Inclisiran sodium

2025年5月7日，中国‌——齐鲁制药与瑞博生物共同宣布，双方合作开发的靶向PCSK9小核酸药物RBD7022/QLC7401(登记号:CTR20244751) II期临床试验已顺利完成全部受试者入组。该试验共纳入204例高脂血症患者，旨在进一步评估药物的安全性、有效性及长效降脂潜力，为后续关键临床研究奠定基础。聚焦全球前沿技术，破解心血管领域“未满足需求”‌RBD7022是瑞博生物基于自主知识产权平台‌RiboGalSTARTM开发的GalNAc-siRNA药物，是继Leqvio后全球第二款进入临床阶段靶向PCSK9的siRNA药物。该药通过精准抑制肝脏PCSK9蛋白表达，长效降低低密度脂蛋白胆固醇（LDL-C）水平。该技术突破传统药物需频繁给药的局限，单次注射即可实现‌数月持续降脂‌，为高脂血症患者提供更优治疗选择。目前，RBD7022已完成I期临床研究，显示其良好的安全性，且临床药效数据达到预期。作为全球首个上市的超长效降脂siRNA药物，诺华的Leqvio上市以来销售高速增长，2024年其销售额为7.54亿美元（+114%）。2025 Q1该药销售额为2.57亿美元（约18.7亿元），同比增长72%。强强联合：技术龙头与商业化巨头的“双向赋能”2023年12月15日，瑞博生物将RBD7022在中国内地、香港及澳门的权益授权予齐鲁制药，凸显市场对该产品的信心。齐鲁制药凭借成熟的研发体系和商业化网络，有望快速实现RBD7022的本土化生产与市场覆盖。这是一次技术龙头与商业化巨头的强强联合。瑞博生物作为小核酸药物领域的领军企业，其RiboGalSTARTM平台在肝靶向递送效率、稳定性和安全性上达到国际领先水平，已布局多款代谢性疾病及罕见病管线。同时，齐鲁制药具有全产业链优势与成熟的商业化网络，近年来通过BD合作加速布局核酸药物、ADC等前沿领域，此次合作将进一步巩固其在心血管赛道的话语权。RBD7022这一里程碑不仅印证了‌瑞博生物在小核酸药物源头创新中的全球竞争力‌，更彰显了‌齐鲁制药在复杂临床试验执行与产业化落地上的行业领先优势‌，为国产长效降脂药物冲击百亿市场按下加速键。关于齐鲁制药 齐鲁制药集团是一家领先的全产业链型制药公司，在全国拥有11大生产基地，总占地8916亩，2022年位列中国医药工业百强榜第五。集团依托中美五大研发中心，以临床价值为导向，以患者为中心，持续整合全球优质资源进行创新药物研发。2022年度集团研发投入占销售收入的比重约为11%，在肿瘤、感染、肝病、自身免疫、代谢疾病等未被满足的重大疾病领域已布局丰富且具有竞争力或差异化的产品管线和多种具有竞争力的平台，积极外部创新项目引进及院校合作，与各方融合、融通创新，以有温度的科技创新推动更多高品质药物快速上市，驱动人类更加美好、健康的未来。关于瑞博生物瑞博生物专注于小核酸（siRNA）药物研究和开发的全球领军者。瑞博生物对标国际小核酸技术的创新前沿，致力于小核酸化学修饰和药物递送技术的迭代研发，建立了自主可控、全技术链整合的小核酸药物研发平台，支持小核酸药物从早期研发到产业化的各阶段研究。公司围绕心血管、代谢、肝病、肾病以及其他治疗领域建立了丰富的产品管线。公司坚持差异化靶点和产品布局，拥有国际化的创新研发团队，在全球开展产品研发和商业化，争取早日为患者带来全新的药物治疗手段。公司总部位于江苏昆山，于北京和瑞典Mölndal设有研发中心，更多信息请参见www.ribolia.com

Presentations by leading siRNA CDMOs, Bachem, Nitto Avecia, and ST Pharm, highlight performance and transferability of Codexis double-stranded RNA ligases Management to host conference call today at 8 am Eastern Time to discuss data REDWOOD CITY, Calif., May 22, 2025 (GLOBE NEWSWIRE) -- Codexis, Inc. (NASDAQ: CDXS), a leading provider of enzymatic solutions for the efficient and scalable manufacturing of complex therapeutics, presented data at the TIDES USA annual meeting in San Diego, California. Codexis’ presentations showcased its proprietary ECO Synthesis platform’s ability to support siRNA manufacturing by reducing purification costs, improving process performance, and demonstrating the potential to control stereochemistry. In addition, three presentations from contract development and manufacturing organizations (CDMOs) including Bachem, Nitto Avecia, and ST Pharm validated the transferability of Codexis’ ligation processes to their in-house facilities. “Having been on the ground at TIDES this week, it is clear that siRNA technology has officially arrived,” said Stephen Dilly, MBBS, PhD, Chairman and Chief Executive Officer at Codexis. “With a broader shift in favor of enzymatic manufacturing solutions and recent initiatives to onshore manufacturing, the rollout of our ECO Synthesis technology is quite timely. Our presentations at TIDES, including those from our three CDMO collaborators, validate the performance of our enzymatic approach and that the technology is now deployed at larger scale. We also showed how our technology can address some of the constraints associated with existing chemical synthesis methods, including intensive purification steps and lack of control over stereochemistry.” Kevin Norrett, Chief Operating Officer of Codexis, added, “Since the TIDES Europe meeting last fall, Codexis has emerged as the leader in enzymatic technology for manufacturing siRNA. Customers have gone from asking whether an enzymatic approach can work, to now asking how and when they can incorporate it into their drug development. Our job is to translate this momentum into genuine traction, including a GMP scale-up partnership by the end of the year.” Codexis Showcases Improvements in Performance and Process Conditions of ECO Synthesis Platform During an oral Main Stage presentation, Codexis used inclisiran as an example to showcase how the use of immobilized enzymes in its ECO Synthesis platform delivers a consistent process with improved performance. Using sequential enzymatic synthesis, we: Continued to achieve consistent coupling efficiency of >98%Maintained the delivery of high product qualitySuccessfully decreased average cycle time by approximately 24%, translating into reduced production timeDemonstrated high yields of 30 