Vectura Group Ltd.

编译：沈丹蕾，南京白令信息科技有限公司总经理 吸入产品从开发到生产再到上市的过程复杂繁琐，这促使美国食品药品监督管理局（FDA）和欧洲药品管理局（EMA）开始联合制定思路，以降低成本并节省时间。 与口服制剂相比，仿制吸入产品为开发者带来了更多挑战。开发者不仅需要证明药物本身与原研药具有等效性，还需向监管机构证明药物与其递送装置的组合也具有等效性。 开发时间表也与原研吸入产品有所不同。对于原研药，从研发到上市的整个过程可能需要十年或更长时间，因此不需要在一开始就确定最终的设备设计；然而，对于仿制药，较短的开发时间意味着最终设备设计的选择不能被推迟。 在美国，FDA要求开发任何仿制产品的公司证明其符合三个标准：药学等效性、生物等效性和治疗等效性。 • 药学等效性：仿制药需包含与参比药相同的活性成分，使用相同的剂型和给药途径，具有相同的强度，并符合所有质量、纯度和标识的药典标准。 • 生物等效性：在作用部位上，活性成分的吸收速率和程度没有显著差异。对于吸入产品，作用部位是肺部的黏膜表面，因此直接测量非常困难。 • 治疗等效性：开发者需证明仿制药可以替代参比药，并在标签规定的条件下具有相同的临床效果和安全性。 尽管可能存在等效但不可替代的产品，但要实现替代性，仿制药必须满足上述三个标准。 01 吸入药物等效性挑战 吸入药物是复杂的产品系统，其性能取决于配方、设备和患者的相互作用。此外，生产过程也会影响药物的表现。参比药物的批次间性能差异增加了比较的难度。 02 欧盟的不同方法 欧盟的审批方法与美国略有不同，但两者有一些共同点（见图1）。无论在哪种情况下，都需要使用相同的药物和相同类型的设备。然而，欧盟没有替代性的概念；仿制药需通过药物递送等效性测试，无论是通过实验室测试、药代动力学（PK）或药效动力学（PD）实验，还是通过临床试验来证明。而在美国，除了这些要求外，还需评估产品是否具有与参比药相同的操作说明。符合此要求的产品可按照美国505(j)ANDA路径获得批准；否则则需按照505(b)2新药申请（NDA）路径审批。 03 FDA的权衡证据方法 FDA对生物等效性采用“权衡证据”方法，考虑设备和配方设计，并需通过PK、PD或临床比较研究进行支持。FDA还发布了产品特定指南，例如2013年首个针对吸入产品的指南（沙美特罗/氟替卡松吸入粉末组合）。目前，大多数口服吸入药物产品都有相应的指南，依据权衡证据获得简化新药申请（ANDA）批准。 04 设备设计与开发早期考量 设备的相似性是FDA对吸入仿制药的核心关注点，2017年FDA发布的指南详细说明了设备等效性的信息要求和用户界面的允许差异范围。例如，Mylan公司在 2019年推出的Wixela® Inhub®吸入器（沙美特罗/氟替卡松）是一个成功案例。 05 新兴测试方法 FDA正在研究新的测试方法，例如Q3测试，旨在更好地理解气溶胶药物递送的物理过程、肺部沉积和吸收。Q3测试有望减少临床试验的需求，但其科学依据需在提交前得到FDA验证。 06 欧盟的分步方法 与FDA不同，EMA采用分步方法定义生物等效性，包括体外相似性测试、肺部沉积和PK数据。如果这些不相似，则需要PD数据或三期临床研究。大多数仿制药通过体外和PK数据包获得批准。 — 总结 — 吸入药物的监管环境复杂，但为开发复杂仿制药提供了机会和路径。设备设计、产品稳健性和早期规模化生产是成功的关键。新的体外测试方法和产品表征方法有助于降低开发成本和时间，使开发者能够以前所未有的方式表征产品。 作者介绍 Martin Oliver是Vectura公司的高级副总裁，负责DPI和pMDI平台的交付管理。他同时担任Vectura在中国的合资企业天津康诺华制药有限公司的副董事长，致力于在该地区推广DPI产品。他拥有英国布拉德福德大学的药学学士学位。 文章来源：How the EU and US Are Aligning Guidance for Inhalation Products，PMPS 同写意媒体矩阵，欢迎关注↓↓↓

Major companies in the dry powder inhaler market are focusing on innovation, developing products with muscarinic antagonists for the treatment of chronic obstructive pulmonary disease (COPD), and expanding their product portfolios. Muscarinic antagonists are drugs that inhibit the muscarinic acetylcholine receptor and are used to treat various illnesses, including COPD NEWARK, DE / ACCESSWIRE / December 5, 2024 / The Dry Powder Inhaler Market is poised for significant growth, with a market value of USD 20.83 billion in 2024, projected to climb to USD 29.95 billion by 2034, representing a steady CAGR of 3.70% over the forecast period. This growth is underpinned by rising prevalence of respiratory diseases such as asthma and COPD, combined with technological advancements in drug delivery systems.Regulatory approvals for patient-friendly inhalation devices, increasing awareness of chronic respiratory diseases, and a rising geriatric population are also boosting the adoption of dry powder inhalers globally. With the growing focus on reducing hospital stays and providing efficient home-care treatments, these devices are becoming an essential tool in respiratory disease management.Regionally, the market sees diverse opportunities. Emerging economies in Asia-Pacific demonstrate the fastest growth due to increasing healthcare expenditure and heightened awareness about respiratory health. Developed