UK-based MiNA Therapeutics has started the phase I clinical study (OUTREACH) of MTL-CEBPA for the treatment of patients with severe liver cancer.
The MTL-CEBPA is composed of a double-stranded RNA formulated into a SMARTICLES liposomal nanoparticle, and is intended to activate the CEBPA gene, which encodes the CCAAT / enhancer binding protein alpha (C/EBP-a).
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C/EBP-a is a transcription factor that solely regulates cell lineage determination and differentiation in several tissues including liver, myeloid cells and adipose tissue.
Within the liver, the C/EBP-a is instrumental in the normal hepatocyte function and response to injury.
OUTREACH is the first-in-human, multi-centre, Phase I clinical trial of a small activating RNA (saRNA) and is designed to assess the safety and tolerability of MTL-CEBPA, an saRNA restoring the expression of C/EBP-a, in patients with advanced primary or metastatic liver cancer who are ineligible or resistant to standard therapies.
MiNA Therapeutics CEO Robert Habib said: "Initiation of the Phase I study is an important achievement in our mission to improve patients’ lives with our groundbreaking class of medicines, known as small activating RNAs.
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By GlobalData"There is increasing excitement about the possibility of using RNA to induce therapeutic protein production.
"We believe our unique approach, here applied to the upregulation of C/EBP-a protein, may provide to patients significant benefits over conventional medicines."
The trial will involve a dose-escalation followed by a dose expansion, wherein the MTL-CEBPA will initially administered as an intravenous infusion once weekly for three weeks, followed by one week of rest.