grams of siRNA per liter Using Codexis’ ligase to manufacture siRNA, our data demonstrated the potential to reduce or eliminate costly downstream purification steps. This benefit of ligation has led many companies to explore using this method in siRNA manufacturing. Codexis Highlights Proprietary Machine Learning Tool for Optimized Ligation Codexis’ poster presentation highlighted how the Company’s recently launched machine learning tool is improving the success rate of ligation to produce siRNA. The tool aids process design by guiding the selection of optimal pairings of ligases and RNA fragments and has been shown to significantly outperform traditional fragment selection by four to sixfold. It also increases the probability of identifying fragment designs for which a single ligase can combine all fragments together in one reaction with greater than 90% efficiency. This approach has been validated through test molecules and partner case studies. By accelerating the ligase selection process and maximizing ligation efficiency, this tool demonstrates the potential to reduce process development time, lower costs, and enable an efficient manufacturing process for customers. Three CDMO Collaborator Presentations Feature the Use of Codexis Ligases to Combine Short RNA Fragments In addition to Codexis’ presentations, Bachem, Nitto Avecia, and ST Pharm, three leading CDMOs in siRNA manufacturing, presented data on the use of Codexis ligases to successfully combine short RNA fragments. Each CDMO collaborator performed their ligations in-house, demonstrating that Codexis’ process can be easily transferred and the benefits across yield, purification, and impurity control can be replicated in customers’ hands. Codexis Demonstrates the Potential to Control Stereochemistry in Enzymatic Synthesis of siRNA In a separate TIDES Talk presentation, Codexis highlighted a new feature under development for the ECO Synthesis technology to allow for stereochemical control during oligonucleotide synthesis. Stereochemistry refers to the presence of mirror-image forms, or chirality, of a therapeutic oligonucleotide strand, which can impact the efficacy and stability profile of siRNA therapeutics. Drug developers have little influence over stereochemistry today, as existing chemical manufacturing solutions produce mixtures of these mirror images that vary in terms of therapeutic potency. In contrast, the data in this oral presentation demonstrate how the ECO Synthesis platform can enable control over stereochemistry under process-relevant conditions. This differentiated capability would give customers the choice to define stereochemistry in their oligonucleotide products, potentially benefiting each assets’ therapeutic potency and market position. The slide decks from both of Codexis’ oral presentations and the poster are now available in the Investor Relations section the Codexis corporate website, www.codexis.com/investors. Conference Call and Webcast Codexis management will host a conference call beginning at 8:00 am Eastern Time on Thursday, May 22, 2025, to discuss the data presented during the conference. The live call can be accessed by dialing 877-705-2976 (domestic) or 201-689-8798 (international). A live webcast to accompany the conference call can be accessed on the Codexis Investor Relations website, where a replay will be available for 90 days. A telephone replay of the call will be available for 48 hours by dialing 877-660-6853 (domestic) or 201-612-7415 (international), access ID #13726635. About Codexis Codexis is a leading provider of enzymatic solutions for efficient and scalable therapeutics manufacturing, leveraging its proprietary CodeEvolver® technology platform to discover, develop and enhance novel, high-performance enzymes. Codexis enzymes solve for real-world challenges associated with small molecule pharmaceuticals manufacturing and nucleic acid synthesis. The Company is currently developing its proprietary ECO Synthesis™ manufacturing platform to enable the scaled manufacture of RNAi therapeutics through an enzymatic route. Codexis’ unique enzymes can drive improvements such as higher yields, reduced energy usage and waste generation, improved efficiency in manufacturing and