Amicus Therapeutics (FOLD) Q1 2025 Earnings Call May 01, 2025 08:30 AM ET Company Participants Andrew Faughnan - Vice President, Investor RelationsBradley Campbell - CEO, President & DirectorSébastien Martel - Chief Business OfficerJeffrey Castelli - Chief Development OfficerSimon Harford - CFOJoshua Fleishman - Biotech Equity Research Associate Conference Call Participants Anupam Rama - AnalystNone - AnalystDennis Ding - Vice President - Equity Research AnalystRick Miller - AnalystGil Blum - Senior Analyst ...

Financial Data and Key Metrics Changes - The company increased its 2025 financial guidance across all metrics, raising revenue expectations by more than 20% due to strong Q1 results and successful licensing transactions [29][34] - Revenue for Q1 2025 was $132 million, a 10% year-over-year increase, with over half coming from commercial products, which grew 28% compared to the same period last year [29][30] - Non-GAAP operating loss guidance improved by nearly 25% to less than $375 million, with an expected cash balance of $1.9 billion by year-end [37][38] Business Line Data and Key Metrics Changes - The first independent commercial launch, TrINGOLZA, generated over $6 million in product sales in its first full quarter, exceeding expectations [6][30] - SPINRAZA royalties contributed $48 million, reflecting a 25% year-over-year increase, while Waynua royalties were $9 million, with expectations for growth driven by strong U.S. demand [30][31] - The company anticipates additional product revenue from the upcoming launch of Donadolorsen for Hereditary Angioedema (HAE) [36][59] Market Data and Key Metrics Changes - The company is focused on expanding its market presence, particularly in the rare disease space, with significant unmet needs in conditions like familial chylomicronemia syndrome (FCS) and severe hypertriglyceridemia (SHTG) [6][18] - The estimated patient population for FCS in the U.S. is around 3,000, while SHTG has a much larger addressable market of over 1 million patients [70][72] Company Strategy and Development Direction - The company is executing on strategic priorities, including multiple independent launches and partnerships to enhance its product pipeline and revenue potential [5][8] - The focus remains on disease education and patient identification to maximize the potential of TrINGOLZA and prepare for the launch of Donadolorsen [35][96] - The company is well-positioned to leverage its first-mover advantage in both FCS and SHTG markets, aiming for substantial revenue growth and positive cash flow [22][39] Management's Comments on Operating Environment and Future Outlook - Management acknowledged the evolving regulatory environment and potential disruptions due to new tariff policies but expressed confidence in the company's strategic execution [4][5] - The company is optimistic about achieving sustained revenue growth, with late-stage programs expected to deliver significant peak sales potential in the multibillion-dollar range [38][41] - Management emphasized the importance of ongoing patient identification efforts and payer engagement to ensure access to treatments [15][16] Other Important Information - The company completed licensing transactions for sapaglutzin and ex-U.S. commercialization rights for olazarcen, enhancing its financial outlook [33][34] - The company is actively engaging with payers to establish broad access for TrINGOLZA and Donadolorsen, with favorable coverage dynamics reported [12][13] Q&A Session Summary Question: Overlap between physician prescribers for FCS and SHTG - Management indicated significant overlap among cardiologists and endocrinologists treating both FCS and SHTG patients, which could facilitate the transition to SHTG treatment [48][49] Question: Preparation for Donadolorsen launch - The company is actively preparing for the launch of Donadolorsen, focusing on patient advocacy and physician education to facilitate transitions from existing therapies [59][60] Question: Impact of tariffs and FDA interactions - Management reported no significant impact from tariffs or changes in FDA interactions, with all programs on track [61][63] Question: Confidence in FCS patient estimates - The company expressed confidence in the estimated 3,000 FCS patients and is focused on increasing awareness and identification of these patients [68][70] Question: Channel stocking for TrINGOLZA - Management confirmed that TrINGOLZA sales are demand-driven, with no significant channel stocking observed [75][76] Question: Manufacturing footprint and tariff exposure - The company has a robust manufacturing supply chain in place and has not experienced any meaningful impacts from tariffs to date [78][79]