regions, particularly North America and Europe, continue to dominate the market due to their advanced healthcare infrastructure and strong presence of key industry players.The shift toward environmentally friendly propellants and the development of digitalized inhalers equipped with dose-tracking and usage monitoring are reshaping the landscape, offering additional revenue streams for manufacturers.With the utilization of dry powder inhalers in the treatment of a diverse range of diseases, the development of technologically innovative products is surfacing. These "smart technologies" are set to satisfy specific market requirements for the development of cost-effective drug products. The deep penetration of digital systems into the inhaler market has been advantageous in terms of ruling out several device and design errors. The implementation of microprocessors in inhalers for supporting patients with device usage and handling is positively impacting compliance and product satisfaction.The development of next-generation inhalers with the integration of smart technologies is likely to drive the growth of the dry powder inhaler market. The integration is set to establish a breakthrough in drug delivery with the primary basis being a tailored performance based on the patient's/user's variable breathing patterns.Technologically advanced dry powder inhalers with digitally enabled platforms along with a friendly user interface will help in preventing or compensating actuation errors that, in turn, will enhance compliance. The growth of digitally operated inhalers devices is also supported by the recent upsurge in digitalization in the healthcare and industrial sectors. This factor will drive the dry powder inhaler market.The dry powder inhalers are a preferred method of treatment of respiratory ailments over pressurized metered dose inhalers and nebulizers owing to the rising concerns related to the environmental impact of inhaler devices which utilize chloroflouro carbons as propellants.Thus, the production and design of dry powder inhalers is set to witness a growth over the forecast period, and is expected to offer product manufacturers or key players in the dry powder inhaler market with an optimistic growth opportunity.Request a Sample of this Report Now: https://www.fmisamplereport.com/sample/rep-gb-5150Key Takeaways from Market StudyMarket Growth: Projected to grow from USD 20.83 billion in 2024 to USD 29.95 billion by 2034, at a CAGR of 3.70%.Technology Focus: Smart inhalers and eco-friendly propellants are driving innovations in the market.Disease Prevalence: Rising cases of asthma and COPD are a key determinant of demand.Regional Growth: North America and Europe lead the market, while Asia-Pacific exhibits the highest growth rate.Segment Dominance: Capsule-based inhalers continue to capture a significant market share due to their ease of use and cost-effectiveness."Dry powder inhalers are witnessing a transformation driven by the convergence of healthcare and technology. The emergence of smart inhalers, coupled with increasing adoption in developing markets, positions the