related costs as the closing of the proposed Astria acquisition is still pending and the related costs are dependent on various factors that have not yet occurred. The actual amount of stock-based compensation, restructuring and transaction-related costs for the full year 2026 could have a material impact on GAAP reported results for the guidance period. Forward-Looking Statements This press release contains forward-looking statements, including statements regarding preliminary, unaudited results and future results, performance or achievements, expectations regarding BioCryst’s growth and expenses, and statements related to BioCryst’s acquisition of Astria Therapeutics, Inc. (the “Merger”), including the expected benefits of the Merger, anticipated timing of the closing of the Merger, the anticipated financial impact of the Merger, BioCryst’s or the combined company’s performance following the Merger, including future financial and operating results, and anticipated approval and commercialization of navenibart. These statements involve known and unknown risks, uncertainties and other factors which may cause BioCryst’s actual results, performance, or achievements to be materially different from any preliminary, unaudited results and future results, performance, or achievements expressed or implied by the forward-looking statements. These statements reflect our current views with respect to future events and are based on assumptions, including assumptions related to the expected date of closing of the Merger and the potential benefits thereof, and are subject to risks and uncertainties. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Some of the factors that could affect the forward-looking statements contained herein include: BioCryst’s ability to successfully implement or maintain its commercialization plans for ORLADEYO; BioCryst’s ability to successfully progress its pipeline development plans, including meeting the expected timelines; the results of BioCryst’s partnerships with third parties may not meet BioCryst’s current expectations; risks related to government actions, including that decisions and other actions, including as they relate to pricing, may not be taken when expected or at all, or that the outcomes of such decisions and other actions may not be in line with BioCryst’s current expectations; the commercial viability of ORLADEYO, including its ability to achieve sustained market acceptance and demand; ongoing and future preclinical and clinical development of product candidates may take longer than expected and may not have positive results; the outcome of preclinical testing and early clinical trials may not be predictive of the success of later clinical trials, and interim results of a clinical trial do not necessarily predict final results; BioCryst may not be able to enroll the required number of subjects in planned clinical trials of product candidates; BioCryst may not advance human clinical trials with product candidates as expected; the FDA or other applicable regulatory agency may require additional studies beyond the studies planned for products and product candidates, may not provide regulatory clearances which may result in delay of planned clinical trials, may