公司代码：688710 公司简称：益诺思 第一节 重要提示 登录新浪财经APP 搜索【信披】查看更多考评等级 1、本年度报告摘要来自年度报告全文，为全面了解本公司的经营成果、财务状况及未来发展规划，投 资者应当到http://www.sse.com.cn/网站仔细阅读年度报告全文。 2、重大风险提示 本公司已在本年度报告中详细阐述在生产经营过程中可能面临的相关风险，详情请查阅本报告第三节、 四、"风险因素"部分的相关内容。 3、本公司董事会、监事会及董事、监事、高级管理人员保证年度报告内容的真实性、准确性、完整 性，不存在虚假记载、误导性陈述或重大遗漏，并承担个别和连带的法律责任。 4、公司全体董事出席董事会会议。 5、立信会计师事务所（特殊普通合伙）为本公司出具了标准无保留意见的审计报告。 6、公司上市时未盈利且尚未实现盈利 □是 √否 7、董事会决议通过的本报告期利润分配预案或公积金转增股本预案 经立信会计师事务所（特殊普通合伙）审计，截至2024年12月31日，2024年度实现归属于母公司所有者 的净利润为人民币147,775,836.46元，其中母公司实现净利润人民币60,227,336.70元，母公 ...

证券代码：300149 证券简称：睿智医药 睿智医药科技股份有限公司投资者关系活动记录表 编号：2025-01 | | □特定对象调研 □分析师会议 | | --- | --- | | 投资者关系活动 | □媒体采访 ■业绩说明会 | | 类别 | □路演活动 □新闻发布会 | | | □现场参观 □其他（机构交流活动） | | 参与单位名称及 | 东方证券、国泰海通、兴业证券、华安证券、中信证券、开源证券、中邮证 | | 人员姓名 | 券、华福证券、国盛证券 | | 时间 | 2025 年 04 月 29 日 20:00-21:00 | | 地点 | 电话会议 | | 上市公司接待人 | 睿智医药董事长、首席执行官（CEO）：WOO SWEE LIAN | | | 上海睿智联席总裁：马兴泉 | | 员姓名 | 睿智医药首席财务官（CFO）：查胤群 | | | 睿智医药董事会秘书：许剑 | | | 睿智医药投资者关系负责人：朱子白 | | | 一、睿智医药董事长、首席执行官（CEO）WOO SWEE LIAN 先生介绍公司 | | 投资者关系活动 | 年度及 年第一季度经营情况 2024 2025 1、经营概 ...

Group 1: Drug Development and Clinical Trials - Pinegabine is being developed using a stepwise approach, first assessing its efficacy in combination therapy before evaluating its effectiveness as a monotherapy [2] - The company is currently conducting Phase IIa clinical trials for Pinegabine, with low-dose cohort enrollment completed and mid-dose cohort enrollment ongoing [2] - Pinegabine is positioned as a me-better drug compared to Retigabine, which was withdrawn from the market due to safety concerns [3] Group 2: Market Analysis and Product Characteristics - Epilepsy is the second most common neurological disorder after stroke, with a significant portion of patients experiencing treatment-resistant epilepsy [4] - There is a lack of innovative antiepileptic drugs in recent years, particularly for special types of epilepsy and acute seizures [4] - Pinegabine demonstrates superior chemical stability and higher brain distribution compared to Retigabine, enhancing its efficacy and safety profile [4] Group 3: Patent and Market Strategy - The patent for Pinegabine is held by the Shanghai Institute of Materia Medica, with exclusive rights for clinical development and commercialization in mainland China [5] - The company plans to explore international rights transfer and partnerships based on the outcomes of Phase II clinical trials [5] Group 4: Future Prospects and Expansion - Pinegabine may expand its indications based on Phase II clinical results and market needs, potentially targeting conditions like severe depression due to KCNQ mutations [4] - Fluorofenone, another drug in development, is expected to enter Phase III clinical trials in the second half of 2025, having been included in the breakthrough therapy list by the National Medical Products Administration [6] - The company aims to focus on anti-fibrosis indications initially, with potential future expansions depending on clinical trial outcomes [6]