industry for robust growth over the next decade. Companies that can successfully innovate while addressing environmental concerns will lead the charge in shaping the future of this vital healthcare segment." SaysSabyasachi Ghosh (Associate Vice President at Future Market Insights, Inc.)What Are The Key Opportunities For Dry Powder Inhalers Manufacturers?Confounding problems are existent in clinical trials for dry powder inhalers, specifically, where the possibility of errors by patients with respect to inhaler handling can be high. Distinguishing handling errors by patients from existing clinical effects is complex, particularly in phase III trials where support to patients cannot be contracted.The impact of digital systems thus presents an opportunity to assist with monitoring and control over all phases of clinical studies. Digital technology can improve clinical outcomes by improving adherence and reducing patient errors through mobile applications connected to the device, which will deliver useful tools including reminders and step-based guidance for the appropriate technique of usage.On the other hand, medical providers can track patient compliance to the therapeutic regimen in real time. Additionally, the monitoring of symptoms and their worsening is registered along with the compliance tracker and it assists with the prevention of severe disease exacerbations, thus leading to a satisfying patient outcome.The development of inhalation products poses the possibility of significant errors for drug-device combinations related to the usage of the device by patients, variations in the delivery of drugs, as well as flawed data collection.These errors may deliver distorted information regarding the therapeutic efficacy of drug formulations and device-based effects in clinical trials. The introduction of programmed tools for use in clinical trials will contribute to an improved understanding of drug-device combinations and their efficiency.These tools may assist with enhanced data collection processes without being influenced by poor patient compliance as well as administration issues. These automated tools can include incorporated audio-based technologies and devices for monitoring medication compliance.Connected wearable devices can be beneficial in tracking patient activity and thus evaluating the effect on clinical efficacy with integrated companion applications to capture patient-reported outcomes.Thus, the change in the landscape of clinical trials is set to be positively impacted by the monitoring of data all through the product life cycle along with patient-reported data in real-time conditions, thus presenting an opportunistic growth for the dry powder inhaler market.What Are The Factors Restraining Demand For Dry Powder Inhalers?Technological advancements are progressively growing in terms of increasing novel and innovative, integrated, alternative good manufacturing practice-ready approaches, which are associated with next-generation analytical and modelling skills. However, regulations encompassing such progressions are yet to catch up with the developing