--Data Published in Molecular Therapy--
LONDON--(BUSINESS WIRE)--MiNA Therapeutics, the pioneer in RNA activation therapeutics, today announced the publication of new pre-clinical data supporting the on-target mechanism of action of drug candidate MTL-CEBPA. MTL-CEBPA is the first development candidate to emerge from MiNA’s RNA activation platform, consisting of CEBPA-51 small activating RNAs encapsulated in SMARTICLES® nanoparticles, and is currently being evaluated in a Phase I clinical study in patients with liver cancer.
The paper, entitled “Development and mechanism of small activating RNA targeting CEBPA, a novel therapeutic in clinical trials for liver cancer”, was published in Molecular Therapy by researchers at MiNA Therapeutics in collaboration with scientists at Imperial College London, Norwegian University of Science and Technology, City of Hope and the Scripps Research Institute.
A number of scientific findings of CEBPA-51 were reported including a direct gene activation mechanism evidenced by physical interaction of CEBPA-51 at the CEBPA gene and increased transcriptional activity of the CEBPA gene. These findings are consistent with externally published mechanisms of small activating RNA including the presence and dependence of a cell’s endogenous gene regulation machinery. CEBPA-51 was shown to be a highly precise activator of CEBPA, supported by a sequence specific mechanism. In addition, a favourable safety profile of CEBPA-51 was evidenced by the absence of potential off-target effects.
“These comprehensive research findings elegantly support an on-target gene activation mechanism of CEBPA-51, supporting its selection as a component of our first clinical candidate MTL-CEBPA” commented Robert Habib, CEO of MiNA Therapeutics.
The paper is available on the Company’s website in the publications section under “Media”.
About MTL-CEBPA
MTL-CEBPA consists of a double stranded RNA
formulated into a SMARTICLES® liposomal nanoparticle and is
designed to activate the CEBPA gene. By restoring CEBPA expression to
normal levels, MTL-CEBPA has been demonstrated to attenuate or reverse
liver disease in a range of pre-clinical studies including models of
liver cancer, liver cirrhosis, non-alcoholic fatty liver disease (NAFLD)
and non-alcoholic steatohepatitis (NASH). MTL-CEBPA is currently under
evaluation in OUTREACH, a first-in-human Phase I clinical study in
patients with severe liver cancer. The multi-centre Phase I study is
assessing the safety and tolerability of MTL-CEBPA in patients with
advanced liver cancer who are ineligible or resistant to standard
therapies. To learn more about the OUTREACH clinical study, please visit
our listing at clinicaltrials.gov
About MiNA Therapeutics
Harnessing the 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 severe liver and other diseases. www.minatx.com
MiNA Therapeutics
Robert Habib, CEO
Phone: +44 208 811
6700
E-Mail: info@minatx.com
or
Media
requests:
MacDougall Biomedical Communications
Stephanie
May
Phone: +49 89 2424 3494
E-Mail: smay@macbiocom.com
or
Gretchen
Schweitzer
+49 175 571 1562