Ixoberogene soroparvovec

REDWOOD CITY, Calif., April 15, 2025 (GLOBE NEWSWIRE) -- Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage company pioneering the use of gene therapy to preserve sight for life in highly prevalent ocular diseases, today reported financial results for the fourth quarter and full year 2024. The company also provided pipeline highlights and updates to anticipated milestones. “We believe Ixo-vec is poised to redefine the standard of care for patients with wet AMD as a potential best-in-class, one-and-done gene therapy. Ixo-vec offers the potential for life-long vision preservation and a favorable safety profile and is overwhelmingly preferred by patients over today’s treatment, as evidenced by our LUNA 52-week and OPTIC 4-year data,” stated Laurent Fischer, M.D., president and chief executive officer of Adverum Biotechnologies. “2025 is off to a strong start with the initiation of our first pivotal trial, ARTEMIS. Designed to enhance the potential for clinical, regulatory and commercial success, ARTEMIS will evaluate a broad patient population, including treatment naïve patients and patients with a high treatment burden, to provide confidence to retina specialists to prescribe Ixo-vec, if approved, across the spectrum of wet AMD patients. We look forward to sharing the design of our second pivotal trial, AQUARIUS, and to presenting long-term follow-up data from LUNA later this year.” Ixo-vec Program Highlights Initiated ARTEMIS, the first-ever registrational intravitreal gene therapy trial in patients with wet AMD ARTEMIS is designed to derisk the clinical, regulatory and commercial path to approval. The inclusion of both treatment-experienced and treatment-naïve patients is expected to increase the speed of enrollment and generate a broad registrational dataset representative of real-world patient demographics to inform prescribers.ARTEMIS is a US-based study evaluating a single administration of Ixo-vec (6E10 vg/eye) compared to on-label aflibercept (2mg) every 8 weeks in approximately 284 patients with wet AMD.The primary endpoint is mean change from baseline of best corrected visual acuity (BCVA) at one year (average of weeks 52 and 56) with a non-inferiority margin of -4.5 letters.Per FDA guidance, all patients will receive three loading doses of aflibercept prior to receiving Ixo-vec. Patients in both arms will be eligible for supplemental injections of aflibercept and will receive prophylactic steroid eye drops.ARTEMIS is the first of two planned Phase 3 registrational trials to evaluate Ixo-vec in patients with wet AMD. Details on the second similar global study, AQUARIUS, are forthcoming. Presented Phase 2 LUNA 52-week trial results LUNA data support selection of 6E10 dose with topical eyedrops for pivotal program, providing evidence of potential best-in-class efficacy with favorable long-term safety.6E10 dose in LUNA maintained visual and anatomic endpoints and continued to demonstrate potential best-in-class injection-free rates and reduction in injection burden.Aflibercept levels were in the therapeutic