Eganelisib induced complete remissions and hematologic improvements as a single-agent via myeloid differentiation in a Phase 1 study in relapsed/refractory patients with myeloid malignancies (MDS/AML) who show high expression of PI3K-gamma in bone marrow blasts.

在一项针对骨髓原始细胞PI3K-γ高表达的复发/难治性髓系恶性肿瘤（MDS/AML）患者的1期研究中，艾加列司布作为单药通过髓系分化诱导了完全缓解与血液学改善。

The improved overall survival in patients with high expression of PI3K-gamma in this Phase 1 study is internally consistent with the observed responses and is externally validated by the TCGA database, which suggests a poor prognosis for patients with high expression of PI3K-gamma.

本项I期研究中，PI3K-γ高表达患者总生存期的改善与观察到的疗效在内部保持一致，并得到了TCGA数据库的外部验证，该数据库表明PI3K-γ高表达患者预后较差。

Eganelisib generated a striking efficacy signal while demonstrating a favorable safety and tolerability profile in this Phase 1 study.

在这项1期研究中，Eganelisib显示出显著的疗效信号，同时展现出良好的安全性和耐受性。

WALTHAM, Mass.

沃尔瑟姆，马萨诸塞州

,

，

June 2, 2026

2026年6月2日

/PRNewswire/ -- Stelexis BioSciences, Inc. (Stelexis) today announced results from a Phase 1 clinical trial (NCT06533761) of eganelisib, an oral, first-in-class PI3K-gamma inhibitor, in 21 patients with relapsed/refractory (R/R) acute myeloid leukemia (AML) or higher-risk myelodysplastic syndrome (HR-MDS).

/美通社/ -- Stelexis BioSciences公司（Stelexis）今日公布了eganelisib（一种口服、首创的PI3K-γ抑制剂）在一项针对21例复发/难治性（R/R）急性髓系白血病（AML）或高危骨髓增生异常综合征（HR-MDS）患者的1期临床试验（NCT06533761）中的结果。

The data were presented in an oral session at the 2026 ASCO Annual Meeting by Mendel Goldfinger, M.D., of Montefiore Einstein Comprehensive Cancer Center..

这些数据由来自蒙蒂菲奥里爱因斯坦综合癌症中心的门德尔·戈尔德芬格（Mendel Goldfinger）医学博士在2026年美国临床肿瘤学会（ASCO）年会口头报告环节中进行展示。

Key Findings

主要发现

The trial recruited a heavily pretreated patient population that was uniformly characterized by features that convey an unfavorable prognosis:

该试验招募的患者人群均接受过大量既往治疗，且均具有提示预后不良的特征：

29% TP53-deleted or -mutated

29% TP53缺失或突变

90% abnormal cytogenetics

90% 细胞遗传学异常

76% received more than two prior therapies

76%接受过两种以上既往治疗。

76% received prior venetoclax

76% 既往接受过维奈克拉治疗

A modified objective response rate (defined as CR + Stable Disease with neutrophil count improvement) was achieved in:

改良客观缓解率（定义为CR+伴中性粒细胞计数改善的疾病稳定）在……中达到：

50% (6/12) of patients with high PI3K-gamma expression vs.

50%（6/12）的PI3K-γ高表达患者与……相比

0% (0/8) of patients with low PI3K-gamma expression (p<0.05, Fisher's exact test), using immunohistochemical staining of bone marrow biopsies

通过对骨髓活检标本进行免疫组织化学染色，0%（0/8）的患者呈低 PI3K-γ 表达（p<0.05，Fisher精确检验）。

Responses included molecular and major cytogenetic remissions in two patients in CR

应答包括2例完全缓解（CR）患者达到分子学缓解和主要细胞遗传学缓解。

Five of six responding patients had severe or life-threatening neutropenia at baseline, and all five patients demonstrated rapid neutrophil count improvements, including a rapid

六名有应答的患者中有五名在基线时患有重度或危及生命的中性粒细胞减少症，且这五名患者均表现出中性粒细胞计数的快速改善，包括快速的

normalization

标准化（或 归一化/规范化/正常化，视具体领域而定）

of neutrophil counts in four patients, consistent with induction of myeloid differentiation

……四名患者的中性粒细胞计数，与诱导髓系分化一致

Median overall survival (OS) was 27 weeks in PI3K-gamma-high patients vs. 9.9 weeks in low expressors (HR 0.22; 95% CI 0.07–0.75)

PI3K-γ高表达患者的中位总生存期（OS）为27周，而低表达患者为9.9周（HR 0.22；95% CI 0.07–0.75）。

Based on historical patient data from the publicly available TCGA database, PI3K-gamma-high expression is an adverse prognostic factor for OS in patients receiving conventional therapy, thereby supporting a substantial clinical benefit observed following treatment with eganelisib in this study.

