Veligrotug

- 70% of patients treated with veligrotug in THRIVE who were proptosis responders at week 15 maintained their response at week 52 - - Veligrotug recently received Breakthrough Therapy Designation (BTD), supporting eligibility for Priority Review; the BTD request was based on veligrotug’s (i) consistent and robust improvement and resolution of diplopia in chronic TED, and (ii) rapid onset of proptosis response - - Biologics License Application (BLA) submission for veligrotug is on track for second half 2025 - - Actively preparing organization for planned U.S. commercial launch in 2026 - WALTHAM, Mass.--(BUSINESS WIRE)-- Viridian Therapeutics, Inc. (Nasdaq: VRDN), a biopharmaceutical company focused on discovering, developing, and commercializing potential best-in-class medicines for serious and rare diseases, today announced positive long-term durability data from the THRIVE phase 3 clinical trial of veligrotug (“veli”), an intravenously delivered anti-insulin-like growth factor-1 receptor (IGF-1R) antibody, in patients with active thyroid eye disease (TED). TED is an autoimmune condition characterized by inflammation, growth, and damage to tissues around and behind the eye. The THRIVE phase 3 clinical trial in active TED evaluated 5 infusions of veli or placebo every three weeks with primary topline analysis at week 15 and then followed patients through week 52. Positive Veligrotug Durability at 52 Weeks 70% of veligrotug patients (21/30) in THRIVE, who were proptosis responders at week 15 and continued follow-up to the end of the study at week 52, maintained their proptosis response. Maintenance of response is defined as responders at week 15 who still had at least a 2-millimeter (mm) reduction in proptosis compared to baseline at week 52, without worsening in the fellow eye (≥2 mm increase), as measured by exophthalmometry. There were no changes to the safety profile in the follow-up period. The vast majority of adverse events reported at the week 15 primary analysis had resolved by week 52. “We view the strength of today’s durability and safety resolution data as reinforcing veli’s strong and consistently robust clinical profile,” said Steve Mahoney, Viridian’s President and CEO. “We believe that the totality of veligrotug’s clinical data continues to demonstrate its potential to be the treatment-of-choice for patients living with TED. We believe these data, together with a streamlined dosing regimen of five infusions, position veli to become a market leading TED therapeutic, if approved. We continue to make great progress towards submitting the BLA in the second half of this year and preparing for a potential launch in 2026.” Robust Veligrotug Topline Clinical Profile for Active and Chronic TED As announced in late 2024, veligrotug met all of its primary and secondary endpoints and was generally well-tolerated in its pivotal phase 3 clinical trials, THRIVE and THRIVE-2, for active