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Apellis to Receive up to $300 Million from Royalty Purchase Agreement with Sobi for Ex-U.S. Royalties of Aspaveli® (systemic pegcetacoplan)
GlobeNewswire News Room· 2025-07-01 11:00
Core Viewpoint - Apellis Pharmaceuticals has entered into a capped royalty purchase agreement with Sobi, allowing Apellis to receive up to $300 million in exchange for 90% of its future ex-U.S. royalties for Aspaveli, which is a treatment for rare diseases [1][2][3] Transaction Details - Apellis will receive $275 million upfront and may earn up to $25 million in milestone payments upon EMA approval for Aspaveli in treating C3G and IC-MPGN [3][7] - The agreement includes defined caps tied to Aspaveli's performance, after which 100% of ex-U.S. royalties will revert to Apellis [4] Product Information - Aspaveli (systemic pegcetacoplan) is approved in the EU and the U.S. for treating paroxysmal nocturnal hemoglobinuria (PNH) and is under review for C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) [2][5] - Approximately 50% of patients with C3G and IC-MPGN may experience kidney failure within five to ten years of diagnosis, highlighting the urgency for effective treatments [6] Strategic Implications - The transaction is seen as a non-dilutive funding method that strengthens Apellis' balance sheet and provides operational flexibility as the company approaches sustainable profitability [2][7] - Sobi's expertise in rare diseases and its commercial footprint are expected to enhance the reach of Aspaveli globally following regulatory approvals [3]
翰宇药业上半年净利润同比大幅增长近1664% 国际化与创新药实现双轮驱动
翰宇药业(300199)6月30日晚间发布2025年半年度业绩预告显示,公司业绩延续了一季度强劲增长态 势。期内,该公司预计实现归属净利润1.42亿元至1.62亿元,较去年同期大幅增长1470.82%至 1663.89%。 目前,翰宇药业国际化业务已遍布全球核心市场,原料药销往20余个国家和地区,制剂通过对外授权签 约90余个国家和地区。 在创新药研发领域,翰宇药业以"多肽+小核酸"为技术核心,始终聚焦前沿,成果丰硕。公司开发的 HY3003项目,作为一款利用"硅基多肽芯片+AI算法技术"筛选的GLP-1R/GIPR/GCGR三靶点受体激动 剂,目前已完成临床前候选化合物筛选,推进原料药中试放大阶段。该项目采用多剂型并行的开发策 略,开发方向包括周制剂、超长效月制剂、口服制剂。其中,每月注射一次的超长效制剂依托公司自主 长效递送平台,降低给药频率,平稳血药浓度,以期减少胃肠道不良反应,从而提高用药依从性。 翰宇药业表示,除HY3003外,公司在多肽和小核酸制剂的未来管线储备丰富,接下来会陆续面向全球 市场推出不少于20种的制剂,包括原创新药、微创新药、ANDA等,丰富的管线储备为公司持续发展注 入强劲动力。包 ...
优质教育资源加速集聚南沙,打造跨域合作“湾区样本”
Nan Fang Du Shi Bao· 2025-06-25 07:06
日前,香港恒生大学(南沙)中心正式启用。作为香港恒生大学在内地设立的首个办公室,南沙中心的 启用不仅是恒生大学粤港澳大湾区的重要支点,也是构建粤港青年联系交流的实体枢纽。 《广州南沙深化面向世界的粤港澳全面合作总体方案》(简称《南沙方案》)发布3年来,广州市南沙 区不断加强教育领域的跨地区交流合作,推动教育对外合作开放有新阶段、新增量、新突破。 随着教育等优质公共资源的加速集聚,作为"湾区之心"的南沙,正在打造港澳青年安居乐业新家园,成 为越来越多港澳青年追求更广阔发展空间的内地第一站。 广州南沙民心港人子弟学校 港籍学生超80% 香港通识与内地国情教育双线并行 《南沙方案》要求,"规划建设外籍人员子女学校或国际化程度较高的中小学校"。 2022年9月,广州南沙民心港人子弟学校正式开学,成为南沙首家港人子弟学校,参照外籍人员子女学 校管理。2025年5月,民心港人子弟学校举办首届中学部毕业典礼,42位毕业生顺利完成学业。目前, 该校共有1100余名在校生,其中港籍学生超80%。 广州南沙民心港人子弟学校提供从小学到高中的完整教育体系,开设HKDSE、GCE(AS & A Levels)、IGCSE、IBDP等 ...
