WALTHAM, Mass.--(BUSINESS WIRE)-- Viridian Therapeutics, Inc. (NASDAQ: VRDN), a biotechnology company focused on discovering and developing potential best-in-class medicines for serious and rare diseases, today reported details of its plans to initiate a phase 3 clinical trial program for its SC VRDN-003 product candidate for patients with moderate-to-severe TED..

马萨诸塞州沃尔瑟姆。--（商业新闻短讯）--Viridian Therapeutics，Inc.（纳斯达克：VRDN），一家专注于发现和开发潜在的治疗严重和罕见疾病的一流药物的生物技术公司，今天报道了其为中重度TED患者启动SC VRDN-003候选产品3期临床试验计划的详细信息。。

“We are very pleased to have completed a positive Type C meeting with the FDA and to take this next step towards rapidly bringing a highly differentiated treatment option to patients living with TED,” said Steve Mahoney, Viridian’s President and Chief Executive Officer. “We view VRDN-003 as a potentially best-in-class anti-IGF-1R product candidate that is designed to preserve the compelling IGF-1R clinical response we have seen in our earlier proof-of-concept studies of VRDN-001.

Viridian总裁兼首席执行官史蒂夫·马奥尼（SteveMahoney）表示：“我们很高兴与FDA完成了一次积极的C型会议，并迈出了下一步，为TED患者快速带来高度分化的治疗选择。”。“我们认为VRDN-003是一种潜在的同类最佳抗IGF-1R候选产品，旨在保持我们在早期的VRDN-001概念验证研究中看到的引人注目的IGF-1R临床反应。

We believe this product profile could maximize convenience as a low-volume, infrequent subcutaneous injection and provide better access to treatment for patients.”.

我们相信这种产品简介可以最大限度地提高低容量，不频繁的皮下注射的便利性，并为患者提供更好的治疗途径。”。

Phase 3 Clinical Trials in Active and Chronic TED

活动性和慢性TED的3期临床试验

Viridian is planning to initiate two randomized, double-masked, placebo-controlled phase 3 clinical trials designed to evaluate the efficacy and safety of subcutaneously administered VRDN-003 in patients with active and chronic TED, named REVEAL-1 and REVEAL-2, respectively. These clinical trials are expected to initiate in August 2024..

Viridian计划启动两项随机，双盲，安慰剂对照的3期临床试验，旨在评估皮下注射VRDN-003对活动性和慢性TED患者的疗效和安全性，分别命名为REVEAL-1和REVEAL-2。这些临床试验预计将于2024年8月开始。。

In REVEAL-1, approximately 84 patients will be randomized in a 1:1:1 ratio to receive VRDN-003 SC or placebo every 4 weeks or every 8 weeks. Patients will receive an initial 600mg loading dose given as two 300mg injections, followed by single injections of 300mg thereafter for a total of 6 administrations in the 4-week dosing regimen and a total of 3 administrations in the 8-week regimen.

在REVEAL-1中，大约84名患者将以1:1:1的比例随机接受VRDN-003 SC或安慰剂治疗，每4周或每8周一次。患者将接受初始600mg负荷剂量，作为两次300mg注射，然后单次注射300mg，在4周给药方案中总共6次给药，在8周方案中总共3次给药。

In REVEAL-2, approximately 126 patients will be randomized in the same manner for the same dosing regimens. The primary endpoint in each clinical trial will be proptosis responder rate, based on the achievement of at least 2mm improvement in proptosis from baseline at week 24, versus placebo. Subsequently, patients will be followed for an additional 28 weeks.

在REVEAL-2中，大约126名患者将以相同的方式随机分配相同的给药方案。每个临床试验的主要终点将是突眼反应率，基于在第24周与安慰剂相比，突眼从基线至少改善2mm。随后，将对患者进行另外28周的随访。

Additional outcome measures in each trial will include changes from baseline in proptosis, clinical activity score (CAS) and diplopia..

每个试验中的其他结果指标将包括眼球突出，临床活动评分（CAS）和复视的基线变化。。

“The current standard of care in TED requires 8 intravenous doses, representing a significant burden for patients,” said Tom Ciulla, Viridian’s Chief Medical Officer. “Subcutaneous VRDN-003 could transform the treatment experience for patients with TED.”

