Ionis Licences New Antisense Drug for Kidney Disease to AstraZeneca

Ionis earns $30 million license fee

CARLSBAD, CA, USA I February 20, 2018 I Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced today that it has licensed IONIS-AZ5-2.5Rx (AZD2373) to the global, science-led biopharmaceutical company AstraZeneca (NYSE: AZN). IONIS-AZ5-2.5Rx is a Generation 2.5 antisense drug, discovered by Ionis and designed to inhibit an undisclosed target to treat a genetically associated form of kidney disease. AstraZeneca will be responsible for developing and commercializing IONIS-AZ5-2.5Rx.

"This is the second drug to enter development under our strategic collaboration with AstraZeneca in cardiovascular, metabolic and renal diseases. IONIS-AZ5-2.5Rx is being developed to treat a genetically associated form of kidney disease with a high unmet medical need. We were able to move this program from target validation to a clinical candidate quickly, exemplifying the efficiency of our antisense platform," said Brett P. Monia, Ph.D., chief operating officer and senior vice president of antisense drug discovery and translational medicine at Ionis. "AstraZeneca is a great partner to work with in the cardiometabolic and renal therapeutic area, and we look forward to them moving this program through development."

Ionis will receive a $30 million upfront payment from AstraZeneca for IONIS-AZ5-2.5Rx and may also receive up to $300 million in additional development and regulatory milestone payments and tiered, low double-digit royalties from sales of this potential new medicine.

ABOUT IONIS AND ASTRAZENECA
AstraZeneca and Ionis have a strategic alliance focused on leveraging antisense technology to discover and develop antisense therapies for cardiovascular, metabolic and renal diseases. The collaboration builds on a broad existing relationship between the two companies and is supported by Ionis' expertise in cardiovascular disease and antisense technology and AstraZeneca's disease area knowledge as well as drug development expertise and infrastructure. Ionis has generated more than $220 million to date from the broad strategic collaboration with AstraZeneca in oncology and cardiometabolic and kidney diseases. Beyond AZD8233, and AZD2373, AstraZeneca has the option to license additional antisense drugs upon development candidate nomination and will pay a license fee for each drug licensed plus development and regulatory milestones and sales royalties for each program that AstraZeneca advances. Ionis and AstraZeneca are also collaborating to discover and develop antisense drugs to treat cancer.

ABOUT IONIS PHARMACEUTICALS, INC.
Ionis is the leading company in RNA-targeted drug discovery and development focused on developing drugs for patients who have the highest unmet medical needs, such as those patients with severe and rare diseases. Using its proprietary antisense technology, Ionis has created a large pipeline of first-in-class or best-in-class drugs, with over three dozen drugs in development. SPINRAZA® (nusinersen) has been approved in global markets for the treatment of spinal muscular atrophy (SMA). Biogen is responsible for commercializing SPINRAZA. Drugs that have successfully completed Phase 3 studies include inotersen, an antisense drug Ionis is developing to treat patients with hereditary TTR amyloidosis (hATTR), and volanesorsen, an antisense drug discovered by Ionis and co-developed by Ionis and Akcea Therapeutics to treat patients with either familial chylomicronemia syndrome or familial partial lipodystrophy. Akcea, an affiliate of Ionis, is a biopharmaceutical company focused on developing and commercializing drugs to treat patients with serious cardiometabolic diseases caused by lipid disorders. If approved, volanesorsen will be commercialized through Ionis' affiliate, Akcea. Inotersen filings for marketing approval have been submitted in the U.S. and EU. Volanesorsen filings for marketing approval have been submitted in the U.S., EU, and Canada. Ionis' patents provide strong and extensive protection for its drugs and technology. Additional information about Ionis is available at www.ionispharma.com.

SOURCE: Ionis Pharmaceuticals

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