HOUSTON, Nov. 12, 2024 — Moleculin Biotech, Inc., (Nasdaq: MBRX) (“Moleculin” or the “Company”), a late-stage pharmaceutical company with a broad portfolio of drug candidates targeting hard-to-treat tumors and viruses, today announced it has received Institutional Review Board (IRB) approval for its Phase 3 pivotal trial protocol evaluating Annamycin in combination with Cytarabine (also known as “Ara-C” and for which the combination of Annamycin and Ara-C is referred to as “AnnAraC”) for the treatment of AML patients who are refractory to or relapsed after induction therapy (R/R AML) (MB-108).

休斯顿，2024年11月12日-Moleculin Biotech，Inc.（Nasdaq:MBRX）（“Moleculin”或“公司”）是一家晚期制药公司，拥有针对难治性肿瘤和病毒的广泛候选药物组合，今天宣布已获得机构审查委员会（IRB）批准其第三阶段关键试验方案，该方案评估阿霉素联合阿糖胞苷（也称为“Ara-C”，其中阿霉素和Ara-C的组合被称为“AnnAraC”）用于治疗诱导治疗（R/R AML）（MB-108）后难治或复发的AML患者。

This Phase 3 “MIRACLE” trial (derived from Moleculin R/R AML AnnAraC Clinical Evaluation) will be a global trial, including sites in the US..

这项3期“奇迹”试验（源自Moleculin R/R AML AnnAraC临床评估）将是一项全球试验，包括美国的网站。。

“IRB approval marks an important milestone towards the launch of our MIRACLE pivotal Phase 3 trial of Annamycin in AML patients. Our team is focused on getting clinical trial sites up and running, and we believe we will be in a position to commence enrollment in the first quarter of 2025,” commented Walter Klemp, Chairman and Chief Executive Officer of Moleculin.

Moleculin董事长兼首席执行官Walter Klemp评论道：“IRB的批准标志着我们在AML患者中启动安那霉素奇迹关键3期试验的一个重要里程碑。我们的团队专注于建立和运行临床试验站点，我们相信我们将能够在2025年第一季度开始注册。”。

“We have been extremely busy meeting with potential investigators for this study and are highly encouraged by these conversations. The positive feedback from the clinicians on the strength of our data generated to date, along with our alignment with FDA on the strategic design of the MIRACLE study, gives us a high level of confidence as we take these next steps toward potentially bringing a much needed solution to AML patients.” .

“我们一直忙于与这项研究的潜在研究人员会面，并受到这些对话的高度鼓舞。临床医生对我们迄今为止生成的数据的积极反馈，以及我们与FDA在MIRACLE研究的战略设计上的一致性，为我们采取下一步可能为AML患者带来急需的解决方案提供了高度的信心。”。

The MIRACLE study, subject to appropriate future filings with and potential additional feedback from the FDA and their foreign equivalents, is expected to initially utilize an adaptive design whereby the first 75 to 90 subjects will be randomized in Part A of the trial to receive high dose cytarabine (HiDAC) combined with either placebo, 190 mg/m2 of Annamycin, or 230 mg/m2 of Annamycin, such doses were specifically recommended by the FDA in our end of Phase 1B/2 meeting.

MIRACLE研究将在未来向FDA及其国外等效机构提交适当的申请并获得潜在的额外反馈，预计最初将采用适应性设计，将前75至90名受试者随机分配到试验的A部分，以接受高剂量阿糖胞苷（HiDAC）联合安慰剂，190 mg/m2的Annamycin或230 mg/m2的Annamycin，这些剂量是FDA在1B/2期会议结束时特别推荐的。

At that point, the trial will be unblinded to select the optimum dose for Annamycin. For Part B of the trial, approximately 240 additional subjects will be randomized to receive either HiDAC plus placebo or HiDAC plus the optimum dose of Annamycin. The selection of the optimum dose will be based on the overall balance of safety, pharmacokinetics and efficacy, consistent with the FDA’s new Project Optimus initiative..

届时，该试验将公开选择阿纳霉素的最佳剂量。对于试验的B部分，大约240名额外的受试者将被随机分配接受HiDAC加安慰剂或HiDAC加最佳剂量的安娜霉素。最佳剂量的选择将基于安全性，药代动力学和功效的整体平衡，与FDA的新项目Optimus倡议一致。。

Annamycin currently has Fast Track Status and Orphan Drug Designation from the FDA for the treatment of relapsed or refractory acute myeloid leukemia, in addition to Orphan Drug Designation for the treatment of soft tissue sarcoma. Furthermore, Annamycin has Orphan Drug Designation for the treatment of relapsed or refractory acute myeloid leukemia from the European Medicines Agency (EMA)..

除了用于治疗软组织肉瘤的孤儿药外，Annamycin目前还具有快速通道状态和FDA指定的孤儿药，用于治疗复发或难治性急性髓细胞白血病。此外，Annamycin还被欧洲药品管理局（EMA）指定为孤儿药，用于治疗复发或难治性急性髓细胞白血病。。

About Moleculin Biotech, Inc.

关于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.

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 pathogenic 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。Moleculin还参与开发一系列抗代谢物，包括用于潜在治疗致病病毒的WP1122，以及某些癌症适应症。。