HOUSTON, Sept. 9, 2024 — Moleculin Biotech, Inc., (Nasdaq: MBRX) (“Moleculin” or the “Company), a Phase 3 clinical-stage pharmaceutical company with a broad portfolio of drug candidates targeting hard-to-treat tumors and viruses, today announced the enrollment and treatment of patients in an Investigator-initiated Phase 2 study evaluating WP1066 in combination with radiation therapy for the treatment of adults with glioblastoma (NU 21C06).

Moleculin Biotech, Inc.（纳斯达克股票代码：MBRX）（以下简称 “Moleculin ”或 “公司”）是一家处于 3 期临床阶段的制药公司，拥有针对难治性肿瘤和病毒的广泛候选药物组合。

The study is being conducted under Northwestern University’s Investigative New Drug application (IND) which cross references the Company’s own IND, which received clearance from the U.S. Food and Drug Administration (FDA) in April 2022. This trial is funded by the National Institutes of Health (NIH) and BrainUp®, a non-profit organization dedicated to bringing awareness to brain cancer..

这项研究是在西北大学的调查性新药申请（IND）下进行的，该申请交叉引用了该公司自己的IND，该IND于2022年4月获得了美国食品和药物管理局（FDA）的批准。该试验由美国国立卫生研究院（NIH）和BrainUp®资助，BrainUp®是一家致力于提高脑癌意识的非营利组织。。

The NU 21C06 trial is a Phase 2, open-label, multi-arm trial of radiation therapy in combination with WP1066 in newly diagnosed IDH (isocitrate dehydrogenase) wild-type, MGMT-unmethylated glioblastoma patients. The primary outcome measure for the study is progression-free survival and secondary outcome measures include tumor microenvironment analysis..

NU 21C06试验是一项针对新诊断的IDH（异柠檬酸脱氢酶）野生型MGMT非甲基化胶质母细胞瘤患者的放射治疗与WP1066联合的2期开放标签多臂试验。该研究的主要结局指标是无进展生存期，次要结局指标包括肿瘤微环境分析。。

Dr. Priya Kumthekar, Associate Professor at Northwestern University and the Co-Investigator for the study commented, “The start of patient dosing is a significant step towards addressing the unmet need in the treatment of glioblastoma. We were very encouraged by the preclinical data that demonstrated by WP1066 in combination with radiation therapy increased survival and induced anti-tumor immune responses and are excited to finally be treating patients with this approach.”.

西北大学副教授兼该研究的共同研究者Priya Kumthekar博士评论道：“开始给患者服用药物是解决胶质母细胞瘤治疗中未满足需求的重要一步。我们对WP1066与放射治疗相结合所证明的临床前数据感到非常鼓舞，这些数据提高了生存率并诱导了抗肿瘤免疫反应，并对最终用这种方法治疗患者感到兴奋。”。

Dr. Amy Heimberger, Co-Investigator and Vice Chair for Research, Department of Neurological Surgery for Northwestern University added, “The significance of the unmet need can be seen in the pace of recruitment for this trial. Within the first few months of opening the trial, we have four subjects active in the study and expect to fill the safety lead-in group of six subjects soon.

西北大学神经外科联合研究员兼研究副主席艾米·海姆伯格博士补充说：“未满足需求的重要性可以从该试验的招募速度中看出。在试验开始的头几个月内，我们有四名受试者积极参与该研究，预计很快将填补由六名受试者组成的安全引导组。

If treatment is well tolerated in these subjects, we will continue enrollment for the balance of the trial.”.

如果这些受试者的治疗耐受性良好，我们将继续参加试验的平衡。”。

WP1066 is Moleculin’s flagship Immune/Transcription Modulator designed to stimulate the immune response to tumors by inhibiting the errant activity of regulatory T cells while also inhibiting key oncogenic transcription factors, including p-STAT3 (phosphorylated signal transducer and activator of transcription 3), c-Myc (a cellular signal transducer named after a homologous avian virus called Myelocytomatosis) and HIF-1α (hypoxia-inducible factor 1α).

WP1066是Moleculin的旗舰免疫/转录调节剂，旨在通过抑制调节性T细胞的错误活性来刺激对肿瘤的免疫反应，同时还抑制关键的致癌转录因子，包括p-STAT3（磷酸化信号转导和转录激活因子3），c-Myc（以称为骨髓细胞瘤病的同源禽病毒命名的细胞信号转导）和HIF-1α（缺氧诱导因子1α）。

These transcription factors are widely sought targets because of their role in cancer cell survival and proliferation, angiogenesis (coopting vasculature for blood supply), invasion, metastasis, and inflammation associated with tumors..

这些转录因子因其在癌细胞存活和增殖，血管生成（协调血液供应的脉管系统），侵袭，转移和与肿瘤相关的炎症中的作用而被广泛寻找。。

“The NU 21C06 study is a critical next step in the development of STAT3-targeting technologies. STAT3 has long been a sought-after but elusive cell-signaling target in cancer therapy but the work we are doing in collaboration with Northwestern University supported by the NIH and BrainUp® could change all that.

