同时，诺泰生物在寡核苷酸领域的布局也初见成效。公司寡核苷酸吨级商业化生产车间于2025年内完成 建设，形成年产寡核苷酸1000公斤、PMO100公斤、PEG偶联环肽200公斤的优质产能，有望充分获益 后续下游相关药物研发生产需求的增长。 童梓权介绍，在市场拓展方面，诺泰生物凭借高质量的产品和优质的服务，与多家头部仿制药企和创新 药企建立了稳固的合作关系。公司全资子公司杭州澳赛诺生物科技有限公司与国外某客户签订的重大合 同，2025年度实际履行金额约2000万美元，远超合同约定的采购指引，体现了公司在全球医药供应链中 的技术实力与市场竞争力。 童梓权表示，生物医药是守护生命的事业，更是新质生产力的重要赛道。未来，诺泰生物将坚持"与势 为伍"的经营理念，继续以"打造百年企业"为初心，深耕专业、全球布局，把论文写在生产线上，为医 药产业高质量发展、为健康中国建设贡献更多力量。 □ 本报记者李达 近日，经国家知识产权局公示，江苏诺泰澳赛诺生物制药股份有限公司(以下简称"诺泰生物")入选"国家 知识产权示范企业"创建对象名单。此外，诺泰生物近期还在多肽药物、寡核苷酸等多个领域实现新突 破，成为生物制药新质生产力的典型代 ...

Core Viewpoint - ST诺泰 (688076.SH) has received an administrative penalty decision, but this has not negatively impacted its current business performance, with significant revenue and profit growth reported for the first three quarters of 2025 [2] Group 1: Financial Performance - For the first three quarters of 2025, the company achieved revenue of 1.527 billion yuan, representing a year-on-year increase of 21.95% [2] - The net profit attributable to shareholders reached 445 million yuan, up 26.92% year-on-year, while the net profit excluding non-recurring items was 437 million yuan, reflecting a 23.20% increase [2] Group 2: Business Growth and Strategy - The company's peptide raw material drug business continues to grow significantly, driven by the expanding GLP-1 market, and is a core driver of its performance [2] - The company is implementing a development strategy focused on time and technology leadership, with a foundation in peptide drugs and small molecule drugs, while strategically expanding into oligonucleotide business and cutting-edge fields like synthetic biology and AI drug development [2] Group 3: Innovation and R&D - The company has made a significant breakthrough in innovative R&D, with its subsidiary, Hangzhou Nuoao Biopharmaceutical Technology Co., Ltd., receiving acceptance for a clinical trial application for the new drug Semaglutide tablets, marking a milestone in oral peptide delivery technology for weight management [2] - Currently, there are no approved oral Semaglutide formulations for weight management in the domestic and international markets, indicating a substantial market opportunity for the company [2] Group 4: Market Position and Collaborations - As a leading enterprise in the peptide raw material drug sector, the company has overcome technical bottlenecks in large-scale production of long-chain peptide drugs, achieving single-batch production of over 10 kilograms for drugs like Semaglutide and Tirzepatide [3] - The company has established collaborations with several leading generic drug companies for the raw materials and formulations of Semaglutide and Tirzepatide, maintaining high growth in peptide raw material sales [3] - The company has also announced comprehensive coverage of its GLP-1 pipeline products, with ongoing development of new targets like Retatrutide, which is set to begin development in early 2024 with an investment of approximately 5.08 million yuan [3] Group 5: Oligonucleotide and New Technologies - The company is actively involved in the oligonucleotide field, with plans for commercial production capacity expected to be completed by 2025, aiming for an annual output of 1,000 kilograms of oligonucleotides and 200 kilograms of PEG-conjugated cyclic peptides [4] - Collaborations with companies like Novogene and DeepTech in synthetic biology and AI-enabled areas are accelerating the exploration of new business fields, positioning the company for high-quality development across multiple tracks [4]

