Amyndas Pharmaceuticals LLC

关键词C3；Pegcetacoplan；研究进展补体系统是连接先天性和适应性免疫的桥梁，激活后参与机体的防御反应及免疫调节，包括增强抗体反应并具有免疫记忆，裂解外来细胞，清除免疫复合物和凋亡细胞等。但补体系统一旦失衡或过度激活会导致多种疾病，如眼部疾病、牙周疾病等急性炎症，自身免疫系统疾病、肿瘤、肾脏疾病、慢性溶血性疾病以及神经退行性疾病等。补体C3是血清中含量最高的补体成分，主要由肝细胞和巨噬细胞合成，通过活化和裂解参与补体经典激活途和旁路激活途径，从而介导补体级联反应。目前全球针对C3靶点仅批准一款药物，即Pegcetacoplan。首款C3靶向药前途不明Pegcetacoplan是一种由13个氨基酸组成的环肽类药物，能够抑制C3转化酶的形成，进而在源头抑制补体通路。2020年10月，Sobi与Apellis就Pegcetacoplan达成合作。据协议，Sobi获得该药美国以外市场的商业权益，同时将向Apellis支付对价为2.5亿美元预付款+9.15亿美元里程碑金额+8000万美元研发费用+销售额两位数的特许权使用费。2021年5月，Pegcetacoplan被FDA批准用于治疗成人阵发性夜间血红蛋白尿(PNH)，用于治疗初治阵发性睡眠性血红蛋白尿症（PNH）成人患者，以及先前接受C5抑制剂治疗的PNH患者，商品名为Empaveli。2021年12月，该药获欧盟批准，用于治疗接受C5抑制剂至少3个月后贫血的PNH成人患者，商品名为Aspaveli。2023年2月，该药又被FDA批准用于治疗继发性干性年龄相关黄斑变性（AMD）及黄斑地理萎缩（GA）。Pegcetacoplan是FDA批准的首个用于治疗PNH的C3靶向疗法，同时也是全球首款获批治疗黄斑地图状萎缩的药物。值得一提的是，该药成功入围2023年初市场调研机构Evaluate Vantage预测的十大重磅新药名单。然而，目前Pegcetacoplan的前景并不明朗，今年7月美国视网膜专家协会称Syfovre具有罕见但严重的副作用。而且，受此消息影响，Apellis股价自7月14日起连跌五日，几近腰斩。此外，Pegcetacoplan新适应症拓展也不顺利。今年5月，Apellis/Sobi宣布将停止Pegcetacoplan针对肌萎缩侧索硬化症（ALS）适应症的开发，原因是Pegcetacoplan在一项名为MERIDIAN的Ⅱ临床研究中未能达到其主要终点及关键次要终点。在研C3靶向药研究进展除了Pegcetacoplan，目前全球还有多款在研C3靶向药，详见下表。据药渡数据库，在研C3靶向药大多处于临床试验阶段，而且其药物类型非常多元化，涉及多肽（如ZP 10068）、融合蛋白、siRNA（如ARO-C3）、双抗（如LP-005）和化药（如APL-1030）等。全球部分在研C3靶向药资料来源：公开资料具体品种上，ARO-C3、Compstatin 40等处于2期临床试验阶段。ARO-C3ARO-C3是一款RNAi疗法，通过与编码C3蛋白的mRNA结合，抑制C3蛋白的表达。今年3月，Arrowhead Pharmaceuticals宣布ARO-C3在1/2期临床试验中取得积极中期结果：ARO-C3导致血清中C3蛋白水平出现剂量依赖性降低，在检验的最高剂量平均降低88%。而且，ARO-C3还导致生物标志物AH50（替代补体通路溶血活性的生物标志物）的水平出现剂量依赖性降低，在检验的最高剂量平均降低91%。ARO-C3药效持续时间久，支持每季度或更长间隔进行一次皮下注射。此外，ARO-C3安全性和耐受性良好，没有发现剂量限制性毒性以及严重不良事件，或因为不良事件导致研究中断。Compstatin 40Compstatin 40是一种新型合成环肽，其目的是在C3水平上集中抑制补体级联，在猴子中该药局部注射给药被证实可逆转预先存在的、自然发生的牙周炎。2021年3月，Amyndas Pharma公司宣布评估AMY-101治疗牙周炎和牙龈炎的2期临床试验取得阳性顶线结果：与安慰剂相比，AMY-101在消除牙周炎症方面具有统计学意义和临床意义的显著疗效，并且安全性和耐受性良好。NGM621NGM621是一款人源化免疫球蛋白1（IgG1）单克隆抗体，旨在有效结合补体C3并长效抑制其活性。2022年10月，GM Bio宣布NGM621在一项2期临床研究中失败，未能达到地图样萎缩病变面积减少的主要终点。值得一提的是，NGM621是NGM Bio与默沙东战略合作中发现的药物。不过2022年12月，根据NGM向美国证券交易委员会提交的文件，默沙东已经放弃了NGM621。LP-005LP-005等处于1期临床试验阶段。LP-005是天辰生物基于自主研发的iCibitorTM技术平台开发的第一款补体双功能抗体，作用于C3和C5，拟用于治疗多种补体相关罕见病和常见病。今年6月，该药在国内获批临床，用于治疗成人PNH。不过，C3靶向药的研发并不顺利。据悉，Apellis已放弃了APL-1030和APL-2006两个项目，其中APL-1030是一种首创的脑活性C3抑制剂，用于治疗神经系统疾病。APL-2006是一种双特异性C3和血管内皮生长因子（VEGF）抑制剂，用于治疗GA和湿性老年性黄斑变性（AMD）。总结自2007年首款补体药物Soliris获批以来，补体系统药物研发越发火热，越来越多的补体药物靶点被发现。C3靶点整体上相较C5进展相对较慢，首款上市药物Pegcetacoplan进展也不顺利，被发现存在严重副作用。此外，其他在研药物进展除了ARO-C3似乎并未取得可喜进展。新药研发大多九死一生，希望C3靶点布局企业不要经历竹篮打水一场空。END👇关注药渡数据媒体矩阵

Rise in adoption of minimally invasive non-surgical procedures in the management of periodontitis in adults is likely to drive the global periodontal treatment market Asia Pacific market is anticipated to witness substantial rise in revenue during the forecast period WILMINGTON, Del., Jan. 4, 2023 /PRNewswire/ -- Increase in focus of people on oral health and high prevalence of periodontal diseases among the global population are likely to propel periodontal treatment market growth. Governments are offering funding for development of innovative medical devices in dentistry, which is likely to broaden periodontal treatment market outlook. The periodontal treatment market size stood at US$ 7.6 Bn in 2021. Advancements in non-surgical periodontal therapy is set to present attractive opportunities to companies in the periodontal treatment market. Associated risk factors of periodontitis are sedentary lifestyle, unhealthy diet, and tobacco consumption. An understanding of these factors has helped healthcare providers decide the extent of the management required in various forms of periodontitis. High prevalence of chronic periodontitis in adults is expected to create attractive business opportunities for companies in the market. Get Exclusive PDF Sample Copy of Periodontal Treatment Market – Key Findings of Study Rise in Demand for Non-surgical Periodontal Therapies: Demand for non-surgical treatments for periodontal diseases has increased significantly in the past few years. The treatments include periodontal scaling & root planning and usage of