Olvimulogene nanivacirepvec

▎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

– Alignment reached with U.S. Food and Drug Administration on key elements of the approval pathway for Olvi-Vec in Platinum Resistant/Refractory Ovarian Cancer – – Positive Preliminary Phase 1b/2 Data of Olvi-Vec in Advanced Small-Cell Lung Cancer – – Matt Pulisic joined the company as its new Chief Financial Officer – – Closing of $10.5 Million Underwritten Offering of Common Stock – – $30.9 million in cash, cash equivalents and short-term investments – WESTLAKE VILLAGE, Calif. , March 28, 2025 (GLOBE NEWSWIRE) -- Genelux Corporation (NASDAQ: GNLX), a late clinical-stage immuno-oncology company, reported fourth quarter and full year 2024 financial results and business updates. "Our progress in the fourth quarter of 2024 and into 2025 marks a pivotal period for patients, our company, and our investors,” said Thomas Zindrick , President, CEO and Chairman of Genelux . “We are pleased with the continued progress and promise of our clinical development program. The strengthening of our management team, with the addition of Matt, and of our balance sheet, with our recent financing, further positions the Company to execute on our mission to transform the lives of patients who need it most." Pipeline Highlights OnPrime/GOG-3076 Phase 3 Registrational Trial Update The Company continues to enroll patients in the ongoing pivotal Phase 3 OnPrime/GOG-3076 registration trial (Phase 3 trial) in platinum resistant/refractory ovarian cancer (PRROC), with the primary endpoint of progression free survival (PFS) (Clinicaltrials.gov identifier NCT05281471), and continues to have productive discussions with the U.S. Food and Drug Administration (FDA). The Company recently concluded a productive Type D meeting with the FDA for Olvi-Vec in the treatment of PRROC. In response to a question seeking the FDA’s guidance on their expectations regarding a confirmatory trial using the ongoing Phase 3 trial results, the FDA responded that “As stated previously, an interim analysis of overall survival (OS) should be planned at the time of the primary PFS analysis. If a clinically meaningful PFS advantage is demonstrated in the absence of a decrement in OS, this could potentially support traditional approval.” The FDA further recommended Genelux request a pre-BLA meeting with FDA with topline safety and efficacy data following completion of the study to discuss next steps. This could potentially avoid the need for conducting a separate confirmatory study. The trial will enroll a sufficient number of patients to achieve 127 events, with the primary endpoint of PFS and with secondary endpoints including OS. The Company anticipates reporting topline data in the first half of 2026. Recurrent Lung Cancer Trials: Systemic Administration of Olvi-Vec Update Phase 1b/2 Olvi-Vec-SCLC-202 has generated preliminary safety and anti-tumor activity data from the dose escalation Phase 1b portion of the ongoing Phase 1b/2 clinical trial of Olvi-Vec immunochemotherapy in patients with platinum-relapsed or platinum-refractory extensive small cell lung cancer. The trial is co-sponsored by the Company and its licensing partner, Newsoara BioPharma Co., Ltd. (Newsoara). Systemic administration of Olvi-Vec in the initial dose escalation cohorts achieved a 71% disease control rate (5/7), with two partial responders. All participants with disease control experienced a reduction in all target lesions, with one participant achieving a tumor reduction of approximately 79%. Additionally, three participants, including one individual with three prior lines of treatment, achieved stable disease at lower dose cohorts, with tumor size reductions ranging between 24% to 29.2%. Olvi-Vec was generally well-tolerated with a favorable safety profile. Participant enrollment into dose escalation cohorts continues to investigate safety and the recommended intravenous dose of Olvi-Vec for Phase 2 portion of the trial, with updated interim results anticipated in the second half of 2025. Phase 2 VIRO-25 is actively enrolling recurrent non-small cell lung cancer (NSCLC) patients (NCT06463665), with interim data anticipated in second half of 2025. Business Updates Chief Financial Officer Matt Pulisic has joined the Company as its new Chief Financial Officer, effective January 30, 2025 . Mr. Pulisic is an accomplished, senior executive with over 19 years of finance and commercial experience in the biopharmaceutical industry, having worked across the United States , Europe , and Asia . He is recognized for his expertise in financial leadership, corporate strategy and operational execution. Underwritten Offering of Common Stock On March 26, 2025 , the Company closed an underwritten offering of 3,000,000 shares of its common stock at an offering price of $3.50 per share. The gross proceeds from the offering are expected to be $10.5 million , before deducting underwriting discounts and commissions and estimated offering expenses payable by the Company. The net proceeds