Sarepta, Arrowhead Pharmaceuticals partner to advance RNA-based drugs in rare diseases

Sarepta Therapeutics said Tuesday that it will pay Arrowhead Pharmaceuticals $500 million in cash for a series of experimental RNA-based drugs to treat rare diseases in the muscle, nervous system, and lung. 

In addition to the upfront payment, Sarepta will also make a $325 million investment in Arrowhead, pay out $50 million a year for the next five years, and promise $300 million in “near-term” milestone payments related to clinical trial enrollment. Arrowhead could also receive royalties and other milestone payments collectively worth up to $10 billion, although most of those are contingent on sales.

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The deal represents the first significant investment Sarepta has made to expand its pipeline in years. 

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