greater sensitivity in genomic and diagnostic applications. For more information, visit https://www.codexis.com. Forward-Looking Statements This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. In some cases, you can identify forward-looking statements by terminology such as “aim,” “anticipate,” “assume,” “believe,” “contemplate,” “continue,” “could,” “design,” “due,” “estimate,” “expect,” “goal,” “intend,” “may,” “objective,” “plan,” “positioned,” “potential,” “predict,” “seek,” “should,” “suggest,” “target,” “on track,” “will,” “would” and other similar expressions that are predictions of or indicate future events and future trends, or the negative of these terms or other comparable terminology. To the extent that statements contained in this press release are not descriptions of historical facts, they are forward-looking statements reflecting the current beliefs and expectations of management, including but not limited to statements regarding anticipated milestones, including product launches, technical milestones, data releases and public announcements related thereto; whether Codexis will be able to, and the timing of it entering into revenue-generating development contracts with customers regarding its ECO Synthesis™ manufacturing platform; its ability to enter into an agreement with a GMP scale-up partner regarding its ECO Synthesis™ manufacturing platform in 2025; Codexis achieving pilot scale production of GLP-grade siRNA material using the ECO Synthesis™ Innovation Lab in 2025; Codexis’ expectations regarding 2025 product revenues, R&D revenues and gross margin on product revenue, as well as its ability to fund planned operations through the end of 2026; Codexis’ ability to achieve positive cash flow around the end of 2026; potential receipt by Codexis of certain royalty payments pursuant to its recent license agreement with Alphazyme; the potential of the ECO Synthesis™ manufacturing platform, including its ability to be broadly utilized and to enable commercial-scale manufacture of RNAi therapeutics through an enzymatic route; and expectations regarding future demand for dsRNA. You should not place undue reliance on these forward-looking statements because they involve known and unknown risks, uncertainties and other factors that are, in some cases, beyond Codexis’ control and that could materially affect actual results. Factors that could materially affect actual results include, among others: Codexis’ dependence on its licensees and collaborators; if any of its collaborators terminate their development programs under their respective license agreements with Codexis; Codexis may need additional capital in the future in order to expand its business; if Codexis is unable to successfully develop new technology such as its ECO Synthesis™ manufacturing platform and dsRNA ligase; Codexis’ dependence on a limited number of products and customers, and potential adverse effects to Codexis’ business if its customers’ products are not received well in the markets; if Codexis is unable to develop and commercialize new products for its target markets; if competitors and potential competitors who have greater resources and experience than Codexis develop products and technologies that make Codexis’ products and technologies obsolete; Codexis’ ability to comply with debt covenants under its loan facility; if Codexis is unable to accurately forecast financial and operational performance; and market, political and economic conditions may negatively impact Codexis business, financial condition and share price. International trade policies, including tariffs, sanctions and trade barriers, may adversely affect our business. Additional information about factors that could materially affect actual results can be found in Codexis’ Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC) on February 27, 2025 and in Codexis’ Quarterly Report on Form 10-Q filed with the SEC on May 14, 2025, including under the caption “Risk Factors,” and in Codexis’ other periodic reports filed with the SEC. Codexis expressly disclaims any intent or obligation to update these forward-looking statements, except as required by law. For More Information Investor ContactCarrie McKim(336) 608-9706ir@codexis.com