not review regulatory filings on our expected timeline, may impose certain restrictions, warnings, or other requirements on products and product candidates, may impose a clinical hold with respect to product candidates, or may withhold, delay or withdraw market approval for products and product candidates; product candidates, if approved, may not achieve market acceptance; BioCryst’s ability to successfully commercialize its products and product candidates; BioCryst’s ability to successfully manage its growth and compete effectively; timing for achieving or sustainability of profitability and positive cash flow may not meet management’s expectations; statements and projections regarding financial guidance and goals and the attainment of such goals may differ from actual results or may not be achieved on the expected timelines, or at all, based on market factors and BioCryst’s ability to execute its operational and budget plans; actual financial results may not be consistent with expectations, including that revenue, operating expenses and cash usage may not be within management’s expected ranges; the occurrence of any event, change or other circumstances that could give rise to the right of BioCryst or Astria to terminate the definitive agreement governing the Merger; the failure to obtain Astria stockholder approval or to satisfy any of the other conditions to the Merger on a timely basis or at all; the possibility that the anticipated benefits of the Merger, including anticipated synergies, are not realized when expected or at all, including as a result of the impact of, or problems arising from, the integration of the two companies or as a result of the strength of the economy and competitive factors in the areas where BioCryst and Astria do business; the significant indebtedness BioCryst expects to incur in connection with the Merger and the need to generate sufficient cash flows to service and repay such debt; the possibility that the Merger may be more expensive to complete than anticipated; diversion of management’s attention from ongoing business operations and opportunities; potential adverse reactions or changes to business or employee relationships, including those resulting from the completion of the Merger; and risks relating to the potential dilutive effect of shares of BioCryst common stock to be issued in the Merger. Please refer to the documents BioCryst files periodically with the Securities and Exchange Commission (the “SEC”), specifically BioCryst’s most recent Annual Report on Form 10-K, Quarterly Reports on Form 10-Q, and Current Reports on Form 8-K, which identify important factors that could cause actual results to differ materially from those contained in BioCryst’s projections and forward-looking statements. BCRXW Contact: Investors: investorrelations@biocryst.com Media: media@biocryst.com