主要股东信息显示，苏州瑞博生物技术股份有限公司由LIANG ZICAI（梁子才）持股12.9679%、昆山 瑞控企业管理咨询合伙企业（有限合伙）持股9.7862%、先进制造产业投资基金（有限合伙）持股 9.129%、Ionis Pharmaceuticals, Inc.持股6.8907%、智魄有限公司持股6.6461%。 来源：金融界 苏州瑞博生物技术股份有限公司，成立于2007年，位于苏州市，是一家以从事医药制造业为主的企业。 企业注册资本13014.5045万人民币，实缴资本12838.5641万人民币。 通过天眼查大数据分析，苏州瑞博生物技术股份有限公司共对外投资了5家企业，参与招投标项目6次， 知识产权方面有商标信息59条，专利信息119条，此外企业还拥有行政许可12个。 金融界4月30日消息，据CDE官网沟通交流公示，于4月30日收到苏州瑞博生物技术股份有限公司申请 的"II类会议"，当前状态"处理中"。 根据《药物研发与技术审评沟通交流管理办法》（2020年第48号通告），沟通交流会议分为Ⅰ类、Ⅱ类 和Ⅲ类会议，就关键阶段重大问题进行沟通交流。Ⅱ类会议一般安排在申请后60日内召开，系指为药物 在研 ...

场需求，后续将根据市场需求变化提前布局，扩充产能，以充分满足市场 需求。 • 公司构建线上线下全渠道销售网络，覆盖民营连锁机构、公立医院、电商 及互联网医疗平台。截至 2024 年第四季度，推出每盒 40 支的新包装规 格，已与超过 200 家连锁药店达成合作，铺货超过 12,000 家。2025 年 目标是超过 2,000 万人知晓该治疗方案，并且有超过百万级数量的儿童能 够及时接受规范、合理用药。 • 公司在 2024 年 7 月申报的 SQ22,031 滴眼液获得临床试验批准通知书， 适应症为干眼症和神经营养性角膜炎。0.02%和 0.04%浓度硫酸阿托品滴 眼液的三期临床试验结果显示，在主要疗效指标上均优于安慰剂组，并且 安全性良好，患者依从性高，目前正在药品审评中心审评过程中。 • 公司将继续围绕战略目标深化管理，管控成本，优化费用结构，以提升运 营效率和盈利水平。公司预计在销售规模增长的前提下，毛利率水平将保 持相对稳定。展望 2025 年，公司将继续围绕战略目标深化管理，管控成 本，优化费用结构，以提升运营效率和盈利水平，期待在 2025 年保持两 位数以上的增长。 Q&A 请介绍一下兴齐眼药 ...

Core Viewpoint - In 2024, Beijing Kain Technology Co., Ltd. experienced a decline in revenue but an increase in net profit, indicating a shift in operational dynamics and potential risks [1]. Revenue Decline and Market Challenges - The company's revenue for 2024 was 1,230,296,984.43 yuan, a decrease of 12.87% from 2023's 1,412,006,182.85 yuan [2]. - The decline was primarily due to the implementation of centralized procurement policies for key products, leading to price reductions and transitional impacts on revenue [3]. Net Profit Growth and Cost Efficiency - The net profit attributable to shareholders was 142,369,228.09 yuan, up 22.18% from 116,524,994.34 yuan in 2023 [4]. - This growth was driven by the execution of centralized procurement policies and a reduction in overall sales costs, with operating costs decreasing by 4.01% [5]. Non-Recurring Profit Growth and Core Profitability - The non-recurring net profit was 136,983,136.25 yuan, reflecting an 11.75% increase from 122,577,218.12 yuan in 2023 [6]. - The growth was lower than the overall net profit increase due to higher non-recurring gains, but it still indicated enhanced core profitability [7]. Earnings Per Share and Shareholder Returns - Basic earnings per share rose to 0.85 yuan, a 21.43% increase from 0.70 yuan in 2023 [8]. - This increase was attributed to the growth in net profit, indicating improved returns for shareholders [9]. Non-Recurring Earnings Per Share Growth - The non-recurring earnings per share was 0.82 yuan, up 12.33% from 0.73 yuan in 2023 [11]. - This growth suggests an improvement in the quality of earnings after excluding non-recurring items [12]. Expense Changes and Structural Adjustments - Sales expenses decreased by 24.70% to 597,482,346.66 yuan from 793,421,431.11 yuan in 2023, attributed to cost reduction strategies [13]. - Management expenses increased by 11.64% to 114,875,541.78 yuan, possibly due to business adjustments [13]. - Financial expenses were negative, indicating that income from interest exceeded expenses, although the absolute value decreased [13]. Research and Development Investment - R&D expenses totaled 138,157,828.57 yuan, a 2.94% increase from 134,215,207.89 yuan in 2023, representing 12.79% of revenue [14][15]. - The number of R&D personnel decreased to 126, but the commitment to R&D investment remained strong, reflecting a focus on innovation [16][17]. Cash Flow Variability - Net cash flow from operating activities was 104,285,937.46 yuan, down 17.63% from 126,603,431.56 yuan in 2023, influenced by changes in payment collection [18]. - Cash flow from investing activities was -176,916,706.00 yuan, indicating increased outflows for capital projects [18]. - Cash flow from financing activities surged by 848.19% to 84,544,154.88 yuan, primarily due to increased bank borrowings for project funding [19]. Executive Compensation - The chairman received a pre-tax compensation of 4.232 million yuan, while the general manager received 2.015 million yuan [23][24]. - High executive compensation reflects the company's commitment to attracting and retaining talent while holding leadership accountable for performance [27].

