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▎药明康德内容团队编辑本期看点1. 用于治疗广泛期小细胞肺癌的溶瘤病毒免疫疗法Olvimulogene nanivacirepvec在一项早期临床试验中使71%患者的肿瘤得到控制。2. 在研病毒样颗粒偶联药物（VDC）bel-sar在治疗非肌层浸润性膀胱癌（NMIBC）患者的1期临床试验中使多名患者的肿瘤完全消失。3. iECURE公司公布了首个通过基因编辑，在肝脏细胞的DNA中插入整个基因的婴儿的完整数据。药明康德内容团队整理Olvimulogene nanivacirepvec：公布1b/2期临床试验数据Genelux公司和恒翼生物（Newsoara BioPharma）公布了其共同资助的溶瘤病毒免疫疗法Olvi-Vec用于治疗铂类复发或铂难治性广泛期小细胞肺癌的1b/2期临床试验结果。Olvi-Vec是Genelux公司专有的溶瘤病毒疫苗，经过改良以提高其安全性、肿瘤选择性和治疗潜力。临床前研究结果表明，Olvi-Vec具有在体外和体内感染并直接杀死多种肿瘤细胞的潜力。2021年9月，Genelux公司授予恒翼生物在大中华区开发和商业化Olvi-Vec的独家许可。此次公布的结果表明，在初始剂量递增队列中，Olvi-Vec的疾病控制率（DCR）达71%（5/7），其中有两名患者达到部分缓解（PR）。所有实现疾病控制的受试者均观察到所有靶病灶的缩小，其中1名患者的肿瘤缩小了约79%。低剂量组中有3名患者实现疾病稳定（SD），肿瘤缩小幅度在24%-29.2%之间。安全性方面，Olvi-Vec总体安全性和耐受性良好。Bel-sar（AU-011）：公布1期临床试验数据Aura Biosciences公司宣布，其在研病毒样颗粒偶联药物bel-sar在治疗非肌层浸润性膀胱癌患者的1期临床试验中获得积极早期数据。Bel-sar由靶向肿瘤表面表达的糖蛋白的病毒样颗粒（VLP）与可由光激活的细胞毒性药物偶联生成。它被设计为具有双重作用机制，既能诱导肿瘤细胞坏死，又能激发强大且持久的抗肿瘤免疫反应。该试验共纳入了15名患者，主要终点是评估bel-sar单独使用（n=5）和bel-sar结合光激活（n=10）局部给药的安全性和可行性。次要终点是评估生物活性及肿瘤微环境（TME）中的免疫介导变化。所有单独接受bel-sar的患者（n=5）均为中危NMIBC，治疗后没有患者出现临床完全缓解（CR）或肿瘤缩小。在接受bel-sar结合光激活治疗的5名中危疾病患者中，有4例表现出目标病灶的临床CR，在组织病理学评估中未检测到肿瘤细胞。在接受bel-sar结合光激活治疗的5名高危疾病患者中，有1例表现出临床CR，3例在膀胱镜检查中观察到肿瘤缩小。此外，在结合光激活治疗队列中，7名多发性肿瘤患者中有4名（57%）在至少一处非靶病灶中表现出临床CR，伴有CD8+和CD4+效应T细胞浸润。此外，bel-sar具有良好的安全性，仅在不到10%的患者中报告了1级药物相关不良事件。ECUR-506：公布1/2期临床试验中首例患儿的数据iECURE公司公布了其正在进行的1/2期研究OTC-HOPE中的首例患儿的积极数据，该研究旨在评估基因编辑疗法ECUR-506治疗新生儿发病的鸟氨酸氨甲酰转移酶（OTC）缺乏症的效果。ECUR-506依赖于使用两种携带不同有效载荷但具有相同衣壳的腺相关病毒（AAV）载体。一种携带ARCUS核酸酶，靶向已被充分研究的PCSK9基因位点进行基因编辑，另一种是供体载体，用于插入功能性OTC基因。在PCSK9位点的基因编辑为安全插入OTC基因提供了潜在路径，从而可能实现功能性基因的永久表达。此前的新闻稿表示，该患儿是首个通过基因编辑，在肝脏细胞的DNA中插入整个基因的婴儿，代表着体内基因编辑领域的重要里程碑。此次公布的结果表明，这名婴儿接受ECUR-506治疗后总体耐受性良好，在单次给药最低剂量的ECUR-506治疗后12周获得完全临床缓解。该患儿停止使用氨清除药物，且每日平均蛋白质摄入量增加至符合年龄标准的水平。缺乏OTC基因的患儿由于尿素生成受损，其血尿素氮（BUN）水平通常较低。观察到ECUR-506治疗后的平均BUN水平与治疗前的平均值相比明显更高，接近正常下限，表明该患儿的功能性OTC酶的活性可能增加。NBTXR3（JNJ-1900）：公布1期临床试验数据Nanobiotix公司公布了其潜在“first-in-class”的新型放射增敏剂NBTXR3的一项1期临床试验的积极数据，该研究旨在评估NBTXR3作为二线或后线（2L+）疗法，用于适合再接受放疗的局部晚期非小细胞肺癌（NSCLC）患者。NBTXR3由功能化二氧化铪（HfO2）纳米颗粒组成，经由一次性瘤内注射给药并通过放射疗法激活。它的物理作用机制为：旨在通过放疗激活，诱导被注射肿瘤内大量的肿瘤细胞死亡，随后触发适应性免疫反应和长期的抗癌记忆。得益于该物理作用机制，Nanobiotix公司认为NBTXR3可扩展到任何可以通过放疗治疗的实体肿瘤和任何联合治疗方案中，特别是与免疫检查点抑制剂联合。2023年，Nanobiotix宣布与强生旗下杨森公司（现名为强生创新制药）达成全球共同开发和商业化NBTXR3的许可协议。此次公布的研究结果显示，在12名适合再次放疗且既往治疗失败的局部晚期非小细胞肺癌患者中，NBTXR3的安全性良好，并确认了注射的可行性。对生存数据的初步审查显示，患者的12个月的局部无进展生存率（LPFS）为64%，12个月的总生存率（OS）为83%。截至目前，剂量递增部分已完成，扩展研究部分已有5/12名患者接受了注射。STM-01：1期临床试验完成首例患者给药Secretome Therapeutics公司宣布，其干细胞疗法STM-01用于治疗射血分数保留型心力衰竭（HFpEF）的1期临床试验已完成首例患者给药。新闻稿指出，这是全球首个针对HFpEF的同种异体、现货型干细胞疗法的临床研究。STM-01是一种新生儿心脏祖细胞（nCPC），具有减少纤维化和改善线粒体功能的能力，目前正在开发用于治疗HFpEF和扩张型心肌病（DCM）。在临床前研究中，STM-01显著降低了HFpEF中细胞和分子水平的炎症，从而改善心脏功能和运动耐量。VERVE-102：IND申请获得FDA许可Verve Therapeutics公司宣布，其体内碱基编辑药物VERVE-102的IND申请已获得美国FDA批准，用于治疗杂合子家族性高胆固醇血症（HeFH）和/或早发性冠状动脉疾病（CAD）患者。VERVE-102旨在使肝脏中的PCSK9基因永久失活，目前正被开发用于治疗杂合子家族性高胆固醇血症，其最终目的是用于治疗那些因低密度脂蛋白胆固醇（LDL-C）水平高而继续受到影响的动脉粥样硬化性心血管疾病（ASCVD）患者。▲欲了解更多前沿技术在生物医药产业中的应用，请长按扫描上方二维码，即可访问“药明直播间”，观看相关话题的直播讨论与精彩回放参考资料（可上下滑动查看）[1] iECURE Announces Presentation of Full Data for the First Infant Dosed with ECUR-506 in OTC-HOPE Phase 1/2 Clinical Trial at the 2025 ACMG Annual Clinical Genetics Meeting. Retrieved March 28, 2025, from https://iecure.com/news/iecure-announces-presentation-of-full-data-for-the-first-infant-dosed-with-ecur-506-in-otc-hope-phase-1-2-clinical-trial-at-the-2025-acmg-annual-clinical-genetics-meeting/[2] Verve Therapeutics Announces Clearance of Investigational New Drug Application by the U.S. FDA for VERVE-102, an Investigational Gene Editing Medicine Designed to Durably Lower Cholesterol After a Single Dose. Retrieved March 28, 2025, from https://www.globenewswire.com/news-release/2025/03/24/3047698/0/en/Verve-Therapeutics-Announces-Clearance-of-Investigational-New-Drug-Application-by-the-U-S-FDA-for-VERVE-102-an-Investigational-Gene-Editing-Medicine-Designed-to-Durably-Lower-Chole.html[3] Positive