MTL-CEBPA

Findings demonstrate structure-activity relationship of RNAa therapeutics, supporting use in genetic medicine, as well as therapeutic potential of MiNA’s MTL-CEBPA in acute myeloid leukemia LONDON--(BUSINESS WIRE)-- MiNA Therapeutics Limited, the pioneer in small activating RNA (RNAa) therapeutics, highlights preclinical research presented by academic partners at the annual meeting of the Oligonucleotide Therapeutics Society (OTS) in Barcelona, Spain. RNAa therapeutics are a revolutionary new class of medicines that can restore or boost normal function of genes and thereby protein-modulated pathways in cells. Key Data Highlights from OTS In a presentation titled, “Investigating Chemically-Modified Short Activating RNAs to Increase Nuclease Stability and Gene Activation,” findings demonstrated improvement in the medicinal properties of RNAa therapeutics using well established chemical modifications. Another study titled, “Small-Activating RNA Therapy Development for the Inherited genetic Disorder Aniridia,” revealed the potential of RNAa therapeutics to treat a rare and severe genetic eye disease. Finally, the presentation titled, “RNA Activation of a Transcription Factor Induces Terminal Differentiation of Blood Cancer in Preclinical Studies,” detailed findings that identified the potential of MTL-CEBPA to treat acute myeloid leukemia (AML), an aggressive blood cancer with low survival rates and high burden of chemotherapeutic treatment side effects. MiNA is exploring out-licensing opportunities for MTL-CEBPA and its immuno-oncology portfolio which uniquely combines the capability to specifically restore or boost any dysregulated gene target with clinically validated in vivo delivery to myeloid immune cells. “The findings from these three preclinical studies demonstrate continued advances in the development of our novel RNAa therapeutics across a range of therapeutic areas,” said Robert Habib, CEO of MiNA Therapeutics. “We are particularly encouraged by the data that reveals the structure-activity relationship of RNAa medicines which supports our development strategy in genetic medicine.” Following the meeting, the posters for the three studies will be available on the MiNA website: . About MiNA Therapeutics MiNA Therapeutics is the global leader in small activating RNA therapeutics or RNAa. Harnessing innate mechanisms of gene activation, RNAa therapeutics are a revolutionary new class of medicines that can restore or boost normal function of genes and thereby protein-modulated pathways in cells. We are advancing a proprietary pipeline of new medicines with an initial focus on genetic medicine, while collaborating with leading pharmaceutical companies to apply our technology platform across a broad range of other therapeutic areas. Based on our unique know-how in RNA activation, we are expanding the possibilities of RNA-based medicine.

Findings validate clinical proof of mechanism of MTL-CEBPA as a combination therapy in solid tumor cancers Company to initiate out-licensing activities of its immuno-oncology portfolio, including MTL-CEBPA LONDON--(BUSINESS WIRE)-- MiNA Therapeutics Limited, the pioneer in small activating RNA (RNAa) therapeutics, will present positive updated biomarker data from its Phase 1a/b TIMEPOINT study of MTL-CEBPA in combination with the anti-PD1 checkpoint inhibitor, pembrolizumab, in adults with advanced solid tumors at the annual meeting of the American Association of Cancer Research (AACR) in Orlando, Florida. Findings from the Phase 1b portion of the study validate the clinical proof of mechanism of MTL-CEBPA as a combination treatment and identify a novel predictive biomarker of clinical response. The data will be presented on Tuesday, April 18, during a late-breaking research session beginning at 9:00 am EDT. MTL‑CEBPA is the first therapy that specifically up-regulates CCAAT/enhancer binding protein alpha (C/EBP-α), a transcription factor that acts as a master regulator of myeloid cells. Dysregulated myeloid cells are implicated in several diseases including solid tumor cancers. MTL-CEBPA is designed to improve the effectiveness of checkpoint inhibitors and other immunotherapies by enhancing the body’s immune response and ability to attack the cancerous cells. Interim data from an open-label, multi-center study demonstrated the safety, tolerability, immunological and clinical activity of MTL-CEBPA in combination with pembrolizumab. “Anti-PD1 checkpoint inhibition has significantly advanced the treatment of cancer, but many patients are resistant to treatment and present with tumors that resemble an ‘immune-desert’,” said Robert Habib, CEO of MiNA Therapeutics. “The updated analysis of TIMEPOINT shows that MTL-CEBPA used in combination with anti-PD1 checkpoint inhibitors profoundly reprograms the tumor microenvironment, reducing resistance and enabling infiltration of disease-fighting T-cells.” The TIMEPOINT study also identified a novel predictive biomarker of clinical response to the combination treatment, presenting an opportunity for further evaluation as a potential companion diagnostic. The novel biomarker predicted clinical response across multiple tumor types. MiNA plans to explore out-licensing opportunities for its immuno-oncology portfolio, which uniquely combines the capability to specifically restore or boost any dysregulated gene target with clinically validated in vivo delivery to myeloid immune cells. MiNA’s immuno-oncology portfolio includes the following programs: MTL‑CEBPA, which has been cleared for evaluation in a global Phase 2 clinical trial in combination with the tyrosine kinase inhibitor, sorafenib, in advanced hepatocellular carcinoma (HCC or liver cancer) and in a Phase 1 clinical trial in pediatric patients with MPS1 Hurler Syndrome MTL-STING currently in pre-clinical development Additional programs currently in discovery About TIMEPOINT TIMEPOINT is a global Phase 1a/b clinical study in patients with solid tumor malignancies to assess the safety and tolerability of MTL‑CEBPA in combination with pembrolizumab in patients who are ineligible or resistant to standard therapies. The study has received clearance from the U.S. Food and Drug Administration (FDA) and the UK Medicines and Healthcare products Regulatory Agency (MHRA). To learn more about the TIMEPOINT clinical study, visit ClinicalTrials.gov (NCT04105335). The poster presentation at AACR entitled, “MTL-CEBPA in combination with pembrolizumab converts an immune desert to an inflamed TME in solid tumors resistant to checkpoint blockade,” will be available on MiNA’s website in the Publications section under "RNA Activation". About MTL-CEBPA MTL-CEBPA is the first therapy that specifically up-regulates CCAAT/enhancer binding protein alpha (C/EBP-α), a transcription factor that acts as a master regulator of myeloid cell lineage determination and differentiation. Dysregulated myeloid cells have been implicated in several diseases, and in solid tumor cancers these cells have been identified as a critical barrier to induction of clinical response for many therapies. In pre-clinical studies, MTL-CEBPA has been shown to improve the anti-tumor activity of cancer therapies by targeting dysregulated myeloid cells and reducing or eliminating their suppressive effect on immune response and therapies in the tumor microenvironment. About MiNA Therapeutics MiNA Therapeutics is the global leader in small activating RNA therapeutics or RNAa. Harnessing innate mechanisms of gene activation, RNAa therapeutics are a revolutionary new class of medicines that can restore or boost normal function of genes and thereby protein-modulated pathways in patients' cells. We are advancing a proprietary pipeline of new medicines with an initial focus on genetic medicine, while collaborating with leading pharmaceutical companies to apply our technology platform across a broad range of other therapeutic areas. Based on our unique know-how in RNA activation, we are expanding the possibilities of RNA-based medicine for patients.