from the offering are to be used for working capital and for general corporate purposes, including the continued clinical development of Olvi-Vec. Fourth Quarter and 2024 Financial Results Cash, cash equivalents and short-term investments were $30.9 million as of December 31, 2024 . The Company expects its cash, cash equivalents and short-term investments as of December 31, 2024 will provide runway into the first quarter of 2026. Research and development expenses were $19.0 million and $12.8 million for the years ended December 31, 2024 and 2023, respectively, an increase of approximately $6.2 million . Significant variations between periods are primarily a result of a $4.5 million increase in clinical and regulatory expenses relating to increased clinical trial costs associated with the Company’s Phase 3 On Prime Registration trial in 2024 and Phase 2 clinical trial for NSCLC, which the Company’s partner, Newsoara, is obligated to fully reimburse per the terms of their agreement; and a $1.2 million increase in employee compensation in 2024, primarily related to new employee hires in 2024. General and administrative expenses were $12.7 million and $11.6 million for the years ended December 31, 2024 and 2023, respectively, an increase of approximately $1.1 million . Significant variations between periods are primarily a result of a $0.2 million increase in employee compensation in 2024, a $0.8 million increase in stock compensation expense in 2024, due to the increase in the cost of stock options and restricted stock units in 2024, and a $0.4 million increase in consulting and contract labor expenses in 2024, primarily resulting from increased accounting and finance costs in 2024, partially offset by a $0.5 million decrease in professional services, primarily resulting from the decrease in legal expenses in 2024. Net loss was $29.9 million or $0.95 per share for the year ending December 31, 2024 , as compared to $28.3 million or $1.16 for the year ending December 31, 2023 . About Genelux Corporation Genelux is a late clinical-stage biopharmaceutical company focused on developing a pipeline of next-generation oncolytic immunotherapies for patients suffering from aggressive and/or difficult-to-treat solid tumor types. Olvi-Vec currently is being evaluated in two U.S. -based clinical trials: OnPrime/GOG-3076, a multi-center, randomized, open-label Phase 3 registrational trial evaluating the efficacy and safety of Olvi-Vec in combination platinum-doublet + bevacizumab compared with physician's choice of chemotherapy and bevacizumab in patients with platinum-resistant/refractory ovarian cancer; and, VIRO-25, a multi-center, randomized, open-label Phase 2 trial evaluating the efficacy and safety of Olvi-Vec & Platinum-doublet + physician's choice of immune checkpoint inhibitor compared to docetaxel in non-small-cell lung cancer. Additionally, Olvi-Vec currently is being evaluated for dose selection in Olvi-Vec-SCLC-202, a China -based, multi-center, open label Ph1b evaluating the efficacy and safety of Olvi-Vec & Platinum-doublet in recurrent small-cell lung cancer. The core of Genelux's discovery and development efforts revolves around its’ proprietary CHOICE™ platform from which the Company has developed an extensive library of isolated and engineered oncolytic vaccinia virus immunotherapeutic product candidates, including Olvi-Vec. For more information, please visit www.genelux.com and follow us on Twitter @Genelux_Corp and on LinkedIn. Forward-Looking Statements This 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, and such forward-looking statements are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. “Forward-looking statements” describe future expectations, plans, results, or strategies and are generally preceded by words such as “believes,” “anticipates,” “expect,” “may,” “plan” or “will”. Forward-looking statements in this release include, but are not limited to, statements related to Genelux’s future plans and prospects, the planned timing of Genelux’s data results in its ongoing clinical trials and continued development of Olvi-Vec, the potential capabilities advantages, safety and efficacy of Olvi-Vec and the potential regulatory approval pathway of Olvi-Vec. Such statements are subject to a multitude of risks and uncertainties that could cause future circumstances, events, or results to differ materially from those projected in the forward-looking statements. These and other risks are identified under the caption “Risk Factors” in Genelux’s filings with the Securities and Exchange Commission . All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management’s assumptions and estimates as of such date. Genelux does not undertake any obligation to publicly update any forward-looking statements, whether as a result of the receipt of new information, the occurrence of future events or otherwise. Investor and Media Contacts Ankit Bhargava , MD Allele Communications, LLC genelux@allelecomms.com Source: Genelux Corporation Source: Genelux Corporation