Ribometrix Signs Major R&D Deal, Gains New Funding, Names Board Chairman

Ribometrix, a Durham company specializing in RNA-based therapeutics, will partner with Vertex Pharmaceuticals of Boston to discover and develop drug candidates for serious diseases in a deal that could ultimately be worth more than $700 million to Ribometrix.

Under the terms of the agreement, Vertex will pay Ribometrix about $20 million upfront, which includes an equity investment in the company. Ribometrix is eligible to receive more than $700 million in total potential payments if specified research, development, regulatory and commercial milestones are met. In addition, Vertex will pay tiered royalties on future net global sales on any products that result from the collaboration.

“As a company founded on groundbreaking research and expertise in RNA structural biology, we have seen firsthand how new technologies are dramatically expanding the druggability of RNA and the potential for transforming the treatment of previously intractable diseases,” said Michael Solomon, Ph.D., chief executive officer of Ribometrix. “Vertex is a leader in creating transformative medicines through serial innovation, and we are proud to partner with them to rapidly and effectively develop a core discovery platform of small-molecule therapeutics targeting RNA.”

The collaboration will combine Ribometrix’s proprietary discovery platform for identifying and optimizing small-molecule therapeutics that modulate RNA function by targeting three-dimensional RNA structures with Vertex’s scientific, clinical and regulatory capabilities.

The deal covers up to three therapeutic programs, including one ongoing discovery program from Ribometrix. A potential treatment for Huntington’s disease is among the programs.

“Targeting RNA is a potentially powerful new approach for developing small-molecule therapeutics, and Ribometrix has developed an innovative technology for the accelerated discovery of selective candidates in this new space,” said Mark Bunnage, senior vice president and site head of Boston Research at Vertex. “This partnership adds a novel mechanism to our internal toolkit which has the potential to enhance our ongoing research capabilities and support the expansion of our pipeline of transformative medicines in serious diseases.”

New funding and board chairman

The news comes after last week’s announcement of $7.8 million in new funding for Ribometrix from existing investors, the Dementia Discovery Fund and Illumina Ventures, and the appointment of a new board chairman.

“This additional investment from two of our earliest supporters demonstrates confidence in our team and scientific approach,” said Solomon. “We appreciate their backing as we advance toward our goal of expanding the universe of druggable targets by identifying and optimizing functionally active RNA modulators.”

Mike Clayman, M.D., a pharmaceutical veteran, was appointed chairman of the Ribometrix board.

UNC roots with NCBiotech startup help

Ribometrix was founded by Kevin Weeks, Ph.D., Kenan Distinguished Professor of Chemistry at the University of North Carolina at Chapel Hill., and Katie Warner, Ph.D., a UNC graduate who is now vice president of RNA biology at the company.

Ribometrix completed a $30 million Series A financing in late 2018. Pappas Capital and Hatteras Venture Partners, both of Durham, invested in the round.

The investment came two years after Ribometrix received a $75,000 Company Inception Loan from the North Carolina Biotechnology Center in 2016. That support helped the company raise $7.5 million in seed capital that preceded the $30 million Series A round.

Ribometrix lists 32 employees on its website and has nine job openings posted.