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HOUSTON, Sept. 9, 2024. Moleculin Biotech, Inc., ("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). 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. 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. 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." 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. If treatment is well tolerated in these subjects, we will continue enrollment for the balance of the trial." 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). 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. "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. 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. 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 months 2 . 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 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. 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. Annamycin is currently in development for the treatment of relapsed or refractory acute myeloid leukemia (AML) and soft tissue sarcoma (STS) lung metastases. 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. This study is subject to appropriate future filings with potential additional feedback from the FDA and their foreign equivalents. 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. Forward-Looking Statements Some of the statements in this release are forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, Section 21E of the Securities Exchange Act of 1934 and the Private Securities Litigation Reform Act of 1995, which involve risks and uncertainties. Although Moleculin believes that the expectations reflected in such forward-looking statements are reasonable as of the date made, expectations may prove to have been materially different from the results expressed or implied by such forward-looking statements. Moleculin has attempted to identify forward-looking statements by terminology including 'believes,' 'estimates,' 'anticipates,' 'expects,' 'plans,' 'projects,' 'intends,' 'potential,' 'may,' 'could,' 'might,' 'will,' 'should,' 'approximately' or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. These forward-looking statements, include, but are not limited to, the ability for the study to fill the safety lead-in group of six subjects on a timely basis and whether the preclinical results will be demonstrated in the Phase 2 trial. These statements are only predictions and involve known and unknown risks, uncertainties, and other factors, including those discussed under Item 1A. "Risk Factors" in our most recently filed Form 10-K filed with the Securities and Exchange Commission (SEC) and updated from time to time in our Form 10-Q filings and in our other public filings with the SEC. Any forward-looking statements contained in this release speak only as of its date. We undertake no obligation to update any forward-looking statements contained in this release to reflect events or circumstances occurring after its date or to reflect the occurrence of unanticipated events. References 1 Thakkar J, Dolecek TA, Horbinski C, Ostrom QT, Lightner DD, Barnholtz-Sloan JS, et al. Epidemiologic and molecular prognostic review of glioblastoma. Cancer Epidemiol. Biomarkers Rev. 2014;23(10):1985-96. 2 Koshy M, Villano JL, Dolecek TA, Howard A, Mahmood U, Chmura SJ, et al. Improved survival time trends of glioblastoma using the SEER 17 population-based registries. J Neuro Oncol. 2012;107(1):207-12 3 Ostrom QT, Gittleman H, Farah P, Ondracek A, Chen Y, Wolinsky Y, et al. CBTRUS statistical report: Primary brain and central nervous system tumors diagnosed in the United States in 2006-2010. Neuro Oncol. 2013;15 Suppl:2ii-56. 4 De Vleeschouwer S, editor. Brisbane (AU): Codon Publications; 2017 Sep 27. Whatever your topic of interest, subscribe to our newsletters to get the best of Drugs.com in your inbox.

SAN FRANCISCO--( BUSINESS WIRE )-- BIOMAKERS , a Precision Medicine & Biotech Company that supports data-driven Drug Development globally, and TOPAZIUM , an AI-driven healthtech company specializing in drug discovery and human wellbeing, have announced the results of their innovative collaborative project. This partnership showcases the power of AI systems in leveraging real-world DNA sequencing data to identify novel targets aimed at improving the efficacy of immunotherapy for non-small cell lung cancer (NSCLC) treatment. The findings were presented at the prestigious 2024 American Society of Clinical Oncology (ASCO) meeting in Chicago. The study used Whole Exome Sequencing (WES) data from biopsies of squamous and adenocarcinoma NSCLC processed through BIOMAKERS ' advanced DNA sequencing platforms. The data was then analyzed using GFPrint™, a proprietary algorithm developed by TOPAZIUM , that generates virtual representations of tumor exomes in a latent space defined by the genetic features of the samples. “The advent of drugs targeting the PD-1/PD-L1 axis has revolutionized NSCLC treatment. Although promising, individual responses to these therapies can vary, and we believe this ongoing project will help to better understand mechanisms involved in PD1/PD-L1 expression, optimize treatment protocols, and