Ionis Pharmaceuticals, a leader in RNA-targeted therapies, entered into a strategic collaboration agreement with AstraZeneca to develop and commercialize eplontersen, Ionis’ investigational antisense medicine for the treatment of transthyretin amyloidosis (ATTR).
 
Ionis will receive $200 million upfront, up to $485 million in development and approval milestones, and up to $2.9 billion in milestone payments, as well as royalties.
 
Eplontersen, formerly known as IONIS-TTR-LRx, is designed to reduce the production of transthyretin, or TTR protein, to treat ATTR, a systemic, progressive and fatal disease. It uses Ionis’ advanced LIgand-Conjugated Antisense (LICA) technology.
 
The companies will develop a global strategy for developing, manufacturing and commercializing eplontersen.
 
Ionis will continue to lead global Phase 3 clinical trials in patients with hereditary ATTR amyloidosis (hATTR) with polyneuropathy (NEURO-TTRansform) and cardiomyopathy (CARDIO-TTRansform). Ionis will manufacture and supply eplontersen for the ongoing clinical trials and process qualifications. AstraZeneca will be responsible for commercial supply.
 
The companies will share responsibility for medical affairs and commercial activities in the U.S.
AstraZeneca will have an exclusive license for eplontersen outside the U.S. except certain countries in Latin America.