range similar to those observed in OPTIC.Ixo-vec was well tolerated, with local steroids effectively managing inflammation at both the higher 2E11 dose and the “go-forward” 6E10 dose.Results from the LUNA pre-specified Patient Preference Survey demonstrate overwhelming preference for Ixo-vec over prior anti-VEGF therapies and for the acceptability of the topical steroid prophylaxis. Presented OPTIC 4-year trial results Despite significant treatment need at baseline, patients in OPTIC continued to experience long-term benefit from Ixo-vec through at least 4 years of follow up, including maintenance of vision, durability of anatomical improvements and sustained reduction in anti-VEGF treatment burden.Stable aflibercept levels have been demonstrated as long as measured, up to 5 years post treatment.Ixo-vec at 2E11 was generally well tolerated and demonstrated a favorable safety profile. Long-term data establish a 10-fold safety margin from highest dose tested in wet AMD to the 6E10 dose selected for Phase 3. Other Ixo-vec updates “Nonclinical Study of Ixo-vec Gene Therapy for nAMD Supports Efficacy for a Human Dose of 6E10 vg/eye and Staggered Dosing of Fellow Eyes” published in Molecular Therapy – Methods and Clinical Development. This study supports staggered contralateral dosing of Ixo-vec, and dosing Ixo-vec at the 6E10 dose explored in LUNA, in patients with wet AMD. Upcoming Anticipated Milestones 2H 2025 Initiate the global AQUARIUS Phase 3 trial4Q 2025 Present Phase 2 LUNA two-year long-term follow-up data Financial Results for the Three Months Ended December 31, 2024 Cash, cash equivalents and short-term investments were $125.7 million as of December 31, 2024, compared to $153.2 million as of September 30, 2024, and $96.5 million as of December 31, 2023. Adverum expects its cash, cash equivalents and short-term investments to fund operations into the second half of 2025. Research and development expenses were $24.1 million for the three months ended December 31, 2024, compared to $15.3 million for the same period in 2023. Research and development expenses increased due to higher material production and bioanalytics expenses and higher compensation expenses in preparation for Phase 3 clinical trials. Stock-based compensation expense included in research and development expenses was $1.2 million for the fourth quarter of 2024. General and administrative expenses were $18.5 million for the three months ended December 31, 2024, compared to $11.7 million for the same period in 2023. General and administrative expenses increased due to increases in a non-cash tenant improvement allowance made available to a subtenant for our North Carolina premises and higher consultant fees. Stock-based compensation expense included in general and administrative expenses was $2.0 million for the fourth quarter of 2024. Net loss was $40.9 million, or $1.96 per basic and diluted share, for the three months ended December 31, 2024, compared to $24.5 million, or $2.42 per basic and diluted share for the same period in 2023. About Ixo-vec in Wet AMD Adverum is developing