- Roche will work with Ribometrix to develop chemical-based drugs aimed at targets on RNA molecules, the small Durham, North Carolina biotech announced Wednesday.
- The Swiss pharma will pay Ribometrix $25 million to kick off the deal, which is heavily backloaded with conditional payments that could exceed $1 billion if certain milestones are hit. The two will collaborate on preclinical development, after which Roche's Genentech division will be responsible for clinical testing and commercialization.
- The agreement is the second collaboration of its kind Roche has struck since April, when the company partnered with another privately held biotech, Arrakis Therapeutics. Large drugmakers are pushing into the emerging field of research, which offers a way to reach drug targets inside cells that are difficult to get at through other methods.
Not too long ago the only way chemical-based medicines reached RNA molecules was by accident. Scientists had considered it futile to try to do so purposefully, given how much RNA — the molecular strands that turn DNA instructions into proteins — twist and change shape inside cells.
But a change in thinking appears underway. Over the past several years, a long list of startups — among them Ribometrix, Arrakis Therapeutics, Skyhawk Therapeutics, Accent Therapeutics and Expansion Therapeutics — have each formed with plans to use small molecule drugs to effectively freeze RNA molecules. For instance, Ribometrix, which was launched in 2014, identifies three-dimensional RNA structures and hunts for clefts or crevices within them that a small molecule can latch onto.
Frozen in place, the RNA can't shift around and perform a specific task, such as making a potentially harmful protein. The strategy could help companies access targets previously considered "undruggable."
The work behind these startups remains very early. None of them have drug candidates near human testing. Yet interest in their work has picked up. Over the past year, Roche, Vertex Pharmaceuticals and AstraZeneca have each cut deals with companies in the space, adding to a list that already included Biogen and Merck & Co.
Drugging RNA with pills: Select deals since April 2020
|Startup||Partner||Date||Terms (upfront + milestones)|
|Arrakis Therapeutics||Roche||4/8/2020||$190M + "several billion dollars"|
|Accent Therapeutics||AstraZeneca||6/4/2020||$55M + $1.1B|
|Skyhawk Therapeutics||Vertex||12/22/2020||$40M + $2.2B|
|Ribometrix||Roche||1/6/2021||$25M + $1B|
Roche is among the most heavily invested in the field, having now formed alliances with both Ribometrix and Arrakis. Its deal with Arrakis was the field's largest, consisting of $190 million in cash and "several billion dollars" in milestones. The Ribometrix pact is much smaller by comparison, but nonetheless gives Roche access to another technology meant to help drug RNA.
Roche "is committed to exploring innovative approaches to drug targets that are difficult to address with conventional approaches," its head of partnering, James Sabry, said in a statement.
The two companies didn't disclose the diseases or targets they are focusing on. Roche will get exclusive license to any compound that emerges from the partnership.