complex clinical requirements.This trend is intensified by the acceleration of complexity and the cost of approval for the formulation of orally inhaled and nasal drug products and by the scientific disparity that still exists, particularly for locally acting dry powder inhaler products.Considering this, despite existing development, animal models are not applicable to humans and perceptive methods for assessing clinical efficiency along with in-vitro and in-vivo association for inhaled medicines are still inadequate.In this concern, the outcome of the clinical approach is intensely predisposed by the ambiguous correlation between efficiency and selected clinical endpoints. This risk is greater for therapies in which the pattern of deposition of the drug is directly associated with efficiency.In spite of thorough assessments and a focus on advances in device design as well as formulation, the impact on the patient in terms of their adherence to therapy, inspiration rate, and correct use of the inhaler are yet to be quantified. This poses a restrictive impact on the growth of the dry powder inhaler market, given the wide-ranging factors associated with the intensity of the impact of disease on the patient, affecting their inspiration rate.Country-wise InsightsWhat Makes the U.S. the Largest Market for Dry Powder Inhalers?The U.S. dominates the North American region with a total market share of about 90.5% in 2020, and is projected to continue experiencing high growth throughout the forecast period. U.S. is the most lucrative market for dry powder inhalers owing to the combination of a robust healthcare infrastructure accompanied with technologically advanced procedure, an increasing number of patients with respiratory disorders, strictly enforced guidelines by regulatory authorities pertaining to medical devices attributed with respiratory disorder therapeutics, and comparatively high government healthcare spending.Furthermore, due to the increased prevalence of asthma and other respiratory disorders, as well as the growing geriatric population, the dry powder inhaler market in the United States is expected to develop significantly over the next decade.Why is Spain Considered a Lucrative Market for Dry Powder Inhalers in Europe?Spain. is set to exhibit a CAGR of nearly 2.90% in the European dry powder inhaler market during the forecast period. The rising research and development activities to generate a sustainable base of drug delivery devices in the country as well as an established healthcare infrastructure are some of the factors aiding the growth of dry powder inhalers in the country.What Makes China an Emerging Market for Dry Powder Inhalers?China holds 38.9% share in the East Asia dry powder inhaler market in 2020, and is projected to increase at a CAGR rate of 5.4% during the forecast period. This growth is associated with the rising deployment of automated systems and devices into an integrated platform for patient monitoring as well as drug delivery devices, rising healthcare costs and rapidly improving medical infrastructure. All these factors are expected to drive