ixoberogene soroparvovec (Ixo-vec, formerly referred to as ADVM-022), its clinical-stage gene therapy product candidate, for the treatment of wet AMD. Ixo-vec utilizes a proprietary vector capsid, AAV.7m8, carrying an aflibercept coding sequence under the control of a proprietary expression cassette. Unlike other ophthalmic gene therapies that require surgery to administer the gene therapy under the retina (sub-retinal approach), Ixo-vec is designed to be administered as a one-time IVT injection in the physician’s office, deliver long-term efficacy, reduce the burden of frequent anti-VEGF, optimize patient compliance and improve vision outcomes for patients with wet AMD. In recognition of the need for new treatment options for wet AMD, FDA granted Fast Track and Regenerative Medicine Advanced Therapy (RMAT) designations for Ixo-vec for the treatment of wet AMD. Ixo-vec has also received PRIME designation from the EMA and the Innovation Passport from the United Kingdom’s Medicines and Healthcare Products Regulatory Agency for the treatment of wet AMD. About Adverum Biotechnologies Adverum Biotechnologies (NASDAQ: ADVM) is a clinical-stage company that aims to establish gene therapy as a new standard of care for highly prevalent ocular diseases with the aspiration of developing functional cures to restore vision and prevent blindness. Leveraging the capabilities of its proprietary intravitreal platform, Adverum is developing durable, single-administration therapies, designed to be delivered in physicians’ offices, to eliminate the need for frequent ocular injections to treat these diseases. Adverum is evaluating its novel gene therapy candidate, ixoberogene soroparvovec (Ixo-vec, formerly referred to as ADVM-022), as a one-time, IVT injection for patients with neovascular or wet age-related macular degeneration. Additionally, by overcoming the challenges associated with current treatment paradigms for debilitating ocular diseases, Adverum aspires to transform the standard of care, preserve vision, and create a profound societal impact around the globe. For more information, please visit www.adverum.com. Forward-looking Statements Statements contained in this press release regarding events or results that may occur in the future are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements include but are not limited to statements regarding: the potential of Ixo-vec to redefine treatment standards as a potential best-in-class, one-and-done gene therapy; the therapeutic and commercial potential of Ixo-vec; potential benefits of Ixo-vec, including the potential for life-long vision preservation and its favorable long-term safety profile; the ability to generate data that provides confidence to retina specialists to prescribe Ixo-vec; plans and milestones related to Adverum’s product candidates, including the planned initiation of the global AQUARIUS Phase 3 trial and the presentation of LUNA Phase 2 two-year long-term follow-up data; Adverum’s cash sufficiency and runway; the potential of Ixo-vec to redefine the standard of care for patients with wet AMD; the ability to establish gene therapy as a standard of care for wet AMD patients and other statements that are not a historical fact. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, including risks inherent to, without limitation: Adverum’s novel technology, which makes it difficult to predict the timing of commencement and completion of clinical trials; regulatory uncertainties; enrollment uncertainties; the results of early clinical trials not always being predictive of future clinical trials and results; the potential for future complications or side effects in connection with use of Ixo-vec; and risks associated with market conditions. Additional risks and uncertainties facing Adverum are set forth under the caption “Risk Factors” and elsewhere in Adverum’s Securities and Exchange Commission (SEC) filings and reports, including Adverum’s most recent Annual Report on Form 10-K and subsequent filings with the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Adverum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law. Corporate & Investor Inquiries:Adverum Investor RelationsEmail: ir@adverum.com Media:Jason Awe, Ph.D.Executive Director, Corporate CommunicationsEmail: jawe@adverum.com Adverum Biotechnologies, Inc.Selected Consolidated Balance Sheet Data(In thousands) December 31 December 31 2024 2023(Restated) Cash and cash equivalents, and marketable securities$125,691 $96,526 Total assets 179,841 155,768 Total current liabilities 22,898 19,981 Total stockholders' equity 70,714 67,222 Adverum Biotechnologies, Inc.Condensed Consolidated Statements of Operations(In thousands except per share data) Three months ended December 31, Years ended December 31, 2024 2023(Restated) 2024 2023(Restated) (Unaudited) (1) License revenue$- $- $1,000 $3,600 Operating expenses: Research and development 24,095 15,278 77,041 77,486 General and administrative 18,523 11,670 63,118 55,056 Total operating expenses 42,618 26,948 140,159 132,542 Operating loss (42,618) (26,948) (139,159) (128,942) Other income, net 1,687 1,311 8,232 5,748 Net loss before income taxes (40,931) (25,637) (130,927) (123,194) Income tax provision - 1,133 - 1,078 Net loss (40,931) (24,504) (130,927) (122,116) Net loss per share — basic and diluted$(1.96) $(2.42) $(6.62) $(12.11) Weighted-average common shares outstanding - basic and diluted 20,898 10,121 19,782 10,082 (1) Derived from Adverum’s annual audited consolidated financial statements.

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12、3月4日，CDE官网显示，默沙东（MSD）申报的来特莫韦微片上市申请拟纳入优先审评，用于接受异基因造血干细胞移植（HSCT）的巨细胞病毒（CMV）血清学阳性的成人和6个月及以上且体重≥6kg的儿童受者[R+]预防巨细胞病毒感染和巨细胞病毒病。来特莫韦是一种新型非核苷类巨细胞病毒（CMV）抑制剂，片剂和注射剂剂型此前已经在中国获批上市。 13、3月6日，CDE官网显示，诺华1类新药瑞米布替尼片上市申请拟纳入优先审评，适用于H1抗组胺药治疗后仍有症状的成人慢性自发性荨麻疹（CSU）患者。这项上市申请此前已于2025年2月获得CDE受理。瑞米布替尼是诺华在研的BTK抑制剂，首发适应症为治疗慢性自发性荨麻疹，针对该适应症的Ⅲ期临床研究已经取得积极结果。 FDA 上市 批准 14、3月3日，FDA官网显示，罗氏旗下公司基因泰克的替奈普酶（商品名：TNKase）获批新适应症，用于治疗成人急性缺血性中风（AIS）。这是近30年来FDA批准的首个中风药物。替奈普酶并不是一款新疗法。此前2000年，该产品获得FDA批准用于治疗成人急性ST段抬高型心肌梗死。  15、3月4日，FDA官网显示，百济神州的替雷利珠单抗（百泽安）获批新适应症，联合含铂化疗用于肿瘤表达PD-L1（≥1）的不可切除或转移性食管鳞状细胞癌（ESCC）成人患者一线治疗。