MiNA Therapeutics Announces Publication of Phase I Liver Cancer Data in Clinical Cancer Research and Provides Update on Clinical Development and Drug Discovery Programs

May 27, 2020 09:00 UTC

LONDON--(BUSINESS WIRE)-- MiNA Therapeutics, the pioneer in RNA activation therapeutics, announced today the publication of data from its Phase I liver cancer trial, OUTREACH, in Clinical Cancer Research. It is the first publication in which a small activating RNA treatment (MTL-CEBPA) demonstrated clinical benefit. In addition, the Company provided an update on its ongoing clinical trials for lead program MTL-CEBPA and its drug discovery programs.

“This landmark publication in Clinical Cancer Research details for the first time that RNA medicines can activate gene expression, providing clinical benefit to patients,” commented Robert Habib, CEO of MiNA Therapeutics. “As we enter into the second half of 2020, we continue to advance our clinical development objectives and uncover the vast opportunities inherent in our unique drug discovery pipeline.”

Publication and OUTREACH Study Update

The publication in Clinical Cancer Research summarizes the results from MiNA’s Phase I, open-label, dose escalation and dose expansion trial of MTL-CEBPA, OUTREACH, in adults with advanced Hepatocellular Carcinoma (HCC). Overall, MTL-CEBPA was well-tolerated and demonstrated pharmacodynamic target engagement, meeting the primary endpoint of the study. Furthermore, a reduction of suppressive immune cells in the tumour microenvironment as well as initial signs of potential synergistic efficacy when combined with standard of care tyrosine kinase inhibitors in HCC could be observed. These encouraging Phase I data validate the targeting of C/EBP-α as a novel therapeutic strategy in cancer and prompted a Phase Ib study further evaluating MTL-CEBPA in combination with sorafenib in HCC. Enrolment for the Phase Ib part of the OUTREACH trial was completed in Q1 2020 and initial results will be presented during a poster session at the forthcoming American Society of Clinical Oncology (ASCO) on Friday, May 29, 2020. The framework for a subsequent Phase II clinical trial is currently being designed with the objective of initiating this next stage of clinical development in the second half of 2020.

The full Clinical Cancer Research publication is available on the “Publications” page of MiNA’s website. A similar overview of the Phase I data was most recently presented at the European Society for Medical Oncology (ESMO) in September 2019.


In March 2020, TIMEPOINT, a global Phase I/Ib clinical study of MTL-CEBPA in combination with anti-PD1 checkpoint inhibitor pembrolizumab in patients with advanced solid tumours was initiated and the first patient was treated. The study is designed to assess the safety, tolerability, pharmacology and clinical activity of MTL-CEBPA in combination with pembrolizumab in these patients. Recruitment for the study is expected to continue through 2021.

Discovery Programs

In parallel to the clinical trial developments, MiNA is further expanding its drug discovery pipeline with a focus on developing new drug candidates that can address a range of indications including genetic and metabolic diseases. Most recently in January 2020, MiNA validated its metabolic disease capabilities through the entry into a research collaboration with AstraZeneca, a global leader in the discovery and development of prescription medicines to treat metabolic diseases. MiNA remains well-positioned to build out its early-stage pipeline based on its saRNA approach which, through transcriptional activation, enables the modulation of previously undruggable targets.


MTL-CEBPA is the first therapy to specifically up-regulate 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 identified as a critical barrier for many therapies to induce clinical responses in solid tumour cancers. In pre-clinical studies MTL-CEBPA has been shown to improve the anti-tumour activity of cancer therapies by targeting dysregulated myeloid cells and reducing their suppression in the tumour microenvironment.

About MiNA Therapeutics

Harnessing an innate mechanism of gene activation, MiNA Therapeutics' platform enables the development of new medicines that restore normal function to patients’ cells. We are applying our technology and clinical know-how to transform the therapy landscape of cancer and other severe diseases. www.minatx.com

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MiNA Therapeutics
Robert Habib, CEO
Phone: +44 208 811 6700
E-Mail: info@minatx.com

Media requests:
Joanne Tudorica or Gretchen Schweitzer
Trophic Communications
Phone: +49 171 351 2733
E-Mail: tudorica@trophic.eu


Source: MiNA Therapeutics

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