Data from Phase 1 Trial of Bel-sar in Patients with Non-Muscle-Invasive Bladder Cancer (NMIBC) Presented at the 40th Annual European Association of Urology Congress. Retrieved March 28, 2025, from https://www.globenewswire.com/news-release/2025/03/24/3047703/0/en/Positive-Data-from-Phase-1-Trial-of-Bel-sar-in-Patients-with-Non-Muscle-Invasive-Bladder-Cancer-NMIBC-Presented-at-the-40th-Annual-European-Association-of-Urology-Congress.html[4] Genelux and Newsoara Announce Positive Preliminary Phase 1b/2 Data of Olvi-Vec in Advanced Small-Cell Lung Cancer. Retrieved March 28, 2025, from https://www.globenewswire.com/news-release/2025/03/25/3048735/0/en/Genelux-and-Newsoara-Announce-Positive-Preliminary-Phase-1b-2-Data-of-Olvi-Vec-in-Advanced-Small-Cell-Lung-Cancer.html[5] Secretome Therapeutics Announces First Patient Dosed in Groundbreaking Phase 1 Study of STM-01 in Heart Failure with Preserved Ejection Fraction. Retrieved March 28, 2025, from https://secretometherapeutics.com/2025/03/secretome-therapeutics-announces-first-patient-dosed-in-groundbreaking-phase-1-study-of-stm-01-in-heart-failure-with-preserved-ejection-fraction/[6] Nanobiotix Announces First Data From the Completed Dose Escalation Part of a Phase 1 Study Evaluating NBTXR3 (JNJ-1900) as a 2L+ Therapy for Patients With Locally Advanced NSCLC. Retrieved March 28, 2025, from https://www.globenewswire.com/news-release/2025/03/27/3051020/0/en/Nanobiotix-Announces-First-Data-From-the-Completed-Dose-Escalation-Part-of-a-Phase-1-Study-Evaluating-NBTXR3-JNJ-1900-as-a-2L-Therapy-for-Patients-With-Locally-Advanced-NSCLC.html[7] Nouscom to Present Final Results from Successful Phase Ib/II Trial of NOUS-209 in People Living with Lynch Syndrome, Demonstrating Powerful Potential to Intercept Cancer in its Earliest Stages of Development. Retrieved March 28, 2025, from https://nouscom.com/2025/03/25/nouscom-to-present-final-results-from-successful-phase-ib-ii-trial-of-nous-209-in-people-living-with-lynch-syndrome-demonstrating-powerful-potential-to-intercept-cancer-in-its-earliest-stages-of-deve/[8] Calidi Biotherapeutics and City of Hope Provide Update on a Phase 1 Clinical Trial with CLD-101 Virotherapy in Patients with Recurrent High-Grade Glioma. Retrieved March 28, 2025, from https://www.globenewswire.com/news-release/2025/03/26/3049641/0/en/Calidi-Biotherapeutics-and-City-of-Hope-Provide-Update-on-a-Phase-1-Clinical-Trial-with-CLD-101-Virotherapy-in-Patients-with-Recurrent-High-Grade-Glioma.html[9] Helicore Biopharma Announces First Participant Dosed in Phase 1 Clinical Trial of HCR-188, a First-in-Class GIP Antagonist for the Treatment of Obesity. Retrieved March 28, 2025, from https://www.globenewswire.com/news-release/2025/03/25/3048810/0/en/Helicore-Biopharma-Announces-First-Participant-Dosed-in-Phase-1-Clinical-Trial-of-HCR-188-a-First-in-Class-GIP-Antagonist-for-the-Treatment-of-Obesity.html[10] Oncotelic Therapeutics Announces Successful Completion of Phase 1 Trial of OT-101 and IL-2, Highlights Findings at SWCR 2025 Conference. Retrieved March 28, 2025, from https://www.oncotelic.com/press-releases/?