market growth in the coming years in China.CountriesForecast CAGRs from 2024 to 2034India5.20%Indonesia4.7%Canada3.1%Spain2.90%France2.40%Competitive LandscapeThe dry powder inhaler market is marked by robust competition as both established pharmaceutical leaders and emerging biotech innovators vie to capture market share. Key players drive innovation by focusing on research and development, improving device functionality, and expanding the therapeutic scope of their products.Strategic collaborations, regulatory compliance, and technological progress are crucial factors shaping this competitive environment. With an increasing emphasis on sustainability and patient-centric solutions, companies strive to differentiate themselves in a rapidly evolving market. As global demand for respiratory medications rises, competition fuels innovation, propelling advancements in dry powder inhaler technologies.Product PortfolioGlaxoSmithKline Plc is a global leader in healthcare innovation, offering a wide range of pharmaceuticals, vaccines, and consumer health products. With a strong commitment to patient-focused solutions, GSK delivers cutting-edge therapies that enhance well-being and improve quality of life worldwide.AstraZeneca Plc is a renowned biopharmaceutical company focused on transforming healthcare through advanced treatments in respiratory, oncology, cardiovascular, and other critical areas. Leveraging research and collaboration, AstraZeneca addresses unmet medical needs to improve patient outcomes globally.Novartis AG redefines medicine with its diverse portfolio that spans pharmaceuticals, generics, and eye care. By harnessing cutting-edge technology and science, Novartis creates innovative solutions aimed at tackling the most urgent healthcare challenges and delivering better health outcomes.Key Companies ProfiledGlaxoSmithKline PlcAstraZeneca Plc;Novartis AG;Teva Pharmaceutical Industries Limited;Boehringer Ingelheim GmbH;Cipla Limited;Chiesi Farmaceutici S.p.A.;Elpen S.A.;Vectura Group Plc;Beximco Pharmaceuticals Ltd.;Hovione;AptarGroup, Inc.;Iconovo AB (Stevanato Group);Sava Healthcare Ltd.;MannKind Corporation;Mylan N.V;PharmaxisSegmentation Analysis of the Dry Powder Inhaler IndustryBy Product:Single Dose Dry Powder InhalersMulti Dose Dry Powder InhalersBy Indication:AsthmaChronic Obstructive Pulmonary Disease (COPD)Pulmonary Arterial HypertensionDiabetesCystic FibrosisBy Function:Manually Operated Inhaler DevicesDigitally Operated Inhaler DevicesBy End User:Institutional SalesHospital PharmaciesCancer ResearchOffice based Specialty ClinicsRetail SalesRetail PharmaciesDrug StoresMail Order PharmaciesBy Region:North AmericaLatin AmericaWestern EuropeEastern EuropeSouth Asia & PacificEast AsiaThe Middle East & AfricaAuthorSabyasachi Ghosh (Associate Vice President at Future Market Insights, Inc.) holds over 12 years of experience in the Healthcare, Medical Devices, and Pharmaceutical industries. His curious and analytical nature helped him shape his career as a researcher.Identifying key challenges faced by clients and devising robust, hypothesis-based solutions to