A company developing small-molecule drugs that target RNA will receive nearly $200 million from Roche under a new partnership between the two.

Waltham, Massachusetts-based Arrakis Therapeutics said Wednesday that it had signed a deal with the Basel, Switzerland-based drugmaker to use the former’s platform technology to discover RNA-targeted small molecule drugs in the latter’s research and development areas. Roche will pay $190 million to Arrakis upfront, along with potential preclinical, clinical, commercial and sales milestones. In aggregate, Arrakis could end up receiving several billion dollars, the company said.

“We are excited to partner with Roche’s strong research and development teams,” Arrakis CEO Michael Gilman said in a statement. “Together, we share a common vision of accessing new drug targets at the RNA level and thereby discovering novel medicines to treat diseases with high unmet medical need.”

Arrakis is a private company and raised $75 million in a Series B financing round about a year ago. Its pipeline page includes several potential targets, including five in oncology, one in dyslipidemia, two in rare diseases and one in neurology. All of the possible targets listed are in the screening and confirmation or hit-to-lead stage of development.

Several of Arrakis’ areas of focus in its R&D efforts overlap with those of Roche, including cardiovascular and metabolism, oncology, neuroscience and rare diseases. Others include ophthalmology, respiratory diseases, women’s health, immunology, infectious diseases and hematology.

The company is one of several that have focused on exploring RNA as a drug target. Several other firms are developing drugs that go after RNA in various ways as well, including companies that are publicly traded and already have medicines undergoing clinical trials, such as Alnylam Pharmaceuticals, Moderna and Dicerna. Arrakis focuses on known targets that have long been considered “undruggable” with conventional medicines.

“Through our pioneering work in RNA biology, we have built a platform for creating and adapting drug discovery tools that allow us to predict and validate the structure of RNA targets, locate druggable pockets, identify drug-like hits and conduct medicinal chemistry programs to discover a new class of RNA-targeted medicines optimized for potency, selectivity and safety,” said Jennifer Petter, Arrakis’ chief scientific officer, in a statement.

Photo: mediaphotos, Getty Images



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