Core Insights - Shanghai Haoyuan Pharmaceutical Co., Ltd. reported a revenue of 606 million yuan for Q1 2025, representing a year-on-year growth of 20.05%, and a net profit attributable to shareholders of 62.38 million yuan, up 272.28% [1] - The total assets of the company reached 5.714 billion yuan by the end of Q1 [1] - The global trade environment is undergoing significant changes, with high-frequency repurchase characteristics of research reagents likely accelerating domestic substitution [1] Business Performance - The front-end life science reagent business of the company showed strong growth, with its revenue share significantly increasing compared to the same period last year [2] - The backlog of orders in the back-end business increased by over 30% year-on-year, indicating a notable rise in customer demand [2] Innovation and R&D - The company is actively exploring the application of AI technology in new drug development, aiming to establish an "AI + Biomedicine" innovation hub [2] - A partnership has been formed with East China Normal University to create an "AI Drug Exploration Joint Laboratory," focusing on ADC drug database construction and AI drug intelligent manufacturing [2] - The initiative aims to enhance the company's technological barriers in the entire drug development process and accelerate the transition of research results from the laboratory to production [2]

Core Insights - Recently, Yuyuan Pharmaceutical achieved a breakthrough in new drug development with its mRNA vaccine YKYY025 for respiratory syncytial virus (RSV) receiving clinical trial approval from the National Medical Products Administration (NMPA) in China, following prior approval from the FDA in the United States, showcasing the company's strong innovation capabilities and international competitiveness [1][3] Group 1: Innovation in mRNA Vaccine Development - Yuyuan Pharmaceutical's subsidiary, Hangzhou Tianlong Pharmaceutical, has successfully submitted clinical trial applications for multiple mRNA vaccines, including YKYY026 for shingles, which has also been accepted by the NMPA [3] - The company has received clinical approvals for its broad-spectrum anti-coronavirus peptide YKYY017 aerosol inhalation and long-acting lipid-lowering nucleic acid drug YKYY015 in both China and the U.S. [3] - Yuyuan's proprietary nucleic acid delivery system has been granted patents in multiple countries, including the U.S., Japan, and Israel, establishing a solid foundation for its mRNA vaccine and nucleic acid drug development [3] Group 2: Comprehensive R&D Platform - The company has built a complete platform system for nucleic acid drug development, covering target discovery, high-throughput screening, process development, and quality control, which is rare among domestic pharmaceutical companies [4] - Yuyuan Pharmaceutical has multiple innovative products nearing commercialization, with three class 1 innovative drugs receiving acceptance for market application, and its peptide drug YKYY017 entering phase III clinical trials [4] - The anti-liver cancer antisense oligonucleotide drug CT102 has completed phase IIa clinical trials, indicating a strong pipeline that could provide new treatment options for patients and drive future growth for the company [4]