empower them with strategic decision-making capabilities come naturally to him. His primary expertise lies in areas such as Market Entry and Expansion Strategy, Feasibility Studies, Competitive Intelligence, and Strategic Transformation.Holding a degree in Microbiology, Sabyasachi has authored numerous publications and has been cited in journals, including The Journal of mHealth, ITN Online, and Spinal Surgery News.Explore FMI's Extensive Coverage in the Healthcare Domain:The global respiratory inhaler devices market size is anticipated to attain an impressive valuation of USD 37,258.5 million in 2023 and is projected to reach USD 60,114.7 million by 2033The global Dry Eye Syndrome Treatment Market is estimated to capture a CAGR of 6.4% over the forecast period. The global market is worth USD 5,120 million in 2023The anticipated size of the metered dose inhalers market in 2022 was USD 25,208.9 million and is estimated to be USD 26,322.9 million in 2023.Expanding at a high CAGR of 8.2%, the global Dual and Multi-energy Computed Tomography (CT) Market is projected to increase from a valuation of USD 1.5 Billion in 2022 to USD 2.6 Billion by 2029.The positron emission tomography (PET) scanners market is estimated to be valued at USD 1.3 billion in 2023 and is rising to USD 2.1 billion by 2033.The global optical coherence tomography market size reached USD 1,311.70 million in 2023.The global photoacoustic tomography market is projected to reach USD 79.2 million in 2022.A 5.8% compound annual growth rate (CAGR) is projected for the global mobile tomography market from 2022 to 2032, with 2022 revenues of USD 295.2 Million and 2032 revenues of USD 595.1 Million.The global medical automation market is estimated to secure a valuation of USD 48,626.2 million in 2023 and reach up to USD 103,052.3 million by 2033.As per the analysis, the electronic medical record market is set to account for a current valuation of USD 18.82 billion in 2024.About Future Market Insights (FMI)Future Market Insights (FMI) is a leading provider of market intelligence and consulting services, serving clients in over 150 countries. FMI is headquartered in Dubai and has delivery centres in the United Kingdom, the United States, and India. FMI's latest market research reports and industry analysis helps businesses navigate challenges and make critical decisions with confidence and clarity amidst breakneck competition. Our customized and syndicated market research reports deliver actionable insights that drive sustainable growth. A team of expert-led analysts at FMI continuously tracks emerging trends and events in a broad range of industries to ensure that our clients prepare for the evolving needs of their consumers.Join us as we commemorate 10 years of delivering trusted market insights. Reflecting on a decade of achievements, we continue to lead with integrity, innovation, and expertise.Contact UsFuture Market Insights Inc.Christiana Corporate, 200 Continental Drive,Suite 401, Newark, Delaware - 19713, USAT: +1-347-918-3531For Sales Enquiries: [email protected]Website:https://www.futuremarketinsights.comLinkedIn| Twitter| Blogs | YouTubeSOURCE: Future Market Insights, Inc.