Core Insights - Capricor Therapeutics has announced new data on a scalable framework for loading therapeutic siRNAs and PMOs into exosomes, which is crucial for future clinical development [1][2][4] Group 1: Exosome Technology - The poster presented at the AAEV 2025 highlighted Capricor's proprietary exosome-based technology, showcasing strategies for scale-up and scale-out electroporation to achieve larger yields of engineered exosomes [2][4] - Key findings indicate that engineered exosomes derived from 293F cells can be successfully loaded with therapeutic siRNA and PMO using optimized electroporation conditions, with comparable loading efficiencies to standard methods [7] Group 2: Clinical Development - Establishing a scalable and reproducible loading process is essential for advancing Capricor's exosome pipeline for therapeutic development [4] - The framework developed provides a feasible pathway for producing clinically relevant quantities of loaded exosomes to support future clinical development [7] Group 3: Company Overview - Capricor Therapeutics is focused on advancing cell and exosome-based therapeutics, with its lead product candidate, Deramiocel, in late-stage clinical development for Duchenne muscular dystrophy (DMD) [5][9] - The company is leveraging its proprietary StealthX™ platform for preclinical development aimed at targeted delivery of therapeutics, with potential applications across a wide range of diseases [5]

Financial Data and Key Metrics Changes - In Q1 2025, the company achieved total net product revenue of $612 million, representing a 70% growth year-over-year [6][38] - Elevitus sales reached $375 million, marking a 180% increase compared to the same quarter last year [6][38] - The PMO franchise generated $237 million, up 5% year-over-year [6][38] - The company revised its net product revenue guidance for 2025 to a range of $2.3 billion to $2.6 billion, reflecting a 37% increase from 2024 at the midpoint [7][42] Business Line Data and Key Metrics Changes - The PMO franchise grew by 5%, while Elevitus saw a significant increase of 180% in sales [6][38] - Collaboration and other revenues amounted to $133 million, primarily from Roche [38] Market Data and Key Metrics Changes - The company noted that 60% of current revenue comes from top sites, which are experiencing high demand and long wait times for appointments [12] - The administrative process for gene therapy has become more complex, leading to longer turnaround times for infusions [10][18] Company Strategy and Development Direction - The company is focusing on addressing administrative challenges and enhancing site capacity to improve patient access to Elevitus [19] - A comprehensive promotional campaign for Elevitus was launched to increase awareness and understanding among healthcare providers and patients [20] - The company aims to expand its treatment network by engaging more community sites to alleviate capacity issues at top centers [19] Management's Comments on Operating Environment and Future Outlook - Management acknowledged the challenges faced in Q1 2025, including a severe flu season and a tragic safety event that impacted patient infusions [5][16] - The company remains optimistic about long-term demand for Elevitus, expecting a recovery in sales starting in the summer [17][64] - Management emphasized the importance of education and communication to address concerns stemming from the recent safety event [52][60] Other Important Information - The company reported a GAAP net loss of $448 million for Q1 2025, primarily due to increased R&D expenses related to a collaboration with Arrowhead [41] - The company has a strong cash position with $647 million in cash and equivalents, plus an additional $600 million available through a revolving credit facility [36][42] Q&A Session Summary Question: What has been the biggest driver of potential downside pressure leading to revised guidance? - Management indicated that it is a mix of all three factors: cycle times, administrative processes, and the safety event, with cycle times being the most mechanically impactful [47][48] Question: How does the company direct patients to sites with more capacity? - Management clarified that it is more about focus and education rather than the number of sites, emphasizing the need to engage secondary sites more effectively [57][58] Question: What percentage of sales does Elevitus represent in the new guidance? - Management confirmed that the revision in total net product revenue was exclusively related to Elevitus, which is the primary driver of the guidance change [66] Question: What are the expectations regarding the potential label update for Elevitus? - Management stated that a labeling supplement was submitted in April, with a target review date set for no later than Q4 2025 [76]