antibiotics, especially in severe forms of periodontal disease. Advances in pharmacologic therapies are expected to create significant opportunities for companies in the market in the next few years. Patient Preference for Dental Clinics: Dental clinics are the most preferred healthcare setting for the management of periodontal diseases. The segment held major share of the market in 2021. Patients suffering from oral health conditions prefer dental clinics to hospitals. Rise in prevalence of periodontal diseases among adults presents significant untapped opportunities for companies. Incidence of these disease is high in older adults (aged 65 years and above). Adoption of periodontal regeneration therapies has led to increased commercialization of advanced bone graft products, thus fueling market growth. Key Drivers Advancements in the treatment modalities for periodontitis, including adoption of advanced dental bone graft products, is a key driver of the periodontal treatment market. Companies are likely to record higher revenue due to increasing commercialization of bone graft substitutes. Rise in awareness about oral health along with patient-specific risk factors of periodontal diseases is expected to propel market development. These risk factors include smoking and inadequate oral health practices. Furthermore, epidemiologic associations between diabetes mellitus and periodontal diseases presents untapped avenue for companies in the periodontal treatment industry. This is because patients with diabetes mellitus are at a greater risk of developing periodontal disease. Ask for References – Regional Growth Dynamics North America held major market share in 2021. Development of novel technologies and rise in R&D in advanced periodontal therapies have fueled market growth in the region in the past few years. The market in Asia Pacific is likely to grow at a rapid pace from 2022 to 2031. High burden of periodontal diseases, especially in Southeast Asia, and rise in prevalence of oral diseases are expected to propel the market in the region. Competition Landscape The global periodontal treatment market in consolidated, with the presence of small number of leading players. These players are restructuring product portfolio and engaging in merger & acquisition in order to consolidate their positions in the market. Prominent companies in the periodontal treatment market are Planmeca Oy, Novartis AG, Medtronic, Karl Schumacher, Dentsply Sirona, Danaher, Carl Martin GmbH, Biolase, Inc., ASA Dental S.p.A, 3M, and Amyndas Pharmaceuticals. Periodontal Treatment Market Segmentation Treatment Type Non-surgical Treatment Scaling & Root Planning Antibiotics Systemic Antibiotics Local Antibiotics Surgical Treatment Pocket Reduction Surgery Soft Tissue Grafts Bone Grafts Others End-user Hospitals Dental Clinics Others Regions Covered North America Latin America Europe Asia Pacific Middle East & Africa Countries Covered U.S. Canada Germany U.K. France Spain Italy Japan China India ASEAN Brazil Mexico GCC Countries Buy this Premium Research Report | Immediate Delivery Available – Modernization of healthcare in terms of both infrastructure and services have pushed the healthcare industry to new heights, Stay Updated with Latest Healthcare Research Reports by Transparency Market Research: Oral Thin Films Market Consumer Genomics Market Acute Migraine Treatment Market Dental Anesthetics Market Ceftriaxone Market Fusion Inhibitors Market Anti-radiation Drugs Market Asthma Treatment Market About Transparency Market Research Transparency Market Research, a global market research company registered at Wilmington, Delaware, United States, provides custom research and consulting services. Our exclusive blend of quantitative forecasting and trends analysis provides forward-looking insights for thousands of decision makers. Our experienced team of Analysts, Researchers, and Consultants use proprietary data sources and various tools & techniques to