本周，生物医药创投领域依然活跃。Insight 数据库显示，本周全球范围内共发生 24 项医药交易和 12 起新药投融资事件（截至9月20日）。 在授权合作方面，诺和诺德、艾伯维、礼来等跨国药企均有合作开发创新疗法。其中： 诺和诺德先后达成两次合作，总金额超过 11 亿美元； 欧加隆 12 亿美元收购自免新锐也备受关注，因为后者核心产品来自中国； 贝达药业、优锐医药等中国公司也分别就新药签订经销协议； 另外，还有多家海外公司就技术、平台或产品达成合作，涉及 3D 技术、药物递送、基因编辑疗法、双特异性抗体、TCR 蛋白药等方向。 在新锐融资方面： 从药物类型来看，除了传统的小分子和抗体药物，细胞疗法、癌症疫苗、RNA 疗法等新分子领域均获得资本支持。 从地区分布来看，本周获得融资的 Biotech 主要来自美国，占比超过 83%。 从金额来看，以靶向疗法开发新锐 Nuvalent 融资金额最高，该公司完成 5.75 亿美元增发。 下面，Insight 数据库将分别节选部分备受关注的融资交易案例做介绍，仅供读者参阅。 多家跨国药企达成新合作    诺和诺德 5.3 亿美元合作开发 RNA 编辑药物 Korro Bio 与诺和诺德达成合作，旨在开发针对两个未公开靶点的 RNA 编辑候选药物，这些靶点可用于治疗肥胖、糖尿病和心血管疾病等疾病。根据协议，诺和诺德将支付 5.3 亿美元的预付款和里程碑付款，外加两个项目研发活动的分级特许权使用费和研发资金。Korro Bio 将负责这两个项目的临床前开发，随后诺和诺德可以选择进行临床测试。 Korro Bio 表示，其 OPERA 平台使用寡核苷酸来「利用人体内的自然过程」，通过对编码 mRNA 进行改变来调节蛋白质功能。由于该技术不影响 DNA，因此其 RNA 编辑方法精确且可调，具有提高特异性和长期耐受性的潜力。   诺和诺德 6 亿美元布局肝外 LNP 递送技术 NanoVation Therapeutics 宣布与诺和诺德达成合作，旨在开发创新基因药物以治疗心脏代谢性疾病和罕见病。根据协议，NanoVation 公司将获得研究资金，并有资格在多年合作中获得高达约 6 亿美元的预付款及潜在里程碑付款。诺和诺德将获得该公司 LNP 技术在两个主要项目中的全球独家使用许可，并在未来扩展至多达五个针对心脏代谢性和罕见病的额外靶点。 NanoVation 公司拥有一个广泛且不断扩展的创新脂质和 LNP 成分库，并已开发出专有长循环脂质纳米粒子（lcLNP）RNA 递送技术。在临床前研究中，该技术展示了比传统递送系统更高的效力、安全性和稳定性，能够将核酸递送至肝脏以外的各种细胞类型。   艾伯维超 3 亿美元合作开发青光眼疗法 Ripple Therapeutics 宣布与艾伯维合作并签署许可协议，以开发下一代青光眼治疗药物 RTC-620。根据协议，Ripple 公司将领导 RTC-620 的临床前开发，行使期权后，艾伯维将领导其临床和商业化活动。为此，Ripple 公司将获得 2180 万美元的预付款，并有资格获得高达 2.9 亿美元的期权费和里程碑付款，以及净销售额的分级特许权使用费。 RTC-620 是一种可生物降解的缓释药物递送前房内植入物，具有重复给药的特点，可降低开角型青光眼或高眼压患者的眼内压。Ripple 公司的创新药物递送平台可使药物在不使用聚合物或赋形剂的情况下，被设计成控释药物。   Axcelead DDP 与礼来合作开发多个药物 Axcelead DDP 宣布，已与礼来就多个药物发现项目达成战略研究与合作协议。Axcelead DDP 通过其完善的药物发现能力和创新技术（包括人工智能）生成候选药物，致力于推动药物发现的创新进步。根据协议，Axcelead DDP 将获得预付款，并有资格根据每个药物发现项目的进展获得额外的里程碑付款。   欧加隆 12 亿美元收购自免新锐，后者核心产品来自中国 欧加隆与 Dermavant Sciences 宣布签署最终协议，将收购后者并囊获其创新疗法 Vtama（tapinarof）乳膏。Dermavant Sciences 是一家专门从事免疫皮肤病学的生物制药公司。根据协议，欧加隆将支付 1.75 亿美元的前期付款和 7500 万美元的里程碑付款，以及高达 9.5 亿美元的商业里程碑付款。 Vtama 乳膏是一种非类固醇的每日一次局部治疗药物，通过激活皮肤中的芳烃受体来减少炎症并恢复皮肤屏障。该产品已于 2022 年 5 月获得美国 FDA 批准，用于治疗成人轻度、中度和重度斑块型银屑病。FDA 还在审评该药另一份补充新药申请，以评估其作为治疗成人和两岁及以上儿童特应性皮炎的潜在疗法。 值得一提的是，Vtama 中文通用名为本维莫德，该药最初由中国天济医药和中昊药业联合开发，天济医药于 2012 年将本维莫德的境外开发权授予 GSK，后者又于 2018 年 7 月转让给 Dermavant 公司。在中国，本维莫德乳膏已通过优先审评审批程序获批上市，用于适合局部治疗的成人轻至中度稳定性寻常型银屑病，并已纳入国家医保目录。 贝达药业、优锐医药等中国公司获得新药商业化权益   贝达药业与禾元生物签订 1 类新药经销协议 贝达药业拟与禾元生物签订药品区域经销协议，禾元生物委托贝达药业在约定区域内独家经销植物源重组人血清白蛋白注射液（商品名为奥福民，HY10010）。 HY1001 是利用水稻胚乳细胞表达，经提取、纯化的治疗用生物制品 1 类新药，其新药上市申请被 CDE 以「临床急需的短缺药品、防治重大传染病和罕见病等疾病的创新药和改良型新药」为由纳入优先审评，拟用于治疗低白蛋白血症。   Altamira 扩大与优锐医药的许可和分销协议 Altamira Therapeutics 宣布，其关联公司 Altamira Medica 已与其合作伙伴优锐医药达成协议，将其 Bentrio 鼻喷雾剂独家许可和分销协议所涵盖的地区扩展至东南亚和东亚的多个国家。根据修订后的协议，优锐医药的权益将从中国大陆、香港、澳门地区及韩国，扩展到新加坡、马来西亚、泰国、菲律宾、印度尼西亚、越南和中国台湾。 Bentrio 是一种不含药物和防腐剂的鼻腔喷雾剂，可用于帮助防护空气传播的过敏原（如花粉或室内尘螨）。自 2022 年底以来，优锐医药一直在中国香港营销 Bentrio，并于最近在 CDE 提交注册申请。   奥赛瑞生物与普利药业签署合作 奥赛瑞生物与普利药业宣布，双方签署关于铁纳米磁共振造影剂 IND 阶段的研发合作协议。此次签约是在普利制药已基本完成聚多巴氨基酸胶束的新型磁共振对比剂，及肿瘤诊疗一体化药物早期研究-候选化合物（PCC）确认的基础上进行。签约后，该公司将继续开发候选化合物，并按照中美申报要求进行 IND 申请。 其它授权合作   日本新药株式会社 15 亿美元获 DMD 新药分销权益 日本新药株式会社（Nippon Shinyaku）和 Capricor Therapeutics 宣布签署一份意向书，规定在未来几个月内独家谈判 CAP-1002（Deramiocel）在欧洲境内治疗杜氏肌营养不良症（DMD）的独家分销协议。