- 70% of patients treated with veligrotug in THRIVE who were proptosis responders at week 15 maintained their response at week 52 -

- 在THRIVE试验中，使用Veligrotug治疗的患者中，70%的突眼反应者在第15周时维持了其反应至第52周 -

- 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 -

- Veligrotug最近获得了突破性疗法认定（BTD），支持其优先审评资格；该BTD申请基于以下两点：(i) veligrotug在慢性TED中对复视的一致且显著的改善和缓解，以及 (ii) 眼球突出反应的快速起效。

- Biologics License Application (BLA) submission for veligrotug is on track for second half 2025 -

- Veligrotug的生物制品许可申请（BLA）按计划将于2025年下半年提交 -

- Actively preparing organization for planned U.S. commercial launch in 2026 -

- 积极筹备计划于2026年进行的美国商业发布 -

WALTHAM, Mass.--(BUSINESS WIRE)--

马萨诸塞州沃尔瑟姆--（商业资讯）--

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).

Viridian Therapeutics, Inc.（纳斯达克股票代码：VRDN）是一家专注于发现、开发和商业化用于治疗严重和罕见疾病的潜在最佳药物的生物制药公司，今天宣布了其在研药物veligrotug（“veli”）的3期临床试验THRIVE的积极长期持久性数据。Veligrotug是一种通过静脉注射的抗胰岛素样生长因子-1受体（IGF-1R）抗体，用于治疗活动性甲状腺眼病（TED）患者。

TED is an autoimmune condition characterized by inflammation, growth, and damage to tissues around and behind the eye..

TED是一种自身免疫性疾病，其特征是眼部周围和后方的组织发生炎症、增生并受到损害。

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.

在活跃的TED中进行的THRIVE第三阶段临床试验评估了每三周一次的5次veli或安慰剂输注，在第15周进行了初次分析，然后随访患者至第52周。

Positive Veligrotug Durability at 52 Weeks

52周时Veligrotug的阳性耐久性

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..

在THRIVE试验中，70%的维利珠单抗患者（21/30）在第15周为眼球突出缓解者并继续随访至第52周结束时，保持了他们的眼球突出缓解效果。缓解效果的维持定义为：第15周的缓解者在第52周时相比基线仍至少有2毫米（mm）的眼球突出减少，并且另一只眼未出现恶化（≥2毫米增加），这是通过眼球突出测量法测量的结果。

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.

在随访期间，安全性特征没有变化。在第15周的主要分析中报告的绝大多数不良事件在第52周时已得到解决。

“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.

“我们认为，当前耐久性和安全性决议数据的强度进一步巩固了veli一贯强劲且稳健的临床表现，”Viridian总裁兼首席执行官史蒂夫·马奥尼表示。“我们相信，veligrotug的临床数据总体上继续展示其有望成为治疗TED患者的首选方案。”

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.”.

我们相信，这些数据加上五次输注的简化剂量方案，将使veli有望成为市场领先的TED疗法（如果获得批准）。我们继续在推进于今年下半年提交生物制品许可申请（BLA）方面取得巨大进展，并为2026年可能的上市做准备。"

Robust Veligrotug Topline Clinical Profile for Active and Chronic TED

针对活动性和慢性TED的稳健Veligrotug顶级临床概况

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.

正如 2024 年底宣布的那样，veligrotug 在其关键的 3 期临床试验 THRIVE 和 THRIVE-2 中分别针对活动性和慢性 TED 达到了所有主要和次要终点，并且总体耐受性良好。Veligrotug 在这两项临床试验中均显示出快速的治疗效果，以及在复视方面统计学显著且临床意义重大的减少和消解。

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..

THRIVE-2 是首个来自全球三期临床试验的数据集，展示了慢性甲状腺相关眼病（TED）患者在复视反应和消退方面具有统计学意义的显著效果。THRIVE 和 THRIVE-2 共同构成了迄今为止 TED 领域规模最大的关键项目。

About Veligrotug

关于维利格罗图格

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.

Veligrotug 是一种静脉注射（IV）的抗胰岛素样生长因子-1受体（IGF-1R）抗体，目前处于治疗甲状腺眼病的第三阶段开发中，有望成为活动性和慢性TED患者的首选静脉注射治疗方案。IGF-1R 是一个在临床和商业上均已验证的甲状腺眼病（TED）靶点，并已在美国获得认可。

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..

2024年收入约为20亿美元。Veligrotug 有可能通过差异化的剂量方案改善患者体验，其特点是输注时间较短且输注次数较少，相比目前获批并上市的 IGF-1R 抑制剂。

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.

在关键的 III 期临床试验 THRIVE 和 THRIVE-2 中，veligrotug 达到了所有主要和次要终点。Veligrotug 在两项临床试验中均显示出治疗效果的快速起效，并且复视得到了统计学显著且临床意义上的减轻和消退。THRIVE-2 是首个在全球 III 期临床试验中证明慢性 TED 患者复视反应和消退具有统计学显著性的试验。

Veligrotug was generally well tolerated..

