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AstraZeneca Teams with Silence Therapeutics to Develop siRNA Therapeutics

March 25, 2020

AstraZeneca and Silence Therapeutics said they will collaborate to discover, develop and commercialize small interfering RNA therapeutics for the treatment of cardiovascular, renal, metabolic, and respiratory diseases.

Photo: Iain Ross, executive chairman of Silence

Small interfering RNA (siRNA) are double-stranded RNA molecules that offer new opportunities for therapeutic intervention by targeting RNA to prevent the production of disease-causing proteins inside the cell.

The multi-target collaboration will harness Silence’s siRNA platform to identify and progress liver-based targets, as well as develop new delivery approaches for targeting other tissues such as the heart, lung and kidney. Targeted delivery to these other tissues represents a new opportunity to target novel pathways not amenable to more traditional drug discovery approaches, said Mene Pangalos, executive vice president of biopharmaceutical R&D at AstraZeneca.

Under the terms of the agreement, AstraZeneca will pay Silence Therapeutics $80 million upfront, comprised of $60 million in cash and $20 million in an equity investment. Silence will be responsible for designing siRNA molecules against gene targets selected by AstraZeneca, and for manufacturing of material to support GLP toxicology studies and phase 1 clinical studies. The companies will collaborate during the discovery phase while AstraZeneca will lead clinical development and commercialization of molecules arising from the collaboration. Silence will have the option to negotiate for co-development of two programs of their choice starting from phase 2.

AstraZeneca will pay Silence an option fee of $10 million for each selected target at the point of candidate nomination. Thereafter for each target selected, Silence will be eligible for up to $140 million in development milestones and up to $250 million in commercialization milestones as well as tiered royalties.

“It not only provides us with an opportunity to collaborate on specific liver expressed gene targets but also to work with a leading company to achieve targeted delivery of siRNA molecules to other tissues including heart, kidney and lung,” said Iain Ross, executive chairman of Silence.

Silence’s liver-focused pipeline is focused on unmet needs in hematology, cardiovascular, and rare diseases. In July 2019, Silence entered into a collaboration with the specialty pharmaceutical Mallinckrodt to develop and commercialize its discovery-stage experimental siRNA therapeutic targeting complement-mediated rare diseases.

Author: Rare Daily Staff

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