基于公开的TCGA数据库历史患者数据，在接受常规治疗的患者中，PI3K-γ高表达是总生存期（OS）的不良预后因素，从而印证了本研究中观察到的eganelisib治疗所获得的显著临床获益。

Eganelisib was well tolerated at both dose levels (45 mg and 60 mg, once daily) with no dose-limiting toxicities and no intrinsic hematologic toxicity. This is consistent with the myeloid-restricted expression of PI3K-gamma, and with the observation that PI3K-gamma is dispensable for normal hematopoiesis (Gu et al., Blood 2024)..

Eganelisib在两种剂量水平（45 mg和60 mg，每日一次）下均表现出良好的耐受性，未出现剂量限制性毒性，亦无固有的血液学毒性。这与PI3K-γ的髓系限制性表达相符，并与PI3K-γ对正常造血非必需的观察结果一致（Gu等，《Blood》2024）。

The majority of adverse events in this study were disease-related rather than treatment-related, consistent with the underlying patient population. Treatment-related AEs were predominantly low-grade with no Grade 5 related AEs. Of note, no eganelisib-related SAEs were observed and no patient discontinued treatment due to an eganelisib-related AE..

本研究中大多数不良事件与疾病相关而非与治疗相关，这与该患者人群的特征相符。治疗相关不良事件主要为低级别，且无5级相关不良事件。值得注意的是，未观察到与伊加奈利西布（eganelisib）相关的严重不良事件，且无患者因伊加奈利西布相关不良事件而终止治疗。

Published outcomes for patients with relapsed/refractory disease who failed frontline venetoclax and hypomethylating agent therapy have shown a median overall survival of approximately 2.4 months (Maiti et al., Haematologica 2021), underscoring the high unmet need in this population.

已发表的针对一线维奈克拉联合去甲基化药物治疗失败的复发/难治性疾病患者的数据显示，其中位总生存期约为2.4个月（Maiti等，《Haematologica》2021），这凸显了该人群巨大的未满足临床需求。

'The preliminary data generated by eganelisib in patients with heavily pretreated AML and higher-risk MDS is encouraging,' said Konstantinos N. Aprilakis, M.D., Board Member at Stelexis and Partner at Deerfield Management. 'In addition to a favorable safety and tolerability profile, Stelexis has observed evidence of biologic and clinical activity for eganelisib in a particularly difficult-to-treat patient population.

“eganelisib在经多线前期治疗的急性髓系白血病（AML）和高危骨髓增生异常综合征（MDS）患者中取得的初步数据令人鼓舞，”Stelexis董事会成员兼Deerfield Management合伙人康斯坦丁诺斯·N·阿普里拉基斯医学博士表示。“除具备良好的安全性和耐受性外，Stelexis还在这一治疗难度极高的患者群体中，观察到了eganelisib具有生物学和临床活性的证据。”

I am especially delighted that Stelexis has identified a biomarker that may be used to identify patients most likely to benefit from treatment with eganelisib. These clinical data suggest that eganelisib has the potential to be effective across multiple hematologic malignancies of high unmet need.'.

我尤为欣喜的是，Stelexis 已发现一种生物标志物，可用于识别最有可能从 eganelisib 治疗中获益的患者。这些临床数据表明，eganelisib 有望在多种高度未满足临床需求的血液系统恶性肿瘤中展现疗效。

'For nearly 20 years now, the field has been searching for therapies that offer a better prognosis to MDS-patients than hypomethylating agents alone. For far too long, clinicians had to primarily rely on classifying and selecting patients for investigational treatment based on prognostic scores or bone marrow blast counts, alone.

近20年来，该领域一直在寻求能为MDS患者提供优于单用去甲基化药物预后的治疗方案。长期以来，临床医生在分类和筛选患者接受试验性治疗时，往往只能单纯依赖预后评分或骨髓原始细胞计数。

It is time we begin to classify patients based on disease-specific, functional, biological vulnerabilities and offer therapies that selectively target those biological vulnerabilities' said Manuel Aivado, MD, PhD, President and Chief Executive Officer of Stelexis BioSciences. 'We believe that myeloid malignancies may find in PI3K-gamma an analog to HER2 in breast cancer, and we are thrilled that the ground-breaking discoveries of our co-founders have paved Stelexis' way to this doorstep.'.

