DALLAS, Feb. 05, 2025 (GLOBE NEWSWIRE) -- Gradalis, Inc., a clinical-stage biotechnology company developing personalized anti-cancer therapies, today announced that the U.S. Food and Drug Administration (FDA) has granted Gradalis’ personalized investigational cellular immunotherapy, Vigil® (Gemogenovatucel-T) Regenerative Medicine Advanced Therapy (RMAT) designation based on favorable clinical results from the ongoing Phase 2b VITAL trial.

达拉斯，2025年2月5日（环球通讯社）--Gradalis，Inc.，一家开发个性化抗癌疗法的临床阶段生物技术公司，今天宣布，美国食品和药物管理局（FDA）已经批准Gradalis的个性化研究性细胞免疫疗法，Vigil®（Gemogenovatucel-T）再生医学高级疗法（RMAT），基于正在进行的2b期VITAL试验的良好临床结果。

Vigil is being developed as a maintenance treatment for women with newly diagnosed, advanced Stage IIIb/IV ovarian cancer who are homologous recombination proficient (HRP), have a high clonal tumor mutation burden (cTMB-H) and who are in complete response after debulking surgery and frontline platinum-based doublet chemotherapy.

Vigil正在开发用于新诊断的晚期IIIb/IV期卵巢癌患者的维持治疗，这些患者具有同源重组能力（HRP），具有高克隆肿瘤突变负荷（cTMB-H），并且在减瘤手术和一线铂类双重化疗后完全缓解。

This recognition underscores the potential value for Vigil to address a critical unmet need in this patient population..

这一认识强调了守夜在解决该患者群体中未满足的关键需求方面的潜在价值。。

RMAT designation is reserved for advanced cellular and engineered therapies that show potential to address serious or life-threatening conditions based on preliminary clinical evidence. The designation provides benefits such as increased FDA interactions and guidance on efficient product development.

RMAT指定用于先进的细胞和工程疗法，这些疗法根据初步临床证据显示出解决严重或威胁生命的疾病的潜力。该名称提供了一些好处，例如增加了FDA的互动，并为有效的产品开发提供了指导。

RMAT designated products may qualify for FDA approval pathways that could expedite the delivery of Vigil to cancer patients..

RMAT指定的产品可能符合FDA的批准途径，可以加速向癌症患者提供守夜服务。。

Vigil is a first in class immunotherapy designed to target clonal mutation signals which are contained within all cancer cells, setting it apart from other treatments that focus on sub-clonal signals. Receipt of RMAT designation was based on analysis of promising clinical results from the VITAL study that demonstrated clinically meaningful and statistically significant overall survival benefits in patients with HRP ovarian cancer and cTMB-H who achieved complete response after standard of care frontline therapy..

Vigil是一种一流的免疫疗法，旨在针对所有癌细胞中包含的克隆突变信号，使其与其他专注于亚克隆信号的治疗方法不同。接受RMAT指定是基于对VITAL研究中有希望的临床结果的分析，该研究表明HRP卵巢癌和cTMB-H患者在标准护理前线治疗后获得完全缓解，具有临床意义和统计学意义的总体生存益处。。

“The RMAT designation for Vigil highlights the transformative capacity of our unique immunotherapy to benefit women battling advanced ovarian cancer,” said David Shanahan, CEO of Gradalis. “This important recognition affirms that Vigil has the potential to extend patient survival and may offer a safer, more precise therapeutic approach to a population in urgent need of innovative solutions.

Gradalis首席执行官大卫·沙纳汉（DavidShanahan）表示：“为守夜而设立的RMAT突显了我们独特的免疫疗法的变革能力，有助于女性对抗晚期卵巢癌。”。“这一重要认识证实，守夜有可能延长患者的生存期，并可能为迫切需要创新解决方案的人群提供更安全，更精确的治疗方法。

We continue to advance our Vigil development efforts as we work to bring this investigational therapy to patients as rapidly as possible.”.

我们继续推进我们的守夜发展努力，因为我们致力于尽快将这种研究性治疗带给患者。”。

Ovarian cancer remains one of the deadliest cancers among women, with a lifetime risk of 1 in 87. Standard treatments, such as surgery and chemotherapy, often yield initial responses, but up to 75% of patients experience disease recurrence within two years. For women with HRP ovarian cancer, current treatment options are even less effective, with significantly shorter progression-free survival compared to other molecular profiles.

卵巢癌仍然是女性中最致命的癌症之一，其终生风险为87分之一。手术和化疗等标准治疗通常会产生初步反应，但高达75%的患者会在两年内复发。对于患有HRP卵巢癌的女性，目前的治疗方案甚至效果不佳，与其他分子谱相比，无进展生存期明显缩短。

Despite advancements, no current therapies have shown meaningful clinical benefits for this subgroup, leaving a critical gap in care..

尽管取得了进展，但目前没有任何疗法对该亚组显示出有意义的临床益处，从而在护理方面留下了严重的差距。。

Gradalis is continuing its efforts to bring Vigil to patients as quickly as possible.

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About Vigil

关于Vigil

Vigil® is a novel, triple function immunotherapy platform that modifies a patient’s tumor by using bi-shRNA to reduce furin, an enzyme which facilitates immunosuppressive TGF beta protein production, and to maximize DNA expression of GM-CSF, which stimulates the immune system and attracts key immune system effector cells, including T-cells.