and chronic TED, respectively. Veligrotug demonstrated a rapid onset of treatment effect and statistically significant and clinically meaningful reduction and resolution of diplopia in both clinical trials. THRIVE-2 was the first data set from a global phase 3 clinical trial in chronic TED patients to demonstrate statistically significant diplopia response and resolution. Together, THRIVE and THRIVE-2 comprise the largest pivotal program to date in TED. About Veligrotug Veligrotug is an intravenously (IV) delivered, anti-insulin-like growth factor-1 receptor (IGF-1R) antibody in phase 3 development for thyroid eye disease, with the potential to be the IV treatment-of-choice for active and chronic TED patients. IGF-1R is a clinically and commercially validated target for thyroid eye disease (TED) with U.S. revenues of approximately $2 billion in 2024. Veligrotug has the potential to improve patient experience with a differentiated dosing regimen that features a shorter infusion time and fewer infusions compared to the currently approved and marketed IGF-1R inhibitor. In its pivotal phase 3 clinical trials, THRIVE and THRIVE-2, veligrotug met all of its primary and secondary endpoints. Veligrotug demonstrated a rapid onset of treatment effect and statistically significant and clinically meaningful reduction and resolution of diplopia in both clinical trials. THRIVE-2 was the first demonstration in a global phase 3 clinical trial of a statistically significant diplopia response and resolution in chronic TED patients. Veligrotug was generally well tolerated. Viridian believes that the robust veligrotug clinical profile has the potential to establish a strong position in the TED commercial market, if approved, and may help facilitate the introduction of VRDN-003, its potential best-in-class subcutaneous IGF-1R antibody for TED. About Viridian Therapeutics Viridian is a biopharmaceutical company focused on discovering, developing and commercializing potential best-in-class medicines for patients with serious and rare diseases. Viridian’s expertise in antibody discovery and protein engineering enables the development of differentiated therapeutic candidates for previously validated drug targets in commercially established disease areas. Viridian is advancing multiple candidates in the clinic for the treatment of patients with thyroid eye disease (TED). The company is conducting a pivotal program for veligrotug (VRDN-001), including two global phase 3 clinical trials (THRIVE and THRIVE-2), to evaluate its efficacy and safety in patients with active and chronic TED. Both THRIVE and THRIVE-2 reported positive topline data, meeting all the primary and secondary endpoints of each study. Viridian is also advancing VRDN-003 as a potential best-in-class subcutaneous therapy for the treatment of TED, including two ongoing global phase 3 pivotal clinical trials, REVEAL-1 and REVEAL-2, to evaluate the efficacy