“NU 21C06研究是STAT3靶向技术发展的关键下一步。STAT3长期以来一直是癌症治疗中备受追捧但难以捉摸的细胞信号传导靶点，但我们与美国国立卫生研究院和BrainUp®支持的西北大学合作所做的工作可能会改变这一切。

We believe that based on the data demonstrated to date, WP1066 has the potential for significant anti-tumor activity in a wide range of hard-to-treat cancers,” added Mr. Walter Klemp, Chairman and Chief Executive Officer of Moleculin..

Moleculin董事长兼首席执行官Walter Klemp先生补充道：“我们相信，根据迄今为止证明的数据，WP1066在各种难以治疗的癌症中具有显着的抗肿瘤活性。”。。

Glioblastoma is a common type of tumor originating in the brain. The average annual age-adjusted incidence rate of glioblastoma is 3.19 per 100,000 persons in the United States.1 Glioblastoma is the most aggressive malignant primary brain tumor with a median survival of only 15 months2. It is the most common malignant primary brain tumor making up 54% of all gliomas and 16% of all primary brain tumors.3 Despite advancements for other cancers, the survival rate for glioblastoma has not changed significantly in the last three decades.4.

胶质母细胞瘤是起源于大脑的常见肿瘤类型。在美国，胶质母细胞瘤的平均年年龄校正发病率为每10万人3.19。胶质母细胞瘤是最具侵袭性的恶性原发性脑肿瘤，中位生存期仅为15个月2。它是最常见的恶性原发性脑肿瘤，占所有胶质瘤的54%，占所有原发性脑肿瘤的16%[3]。尽管其他癌症取得了进展，但胶质母细胞瘤的生存率在过去三十年中没有显着变化。

Moleculin has received Orphan Drug Designation for WP1066 for the treatment of brain tumors, as well as Rare Pediatric Disease designation for three other pediatric indications. For more information about the NU 21C06 Phase 2 study, visit clinicaltrials.gov and reference identifier NCT05879250.

Moleculin已被指定为用于治疗脑肿瘤的WP1066孤儿药，以及用于其他三种儿科适应症的罕见儿科疾病。有关NU 21C06 2期研究的更多信息，请访问clinicaltrials.gov和参考标识符NCT05879250。

About Moleculin Biotech, Inc.Moleculin Biotech, Inc. is a Phase 3 clinical stage pharmaceutical company advancing a pipeline of therapeutic candidates addressing hard-to-treat tumors and viruses. The Company’s lead program, Annamycin, is a next-generation anthracycline designed to avoid multidrug resistance mechanisms and to eliminate the cardiotoxicity common with currently prescribed anthracyclines.

关于Moleculin Biotech，Inc.Moleculin Biotech，Inc.是一家三期临床阶段制药公司，致力于开发治疗难治性肿瘤和病毒的候选治疗药物。该公司的主导项目安那霉素是一种下一代蒽环类药物，旨在避免多药耐药机制，并消除目前处方的蒽环类药物常见的心脏毒性。

Annamycin is currently in development for the treatment of relapsed or refractory acute myeloid leukemia (AML) and soft tissue sarcoma (STS) lung metastases..

Annamycin目前正在开发用于治疗复发或难治性急性髓细胞白血病（AML）和软组织肉瘤（STS）肺转移。。

The Company is initiating the MIRACLE (Moleculin R/R AML AnnAraC Clinical Evaluation) Trial (MB-108), a pivotal, adaptive design Phase 3 trial evaluating Annamycin in combination with cytarabine, together referred to as AnnAraC, for the treatment of relapsed or refractory acute myeloid leukemia. Following a successful Phase 1B/2 study (MB-106), with input from the FDA, the Company believes it has substantially de-risked the development pathway towards a potential approval for Annamycin for the treatment of AML.

该公司正在启动MIRACLE（Moleculin R/R AML AnnAraC临床评估）试验（MB-108），这是一项关键的适应性设计3期试验，评估Annamycin与阿糖胞苷联合治疗复发或难治性急性髓细胞白血病。在FDA的投入下，一项成功的1B/2期研究（MB-106）之后，该公司认为它已经大大降低了开发途径的风险，有望获得批准用于治疗AML的阿霉素。

This study is subject to appropriate future filings with potential additional feedback from the FDA and their foreign equivalents..

这项研究将在未来提交适当的文件，并可能获得FDA及其国外等效机构的额外反馈。。

Additionally, the Company is developing WP1066, an Immune/Transcription Modulator capable of inhibiting p-STAT3 and other oncogenic transcription factors while also stimulating a natural immune response, targeting brain tumors, pancreatic and other cancers. Moleculin is also engaged in the development of a portfolio of antimetabolites, including WP1122 for the potential treatment of viruses, as well as certain cancer indications..

此外，该公司正在开发WP1066，这是一种免疫/转录调节剂，能够抑制p-STAT3和其他致癌转录因子，同时还能刺激自然免疫反应，靶向脑肿瘤，胰腺癌和其他癌症。