1 J.P. MORGAN HEALTHCARE CONFERENCE JON STONEHOUSE PRESIDENT AND CHIEF EXECUTIVE OFFICER JANUARY 2023 ForwardLooking Statements BioCryst's presentation may contain forward-looking statements, including statements regarding future results, unaudited and forward-looking financial information and company performance or achievements. These statements are subject to known and unknown risks and uncertainties which may cause our actual results, performance or achievements to be materially different from any future ...

公司产品和业务发展 - 公司专注于口服药物，针对补体系统的经典、凝集素和末端途径的其他靶点进行研发[35] - 公司通过结构引导的药物设计过程，从与特定疾病相关的酶靶点的详细结构知识中设计合成化合物[31] - 公司的产品包括ORLADEYO® (berotralstat)、BCX10013、RAPIVAB® (peramivir injection)、RAPIACTA® (peramivir injection)和PERAMIFLU® (peramivir injection)[31][32][33][34] - 公司的业务策略是专注于治疗罕见病的口服药物研发，并在获得监管批准后在美国和其他地区高效推广这些药物[37] - 通过结构引导药物设计技术，公司致力于高附加值的药物研发，设计和合成多种抑制同一酶靶点的产品候选物[37] - 公司的商业策略之一是全球推广罕见病产品，已在美国和其他全球市场建立商业团队，继续扩展结构和专业知识以在其他市场高效推广产品[40] - RAPIVAB（peramivir注射液）是在美国卫生和公众服务部（BARDA/HHS）的生物医学先进研究和发展局的2.348亿美元合同下开发的[50] - RAPIVAB在日本以商业名称RAPIACTA上市，用于治疗季节性流感和高风险并发症患者[50] - 2022年和2021年，公司分别交付了20,000和9,980剂RAPIVAB，分别实现了1390万美元和690万美元的收入[51] - Galidesivir是一种广谱抗病毒药物，对包括黄热病、埃博拉病毒、寨卡病毒在内的20多种RNA病毒具有活性[52] - 公司与Torii Pharmaceutical Co., Ltd.签订了商业化和许可协议，授予Torii在日本预防HAE发作的独家权利[53] - 公司与Neopharm Ltd.和NewBridge Pharmaceuticals签订了供应和分销协议，支持ORLADEYO在以色列和阿联酋的商业化工作[55] - 公司与Shionogi & Co., Ltd.签订了许可、开发和商业化协议，授予Shionogi在日本开发和商业化peramivir的独家权利[56] - 公司与Green Cross Corporation签订了协议，在韩国开发和商业化peramivir[58] - 公司与美国政府签订了多项合同，包括向HHS供应RAPIVAB的采购合同和与NIAID/HHS和BARDA/HHS合作开发galidesivir的合同[59] - 公司的成功部分取决于其获得和执行产品、方法、流程和其他专有技术的专利保护，保留商业秘密，并在不侵犯其他方的专有权利的情况下运作[60] - 公司拥有大约31项美国专利，涵盖了kallikrein抑制剂化合物、神经氨酸酶抑制剂化合物、广谱抗病毒化合物等[61] - 公司持有约25项专利合作条约或美国专利申请，涉及kallikrein抑制剂化合物、神经氨酸酶抑制剂化合物等[62] 竞争环境和市场前景 - 公司面临激烈的竞争，包括拥有更多资源和经验的公司，以及学术机构和研究组织[63] - HAE是一种常染色体显性疾病，预计每3.3万至6.7万人中就有1例，可导致40%的死亡率[64] - 公司开发的HAE治疗药物包括Kalbitor、Takhzyro、Firazyr等[65][66][67] - 公司了解到多种HAE治疗药物正在临床开发中，可能与ORLADEYO竞争[68] - 多种罕见疾病由补体系统缺陷引起，包括PNH、aHUS等，Soliris、Ultomiris、Empaveli等是已批准的治疗药物[68] - 多种基于补体途径的产品正在开发中，包括C3抑制剂、C5抑制剂等[69] - 补体系统缺陷引起的疾病，如IgAN，可能会改变治疗格局和未来竞争环境[70] - 公司面临激烈的抗流感药物市场竞争，包括TAMIFLU、RELENZA、XOFLUZA等[71] 法律风险和监管要求 - 公司需遵守各国的医疗欺诈和滥用法律，包括反回扣和虚假索赔法[91] 市场不确定性和法律诉讼 - 公司的财务报表电话会议中提到，特定诉讼必须在特定法院进行，可能限制股东在与公司或董事、高管或员工的争议中获得有利的司法论坛[186] - 公司的财务报表电话会议中指出，不稳定的市场和经济条件可能对公司的业务、运营、临床开发或商业化计划和时间表以及资本获取产生不利影响[190] - 公司可能会面临法律诉讼，可能损害公司声誉或导致其他损失或意外支出时间和资源[191]

Fourth Quarter 2022 Results Call Corporate Update & Financial Results February 21, 2023 Forward-Looking Statements BioCryst's presentation may contain forward-looking statements, including statements regarding future results, unaudited and forward-looking financial information and company performance or achievements. These statements are subject to known and unknown risks and uncertainties which may cause our actual results, performance or achievements to be materially different from any future results or p ...

BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX) Q4 2022 Earnings Conference Call February 21, 2023 8:30 AM ET Company Participants John Bluth - Investor Relations Jon Stonehouse - Chief Executive Officer Anthony Doyle - Chief Financial Officer Charlie Gayer - Chief Commercial Officer Helen Thackray - Chief R&D Officer Conference Call Participants Jessica Fye - JPMorgan Tazeen Ahmad - Bank of America Chris Raymond - Piper Sandler Liisa Bayko - Evercore ISI Jon Wolleben - JMP Securities Ken Cacciatore - Cowen & ...

Table of Contents Title of each class Trading Symbol(s) Name of each exchange on which registered Common Stock BCRX Nasdaq Global Select Market UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q x QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended September 30, 2022 or o TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from _____________________ ...

BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX) Q3 2022 Earnings Conference Call November 1, 2022 8:30 AM ET Company Participants John Bluth - SVP, IR and Corporate Communications Jon Stonehouse - Chief Executive Officer Anthony Doyle - Chief Financial Officer Charlie Gayer - Chief Commercial Officer Helen Thackray - Chief R&D Officer Conference Call Participants Ken Cacciatore - Cowen and Company Chris Raymond - Piper Sandler Daniel Wolle - JPMorgan Joe Spak - RBC Capital Jon Wolleben - JMP Securities Gena Wa ...

Table of Contents Title of each class Trading Symbol(s) Name of each exchange on which registered Common Stock BCRX Nasdaq Global Select Market UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q x QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2022 or o TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from ______________________ to ...

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