翰宇药业制剂矩阵:三靶点减肥创新药及近20种制剂面向全球
Core Insights - Hanyu Pharmaceutical (300199) has recently conducted investor research, discussing the progress of the HY3003 new drug project, its layout in the innovative drug field, and overseas market expansion [1][2] Group 1: HY3003 New Drug Project - The HY3003 project has completed preclinical candidate compound screening and raw material process development, officially entering the raw material pilot scale-up phase, which validates the producibility of the molecular structure for subsequent IND application and clinical transformation [1] - HY3003 is a GLP-1R/GIPR/GCGR receptor agonist developed using AI peptide chip technology, aimed at weight loss indications [1] - The project employs a multi-formulation parallel development strategy, including weekly, ultra-long-acting monthly, and oral formulations, with the ultra-long-acting formulation designed to reduce dosing frequency and improve medication adherence [1] Group 2: siRNA and Other Drug Developments - The company is also developing siRNA drugs such as Inclisiran and Zilebesiran for treating high cholesterol and hypertension, as well as Pegcetacoplan for treating geographic atrophy [1] Group 3: Overseas Market Expansion - Currently, the company's raw materials are sold to over 20 countries and regions, with formulations licensed in over 90 countries and regions [2] - The company has signed cooperation agreements for the semaglutide injection (weekly formulation) and oral tablets in markets including China, Brazil, Egypt, Mexico, Eurasian Union, GCC member countries, and North Africa [2] - The company has completed the enrollment of all participants in the Phase III clinical trial for semaglutide injection as of January this year, with plans to submit for approval in China by 2026, followed by the US and other global markets [2] Group 4: Future Pipeline - Hanyu Pharmaceutical has a rich pipeline of peptide and small nucleic acid formulations, planning to launch no less than 20 formulations over the next 10 years, including innovative drugs, micro-innovative drugs, and ANDA [2]
Apellis and Sobi Announce EMPAVELI® (pegcetacoplan) Showed Sustained Efficacy at One Year in Phase 3 Study for C3G and Primary IC-MPGN
Globenewswire· 2025-06-06 13:15
Core Insights - Apellis Pharmaceuticals and Sobi presented new data from the Phase 3 VALIANT study for EMPAVELI (pegcetacoplan) targeting C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) at the European Renal Association Congress [1][5] Study Results - EMPAVELI showed a statistically significant 68% reduction in proteinuria compared to placebo at Week 26, with effects sustained for one year [2][4] - Patients switching from placebo to EMPAVELI also experienced similar benefits in proteinuria reduction and kidney function stabilization [3] Efficacy and Safety - The study confirmed EMPAVELI's efficacy and safety profile across a diverse patient population, including adults and adolescents with both native and post-transplant kidney disease [4][7] - No new safety signals were reported, indicating favorable safety and tolerability consistent with its established profile [4][7] Clinical Significance - The results highlight the urgent need for effective treatments for patients with C3G and primary IC-MPGN, as approximately 50% of these patients may face kidney failure within five to ten years of diagnosis [6] - The VALIANT study is the largest trial conducted in these populations, involving 124 patients aged 12 and older [8] Future Prospects - Marketing applications for EMPAVELI are currently under review with the FDA and EMA, with an FDA decision expected this summer [4][7] - The collaboration between Apellis and Sobi aims to expedite the availability of EMPAVELI for patients suffering from these rare kidney diseases [10]
Guidewire(GWRE) - 2025 Q3 - Earnings Call Transcript
2025-06-03 22:02