[11] Neuron23 to Present Phase 1 Healthy Volunteer Data of NEU-411, a Brain-penetrant LRRK2 Inhibitor, at AD/PD™ 2025. Retrieved March 28, 2025, from https://neuron23.com/neuron23-to-present-phase-1-healthy-volunteer-data-of-neu-411-a-brain-penetrant-lrrk2-inhibitor-at-ad-pd-2025/[12] Personalized Cancer Vaccine Proves Promising in a Phase 1 Trial at Mount Sinai. Retrieved March 28, 2025, from https://www.globenewswire.com/news-release/2025/03/21/3047122/0/en/Personalized-Cancer-Vaccine-Proves-Promising-in-a-Phase-1-Trial-at-Mount-Sinai.html[13] Entrada Therapeutics Receives Authorization in the United Kingdom to Initiate ELEVATE-45-201, a Phase 1/2 Multiple Ascending Dose Clinical Study of ENTR-601-45 in People Living with Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping. Retrieved March 28, 2025, from https://www.globenewswire.com/news-release/2025/03/24/3047704/0/en/Entrada-Therapeutics-Receives-Authorization-in-the-United-Kingdom-to-Initiate-ELEVATE-45-201-a-Phase-1-2-Multiple-Ascending-Dose-Clinical-Study-of-ENTR-601-45-in-People-Living-with.html免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：本文来自药明康德内容团队，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。分享，点赞，在看，聚焦全球生物医药健康创新

- Recent ODYSSEY Phase 2b Trial of Suprachoroidal CLS-AX in Wet AMD Achieved All Primary and Secondary Outcomes - - Positive Topline Results Support Advancing CLS-AX to Phase 3 Targeting a Differentiated Flexible Dosing Approach Similar to a Biologic with the Potential Extended Duration of a Tyrosine Kinase Inhibitor (TKI) - - Recent Commercial Licensing Agreement for China by a Global Ophthalmic Pharmaceutical Company Provides Strategic Validation of Clearside’s Suprachoroidal Platform - - Compelling Data Presentations at AAO Demonstrated Potential Safety and Efficacy Benefits of Suprachoroidal Delivery Using Clearside’s Proprietary SCS Microinjector® in Multiple Clinical Programs - - Management to Host Webcast and Conference Call Today at 4:30 P.M. ET - ALPHARETTA, Ga., Nov. 12, 2024 (GLOBE NEWSWIRE) -- Clearside Biomedical, Inc. (Nasdaq: CLSD), a biopharmaceutical company revolutionizing the delivery of therapies to the back of the eye through the suprachoroidal space (SCS®), today reported financial results for the third quarter ended September 30, 2024, and provided a corporate update. “We are making outstanding progress advancing our differentiated suprachoroidal delivery pipeline,” said George Lasezkay, PharmD, JD, President and Chief Executive Officer. “The recent positive results from our ODYSSEY trial establish CLS-AX as a Phase 3 ready asset in the large and growing wet AMD market. We are positioning CLS-AX for real-world success by focusing on a Phase 3 program in wet AMD designed to evaluate extended efficacy duration compared to current standard of care intravitreal products and produce data supportive of a prescribing label that enables physicians to take advantage of flexible maintenance dosing between 3 and 6 months. We look forward to conducting an End-of-Phase 2 meeting with the FDA in early 2025 to align on the essential components of our Phase 3 program.” “As we work to expand the overall value of our suprachoroidal drug delivery platform, we are seeing significant interest among the retinal specialist community and from leading biopharmaceutical companies in applying our innovative approach to treating serious retinal diseases. The recent commercial collaboration announced by Santen Pharmaceutical Co. and our Asia-Pacific partner, Arctic Vision, is a compelling validation of our suprachoroidal platform from a well-respected leader in the global ophthalmic industry. The licensing of ARVN001, branded as XIPERE® in the U.S., is part of Santen’s commitment to bringing innovative eyecare solutions to patients in China,” concluded Dr. Lasezkay. Victor Chong, M.D., MBA, Chief Medical Officer and EVP, Head of Research & Development, added, “In addition to our partners’ promising programs, our research team is currently evaluating various small molecules through in vivo models for the potential treatment of geographic atrophy (GA), with a market size valued at over $20 billion in sales. We believe that GA is primarily a choroidal disease. Delivery of small molecules