维利格罗图单抗通常耐受性良好。

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.

Viridian认为，如果获得批准，强有力的veligrotug临床概况有潜力在TED商业市场中确立强势地位，并可能有助于推动其潜在的同类最佳皮下IGF-1R抗体VRDN-003的引入。

About Viridian Therapeutics

关于Viridian治疗公司

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是一家生物制药公司，专注于为患有严重和罕见疾病的患者发现、开发和商业化潜在的同类最佳药物。Viridian在抗体发现和蛋白质工程方面的专业知识使得其能够在已验证的药物靶点上开发出具有差异化的治疗候选药物，涉及商业上已确立的疾病领域。

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.

Viridian公司正在推进多个针对甲状腺眼病（TED）患者的临床候选药物。该公司正在进行veligrotug（VRDN-001）的关键项目，包括两项全球三期临床试验（THRIVE和THRIVE-2），以评估其在活动性和慢性TED患者中的有效性和安全性。

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..

THRIVE 和 THRIVE-2 均报告了积极的顶线数据，达到了每个研究的所有主要和次要终点。Viridian 还正在推进 VRDN-003 作为一种潜在的同类最佳皮下治疗药物，用于治疗 TED（甲状腺眼病），包括两项正在进行的全球 III 期关键临床试验 REVEAL-1 和 REVEAL-2，以评估 VRDN-003 在活动性和慢性 TED 患者中的有效性和安全性。

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.

除了其TED产品组合外，Viridian还在推进一种新型的新生儿Fc受体（FcRn）抑制剂组合，包括VRDN-006和VRDN-008，这些药物有潜力在多种自身免疫疾病中进行开发。

Viridian is based in Waltham, Massachusetts. For more information, please visit

Viridian 总部位于马萨诸塞州沃尔瑟姆。欲了解更多信息，请访问

www.viridiantherapeutics.com

www.viridiantherapeutics.com

. Follow Viridian on

关注Viridian

LinkedIn

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Forward Looking Statements

前瞻性声明

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.

本新闻稿包含1995年《私人证券诉讼改革法案》意义下的前瞻性陈述。这些陈述可以通过使用诸如以下词语来识别，但不限于：“预期”、“相信”、“成为”、“继续”、“可能”、“设计”、“估计”、“期望”、“打算”、“或许”、“可能”、“在正轨上”、“计划”、“潜在”、“预测”、“预计”、“应该”、“目标”、“将”或“会”，或其他类似的术语或表达，涉及我们的期望、计划和意图。

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 potentia.

前瞻性陈述既不是历史事实，也不能保证未来的表现。相反，它们是基于我们当前的信念、期望和假设。前瞻性陈述包括但不限于以下方面的声明：Viridian产品候选物（包括veligrotug（前称VRDN-001）和VRDN-003）的临床开发和预期商业化；veligrotug和VRDN-003的潜在效用、疗效、效力、安全性、临床益处、临床反应、便利性及适应症数量，包括Viridian对THRIVE持久性和安全性解析数据强度及veligrotug强大临床特征的看法；veligrotug有潜力成为活动性和慢性TED的首选静脉注射治疗方案；突破性疗法认定的影响，包括优先审查资格，或任何其他FDA认定；与监管机构的互动及预期的监管提交时间，包括预计在2025年下半年提交veligrotug的BLA申请；Viridian产品候选物的潜在市场规模和市场机会，包括Viridian认为如果获得批准，veligrotug将有望成为市场领先的TED治疗药物；Viridian产品候选物的潜力。

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.

新的风险和不确定性可能会不时出现，无法预测所有风险和不确定性。对于任何此类前瞻性陈述的准确性，不作任何明示或暗示的陈述或保证。此类前瞻性陈述受多项重大风险和不确定性的影响，包括但不限于：Viridian产品候选物的潜在效用、疗效、效力、安全性、临床益处、临床反应及便利性；已完成或正在进行的临床试验的结果或数据可能无法代表正在进行或未来临床试验的结果；初步数据可能无法代表最终数据；关于监管文件时间的预期和变化；与监管机构的互动；临床药物开发相关的不确定性和潜在延迟；我们获得和维持治疗候选物监管批准的时间和能力；来自其他疗法或产品的竞争；市场规模的估计；我们未来的运营结果和财务表现；Viridian的知识产权地位；我们的产品候选物即使获批也可能无法取得商业成功；以及我们在向证券交易委员会（SEC）提交的文件中“风险因素”部分不时描述的其他风险，包括我们最近的年度报告（Form 10-K）或季度报告（Form 10-Q）中描述的风险（视情况而定），并由我们不时提交的当前报告（Form 8-K）进行补充。

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.

任何前瞻性声明仅在其作出之日有效。公司及其附属机构、顾问或代表均无义务公开更新或修改任何前瞻性声明，无论是否。

IR@viridiantherapeutics.com

IR@viridiantherapeutics.com

Media@viridiantherapeutics.com

媒体@维里迪安制药公司.com

Source: Viridian Therapeutics, Inc.

来源：Viridian Therapeutics, Inc.