Stelexis BioSciences 总裁兼首席执行官 Manuel Aivado 医学博士、哲学博士表示：“是时候开始根据疾病特异性、功能性和生物学脆弱性对患者进行分类，并提供能够选择性靶向这些生物学脆弱性的疗法了。我们认为，PI3K-γ 有望成为髓系恶性肿瘤中类似于乳腺癌 HER2 的靶点。我们深感振奋，联合创始人的突破性发现已为 Stelexis 铺平了通往这一目标的大门。”

Based on these results, Stelexis plans to advance eganelisib into a randomized expansion cohort with hypomethylating agents in treatment-naïve, PI3K-gamma-high MDS patients with an IPSS-R category of intermediate, high, or very-high risk or with CMML-2.

基于上述结果，Stelexis计划将eganelisib推进至一项随机扩展队列研究，联合去甲基化药物，用于治疗初治、PI3Kγ高表达且IPSS-R风险分层为中危、高危或极高危的骨髓增生异常综合征（MDS）患者，或慢性粒单核细胞白血病2型（CMML-2）患者。

About Eganelisib

关于艾加尼利西布

Eganelisib is a first-in-class, oral, highly potent, and isoform-selective inhibitor of PI3K-gamma, the myeloid-restricted isoform of the PI3K kinase family. PI3K-gamma is dispensable in normal hematopoiesis and lymphocytes, differentiating it from delta-class PI3K inhibitors that have been associated with significant immune-mediated adverse events..

Eganelisib 是一种同类首创的口服、高效且具有亚型选择性的 PI3K-γ 抑制剂，PI3K-γ 为 PI3K 激酶家族中的髓系限制性亚型。PI3K-γ 在正常造血和淋巴细胞中并非必需，这一特性使其区别于已知会引发严重免疫介导不良反应的 PI3K-δ 抑制剂。

In myeloid malignancies, PI3K-gamma signaling reinforces the differentiation block characteristic of AML and higher-risk MDS through PAK1, identifying PI3K-gamma as a therapeutic target whose inhibition can re-engage myeloid differentiation without imposing intrinsic myelosuppression or lymphoid toxicity..

在髓系恶性肿瘤中，PI3K-γ信号通路通过PAK1强化了急性髓系白血病（AML）和较高危骨髓增生异常综合征（MDS）特征性的分化阻滞，表明PI3K-γ是一个治疗靶点，抑制该靶点可在不引起固有骨髓抑制或淋巴细胞毒性的情况下重新激活髓系分化。

About Stelexis

关于 Stelexis

Stelexis BioSciences, Inc. is a privately held clinical-stage biopharmaceutical company developing targeted therapies for myeloid malignancies. Stelexis was founded by Deerfield Management and is headquartered in Waltham, Massachusetts.

Stelexis BioSciences, Inc. 是一家私营的临床阶段生物制药公司，致力于开发针对髓系恶性肿瘤的靶向疗法。Stelexis 由 Deerfield Management 创立，总部位于马萨诸塞州沃尔瑟姆。

Stelexis' Co-Founders discovered that PAK1 is a key kinase maintaining the malignant stem cell phenotype responsible for the maturation block in patients with MDS or AML (Pandolfi et al., Blood 2015), later they identified PI3K-gamma as a core vulnerability in myeloid malignancies, and eventually showed that eganelisib-mediated inhibition of PI3K-gamma acts via inhibition of PAK1 to exert its therapeutic effects (Luo et al., Nature 2024). .

Stelexis的联合创始人发现，PAK1是一种关键激酶，负责维持导致MDS或AML患者成熟阻滞的恶性干细胞表型（Pandolfi等，《Blood》2015）；随后，他们鉴定出PI3K-gamma是髓系恶性肿瘤的核心脆弱性，并最终证明，eganelisib介导的PI3K-gamma抑制是通过抑制PAK1来发挥治疗作用的（Luo等，《Nature》2024）。

Stelexis is advancing eganelisib as a first-in-class treatment for HR-MDS and AML, and Stelexis is developing a potential best-in-class anti-IL-1RAP antibody for the treatment of Lower-Risk MDS, and other indications.

Stelexis正在推进eganelisib作为治疗HR-MDS和AML的同类首创药物，同时Stelexis正在开发一种潜在的同类型最优抗IL-1RAP抗体，用于治疗低危MDS及其他适应症。

For more information, please visit

如需更多信息，请访问

www.stelexis.com

www.stelexis.com

.

。

SOURCE Stelexis BioSciences, Inc.

来源：Stelexis BioSciences, Inc.

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