Viridian首席医疗官汤姆·奇拉（TomCiulla）表示：“TED目前的护理标准需要静脉注射8剂，这对患者来说是一个沉重的负担。”。“皮下VRDN-003可以改变TED患者的治疗体验。”

Viridian anticipates topline data for both clinical trials to be available in the first half of 2026 and to file a BLA by the end of 2026. The company plans to launch VRDN-003 with a commercially available autoinjector pen.

Viridian预计这两项临床试验的topline数据将在2026年上半年提供，并在2026年底提交BLA。该公司计划推出带有商用自动注射器笔的VRDN-003。

About VRDN-003

关于VRDN-003

VRDN-003 is a potential best-in-class, subcutaneously administered anti-IGF-1R antibody in development for TED. VRDN-003 has the same binding domain as VRDN-001, was engineered to have a longer half-life, and acts as a full antagonist of IGF-1R. IGF-1R inhibition is the only approved mechanism of action that has been clinically and commercially validated for TED and has shown to be highly effective in treating the disease..

VRDN-003是TED开发中潜在的同类最佳皮下注射抗IGF-1R抗体。VRDN-003具有与VRDN-001相同的结合结构域，被设计为具有更长的半衰期，并且充当IGF-1R的完全拮抗剂。IGF-1R抑制是唯一经临床和商业验证的TED作用机制，已被证明对治疗该疾病非常有效。。

Phase 1 results in healthy volunteers showed a VRDN-003 half-life of 40-50 days which is 4-5x the half-life of VRDN-001. Further, pharmacokinetic modeling predicts that convenient dosing regimens of VRDN-003 (e.g., a low volume subcutaneous injection once every 4 or 8 weeks) could achieve exposure levels of VRDN-003 that are equivalent to those of VRDN-001 that produced clinically meaningful results in TED patients in a phase 2, proof-of-concept clinical trial..

健康志愿者的第一阶段结果显示，VRDN-003的半衰期为40-50天，是VRDN-001半衰期的4-5倍。此外，药代动力学模型预测，VRDN-003的方便给药方案（例如，每4周或8周一次的低容量皮下注射）可以达到VRDN-003的暴露水平，相当于VRDN-001的暴露水平，该暴露水平在2期概念验证临床试验中对TED患者产生临床意义的结果。。

About Viridian Therapeutics

关于Viridian Therapeutics

Viridian is a biopharmaceutical company focused on engineering and developing 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，专注于为严重和罕见疾病患者设计和开发潜在的一流药物。Viridian在抗体发现和蛋白质工程方面的专业知识使其能够在商业确定的疾病领域为先前验证的药物靶标开发出差异化的治疗候选药物。。

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 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正在临床上推广多种候选药物，用于治疗甲状腺眼病（TED）。该公司正在为VRDN-001进行一项关键计划，包括两项全球3期临床试验（THRIVE和THRIVE-2），以评估其对活动性和慢性TED患者的疗效和安全性。

Viridian is also advancing VRDN-003 as a potential best-in-class subcutaneous therapy for the treatment of TED and is planning to initiate a global phase 3 program, REVEAL and REVEAL-2, to evaluate the efficacy and safety of VRDN-003 in patients with active and chronic TED, respectively..

Viridian还将VRDN-003作为治疗TED的潜在最佳皮下疗法进行了改进，并计划启动一项全球3期计划REVEAL和REVEAL-2，以评估VRDN-003在活动性和慢性TED患者中的疗效和安全性。。

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组合之外，Viridian正在开发一种新的新生儿Fc受体（FcRn）抑制剂组合，包括VRDN-006和VRDN-008，它有可能在多种自身免疫性疾病中开发。

Viridian is based in Waltham, Massachusetts. For more information, please visit www.viridiantherapeutics.com. Follow Viridian on LinkedIn and X.

Viridian总部位于马萨诸塞州的沃尔瑟姆。有关更多信息，请访问www.viridiatherapeutics.com。在LinkedIn和X上关注Viridian。