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20 Dec 2016

Ionis and AstraZeneca advance first generation 2.5 LICA drug into preclinical development to treat CV disease

Ionis earns $25 million milestone payment.

Ionis Pharmaceuticals has advanced its lead drug candidate IONIS-AZ4-2.5-LRx, which has now been renamed AZD8233, into preclinical development. AZD8233 is designed to inhibit an undisclosed target to treat cardiovascular (CV) disease and is also the first of a new class of antisense drugs that uses both Ionis' proprietary Generation 2.5 chemistry and its LIgand Conjugated Antisense or LICA technology. In conjunction with this milestone, Ionis earned a $25 million milestone payment from AstraZeneca.

"This is the first drug to enter development under our strategic collaboration with AstraZeneca in CV, metabolic and renal diseases. We are pleased with how quickly this important program has advanced. AZD8233 incorporates many of the recent advancements we have made in antisense technology, including LICA and Generation 2.5 chemistry," said Brett Monia, senior vice president of drug discovery at Ionis. "By combining Generation 2.5 and LICA, we have generated a drug that has the advantages of both higher affinity chemistry and efficient cell-specific targeting. This combination has allowed us to produce a drug that is substantially more potent than either Generation 2.5 or LICA alone, which should support both a very low dose and infrequent dosing."

As AZD8233 advances in development, Ionis will be eligible to receive up to $300 million in additional development and regulatory milestone payments as well as tiered, low double-digit royalties from sales of the drug. AstraZeneca will be responsible for further developing and commercializing AZD8233.

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