gather and analyses information. Our data repository is continuously updated and revised by a team of research experts, so that it always reflects the latest trends and information. With a broad research and analysis capability, Transparency Market Research employs rigorous primary and secondary research techniques in developing distinctive data sets and research material for business reports. For More Research Insights on Leading Industries, Visit Our YouTube Channel and hit subscribe for Future Update - Contact Nikhil Sawlani Transparency Market Research Inc. CORPORATE HEADQUARTER DOWNTOWN, 1000 N. West Street, Suite 1200, Wilmington, Delaware 19801 USA Tel: +1-518-618-1030 USA – Canada Toll Free: 866-552-3453 Website: Blog: Email: [email protected] Logo: SOURCE Transparency Market Research

DUBLIN--( BUSINESS WIRE )--The "Global Respiratory Partnering 2010-2022: Deal Trends, Players and Financials" report has been added to ResearchAndMarkets.com's offering. The Global Respiratory Partnering 2010 to 2022 report provides comprehensive access to available deals and contract documents for over 1000 respiratory deals. Most of the deals included within the report occur when a licensee obtains a right or an option right to license a licensor's product or technology. More often these days these deals tend to be multi-component including both a collaborative R&D and a commercialization of outcomes element. The report takes readers through the comprehensive Respiratory disease deal trends, key players and top deal values allowing the understanding of how, why and under what terms companies are currently entering Respiratory deals. The report presents financial deal terms values for Respiratory deals, where available listing by overall headline values, upfront payments, milestones and royalties enabling readers to analyse and benchmark the value of current deals. The initial chapters of this report provide an orientation of Respiratory dealmaking trends. Chapter 1 provides an introduction to the report. Chapter 2 provides an overview of the trends in Respiratory dealmaking since 2010 covering trends by year, deal type, stage of development, technology type and therapeutic indication. Chapter 3 includes an analysis of financial deal terms covering headline value, upfront payment, milestone payments and royalty rates. Chapter 4 provides a review of the leading Respiratory deals since 2010. Deals are listed by headline value. The chapter includes the top 25 most active Respiratory dealmakers, together with a full listing of deals to which they are a party. Where the deal has an agreement contract published at the SEC a link provides online access to the contract. Chapter 5 provides comprehensive access to Respiratory deals since 2010 where a deal contract is available, providing the user with direct access to contracts as filed with the SEC regulatory authorities. Each deal title links via Weblink to an online version of the deal record contract document, providing easy access to each contract document on demand. Chapter 6 provides a comprehensive directory of all Respiratory partnering deals by specific Respiratory target announced since 2010. The chapter is organized by specific Respiratory therapeutic target. Each deal title links via Weblink to an online version of the deal record and where available, the contract document, providing easy access to each contract document on demand. In addition, a comprehensive appendix is provided with each report of all Respiratory partnering deals signed and announced since 2010. The appendices are organized by company A-Z, stage of development at signing, deal type (collaborative R&D, co-promotion, licensing etc) and technology type. Each deal title links via Weblink to an online version of the deal record and where available, the contract document, providing easy access to each contract document on demand. The report also includes numerous tables and figures that illustrate the trends and