and safety of VRDN-003 in patients with active and chronic TED. In addition to its TED portfolio, Viridian is advancing a novel portfolio of neonatal Fc receptor (FcRn) inhibitors, including VRDN-006 and VRDN-008, which has the potential to be developed in multiple autoimmune diseases. Viridian is based in Waltham, Massachusetts. For more information, please visit www.viridiantherapeutics.com. Follow Viridian on LinkedIn and X. Forward Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may be identified by the use of words such as, but not limited to, “anticipate,” “believe,” “become,” “continue,” “could,” “design,” “estimate,” “expect,” “intend,” “may,” “might,” “on track,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” or “would” or other similar terms or expressions that concern our expectations, plans and intentions. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based on our current beliefs, expectations, and assumptions. Forward-looking statements include, without limitation, statements regarding: clinical development and anticipated commercialization of Viridian’s product candidates, including veligrotug (formerly VRDN-001) and VRDN-003; the potential utility, efficacy, potency, safety, clinical benefits, clinical response, convenience, and number of indications of veligrotug and VRDN-003, including Viridian’s view of the strength of the THRIVE durability and safety resolution data and veligrotug’s robust clinical profile; veligrotug’s potential to be the IV treatment-of-choice for active and chronic TED; the impact of Breakthrough Therapy Designation, including eligibility for Priority Review, or any other FDA designations; regulatory interactions and anticipated timing of regulatory submissions, including the anticipated BLA submission for veligrotug in the second half of 2025; potential market sizes and market opportunities for Viridian’s product candidates, including Viridian’s belief that veligrotug is positioned to become a market leading TED therapeutic, if approved; Viridian’s product candidates potentially being best-in-class; whether veligrotug will serve an unmet need; veligrotug’s potential to improve patient experience over existing therapies; and Viridian’s expectations regarding the potential commercialization of veligrotug and VRDN-003, if approved, including the potential U.S. launch of veligrotug in 2026. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. No representations or warranties (expressed or implied) are made about the accuracy of any such forward-looking statements. Such forward-looking statements are subject to a number of material risks and uncertainties including but not limited to: potential utility, efficacy, potency, safety, clinical benefits, clinical response, and convenience of Viridian’s product candidates; that results or data from completed or ongoing clinical trials may not be representative of the results of ongoing or future clinical