Financial Data and Key Metrics Changes - The company reported a total revenue of $294 million, reflecting a 22% year-over-year growth and exceeding the high end of the outlook [24] - Subscription and support revenue reached $182 million, showing a 32% year-over-year growth [24] - The annual recurring revenue (ARR) finished at $960 million, allowing the company to raise its full-year outlook to exceed $1 billion [10][22] Business Line Data and Key Metrics Changes - The company closed 17 cloud deals in Q3, including 14 for InsuranceSuite applications and 3 for InsuranceNow, indicating strong sales activity [9] - The company secured its first Guidewire Industry Intelligence sale, which is expected to be a significant revenue source [13] - Services revenue finished at $54 million, benefiting from strong bookings and higher utilization rates [24] Market Data and Key Metrics Changes - The company experienced significant international momentum, with cloud expansions in Canada and wins in APAC and EMEA [11] - The company noted strong demand for its cloud offerings, particularly in the high-end market with Tier one and Tier two insurers [9][23] Company Strategy and Development Direction - The company is focused on driving growth through its Guidewire Cloud Platform, which is seen as critical for insurers to modernize their operations [8] - The company is committed to the Japanese market, planning a $60 million investment over the next five years to enhance local operations [17][40] - The acquisition of Quanti is expected to enhance the company's pricing and rating technology, providing more agility to insurance companies [20][92] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in maintaining upper teens growth for fully ramped ARR, emphasizing the importance of Q4 for closing large deals [36] - The company highlighted the agility it provides to insurers, which is crucial for addressing the widening coverage gap in the industry [60] - Management noted that the company is well-positioned to continue its cloud transformation and support innovation in the insurance industry [21] Other Important Information - The company ended the quarter with over $1.2 billion in cash and cash equivalents, with operating cash flow at $32 million [28] - The company raised its ARR outlook for fiscal year 2025 to between $1.012 billion and $1.022 billion, reflecting a growth of 17% to 18% year-over-year [29] Q&A Session Summary Question: What is the fully ramped ARR outlook for this year? - Management indicated strong momentum and confidence in maintaining upper teens growth for fully ramped ARR, with Q4 being critical for large commitments [35][36] Question: What is the secret to winning cloud deals in the Japanese market? - Management emphasized commitment to the Japanese market and the importance of delivering successful outcomes for customers [40][41] Question: Was there any pull forward from Q4 due to Q3 outperformance? - Management clarified that there was no significant pull forward, attributing Q3 success to strong execution across all tiers [48][52] Question: How is the company leveraging AI in its operations? - Management discussed broad applications of generative AI across the product suite, particularly in claims workflows and developer productivity [75] Question: What is the incremental functionality from the Quanti acquisition? - The acquisition is expected to enhance pricing and rating technology applicable to all customers, providing more flexibility and agility [92]
RingCentral (RNG) 2025 Conference Transcript
2025-06-03 22:00