替雷利珠单抗是一款抗PD-1单抗，此前已在美国获批作为单药用于治疗既往接受过系统化疗（不含PD-1/PD-L1抑制剂）后不可切除或转移性ESCC的成人患者，以及联合化疗用于胃和胃食管结合部（G/GEJ）癌成人患者的一线治疗。 16、3月6日，FDA官网显示，Neurotech Pharmaceuticals的revakingene taroretcel-lwey（商品名：Encelto）批准上市，用于治疗2型黄斑毛细血管扩张症（MacTel）。该药是首个且唯一获得FDA批准的MacTel治疗药物。 申请 17、3月3日，FDA官网显示，GSK的Depemokimab递交生物制品许可申请。拟定的适应症为：对于患有以嗜酸性粒细胞表型为特征的2型炎症的哮喘成人和12岁及以上的儿童患者，使用中至高剂量吸入皮质类固醇(ICS)加另一种哮喘控制药物进行辅助维持治疗，以及对于控制不佳的慢性鼻窦炎伴鼻息肉(CRSwNP)成人患者进行辅助维持治疗。PDUFA日期为2025年12月16日。 18、3月4日，FDA官网显示，Capricor Therapeutics的Deramiocel申报上市，用于治疗患有杜氏肌营养不良症（DMD）心肌病的患者。该申请已被FDA授予优先审评，PDUFA目标行动日期为2025年8月31日。Deramiocel（也称为CAP-1002）由同种异体心脏球衍生细胞（CDC）组成，每季度给药一次。 19、3月5日，FDA官网显示，罗氏的奥妥珠单抗递交补充生物制品许可申请(sBLA)，用于治疗狼疮性肾炎。FDA预计将在2025年10月之前做出审批决定。奥妥珠单抗是一种靶向CD20抗体，奥妥珠单抗此前已获批治疗多种淋巴瘤适应症，本次是该药递交的首个非肿瘤适应症上市申请。 研发 临床状态 20、3月3日，药物临床试验登记与信息公示平台显示，翰森制药启动了一项多中心、随机、开放、阳性药物对照临床试验（n=468），旨在评估HS-20089对比研究者选择的化疗（托泊替康、紫杉醇、多柔比星脂质体）治疗铂耐药复发上皮性卵巢癌、输卵管癌或原发性腹膜癌患者的Ⅲ期临床试验。HS-20089是翰森制药开发的一款B7-H4ADC。 21、3月3日，ClinicalTrials官网显示，诺华启动了AAA817的Ⅲ期临床试验（AcTFirst）。该研究是一项多中心、随机、开放标签、阳性药物对照临床试验（n=486），旨在评估AAA817联合雄激素受体信号通路抑制剂（ARPI）对比研究者选择的标准治疗（SoC）治疗未接受过含紫杉醇化疗方案和RLT治疗的PSMA阳性转移性去势抵抗性前列腺癌（mCRPC）患者的有效性和安全性，主要终点是影像学无进展生存期（rPFS）。AAA817是一款α粒子核药。 22、3月4日，Clinicaltrials官网显示，Adverum Biotechnologies启动了Ixo-vec的首个Ⅲ期临床试验（ARTEMIS）。Ixo-vec是Adverum Biotechnologies开发的一款携带阿柏西普编码序列的基因疗法，旨在通过单次玻璃体内注射给药即可实现持续产生阿柏西普的效果，进而治疗湿性年龄相关性黄斑变性（wAMD）等眼科疾病。 23、3月5日，Clinicaltrials官网显示，安进启动了AMG133的两项Ⅲ期临床（MARITIME-1和MARITIME-2）。MARITIME-1、MARITIME-2均是一项随机、双盲、安慰剂对照临床，旨在评估AMG133（低中高剂量）在不伴2型糖尿病的肥胖或超重成人患者中的有效性、安全性和耐受性，主要终点均是第72周患者的体重相较于基线的百分比变化。MARITIME-1的n=3501，MARITIME-2的n=999，AMG133是一款每4周注射1次的FIC抗体多肽偶联药物。 24、3月6日，药物临床试验登记与信息公示平台显示，恒瑞启动了一项注射用SHR-A1811、贝伐珠单抗注射液、阿得贝利单抗注射液、SHR-8068注射液联合方案治疗晚期实体肿瘤的开放、多中心Ⅰb/Ⅱ期临床研究。SHR-A1811是恒瑞医药自主研发的HER2ADC，贝伐珠单抗是一种人源化抗VEGF单克隆抗体，阿得贝利单抗是人源化抗PD-L1单克隆抗体，SHR-8068是恒瑞引进的一款全人源抗CTLA-4单抗。 25、3月7日，药物临床试验登记与信息公示平台显示，圣因生物启动了一项靶向补体C3的小核酸（siRNA）药物SGB-9768注射液的Ⅱ期临床试验。该临床试验是一项评估SGB-9768在原发性IgA肾病患者、C3肾小球病患者和免疫复合物介导的膜增生性肾小球肾炎患者中的有效性和安全性的多中心、开放性Ⅱ期研究。 临床数据 26、3月3日，Biohaven公布了IgG降解剂BHV-1300（4个剂量，皮下注射，每周1次）在健康受试者中的I期研究数据。数据显示，治疗4周后，1000mg BHV-1300组受试者的总IgG水平快速、深度且持续降低，最高降幅达到84%，中位数为80%。并且，BHV-1300在单次给药后数小时内即可快速且持续降低受试者的总IgG水平。 27、3月3日，天辰生物公布了新一代抗IgE抗体LP-003治疗慢性荨麻疹（CSU）的Ⅱ期临床中期数据。结果显示，LP-003在关键疗效指标上优于现有抗IgE疗法奥马珠单抗，且展现出长效优势，或将成为全球抗IgE治疗领域的颠覆性药物。 28、3月4日，基石药业宣布，该公司自主研发的PD-1/VEGF/CTLA-4三特异性抗体CS2009的全球多中心Ⅰ期临床试验顺利完成首例患者给药，未发生输液反应或其他不良事件。该试验将深入评估CS2009在多种晚期实体瘤中的临床应用潜力，包括非小细胞肺癌、肝癌、胃癌、子宫内膜癌、卵巢癌、肾细胞癌及宫颈癌等。 29、3月7日，阿斯利康公布了Ⅲ期MATTERHORN研究。结果显示，Imfinzi（度伐利尤单抗）联合标准治疗FLOT（氟尿嘧啶、亚叶酸钙、奥沙利铂和多西他赛）化疗作为可切除的早期和局部晚期（Ⅱ期、Ⅲ期、ⅣA期）的胃癌和胃食管交界处癌（GEJ）患者围手术期治疗方案，主要终点无事件生存期（EFS）具有统计学意义和临床意义的改善。MATTERHORN是首个证明免疫疗法联合治疗优于标准治疗的全球随机Ⅲ期临床研究。 交易及投融资 30、3月3日，艾伯维宣布，与Gubra达成一项许可协议，共同开发GUB014295，这是一种潜在治疗肥胖症的同类最佳长效胰淀素（amylin）类似物。根据协议，艾伯维将在全球范围内领导GUB014295的开发和商业化活动。Gubra将获得总额为3.5亿美元的预付款，并有资格获得高达18.75亿美元的开发、商业和销售里程碑付款，以及全球净销售额的分级版税。 31、3月3日，丹擎医药宣布完成新一轮1.5亿元人民币融资。本轮融资由康君资本领投，阿斯利康中金医疗产业基金、国方创新、磐霖资本等多家知名投资机构跟投，老股东红杉中国持续加注。本次融资将用于加速核心管线的临床研究、拓展创新技术平台及全球化战略布局。 【2025CPHI思享会预告】 扫码领取CPHI & PMEC China 2025展会门票 来源：CPHI制药在线 声明：本文仅代表作者观点，并不代表制药在线立场。本网站内容仅出于传递更多信息之目的。如需转载，请务必注明文章来源和作者。 投稿邮箱：Kelly.Xiao@imsinoexpo.com ▼更多制药资讯，请关注CPHI制药在线▼ 点击阅读原文，进入智药研习社~