via suprachoroidal injection enables comprehensive drug coverage of both the retina and choroid, while potentially minimizing systemic and anterior segment side effects.” Key Highlights The ODYSSEY Phase 2b clinical trial of CLS-AX (axitinib injectable suspension) for the treatment of neovascular age-related macular degeneration (wet AMD) achieved its primary and secondary outcomes, demonstrated extended duration, stable vision and anatomic measures and a well-tolerated safety profile.Clearside’s Asia-Pacific partner, Arctic Vision, signed a new commercial collaboration with Santen Pharmaceutical Co., Ltd. for commercial rights in China to ARVN001, Arctic Vision’s triamcinolone acetonide injectable suspension for suprachoroidal use, in the treatment of uveitic macular edema (UME) and certain other ophthalmic indications under development. ARVN001 is branded as XIPERE in the U.S. and ARCATUS® in China.Arctic Vision reported positive topline results from its Phase 3 clinical trial of ARCATUS for the treatment of UME in China and announced that New Drug Applications for ARCATUS are under review by regulators in Australia and Singapore.Tony Gibney was appointed Chair of Clearside’s Board of Directors, effective November 1, 2024, succeeding Clay Thorp, who will continue serving as a member of the Board. Mr. Gibney joined Clearside’s Board as an independent director in April 2024 and is an experienced biotechnology executive and former investment banker, most recently serving as Executive Vice President, Chief Business & Strategy Officer, of Iveric Bio, Inc. until the company’s acquisition by Astellas Pharma Inc. in July 2023.Glenn Yiu, MD, PhD, Professor of Ophthalmology at the University of California, Davis, was appointed to Clearside’s Scientific Advisory Board in July 2024. Dr. Yiu, a board-certified vitreoretinal surgeon, leads the translational research program at UC Davis studying AMD and other retinal diseases, with a focus on ocular imaging technologies, gene editing and delivery, and animal models of retinal disease.An article was published titled “Early Adoption of Triamcinolone Acetonide Suprachoroidal Injection for UME: A Physician Survey” by Christopher R. Henry et al. that summarizes physicians’ “real-world” perspectives on early experiences with XIPERE for the treatment of patients with UME. Findings from this survey indicate that the suprachoroidal injection technique was easy to learn (92% found the injection procedure relatively easy post-training) and resulted in favorable patient outcomes consistent with clinical trial data. The full publication can be accessed here.Clearside’s gene therapy partner, REGENXBIO, reported on both of their programs administering ABBV-RGX-314 via Clearside’s SCS Microinjector. Based on positive interim results to date from the Phase 2 ALTITUDE® trial in diabetic retinopathy (DR), AbbVie and REGENXBIO announced that they have accelerated a planned End-of-Phase 2 meeting with the FDA expected this quarter. REGENXBIO expects to initiate the first global pivotal trial in DR in the first half of 2025. In addition, the ALTITUDE trial is enrolling a new cohort of patients with center-involved diabetic macular edema. In wet AMD, based on a favorable safety profile and to evaluate dose levels for a planned pivotal program, the Phase 2 AAVIATE® trial is enrolling a new cohort.In September 2024, Clearside’s ocular oncology partner, Aura Biosciences, presented positive Phase 2 end-of-study results evaluating bel-sar (AU-011) for the first-line treatment of early-stage choroidal melanoma at The Retina Society Annual Meeting. Bel-sar is being administered via Clearside’s SCS Microinjector.Multiple presentations were delivered at the 2024 Annual