/PRNewswire/ -- IDEAYA Biosciences, Inc. (NASDAQ:IDYA), a precision medicine oncology company committed to the discovery and development of targeted therapeutics, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation (BTD) for darovasertib, a potential first-in-class protein kinase C (PKC) inhibitor, for the neoadjuvant treatment of adult patients with primary uveal melanoma (UM) for whom enucleation has been recommended..

/PRNewswire/ -- IDEAYA Biosciences, Inc.（纳斯达克股票代码：IDYA），一家致力于精准医疗的肿瘤学公司，专注于发现和开发靶向治疗药物，今日宣布美国食品药品监督管理局（FDA）已授予darovasertib突破性疗法认定（BTD）。Darovasertib是一种潜在的首创蛋白激酶C（PKC）抑制剂，用于推荐摘除眼球的成年原发性葡萄膜黑色素瘤（UM）患者的术前治疗。

'We are pleased to receive FDA Breakthrough Therapy designation as we prepare to advance neoadjuvant darovasertib into a potential Phase 3 registrational trial in patients with primary UM. This designation highlights the potential of monotherapy darovasertib in a patient population with significant unmet medical need where there are currently no FDA-approved systemic therapies,' said Dr.

“我们很高兴在准备将新辅助darovasertib推进到潜在的III期注册试验时获得了FDA突破性疗法认定。这一指定突显了单药darovasertib在目前尚无FDA批准的系统治疗方案、存在显著未满足医疗需求的患者群体中的潜力，”博士表示。

Darrin Beaupre

达林·博普雷

, M.D., Ph.D., Chief Medical Officer of IDEAYA Biosciences. 'We are targeting to present the updated Phase 2 clinical data in neoadjuvant UM that was provided as part of the BTD application at multiple medical conferences in 2025,' said

医学博士、哲学博士，IDEAYA生物科学首席医学官。“我们计划在2025年的多个医学会议上展示作为BTD申请一部分提供的新辅助UM的二期临床数据更新，”他表示。

Yujiro S. Hata

服部裕次郎

, President and Chief Executive Officer, IDEAYA Biosciences.

，总裁兼首席执行官，IDEAYA生物科学公司。

This U.S. FDA BTD designation, follows the Fast Track designation granted by the U.S. FDA for evaluation of darovasertib in combination with crizotinib in adult patients being treated for metastatic uveal melanoma (MUM), where a Phase 2/3 registration-enabling trial of the darovasertib and crizotinib combination in 1L HLA-A2-negative MUM is ongoing.

这一美国FDA的突破性疗法认定（BTD）之前，美国FDA还授予了darovasertib与crizotinib联合用于治疗转移性葡萄膜黑色素瘤（MUM）成人患者的快速通道资格（Fast Track），目前正在进行一项针对1L HLA-A2阴性MUM的darovasertib和crizotinib联合疗法的2/3期注册试验。

Darovasertib has also been designated as an Orphan Drug by the U.S. FDA in UM, including in MUM, entitling IDEAYA to certain potential tax credits, exemptions from user fees, and statutory marketing exclusivity..

达罗伐塞替也被美国FDA指定为UM（葡萄膜黑色素瘤）中的孤儿药，包括转移性葡萄膜黑色素瘤（MUM），使IDEAYA有资格获得某些潜在的税收抵免、用户费用豁免以及法定的市场独占权。

The BTD application was supported by updated interim clinical data from an ongoing Phase 2 open-label trial (NCT05907954) evaluating darovasertib monotherapy in the neoadjuvant setting for localized UM. IDEAYA presented interim clinical data demonstrating an 82% ocular tumor shrinkage rate and a 61% eye preservation rate in UM patients in .

BTD申请得到了来自正在进行的2期开放标签试验（NCT05907954）的更新中期临床数据的支持，该试验评估了darovasertib单药在局部UM新辅助治疗中的效果。IDEAYA展示了中期临床数据，显示在UM患者中，眼部肿瘤缩小率达到82%，眼球保留率为61%。

), and neoadjuvant UM data was also presented as an oral presentation at ASCO 2024. Updated clinical data in neoadjuvant UM, including efficacy, safety, radiation reduction, eye preservation, and vision preservation / improvement on treatment, were submitted as part of the BTD application that we plan to present at medical conferences in 2025.

），并且新辅助UM数据也在2024年ASCO年会上进行了口头报告。我们计划在2025年的医学会议上展示的新辅助UM更新临床数据，包括疗效、安全性、放射剂量减少、眼球保存以及治疗中的视力保存/改善，已作为BTD申请的一部分提交。

Multiple clinical data updates in neoadjuvant UM and MUM, including median overall survival (mOS) from the Phase 2 study (IDE196-001), are targeted to be presented at medical conferences in mid-year 2025 and the second half of 2025. A median progression free survival (mPFS) readout for the Phase 2/3 registration-enabling trial of the darovasertib and crizotinib combination in 1L HLA-A2-negative MUM is targeted by year-end 2025.

预计将在2025年年中和2025年下半年的医学会议上展示新辅助UM和MUM的多项临床数据更新，包括来自2期研究（IDE196-001）的中位总生存期（mOS）。针对darovasertib和crizotinib联合用于一线HLA-A2阴性MUM的2/3期注册试验，预计将在2025年底获得中位无进展生存期（mPFS）数据。

The Company also intends to initiate a Phase 3 randomized registrational trial in neoadjuvant UM in the first half of 2025..

公司还计划在2025年上半年启动新辅助UM的III期随机注册试验。

A potential Phase 3 registrational study would evaluate neoadjuvant darovasertib in primary UM patients who are eligible for enucleation (Cohort 1) or plaque brachytherapy (Cohort 2). Neoadjuvant UM has a projected annual incidence for

潜在的三期注册研究将评估新辅助达罗伐塞替在适合眼球摘除术（队列1）或斑块近距离放射治疗（队列2）的原发性葡萄膜黑色素瘤患者中的效果。新辅助治疗的葡萄膜黑色素瘤预计年度发病率为。

of approximately 12,000 patients, and is a high unmet medical with no FDA approved systemic therapies.

大约有12,000名患者，这是一种未满足的高医疗需求，没有FDA批准的系统性疗法。

BTD is designed to expedite the development and regulatory review of promising therapies for serious or life-threatening conditions where preliminary clinical evidence suggests substantial improvement over existing treatments. The designation facilitates more intensive FDA guidance, cross-disciplinary collaboration, and eligibility for rolling submission and priority review..

BTD旨在加速针对严重或危及生命状况的有前景疗法的开发和监管审查，其中初步临床证据表明其较现有治疗有显著改善。该指定有助于加强FDA指导、跨学科协作，并符合滚动提交和优先审查的资格。

About IDEAYA Biosciences

关于IDEAYA生物科学

IDEAYA is a precision medicine oncology company committed to the discovery and development of targeted therapeutics for patient populations selected using molecular diagnostics. IDEAYA's approach integrates capabilities in identifying and validating translational biomarkers with drug discovery to select patient populations most likely to benefit from its targeted therapies.

IDEAYA是一家精准医疗肿瘤公司，致力于利用分子诊断技术为特定患者群体发现和开发靶向治疗药物。IDEAYA的方法整合了识别和验证转化生物标志物的能力，并结合药物研发来选择最有可能从其靶向疗法中受益的患者群体。

IDEAYA is applying its research and drug discovery capabilities to synthetic lethality – which represents an emerging class of precision medicine targets..

IDEAYA正在将其研究和药物发现能力应用于合成致死性——这代表了一类新兴的精准医学靶点。