Vigil®是一种新型的三功能免疫治疗平台，可通过使用双shRNA来减少弗林蛋白酶（一种促进免疫抑制性TGF-β蛋白产生的酶）来修饰患者的肿瘤，并最大化GM-CSF的DNA表达，从而刺激免疫系统并吸引关键的免疫系统效应细胞，包括T细胞。

By utilizing the patient's own tumor as the antigen source, Vigil is designed to elicit an immune response that is specifically targeted and broadly relevant to each patient's unique “clonal” tumor neoantigens. Vigil therapy has been well tolerated in Phase 1, 2a and 2b clinical studies..

通过利用患者自身的肿瘤作为抗原来源，Vigil旨在引发特异性靶向的免疫反应，并与每位患者独特的“克隆”肿瘤新抗原广泛相关。守夜疗法在1、2a和2b期临床研究中具有良好的耐受性。。

In VITAL, a multicenter, randomized, double-blind, placebo-controlled Phase 2b trial in Stage III/IV newly diagnosed, frontline ovarian cancer patients, Vigil showed a positive trend in the primary endpoint of recurrence free survival (RFS) in the overall population and a statistically significant improvement in the secondary endpoint of recurrence free survival and overall survival (OS) in patients with the BRCAwt molecular profile.

VITAL是一项针对III/IV期新诊断的一线卵巢癌患者的多中心，随机，双盲，安慰剂对照的2b期临床试验，Vigil在总体人群中无复发生存期（RFS）的主要终点呈阳性趋势，BRCAwt分子谱患者的无复发生存期和总生存期（OS）的次要终点有统计学意义的改善。

In patients with HRP ovarian cancer and cTMB-H where there is a high unmet medical need, Vigil has been designated a Regenerative Medicine Advanced Therapy (RMAT) by the FDA on the basis of a statistically significant and clinically meaningful improvement in OS..

在HRP卵巢癌和cTMB-H患者中，医疗需求未得到很高的满足，Vigil已被FDA指定为再生医学高级疗法（RMAT），其基础是OS具有统计学意义和临床意义的改善。。

Additionally, Phase 1 results in an “all-comer” clinical trial have shown positive signals of activity in 19 different tumor types and some patients treated with Vigil remain in the study 48 months later.

此外，一项“全方位”临床试验的第一阶段结果显示，19种不同肿瘤类型的活性呈阳性信号，一些接受守夜治疗的患者在48个月后仍留在研究中。

Vigil is the first cellular immunotherapy to demonstrate longer-term survival benefits in a randomized controlled trial of patients with solid tumors. The results of the company’s Phase 2b study have been published in Lancet Oncology, Gynecologic Oncology and presented at the American Society of Clinical Oncology..

Vigil是第一个在实体瘤患者的随机对照试验中证明长期生存益处的细胞免疫疗法。该公司的2b期研究结果已发表在《柳叶刀肿瘤学》、《妇科肿瘤学》上，并在美国临床肿瘤学会上发表。。

About Gradalis, Inc.

关于Gradalis，Inc。

Founded in 2006, Gradalis is a privately held, clinical-stage biotechnology company developing a new category of personalized therapies based on proprietary bi-shRNAi technology. Its lead product, called Vigil, is an immunotherapy that has been tested in multiple studies in ovarian and other cancer tumor types..

Gradalis成立于2006年，是一家私营的临床阶段生物技术公司，基于专有的bi-shRNAi技术开发了一类新的个性化疗法。它的主要产品Vigil是一种免疫疗法，已经在卵巢癌和其他癌症肿瘤类型的多项研究中进行了测试。。

Gradalis’ Vigil platform uses the patient’s immune system to target the entire tumor. Based on multiple clinical studies, Gradalis has developed a pioneering oncology platform that is designed to decloak the patient’s tumor clonal neoantigens, reactivate the immune system, and summon key effector cells to deliver a durable clinical response.

Gradalis的守夜平台使用患者的免疫系统来靶向整个肿瘤。基于多项临床研究，Gradalis开发了一个开创性的肿瘤学平台，旨在去除患者的肿瘤克隆新抗原，重新激活免疫系统，并召唤关键效应细胞以提供持久的临床反应。

When combined, these are a powerful Trifecta of anti-cancer activities, potentially eliminating even the elusive metastatic cells, and as shown in Phase 2 clinical studies in ovarian cancer. Our clinical trials have also demonstrated that Gradalis’ platform is better tolerated compared to standard cancer treatments since Vigil uses the patient’s immune system operating within its natural state of balance rather than in an artificial overdrive as with some technologies.

当结合起来时，这些是抗癌活性的强大三联疗法，甚至可能消除难以捉摸的转移细胞，如卵巢癌的2期临床研究所示。我们的临床试验还表明，与标准癌症治疗相比，Gradalis平台的耐受性更好，因为Vigil使用患者的免疫系统在其自然平衡状态下运行，而不是像某些技术那样在人工超速状态下运行。

Vigil utilizes proprietary bi-shRNA technology that has been proven to silence multiple genes EWS/FLI1, KRAS, STMN1, PDX-1 in a variety of cancers and has the potential to be used in other diseases. For additional information, visit gradalisinc.com..

Vigil利用专有的双shRNA技术，已被证明可以沉默多种癌症中的多个基因EWS/FLI1，KRAS，STMN1，PDX-1，并有可能用于其他疾病。有关更多信息，请访问gradalisinc.com。。