trials; that preliminary data may not be representative of final data; expectations and changes regarding the timing for regulatory filings; regulatory interactions; uncertainty and potential delays related to clinical drug development; the timing of and our ability to obtain and maintain regulatory approvals for our therapeutic candidates; competition from other therapies or products; estimates of market size; our future operating results and financial performance; Viridian’s intellectual property position; that our product candidates may not be commercially successful, if approved; and other risks described from time to time in the “Risk Factors” section of our filings with the Securities and Exchange Commission (SEC), including those described in our most recent Annual Report on Form 10-K or Quarterly Report on Form 10-Q, as applicable, and supplemented from time to time by our Current Reports on Form 8-K. Any forward-looking statement speaks only as of the date on which it was made. Neither the company, nor its affiliates, advisors, or representatives, undertake any obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law. These forward-looking statements should not be relied upon as representing the company’s views as of any date subsequent to the date hereof. IR@viridiantherapeutics.com Media@viridiantherapeutics.com

- 针对IgG4相关疾病的 INDIGO 3 期关键试验预计将于2025年年底左右得到顶线结果- 针对复发性多发性硬化症的MoonStone 2 期试验的招募工作已经结束；预计将于 2025年第四季度初得到顶线结果- 针对系统性红斑狼疮的SunStone 2 期试验的入组工作预计将于2025年年底完成；预计将于2026年年中取得顶线结果-任命研发负责人兼首席医疗官Lisa von Moltke 博士和首席科学官 Haley Laken 博士，加强领导团队- 截至2025年3月31日，现金、现金等价物和投资达3.142亿美元，预计将为2026年第四季度提供资金支持美国马萨诸塞州沃尔瑟姆,2025年5月15日(GlOBE NEWSWIRE)- Zenas BioPharma, Inc.(以下简称 “Zenas” 或 “公司”)(纳斯达克股票代码:ZBIO)是一家处于临床阶段的全球生物制药公司，致力于成为炎症与免疫定向疗法开发和商业化的领导者，今天公布了截至2025年3月31日的第一季度财务业绩，并提供了最新的公司业务更新。Zenas公司创始人兼首席执行官Lonnie Moulder说:"我们很高兴看到我们的Obexelimab项目在正在进行的2期和3期临床试验中继续保持良好势头，我们预计今年晚些时候将公布复发性多发性硬化症和IgG4-RD患者试验的初步结果。随着Lisa和Haley的加入，我们的团队实力得到了增强，我们完全有能力开展临床试验，推动Obexelimab成为一种差异化的B细胞抑制剂，为患者提供更安全、更有效、更便捷的治疗方案。"近期公司要闻Obexelimab-一种 CD-19 x FcγRIIb B 细胞功能抑制剂:IgG4相关性疾病（IgG4-RD）:继续推进INDIGO 3期试验，这是一项全球性的3期、基于注册目的、随机、双盲、安慰剂对照研究，旨在评估Obexelimabd对IgG4-RD患者的疗效和安全性。INDIGO是迄今为止在 IgG4-RD患者中开展的规模最大的临床试验。2024年第四季度，Zenas 完成了INDIGO试验的目标入组，预计将在2025年年底左右报告INDIGO试验的顶线结果。复发性多发性硬化症(RMS)：MoonStone 2期试验的患者筛选工作已经结束，最后一名受试者预计将于6月初入组，该试验是一项多中心、随机、双盲、安慰剂对照研究，旨在评估Obexelimab对RMS患者的疗效和安全性。公司预计在2025年第四季度初报告该试验的顶线结果，包括12 周的主要终点结果。系统性红斑狼疮(SLE)：SunStone 2期试验继续推进中，这是一项多中心、随机、双盲、安慰剂对照研究，旨在评估Obexelimab对系统性红斑狼疮患者的疗效和安全性。公司预计到 2025年底完成这项研究的入组工作，并在2026年年中报告其顶线结果。其他公司要闻除了在Obexelimab方面取得进展之外，公司在第一季度及近期：任命医学博士Lisa von Moltke为研发负责人兼首席医学官，任命Haley     Laken博士为首席科学官，加强了公司的领导团队。von Moltke博士拥有30多年的美国和国际药物开发经验，涉及多个治疗领域，包括自身免疫性疾病、早期和晚期临床开发，以及大型和新兴成长型公司的商业化。Laken博士在研究、开发运营、研发战略和业务开发方面拥有超过25年的领导经验。将甲状腺眼病项目ZB001(胰岛素样生长因子-1 受体(抗 IGF-1R)单克隆抗体)的地区权利授权给Zai Lab(Zai)。公司收到了一笔预付款，并有资格在未来收到里程碑付款和特许权使用费，作为对 Zai 在大中华区开发和商业化 ZB001 及相关项目的独家转授权的对价。2025年第一季度财报截至2025年3月31日，公司的现金、现金等价物和投资为 3.142亿美元。