根据协议，Capricor 公司将获得 1500 万美元的股权投资，并在签署最终协议后获得 2000 万美元的预付款，潜在里程碑付款高达 7.15 亿美元以及两位数的产品收入，合作潜在总额约为 15 亿美元。 CAP-1002 是一种基于外泌体的细胞疗法，由人类同种异体心脏球衍生细胞组成，拟开发用于治疗各种 DMD，无论基因突变类型如何。在美国进行的一项 2 期研究 HOPE-2 显示，该产品可使晚期 DMD 患者的上肢和心脏功能得到改善。目前，该产品正在美国多家医院进行 3 期 HOPE-3 研究。   Vectura Fertin Pharma 近 3 亿英镑出售子公司 Philip Morris International 子公司 Vectura Fertin Pharma 宣布，将其子公司 Vectura 出售给 Molex Asia Holdings，并建立主服务协议（MSA）以开发 Vectura Fertin Pharmas 的吸入疗法专有管线。此次出售包括 1.5 亿英镑的预付现金对价，以及高达 1.48 亿英镑的潜在延期付款。 Vectura 公司擅于吸入药物装置设计和配方。出售后该公司将由 Molex 公司旗下的 Phillips Medisize 运营，这将增强后者向制药客户提供更广泛的吸入组合药物设备和解决方案的能力。   MaxCyte 与 Kamau 合作开发遗传病细胞疗法 MaxCyte 与 Kamau Therapeutics 达成合作，后者将使用前者的 Flow Electroporation 技术和 ExPERT 平台的非独家研究、临床和商业权利，以支持其同源定向修复（HDR）新型基因校正技术开发。对此，MaxCyte 公司有权获得年度许可费和与项目相关的收入。 Kamau 是下一代基因校正公司，其平台建立在 HDR 编辑的基础上，以第一代 CRISPR-Cas9 技术为基础，不仅切割 DNA，还提供修复 DNA 的模板。MaxCyte 公司的 ExPERT 仪器组合是下一代电穿孔技术，用于复杂和可扩展的细胞工程。 新锐融资   Nuvalent 完成 5.75 亿美元增发 Nuvalent 公司宣布完成普通股的大规模公开发行，扣除承销折扣、佣金和其他发行费用后，此次发行的总收益约为 5.75 亿美元。 Nuvalent 公司专注于为癌症患者创造精确的靶向疗法，旨在克服现有疗法对临床证明的激酶靶点的限制。目前，该公司正在推进多种小分子新药管线，包括针对 ROS1 阳性、ALK 阳性和 HER2 突变非小细胞肺癌的候选药，以及多个处于药物发现阶段的研究项目。   Nura Bio 完成超 1.4 亿美元 A 轮融资 Nura Bio 宣布完成超过 1.4 亿美元的 A 轮融资。这包括在 2020 年宣布的首轮 7300 万美元，以及在这基础上增加的 6800 万美元。本轮融资由创始投资人 The Column Group 领投，Samsara Bio Capital、Euclidean Capital、Sanofi Ventures 参与投资。 Nura Bio 正在开发神经保护性小分子疗法用于治疗衰弱性神经疾病。其中，NB-4746 是该公司小分子产品线中的主要资产，这是一种脑渗透性 SARM1 抑制剂，已被证明可以预防轴突退化，并在多种神经损伤和疾病的临床前模型中被证明可提供神经保护。Nura Bio 计划于 2025 年启动该产品的 1b/2 期试验。   GCTx 公司募资 7500 万美元 GC Therapeutics（简称 GCTx）宣布走出隐匿模式。该公司已完成由 Cormorant Asset Management 领投的 6500 万美元 A 轮融资，筹资总资金达到 7500 万美元，其他投资人包括 Mubadala Capital、Andreessen Horowitz (a16z) Bio + Health 等。 GCTx 公司致力于开发 first-in-class 和 best-in-class 的现货型细胞疗法，用于治疗胃肠道、神经和免疫性疾病。其诱导多能干细胞（iPSC）编程平台 TFome 由知名科学家团队发明和开发，可克服与细胞治疗相关的开发和扩展复杂性。   Brenus Pharma 完成 2500 万美元 A 轮融资 Brenus Pharma 宣布完成 2500 万美元 A 轮融资，由投资基金 Angelor 领投，UI Investissement、Fonds Régional Avenir Industrie 等参与投资。 本次融资所得款项将用于全额资助 STC-1010 癌症疫苗的首次人体概念验证，适应症为转移性结直肠癌一线治疗。同时，该产品将扩展到其他类型的胃肠道肿瘤，如胰腺癌、肝癌。Brenus Pharma 还计划加快其在其他实体瘤适应症方面的产品组合的开发，特别是第二个候选产品 STC-1020。   Syntax Bio 筹集 1500 万美元 Syntax Bio 在 Astellas Venture Management 和 Illumina Ventures 的支持下成立，并筹集 1500 万美元，其他投资者包括 DCVCBio、Civilization Ventures、EGB Capital 和 Portal Innovations。 Syntax Bio 专注于利用其新型 Cellgorithm 平台技术从干细胞中产生治疗性细胞。该平台可在表观遗传水平上模拟人类发育过程，使用改良的 CRISPR 系统以自动和时间控制的方式调节内源性基因。通过加速细胞分化，该技术显著减少了产生高价值细胞类型所需的时间，克服了干细胞衍生疗法中长期存在的挑战。   ImmunOs 在 C 轮融资中筹集 1100 万美元 ImmunOs Therapeutics 宣布在 C 轮融资中筹集 1100 万美元。本轮融资由 Gimv、Pfizer Ventures、Mission BioCapital、BioMed Partners 领投，并得到新投资者 Double Point Ventures 和其他现有投资者的支持。 目前，ImmunOs 公司正利用其基于免疫系统调节 HLA 的技术平台，开发用于治疗癌症和自身免疫性疾病的 first-in-class 新药。本次融资将进一步资助其主导项目 IOS-1002 正在进行的临床试验，以完成 1a 期剂量递增临床试验。这是一种针对 LILRB1（ILT2）、LILRB2（ILT4）和 KIR3DL1 的检查点抑制剂，拟用于治疗晚期实体瘤。   Altamira 完成 1200 万美元公开募股 Altamira Therapeutics 宣布完成 1200 万美元的公开募股。该公司正在开发和提供基于肽的纳米粒子技术，用于将 RNA 高效递送到肝外组织。目前，Altamira 公司已有两个使用其专有递送技术的临床前开发阶段 siRNA 项目，分别是用于治疗 KRAS 驱动癌症的 AM-401，以及用于治疗类风湿性关节炎的 AM-411。 封面来源：站酷海洛 Plus 免责声明：本文仅作信息分享，不代表 Insight 立场和观点，也不作治疗方案推荐和介绍。如有需求，请咨询和联系正规医疗机构。 编辑：馨药 PR 稿对接：微信 insightxb 投稿：微信 insightxb；邮箱 insight@dxy.cn 多样化功能、可溯源数据…… Insight 数据库网页版等你体验 点击阅读原文，立刻解锁！