Meeting of the American Academy of Ophthalmology (AAO) and preceding events that highlighted encouraging safety and efficacy data from clinical trials of therapies utilizing Clearside’s SCS Microinjector to deliver drugs into the suprachoroidal space to treat a variety of retinal diseases. Third Quarter 2024 Financial Results License and other revenue for the third quarter of 2024 was $1.0 million, compared to $0.9 million for the third quarter of 2023. The revenue primarily related to payments pursuant to Clearside’s license agreements and revenue for services and the sales of SCS Microinjector kits to licensees.Research and development expenses for the third quarter of 2024 were $4.1 million, compared to $5.1 million for the third quarter of 2023. This decrease was primarily due to a $1.9 million decrease in costs related to the CLS-AX program, which was partially offset by increases in employee-related costs and device development and a research and development tax credit received in the prior year.General and administrative expenses were $2.8 million for the third quarter of 2024, compared to $2.6 million for the third quarter of 2023. This increase was primarily due to an increase in patent-related expenses and consulting fees.Net loss for the third quarter of 2024 was $7.7 million, or $0.10 per share of common stock, compared to net loss of $9.3 million, or $0.15 per share of common stock, for the third quarter of 2023.As of September 30, 2024, Clearside’s cash, cash equivalents and short-term investments totaled $23.6 million. The Company believes it will have sufficient resources to fund its planned operations into the third quarter of 2025. Conference Call & Webcast Details Clearside’s management will host a webcast and conference call today at 4:30 p.m. Eastern Time to discuss the financial results and provide a corporate update. The live and archived webcast may be accessed on the Clearside website under the Investors section: Events and Presentations. The live call can be accessed by dialing (877) 545-0523 (domestic) or (973) 528-0016 (international) and entering conference code: 756568. The Company suggests participants join 15 minutes in advance of the event. About Clearside Biomedical, Inc. Clearside Biomedical, Inc. is a biopharmaceutical company revolutionizing the delivery of therapies to the back of the eye through the suprachoroidal space (SCS®) to improve patient outcomes. Clearside’s SCS injection platform, utilizing the Company’s patented SCS Microinjector®, enables an in-office, repeatable, non-surgical procedure for the targeted and compartmentalized delivery of a wide variety of therapies to the macula, retina, or choroid to potentially preserve and improve vision in patients with sight-threatening eye diseases. Clearside is developing its own pipeline of small molecule product candidates for administration via its SCS Microinjector. The Company’s lead program, CLS-AX (axitinib injectable suspension), for the treatment of neovascular age-related macular degeneration (wet AMD), recently completed a Phase 2b clinical trial, and planning for a Phase 3 program is underway. Clearside developed and gained approval for its first product, XIPERE® (triamcinolone acetonide injectable suspension) for suprachoroidal use, which is available in the U.S. through a commercial partner. Clearside also strategically partners its SCS injection platform with companies utilizing other ophthalmic therapeutic innovations. For more information, please visit clearsidebio.com or follow us on LinkedIn and X. Cautionary Note Regarding Forward-Looking Statements Any statements contained in this press release that do not describe historical facts may constitute forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995. These statements may be identified by words such as “believe”, “expect”, “may”, “plan”, “potential”, “will”, and similar expressions, and are based on Clearside’s current beliefs and expectations. These forward-looking statements include statements regarding the clinical development of CLS-AX, the potential benefits of CLS-AX, Clearside’s suprachoroidal delivery technology and Clearside’s SCS Microinjector® and Clearside’s ability to fund its operations into the third quarter of 2025. These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements. Risks and uncertainties that may cause actual results to differ materially include uncertainties inherent in the conduct of clinical trials, Clearside’s reliance on third parties over which it may not always have full control and other risks and uncertainties that are described in Clearside’s Annual Report on Form 10-K for the year ended December 31, 2023, filed with the U.S. Securities and Exchange Commission (SEC) on March 12, 2024, Clearside’s Quarterly Report on Form 10-Q for the quarter ended June 30, 2024 filed with the SEC on August 12, 2024 and Clearside’s other Periodic Reports filed with the SEC. Any forward-looking statements speak only as of the date of this press release and are based on information available to Clearside as of the date of this release, and Clearside assumes no obligation to, and does not intend to, update any forward-looking statements, whether as a result of new information, future events or otherwise. *References XIPERE® (triamcinolone acetonide injectable suspension) for suprachoroidal use is being commercialized by Bausch + Lomb who has the exclusive license for the commercialization and development of XIPERE in the United States and Canada. Arctic Vision has the exclusive license for the commercialization and development of XIPERE, in Greater China, South Korea, Australia, New Zealand, India and the ASEAN Countries. XIPERE is approved by the U.S. Food and Drug Administration and is commercially available in the U.S. A link to the full prescribing information is available at https://www.xipere.com/hcp/#isi.Source: IMARC Geographic Atrophy Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2024-2034ALTITUDE® and AAVIATE® are registered trademarks of REGENXBIO, Inc. Investor and Media Contacts: Jenny Kobin Remy Bernarda ir@clearsidebio.com(678) 430-8206 -Financial Tables Follow- CLEARSIDE BIOMEDICAL, INC.Selected Financial Data (in thousands, except share and per share data)(unaudited) Statements of Operations Data Three Months EndedSeptember 30, Nine Months EndedSeptember 30, 2024 2023 2024 2023 License and other revenue $1,038 $859 $1,358 $1,881 Operating expenses: Cost of goods sold — 142 — 355 Research and development 4,128 5,134 14,346 14,533 General and administrative 2,844 2,637 8,745 8,922 Total operating expenses 6,972 7,913 23,091 23,810 Loss from operations (5,934) (7,054) (21,733) (21,929)Interest income 338 409 1,104 1,359 Other income, net 365 — 783 — Non-cash interest expense on liability related to the sales of future royalties (2,457) (2,622) (7,200) (7,083)Net loss $(7,688) $(9,267) $(27,046) $(27,653)Net loss per share of common stock — basic and diluted $(0.10) $(0.15) $(0.37) $(0.45)Weighted average shares outstanding — basic and diluted 74,745,415 61,983,987 73,115,896 61,605,648 Balance Sheet DataSeptember 30, December 31, 2024 2023 Cash and cash equivalents$13,888 $28,920 Short-term investments 9,703 — Total assets 29,161 34,018 Liabilities related to the sales of future royalties, net 49,188 41,988 Warrant liabilities 8,757 — Total liabilities 63,950 49,930 Total stockholders’ deficit (34,789) (15,912) Source: Clearside Biomedical, Inc.