公司预计，截至2025年3月31日，其现金、现金等价物和投资将为2026年第四季度的运营支出和资本支出需求提供资金。截至2025年3月31日的季度，许可与合作收入为 1,000 万美元，是与Zai签订的次级许可协议所收到的一次性不可退还的预付现金。截至2024年3月31日的季度，公司未确认任何许可与合作收入。截至2025年3月31日的季度，研发(R&D)费用为3490万美元，而截至2024 年3 月31日的季度为2260万美元。研发费用增加1230万美元，主要与奥Obexelimab的临床开发和生产相关的成本增加有关。截至2025年3月31日的季度，运营和行政支出(G&A)为1240万美元，而截至 2024年3月31日的季度为490万美元。G&A增加750万美元的原因是人事费用(包括股票薪酬费用)、商业化前活动(包括招聘)以及其他费用(包括作为上市公司运营的相关费用)的增加。截至 2025年3月31日的季度净亏损为3360万美元，截至2024年3月31日的季度净亏损为2780万美元。关于ObexelimabObexelimab是一种双功能单克隆抗体，可同时结合CD19和FcγRIIb(广泛存在于B细胞系中)，从而抑制与许多自身免疫性疾病有关的细胞的活性，而不会耗竭它们。Obexelimab的作用机制和慢性给药方案可以广泛而有效地解决B细胞系在慢性自身免疫性疾病中的致病作用。Obexelimab已在五项已完成的临床试验中进行了评估，共有198名患者通过静脉输注或皮下注射接受了Obexelimab治疗。在这五项临床试验中，Obexelimab的耐受性和临床活性均表现良好，为公司提供了一个初步的临床概念验证，即Obexelimab是一种有效的B细胞抑制剂，可用于治疗某些自身免疫性疾病。目前，Zenas正在开展多项Obexelimab治疗自身免疫性疾病的2期和3期试验，包括IgG4相关性疾病、复发性多发性硬化症和系统性红斑狼疮。关于Zenas BioPharmaZenas BioPharma是一家全球生物制药公司，致力于成为为世界各地患者开发和商业化免疫学疗法的领导者。我们的核心业务战略是将经验丰富的领导团队与严谨的候选产品组合相结合，在全球范围内发现、收购和开发我们认为能为自身免疫疾病患者带来卓越临床疗效的候选产品。Zenas的主要候选产品Obexelimab是一种双功能单克隆抗体，能同时结合CD19和FcγRIIb(广泛存在于 B 细胞系中)，从而抑制与许多自身免疫性疾病有关的细胞的活性，而不会消耗这些细胞。我们相信，Obexelimab的作用机制和慢性给药方案可以广泛而有效地解决B细胞系在慢性自身免疫性疾病中的致病作用。欲了解有关Zenas BioPharma的更多信息，请访问网站：www.zenasbio.com，LinkedIn及微信公众号上关注我们。 前瞻性说明本新闻稿包含 “前瞻性声明”，其中涉及风险、不确定性和突发事件，许多风险、不确定性和突发事件超出了公司的控制范围，可能导致实际结果、业绩或成果与预期结果、业绩或成果存在实质性差异。本新闻稿中除历史事实陈述外的所有陈述均为前瞻性陈述。在某些情况下，您可以通过 “可能”、“将要”、“应该”、“期望”、“计划”、“预期”、“也许”、“打算”、“目标”、“项目”、“考虑”、“相信”、“估计”、“预测”、“潜在”或 “继续”等词语或这些词语的反义词或其他类似表述来识别出前瞻性表述，尽管并非所有前瞻性表述都包含这些词语。前瞻性表述包括但不限于有关正在进行的和未来的临床试验的时间和结果的表述，包括对INDIGO试验顶线结果报告时间的预期、MoonStone试验的最后一名入组患者和12周主要终点数据，以及SunStone试验完成入组和顶线结果报告的预期时间；发展战略；现金流信息；本新闻稿中的前瞻性表述仅涉及截至本新闻稿发布之日的情况，受许多已知和未知风险、不确定性和假设的影响，可能导致公司的实际结果与前瞻性表述中的预期结果存在实质性差异，这些风险、不确定性和假设包括但不限于公司的经营历史较短，自公司成立以来的亏损，以及预计在可预见的未来将发生不断增加的亏损；公司需要大量额外融资以实现公司目标；临床开发的不确定性，临床开发耗时长、成本高、结果不确定，以及在完成或未能完成公司现有候选产品或任何未来候选产品的开发和商业化过程中与额外成本或延迟相关的风险；临床试验中患者入组和用药的延迟或困难；公司候选产品引起的任何重大不良事件或不良副作用的影响；潜在的竞争，包括来自大型和专业制药及生物技术公司的竞争，其中许多公司已经在公司目前的适应症方面拥有获得批准的疗法；公司从目前或未来的合作或授权协议中获益的能力，以及成功完成未来合作的能力；公司在美国或任何其他司法管辖区获得任何候选产品商业化的监管批准的能力，以及任何此类批准的适应症可能比公司寻求的更狭窄的风险；公司对公司高级管理人员及其他临床和科研人员服务的依赖性，以及公司留住这些人员或招聘更多管理人员或临床和科研人员的能力；公司发展组织、管理公司发展和扩大公司业务的能力；与公司候选产品生产相关的风险(生产过程非常复杂)，以及公司的第三方生产商在生产过程中可能遇到困难的风险；公司为候选产品或公司未来可能开发的候选产品获得并保持足够的知识产权保护的能力；公司依赖第三方开展临床前研究和临床试验的情况；公司履行其他方授予公司的候选产品开发和商业化权利许可下的义务的情况；重大政治、贸易和监管动态，包括中美关系的变化；与公司供应商运营相关的风险，其中许多供应商位于美国境外，包括公司目前唯一的药物物质和药物产品合同生产机构，位于中国的药明康德生物制品(香港)有限公司；以及公司截至2025年3月31日的10-Q年报中 “风险因素 ”一节所述的其他风险和不确定性，以及我们向美国证券交易委员会提交的其他信息。本新闻稿中的前瞻性陈述本身具有不确定性，仅截至本新闻稿发布之日，并可能被证明是不正确的。尽管公司认为这些信息构成了前瞻性声明的合理基础，但这些信息可能是有限的或不完整的，而且我们的声明不应被理解为表明公司已对所有可能获得的相关信息进行了详尽的调查或审查。由于前瞻性声明本身存在风险和不确定性，其中有些风险和不确定性无法预测或量化，有些风险和不确定性超出了公司的控制范围，因此不应将这些前瞻性声明视为未来事件的保证。前瞻性声明中反映的事件和情况可能无法实现或不会发生，未来的实际结果、活动水平、业绩以及事件和情况可能与前瞻性声明中预测的有实质性差异。此外，公司在不断变化的环境中运营，新的风险和不确定因素可能不时出现，管理层无法预测所有风险和不确定因素。除非适用法律另有规定，否则公司不承诺公开更新或修订此处包含的任何前瞻性声明，无论是否因任何新信息、未来事件、环境变化或其他原因所致。Zenas BioPharma文字商标和Logo是Zenas BioPharma公司或其关联公司的商标。投资及媒体联系： Argot PartnersZenas@argotpartners.com关注我们获得更多相关信息