identify which patients are most likely to benefit from these therapies. In this study, DNA sequencing and GFPrint™ were employed to identify mutations in genes linked to PD-L1 expression in NSCLC adenocarcinoma tumor samples,” said Dr. Rubén Salanova, Chief Medical Officer & Co-Founder of BIOMAKERS . “Using this strategy, we have identified a subpopulation of adenocarcinoma patients with increased PD-L1 expression levels, enriched in non-silent mutations of 10 genes of interest, mostly included in the HIF-1α signaling pathway. Notably, 6 of these mutated genes (PIK3R1, HK1, NOS2, NRAS, BRAF, ITGA1) were uniquely found in adenocarcinomas” said Juan Manuel Domínguez, Head of Preclinical & Biostatistics at TOPAZIUM . “All these newly identified genes deserve further studies to validate them as potential targets for pharmacological intervention concomitant with PD-1/PD-L1 blockade, and as potential biomarkers for patient selection to improve the efficiency of immunotherapy in NSCLC treatment,” added Andrea Mendoza, Head of R&D at BIOMAKERS . For more details, the abstract can be accessed at: https://ascopubs.org/doi/10.1200/JCO.2024.42.16_suppl.e13600 For more information about GFPrint™, visit: https://www.biorxiv.org/content/10.1101/2024.03.08.584090v4 About BIOMAKERS: BIOMAKERS is an Oncology Precision Medicine Company with a Comprehensive Genomic Platform and reach throughout LATAM, delivering care-changing solutions. In addition to its strong focus on physician support and patient care, their Life Science division partners with biopharma companies to advance R&D programs by providing support for Drug Discovery and Drug Development. This innovative work is enabled by direct partnerships with renowned Health Institutions across 17 different countries. contacto@biomakers.net About TOPAZIUM: TOPAZIUM is committed to supporting medical research with artificial intelligence (AI) through state-of-the-art analytical tools. They have developed a comprehensive AI engine that utilizes multiple medical data types to assist in identifying new disease patterns and the most promising therapeutic approaches, striving to achieve one of the most challenging global goals: health for all. info@topazium.com

FARMINGTON HILLS, Mich., June 06, 2024 (GLOBE NEWSWIRE) -- Ocuphire Pharma, Inc. (Nasdaq: OCUP) (“Ocuphire”), a clinical-stage biopharmaceutical company focused on developing small molecule therapies for the treatment of patients with retinal and refractive eye disorders, today announced that clinical updates on the Company’s lead candidate APX3330 for diabetic retinopathy (DR) will be featured in upcoming presentations at the Clinical Trials at the Summit meeting on June 8 in Park City, Utah, and the Retinal Imaging Biomarkers & Endpoints Summit meeting June 25-27 in Boston. APX3330 is an oral small-molecule inhibitor of Ref-1 (reduction oxidation effector factor-1 protein) being developed for the treatment of non-proliferative diabetic retinopathy (NPDR). DR affects approximately 10 million of the 38 million Americans with diabetes and is the leading cause of blindness in working age adults, and 80 percent of DR patients have NPDR. “Clinical Update on Oral APX3330 for Diabetic Retinopathy” will be presented by Veeral Sheth, M.D., M.B.A., a practicing retina specialist at University Retina and Clinical Assistant Professor at the University of Illinois, at the Clinical Trials at the Summit meeting. Clinical Trials at the Summit brings together a diverse group of experts to discuss ongoing clinical trials and the latest data to accelerate advances in vitreoretinal care. “Multiplex Analysis of Clinical Imaging & Biomarker Data to Validate Novel Endpoints for Diabetic Retinopathy” will be presented by Ashwath Jayagopal, Ph.D., Ocuphire’s Chief Scientific and Development Officer, at the second annual Retinal Imaging Biomarkers & Endpoints Summit. The Retinal Imaging Biomarkers & Endpoints Summit is the only industry-dedicated event focusing on streamlining imaging data analysis with the implementation of AI-based predictive measures for earlier detection and diagnosis to optimize patient stratification, selection and recruitment for trials. “APX3330 has the potential to be a novel treatment that addresses multiple established disease pathways for NPDR,” said George Magrath, M.D., M.B.A., M.S., Ocuphire’s Chief Executive Officer. “We are grateful for multiple opportunities to share an update on our APX3330 clinical program and our planned ZETA-2 pivotal trial, including the unmet need for an earlier intervention treatment to delay or prevent DR progression, the value of multimodal imaging in DR, and Phase 2/3 trial optimization and patient selection, with industry and scientific peers.” Ocuphire anticipates initiating the ZETA-2 Phase 2/3 trial of APX3330 for NPDR in early 2025. About Ocuphire Pharma Ocuphire is a clinical-stage ophthalmic biopharmaceutical company focused on developing novel therapies for the treatment of retinal and refractive eye disorders. Ocuphire’s lead retinal product candidate, APX3330, is an oral small-molecule inhibitor of Ref-1 (reduction oxidation effector factor-1 protein) for the treatment of non-proliferative diabetic retinopathy (NPDR). Ref-1 is a regulator of the transcription factors HIF-1α and NF-κB. Inhibiting REF-1 reduces levels of vascular endothelial growth factor (VEGF) and inflammatory cytokines which are known to play key roles in ocular angiogenesis and inflammation. APX3330 is an oral tablet to be administered twice per day for the treatment of diabetic retinopathy (DR). A Phase 2 study in subjects with DR and an End-of-Phase 2 meeting have been completed, and a special protocol assessment (SPA) was submitted to the U.S. Food and Drug Administration (FDA) in February 2024. In addition, Ocuphire has a partnership to develop and commercialize Phentolamine Ophthalmic Solution 0.75% (“PS”), a non-selective alpha-1 and alpha-2 adrenergic antagonist designed to reduce pupil size. PS was approved by the FDA for the treatment for pharmacologically-induced mydriasis under the brand name RYZUMVI™ in September 2023. PS is also in Phase 3 clinical development for the treatment of presbyopia and for the treatment of decreased visual acuity under low light (mesopic) conditions after keratorefractive surgery. Ocuphire is also developing APX2009 and APX2014, second-generation analogs of APX3330. These programs are being evaluated for treating other retinal diseases such as age-related macular degeneration and geographic atrophy. For more information, please visit . Forward Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements include, but are not limited to, statements concerning the efficacy of APX3330 in slowing the progression of diabetic retinopathy, the safety and tolerability of APX3330, ongoing discussions with the FDA regarding various of our drug products, and continued drug development under our partnership agreement. These forward-looking statements relate to us, our business prospects and our results of operations and are subject to certain risks and uncertainties posed by many factors and events that could cause our actual business, prospects and results of operations to differ materially from those anticipated by such forward-looking statements. Factors that could cause or contribute to such differences include, but are not limited to, those described under the heading “Risk Factors” included in our Annual Report on Form 10-K. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this report. In some cases, you can identify forward-looking statements by the following words: “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “aim”, “may,” “ongoing,” “plan,” “potential,” “predict,” “project,” “should,” “will,” “would” or the negative of these terms or other comparable terminology, although not all forward-looking statements contain these words. We undertake no obligation to revise any forward-looking statements in order to reflect events or circumstances that might subsequently arise. These forward-looking statements are based upon Ocuphire’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, including, without limitation: The success and timing of regulatory submissions and pre-clinical and clinical trials, including enrollment and data readouts; Regulatory requirements or developments; Changes to or unanticipated events in connection with clinical trial designs and regulatory pathways; Delays or difficulties in the enrollment of patients in clinical trials; Substantial competition and rapid technological change; Our development of sales and marketing infrastructure; Future revenue losses and profitability; Our relatively short operating history; Changes in capital resource requirements; Risks related to the inability of Ocuphire to obtain sufficient additional capital to continue to advance its product candidates and its preclinical programs; Domestic and worldwide legislative, regulatory, political and economic developments; Employee misconduct; Changes in market opportunities and acceptance; Reliance on third-parties; Future, potential product liability and securities litigation; System failures, unplanned events, or cyber incidents; The substantial number of shares subject to potential issuance associated with our equity line of credit arrangement; Risks that our partnership or other licensing arrangements, may not facilitate the commercialization or market acceptance of Ocuphire’s product candidates; Future fluctuations in the market price of our common stock; The success and timing of commercialization of any of Ocuphire’s product candidates; and Obtaining and maintaining Ocuphire’s intellectual property rights. The foregoing review of important factors that could cause actual events to differ from expectations should not be construed as exhaustive. Readers are urged to carefully review and consider the various disclosures made by us in this report and in our other reports filed with the Securities and Exchange Commission that advise interested parties of the risks and factors that may affect our business. All forward-looking statements contained in this press release speak only as of the date on which they were made. Ocuphire undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made. Contacts Corporate Investor Relations Nirav Jhaveri, M.B.A. CFO ir@ocuphire.com Corey Davis, Ph.D. LifeSci Advisors cdavis@lifesciadvisors.com