activities in Respiratory partnering and dealmaking since 2010. In conclusion, this report provides everything a prospective dealmaker needs to know about partnering in the research, development and commercialization of Respiratory technologies and products. Report scope Global Respiratory Partnering 2010 to 2022 includes: Trends in Respiratory dealmaking in the biopharma industry since 2010 Access to headline, upfront, milestone and royalty data Access to hundreds of Respiratory deal contract documents Comprehensive access to over 1000 Respiratory deal records The leading Respiratory deals by value since 2010 Most active Respiratory dealmakers since 2010 Analyzing actual contract agreements allows assessment of the following: What are the precise rights granted or optioned? What is actually granted by the agreement to the partner company? What exclusivity is granted? What is the payment structure for the deal? How are the sales and payments audited? What is the deal term? How are the key terms of the agreement defined? How are IPRs handled and owned? Who is responsible for commercialization? Who is responsible for development, supply, and manufacture? How is confidentiality and publication managed? How are disputes to be resolved? Under what conditions can the deal be terminated? What happens when there is a change of ownership? What sublicensing and subcontracting provisions have been agreed? Which boilerplate clauses does the company insist upon? Which boilerplate clauses appear to differ from partner to partner or deal type to deal type? Which jurisdiction does the company insist upon for agreement law? Key Topics Covered: Executive Summary Chapter 1 - Introduction Chapter 2 - Trends in Respiratory dealmaking 2.1. Introduction 2.2. Respiratory partnering over the years 2.3. Respiratory partnering by deal type 2.4. Respiratory partnering by industry sector 2.5. Respiratory partnering by stage of development 2.6. Respiratory partnering by technology type 2.7. Respiratory partnering by therapeutic indication Chapter 3 -Financial deal terms for Respiratory partnering 3.1. Introduction 3.2. Disclosed financials terms for Respiratory partnering 3.3. Respiratory partnering headline values 3.4. Respiratory deal upfront payments 3.5. Respiratory deal milestone payments 3.6. Respiratory royalty rates Chapter 4 - Leading Respiratory deals and dealmakers 4.1. Introduction 4.2. Most active in Respiratory partnering 4.3. List of most active dealmakers in Respiratory 4.4. Top Respiratory deals by value Chapter 5 - Respiratory contract document directory 5.1. Introduction 5.2. Respiratory partnering deals where contract document available Chapter 6 - Respiratory dealmaking by therapeutic target 6.1. Introduction 6.2. Deals by Respiratory therapeutic target Appendices Appendix 1 - Directory of Respiratory deals by company A-Z 2010 to 2022 Appendix 2 - Directory of Respiratory deals by deal type 2010 to 2022 Appendix 3 - Directory of Respiratory deals by stage of development 2010 to 2022 Appendix 4 - Directory of Respiratory deals by technology type 2010 to 2022 Companies Mentioned Alnylam Pharmaceuticals Vortran Medical Sichuan University Lung and Blood Institute Food and Drug Administration (FDA) Bill and Melinda Gates Foundation Zhejiang Medicine Jubilant DraxImage Enzyvant Science Altavant Sciences Biostar Pharmaceuticals Scholar Rock Monopar Therapeutics Wockhardt Innovare Celularity Highmark Blue Shield Highmark Health Glenmark Pharmaceuticals 4D Pharma BESPharma IR Pharma SI Pharmaceuticals Wal-Mart Sanovas Amyndas Pharmaceuticals Tunitas Therapeutics Innovata HK Medidata Solutions Advocate Health Care Innovate UK Foundation for Sarcoidosis Research Simulations Plus Innovative Genomics Initiative (IGI) Innovative Medicines Initiative (IMI) Laboratory Corporation of America Novavax Dynavax Technologies Pharmaxis Scarab Genomics Punjab Province For more information about this report visit https://www.researchandmarkets.com/r/tub168