France-based Servier and U.K.-based MiNA Therapeutics are partnering on small activating RNA (saRNA) therapies to treat neurological diseases. saRNA is an entirely new class of drugs. They are small oligonucleotides, similar to siRNAs. This technology is MiNA’s focus, based on inventions from the company’s founder Pål Saetrom, which were assigned to MiNA by the Norwegian University of Science and Technology. The company has also in-licensed fundamental patents from UT Southwestern Medical Center that covers RNA activation therapeutics.
MiNA will leverage its saRNA platform to identify possible treatments to restore normal cell function in neurological diseases. Servier will handle preclinical and clinical development of potential candidates. Servier also will have the option for commercialization of any products coming out of the partnership.
Servier is paying MiNA an undisclosed payment up front and an exclusivity fee on specific neurological targets. MiNA is also up for various milestones and royalties. MiNA is eligible for up to 220 million euros ($268 million, U.S.) in upfront, development and commercial milestone payments for the first target. No specific neurological indications were disclosed.
“MiNA’s innovative approach to activate gene expression through small activating RNAs is an exciting opportunity to unlock potential for the treatment of genetically defined neurodegenerative diseases, for which there are currently limited treatment options,” said Ross Jeggo, Head of the Servier Neurology and Immuno-inflammation Therapeutic Area. “We are delighted to welcome the MiNA team and to combine their unique approach to restoring cellular function with Servier’s focus on treating neurological diseases.”
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Small activating RNAs are short, double-stranded oligonucleotides. They selectively increase gene transcription. RNA activation (RNAa) was first described in 2006. In that study, saRNA that targeted the promoter region of a gene was found to activate transcription of the gene. Since then, research has shown that in mammals, saRNAs can activate a wide variety of genes.
MiNA’s pipeline currently consists of five compounds, three in the discovery stage for undisclosed metabolic, immuno-oncology and genetic targets. The other two programs are for the same drug, MTL-CEBPA, which are in Phase I/II trials, one with Bayer and Onyx Pharmaceuticals Nexavar (sorafenib) for hepatocellular carcinoma (HCC) and the other with Merck’s checkpoint inhibitor Keytruda (pembrolizumab) for advanced solid tumors.
The CEBPA gene encodes CCAAT/enhancer binding protein alpha, a transcription factor. This factor behaves as a master regulator of cell lineage determination and differentiation of myeloid cells, as well as other cell types. In the microenvironment of solid tumors, myeloid cells are often dysregulated. Restoring CEBPA expression in myeloid cells is believed to change immune cell populations in the tumor microenvironment and improve the effectiveness of cancer therapies.
In May 2020, MiNA presented topline data from the Phase Ib dose escalation and cohort expansion study, OUTREACH, of MTL-CEBPA and sorafenib in HCC. The trial hit the primary endpoints of safety and tolerability. Also, five patients experienced objective tumor responses, including two complete responses during the combination regimen.
“Working together with Servier, an established pharmaceutical leader in the treatment of CNS disorders, will enable us to expand the evaluation of our platform beyond our current liver-focused indications in an area of great unmet medical need,” said Robert Habib, chief executive officer of MiNA. “Following our first clinical proof-of-concept in cancer, this partnership in neurodegenerative disorders further demonstrates the potential of saRNAs as a promising treatment modality providing a new path to address previously undruggable targets.”
Source: Biospace