CDE一周药闻 12月8 - 14日，CDE共承办34个1类新药申请，其中： 化药临床试验申请受理品种21个； 治疗用生物制品临床试验申请受理号13个； 15款1类创新药获CDE临床试验默示许可（IND）。 01 NEWS 博奥信生物 - BSI-082注射液 2025 年 12 月 8 日，中国国家药监局药品审评中心（CDE）官网显示，博奥信生物申报的1类新药BSI-082注射液获得CDE的临床试验默示许可，拟开发治疗晚期或转移性实体瘤。 公开资料显示，BSI-082是全人源抗SIRPα拮抗性单克隆抗体，其特异性结合SIRPα和SIRPβ，但不结合SIRPγ，能有效阻断SIRPα与CD47相互作用向巨噬细胞发出“别吃我”信号，从而使巨噬细胞和树突状细胞恢复对肿瘤细胞的吞噬作用，同时有效避免靶向CD47治疗中遇到的毒副作用难题。作为一款高度差异化的抗SIRPα单抗，BSI-082对huSIRPα蛋白变体V1/V2/V8都具有较强的结合活性，因此可以覆盖90%以上的人群。 02 NEWS 科兴药业 - GB10注射液 2025 年 12 月 8 日，中国国家药监局药品审评中心（CDE）官网显示，科兴药业申报的1类新药GB10注射液获得CDE的临床试验默示许可，拟开发治疗新生血管性年龄相关性黄斑变性。 根据科兴制药公告介绍，GB10注射液为抗VEGF/Ang-2双靶点抗体高浓度眼科专用蛋白药物制剂。该产品浓度达到140mg/mL，可减少注射体积或提高给药量，延长给药周期，预期实现每4个月给药一次，极大提高患者依从性。 03 NEWS 阿斯利康 - AZD0516 2025 年 12 月 8 日，中国国家药监局药品审评中心（CDE）官网显示，阿斯利康申报的1类新药AZD0516获得CDE的临床试验默示许可，拟开发治疗转移性前列腺癌。 公开资料显示，AZD0516是一款在研靶向STEAP2的ADC。STEAP2是一种新型肿瘤相关抗原，其质膜表达仅限于前列腺组织，但在前列腺腺癌的所有阶段均高度过表达，因此成为理想的抗癌靶点。AZD0516由抗STEAP2单克隆抗体通过链间半胱氨酸与马来酰亚胺反应性、β-葡萄糖醛酸酶可裂解连接子偶联，该连接子携带有拓扑异构酶1抑制剂（TOP1i）有效载荷exatecan。 04 NEWS Maruho - EVO756片 2025 年 12 月 8 日，中国国家药监局药品审评中心（CDE）官网显示，Maruho申报的1类新药EVO756片获得CDE的临床试验默示许可，拟开发治疗慢性自发性荨麻疹。 公开资料显示，EVO756是一种口服、强效、高选择性的MRGPRX2小分子拮抗剂。MRGPRX2是肥大细胞上表达的一种受体，可由包括感觉神经元和其他细胞类型释放的多种配体激活。通过阻断MRGPRX2和肥大细胞颗粒释放，EVO756具潜力治疗多种肥大细胞介导的疾病。 05 NEWS 诺和诺德（Novo Nordisk） -  NNC6019-0001注射液 2025 年 12 月 9 日，中国国家药监局药品审评中心（CDE）官网显示，诺和诺德申报的1类新药NNC6019-0001注射液（coramitug）获得CDE的临床试验默示许可，拟开发治疗转甲状腺素蛋白淀粉样变性心肌病（ATR-CM）。 公开资料显示，coramitug是一种人源化单克隆抗体，靶向错误折叠的转甲状腺素，旨在通过抗体介导的吞噬作用促进转甲状腺素淀粉样蛋白的清除。ATR-CM是一种由转甲状腺素淀粉样蛋白在心肌中沉积引起的进行性疾病。 06 NEWS 阿斯利康 - 注射用AZD0292 2025 年 12 月 10 日，中国国家药监局药品审评中心（CDE）官网显示，阿斯利康申报的1类新药注射用AZD0292获得CDE的临床试验默示许可，拟开发治疗支气管扩张症。 根据阿斯利康官网资料，这是一款假单胞菌（Pseudomonas）Psl-PcrV双特异性抗体，拟开发治疗非囊性纤维化支气管扩张症。铜绿假单胞菌是支气管扩张症患者呼吸道标本中分离出的常见病原体之一。许多研究揭示了铜绿假单胞菌感染会导致支气管扩张症患者的炎症反应增加、肺功能损害加重、病情加重、生活质量恶化和病死率增加。 07 NEWS Climb Bio -  budoprutug注射液 2025 年 12 月 10 日，中国国家药监局药品审评中心（CDE）官网显示，Climb Bio申报的1类新药budoprutug注射液获得CDE的临床试验默示许可，拟开发治疗系统性红斑狼疮。 公开资料显示，这是一种抗CD19单克隆抗体，具有较长的给药间隔和静脉（IV）和皮下（SC）给药的潜力，可导致有效和持久的B细胞消耗。 08 NEWS 悦康药业 - 注射用YKYY031 2025 年 12 月 12 日，中国国家药监局药品审评中心（CDE）官网显示，悦康药业1类新药注射用YKYY031获得CDE的临床试验默示许可，拟开发治疗晚期实体瘤。 根据悦康药业公告介绍，这是一种通用型mRNA肿瘤疫苗，采用新型阳离子脂质YK-009组成的脂质纳米颗粒（LNP）递送系统，该系统具备递送效率高、安全性良好的特点。注射用 YKYY031所含的mRNA序列编码由多个在实体瘤组织中高表达的肿瘤相关抗原（TAA）及特异性表达的肿瘤特异性抗原（TSA）表位肽串联组成的多肽抗原序列。该疫苗递送至体内后，可诱导机体产生针对这些抗原的特异性杀伤T细胞，抑制肿瘤细胞生长。 全球药闻 12月8 - 14日，FDA已批准13种新药及适应症，以下为其中部分获批的新药及适应症： 01 NEWS Telethon -  etuvetidigene autotemcel 2025 年 12 月 9 日，美国食品药品监督管理局（FDA）宣布批准批准由意大利非营利组织Telethon基金会主导研发的Waskyra（etuvetidigene autotemcel）上市，这是全球首款针对 Wiskott-Aldrich 综合征（WAS）的细胞基基因疗法。 etuvetidigene autotemcel 是一款自体细胞基基因疗法，其核心作用机制是提取患者自身的造血干细胞，通过基因修饰技术引入功能性的 WAS 基因，经减低强度预处理后将基因修正后的细胞静脉输注回患者体内，最终恢复受影响细胞中 WAS 蛋白的正常表达，从根源上解决 WAS 的致病问题。这种自体疗法规避了异基因造血干细胞移植的供体匹配限制与排异风险，专门适配无合适供体的 WAS 患者需求。 02 NEWS 安进（Amgen） -  inebilizumab-cdon 2025年12月11日，安进（Amgen）宣布美国食品药品监督管理局（FDA）批准UPLIZNA（inebilizumab-cdon）用于治疗抗乙酰胆碱受体（AChR）和抗肌肉特异性酪氨酸激酶（MuSK）抗体阳性的成人全身性重症肌无力（gMG）。 inebilizumab-cdon是一款人源化单克隆抗体，作用机制是靶向耗竭产生自身抗体的CD19阳性B细胞（包括浆母细胞和部分浆细胞），从根源上减少驱动gMG发病的AChR和MuSK自身抗体，进而改善神经肌肉传导功能，缓解肌肉无力症状。其给药方案极具优势：患者在接受两次初始负荷输注后，仅需每6个月给药一次，大幅降低了治疗负担，提升了长期治疗的依从性。这是全球首款且唯一一款针对该两类抗体阳性gMG的CD19靶向B细胞疗法，以“初始两剂后每年仅需两次给药”的便捷方案，为患者带来深度持久的症状控制，同时支持类固醇减量，填补了gMG精准治疗的重要空白。 03 NEWS BioCryst制药公司 - berotralstat 2025 年 12 月 12 日，BioCryst制药公司宣布美国食品药品监督管理局（FDA）批准berotralstat口服颗粒剂上市，用于2至<12岁遗传性血管性水肿（HAE）患儿的预防性治疗。 berotralstat是一款每日一次的口服血浆激肽释放酶抑制剂，通过降低血浆激肽释放酶的活性，从根源上预防HAE发作，其核心优势在于靶向机制明确、给药便捷且适用人群广泛。这是全球首款且唯一一款适用于2岁及以上HAE患者的靶向口服预防疗法，填补了低龄儿童HAE口服治疗的空白——此前该年龄段患者仅能依赖静脉或皮下注射疗法。 信息来源：国家药品监督管理局药品审评中心、美国食品药物监督管理局，以及相关公司官方网站等。 · 往期推荐   RECOMMEND 1 FDA历史性认证首款AI药物研发工具，将用于“MASH”临床试验 2 肺炎链球菌多糖及多糖结合疫苗免疫原性评价 | 斯丹姆分享 3 细胞产品的PD检测 免疫原性分析分享｜精彩回顾 点击此链接，阅读关于斯丹姆公众号的法律声明细节