Genentech, a member of the Roche Group, will apply Ribometrix’s discovery platform to discover, develop, and commercialize small molecule drug candidates against RNA targets, through a collaboration that could generate more than $1 billion for the Durham, NC-based developer of RNA-based small molecule therapeutics, the companies said today.

The companies have agreed to partner on the discovery and preclinical development of programs, using Ribometrix’s proprietary discovery platform to identify and optimize small molecule compounds that modulate RNA function by targeting three-dimensional (3D) RNA structures.

Genentech and Ribometrix did not announce how many RNA targets they will pursue, except to say it will be “several.”

Ribometrix’s platform uses structural analytic capabilities and other specialized technologies to identify 3D motifs in RNA molecules, then design small molecule candidates designed to bind to these motifs in order to produce a therapeutic effect.

The company has shown that several RNAs associated with disease contain structural pockets—clefts and crevices—that are amenable to targeting with small molecules. Ribometrix identifies ligands intended to therapeutically modulate these RNAs by binding specifically in these crevices.

“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,” Ribometrix CEO Michael Solomon, PhD, said in a statement.

According to Ribometrix, potential benefits of small molecule binders of RNA over current protein and RNA-based approaches include 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,” added James Sabry, MD, PhD, global head of Pharma Partnering, Roche. “Ribometrix has built capabilities to drug specific RNAs, and we look forward to a productive collaboration to explore this emerging area of science.”

Genentech has agreed to oversee further development and commercialization.

Under the companies’ exclusive collaboration, Genentech has agreed to pay Ribometrix $25 million upfront, in return for which it will receive exclusive rights to several predefined targets including an exclusive global license for the development and commercialization of molecules.

Genentech also agreed to pay Ribometrix more than $1 billion in payments tied to achieving milestones, as well as tiered royalties on future global net sales of products that result from the collaboration.

Privately-held Ribometrix was established in 2014 to commercialize technologies developed by its co-founders, Kevin Weeks, PhD, Kenan Distinguished Professor of Chemistry at the University of North Carolina (UNC) at Chapel Hill, and Katie Warner, PhD, who is the company’s vice president, RNA Biology. The main operations of Ribometrix are in Durham within Research Triangle Park, with offices in Chapel Hill, NC, and Boston.

In September 2019, Ribometrix launched a partnership valued at up to $700 million, plus royalties, with Vertex Pharmaceuticals focused on discovering and developing novel RNA-targeted small molecule therapeutic candidates for up to three therapeutic programs, including one ongoing discovery program from Ribometrix, targeting unspecified “serious” diseases. The partnership with Vertex began with a $20 million upfront payment that included an equity investment by Vertex in Ribometrix.

A year earlier in November 2018, Ribometrix completed a $30 million Series A financing. M Ventures led the round with new investors Amgen Ventures, Pappas Capital and Illumina Ventures joining founding investors SV Health Investors, AbbVie Ventures, Hatteras Venture Partners, MP Healthcare Venture Management, the Dementia Discovery Fund, and Alexandria Venture Investments.