Summary of RingCentral (RNG) 2025 Conference Call Company Overview - RingCentral is a leader in cloud-based business communications, with a revenue of half a billion dollars and over half a billion dollars in annual free cash flow [6][14][22] - The company has transitioned into a multiproduct portfolio, adding AI-driven products and a contact center solution called Ring CX for the SMB market [8][10] Core Industry Insights - The enterprise communications market is estimated to be around $100 billion, with RingCentral holding a 20% share in cloud telephony [12][14] - The contact center market is smaller but growing faster, especially with the integration of AI technologies [16][20] - Voice remains the primary means of communication for consumers, presenting significant opportunities for RingCentral [19] Product Development and AI Integration - RingCentral has introduced several AI-led products, including RingCentral Air (AI receptionist) and AI assistants for calls, enhancing customer interactions [9][10][22] - The company aims to achieve a run rate of $100 million in annual recurring revenue (ARR) from its new AI products by the end of the year [22] Competitive Landscape - RingCentral is positioned as a disruptor in the contact center industry, leveraging its PBX capabilities to differentiate from competitors like Zoom and NICE [24][60] - The company maintains a strong market share, with competitors like Zoom and Microsoft having smaller bases in terms of paying seats [56][58] Partnerships and Market Strategy - RingCentral has over 16,000 channel partners, with significant relationships with global service providers like AT&T, which is refocusing on the SMB market [42][44] - The company sees small businesses as a critical market segment, representing over 40% of the economy [44] Financial Health and Future Outlook - RingCentral plans to reduce gross debt to under $1 billion by the end of next year while investing heavily in R&D [69][70] - The company is considering various options for returning capital to shareholders, including share buybacks and potential dividends [75][77] Key Takeaways - RingCentral is well-positioned to capitalize on the growing demand for AI-driven communication solutions in the SMB market [30][32] - The company is focused on maintaining its market share while expanding its product offerings to address evolving customer needs [29][60] - Future growth will be supported by strategic partnerships and a commitment to innovation in the cloud communications space [46][50]
世界首例:极限操作6个月,他们成功编辑了婴儿突变基因,治疗其罕见遗传病
生物世界· 2025-05-19 03:52
撰文丨王聪 编辑丨王多鱼 排版丨水成文 近日,美国 费城儿童医院和宾夕法尼亚大学医学院的研究团队取得了 一项具有里程碑意义的医学突破 —— 人类首次实现为单个病人定制基因编辑疗法 ,并成功治疗了一名 患有罕见致命遗传疾病的婴儿。 该研究以: Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease 为题,于 2025 年 5 月 15 日发表在了《 新英格兰医学杂志 》 (NEJM) ,详细介绍了这款定制的 体内碱基编辑疗法 的开 发和治疗过程。 这一具有里程碑意义的成功案例可能为基因编辑技术开辟一条成功应用于治疗那些目前尚无医疗手段可用 的罕见病患者的新途径。 婴儿 KJ Muldoon 在出生几天后就被诊断出患有严重的 氨甲酰磷酸合成酶-1 (CPS1) 缺乏症 ,这是一 种罕见且严重的隐性遗传疾病 (在新生儿中发病率为130万分之一) ,是最严重的 尿素循环障碍 疾病。 人体在分解蛋白质时会产生 氨 ,正常情况下,我们的身体会将氨转化为 尿素 ,然后通过排尿将尿素排出 体外。 CPS1 作为一种线粒体酶, 催化尿素循环的第 ...
里程碑突破:首款个性化碱基编辑疗法,成功治疗罕见遗传病,整个开发过程仅6个月
生物世界· 2025-05-16 07:57
该研究以: Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease 为题,于 2025 年 5 月 15 日发表在了《 新英格兰医学杂志 》 (NEJM) ,详细介绍了这款定制的 体内碱基编辑疗法 的开 发和治疗过程。 这一具有里程碑意义的成功案例可能为基因编辑技术开辟一条成功应用于治疗那些目前尚无医疗手段可用 的罕见病患者的新途径。 撰文丨王聪 编辑丨王多鱼 排版丨水成文 近日, 费城儿童医院和宾夕法尼亚大学医学院的研究团队取得了一项具有里程碑意义的医学突破—— 人类 首次实现为单个病人定制基因编辑疗法 ,并成功治疗了一名 患有罕见致命遗传疾病的儿童。 婴儿 KJ 在出生几天后就被诊断出患有严重的 氨甲酰磷酸合成酶-1 (CPS1) 缺乏症 ,这是一种罕见且 严重的遗传疾病 (在新生儿中发病率为130万分之一) ,是最严重的 尿素循环障碍 疾病。 KJ 与为他开发疗法的研究人员在一起 人体在分解蛋白质时会产生 氨 ,正常情况下,我们的身体会将氨转化为 尿素 ,然后通过排尿将尿素排出 体外。然而,患有尿素循环障碍的儿童肝脏中缺 ...
RingCentral(RNG) - 2025 Q1 - Earnings Call Transcript
2025-05-08 22:02
RingCentral (RNG) Q1 2025 Earnings Call May 08, 2025 05:00 PM ET Company Participants Vaibhav Agarwal - Chief Transformation Officer and Deputy CFOVlad Shmunis - Founder, Chairman and Chief Executive OfficerKira Makagon - President & Chief Operating OfficerAbhey Lamba - Chief Financial OfficerBilly Fitzsimmons - VP - Equity ResearchPeter Levine - Managing DirectorJessica Wang - Equity Research Associate Conference Call Participants Meta Marshall - AnalystKash Rangan - AnalystCatharine Trebnick - Senior Rese ...