RARE Daily

Roche and Arrakis Collaborate to Develop RNA-Targeted Small Molecule Drugs

April 8, 2020

Roche has entered into a strategic collaboration and license agreement with Arrakis Therapeutics for the discovery of RNA-targeted small molecule drugs against a broad set of targets across all of Roche’s research and development areas, which includes rare diseases.

Photo: Michael Gilman, CEO of Arrakis

Roche will pay Arrakis $190 million upfront to lead discovery and research activities for each target to a defined point, at which time Roche has the right to exclusively pursue further preclinical and clinical development. Arrakis may also receive preclinical, clinical, commercial, and sales milestone payments, and royalties for any resulting products, in aggregate potentially worth several billion dollars.

“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. The collaboration will increase the number of new treatments for patients arising from our proprietary RNA-targeted small molecule (rSM) discovery platform,” said Michael Gilman, CEO of Arrakis. “In addition to the Roche collaboration, we are further building our capabilities and advancing our wholly-owned rSM programs for diseases unaddressed by today’s medicines.”

Arrakis’ rSM platform targets the RNA lifecycle to identify RNA-targeted small molecules that directly bind to and modulate the function of RNA to predictably impact the biology of disease processes. Its discovery platform integrates RNA bioinformatic and structural tools, curated chemical libraries, RNA-specific assays, and RNA-guided medicinal chemistry.

Arrakis Founder and Chief Scientific Officer Jennifer Petter says the company has built a platform for creating and adapting drug discovery tools that allow it 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.

“This agreement with Roche underscores the value inherent in our rSM platform and will enable us to continue to make leading discoveries and further scientific contributions in the field,” said Petter.

Author: Rare Daily Staff

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