Ribometrix and Genentech Partner in Potential $1 Billion+ RNA Deal

Genentech_Justin Sullivan/Getty Images

Justin Sullivan/Getty Images

Durham, North Carolina-based Ribometrix announced a strategic collaboration deal with Genentech, a Roche company, to identify and advance novel RNA-targeted small molecule therapeutics. Ribometrix’s technology platform focuses on identify 3D “motifs” in RNA molecules so it can then design small molecule drugs that bind to these motifs to treat diseases.

Targeting RNA is believed to be a way to develop therapeutics for so-called undruggable proteins. This is because many proteins do not have small-molecule binding sites. So therapeutics that focus on RNA are designed to stop the DNA from coding for disease-causing proteins.

Two prime examples of proteins that don’t have small-molecule binding sites are KRAS and c-Myc, both common cancer pathways, and what the National Cancer Institute has dubbed “co-conspirators in cancer.”

This is not the first deal for RNA therapeutics for Genentech. In April 2020, Roche inked a deal with Waltham, Massachusetts-based Arrakis Therapeutics to discover RNA-targeted small molecule drugs against a range of targets. Under that deal, Genentech paid Arrakis $190 million in cash with undisclosed milestones.

Under the terms of the Ribometrix-Genentech collaboration, Genentech is paying Ribometrix $25 million up front, gaining exclusive rights to several predefined targets as well as an exclusive global license for the development and commercialization of compounds that come out of the collaboration. Ribometrix is eligible for possible milestone payments that could exceed $1 billion in addition to tiered royalties on future global net sales.

“The ability to specifically target the 3D structure of RNA opens up an array of new possibilities in medicine, and Ribometrix is building the capabilities to deliver on that promise,” said Michael Solomon, chief executive officer of Ribometrix. “This collaboration with Genentech unites our RNA therapeutics leadership with their global reach and broad expertise to accelerate development of this important new class of medicines.”

On September 30, 2019, Ribometrix signed a strategic collaboration deal with Boston’s Vertex Pharmaceuticals. Again, the goal was to discover and develop novel RNA-targeted small molecule therapeutic candidates for serious diseases. That deal was for up to three therapeutic programs, including a program that was ongoing at the time from Ribometrix.

Under that collaboration, Vertex paid Ribometrix $20 million up front, as well as an equity investment in Ribometrix. Ribometrix is eligible for more than $700 million in total potential payments, as well as royalties.

The Ribometrix technology platform leverages multiple specialized technologies. The first identifies the 3D RNA motifs. The second is to discover small molecules that bind to the 3D motifs. It has state-of-the-art proprietary assays to identify ligands that bind specifically to the structural pockets in many RNA molecules. They indicate that the potential benefits over current protein and RNA-based approaches are “potency, selectivity, oral bioavailability, tissue distribution and central nervous system penetration.”

“Genentech is committed to exploring innovative approaches to drug targets that are difficult to address with conventional approaches,” said James Sabry, global head of Pharma Partners, Roche. “Ribometrix has built capabilities to drug specific RNAs, and we look forward to a productive collaboration to explore this emerging area of science.”

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