– IONIS-JBI2-2.5 Rx designed to treat GI autoimmune disease

– Ionis Earns $5 Million License Fee from Janssen

CARLSBAD, CA, USA I November 16, 2017 I Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced today that it has licensed a second orally delivered Generation 2.5 antisense drug to Janssen Biotech, Inc. (Janssen) for which Ionis earned $5 million. IONIS-JBI2-2.5Rx is designed to locally reduce the production of an undisclosed target in the gastrointestinal (GI) tract for the treatment of a GI autoimmune disease. Jansen will now assume all global development, regulatory, and commercialization responsibilities for IONIS-JBI2-2.5Rx for GI diseases.

“We are pleased that IONIS-JBI2-2.5Rx is advancing into development because we believe this drug has the potential to treat autoimmune disorders in the GI tract that are underserved with current therapies,” said Frank Bennett, Ph.D., senior vice president of research at Ionis Pharmaceuticals. “Our collaboration with Janssen has been very productive. This is the second drug Janssen has licensed in little over a year, which is the direct result of the speed and efficiency of our antisense technology.”

Under its global collaboration agreement with Janssen, Ionis is eligible to receive nearly $800 million in development, regulatory and sales milestone payments and license fees. In addition, Ionis will receive tiered royalties that on average are double-digits on sales from any product that is successfully commercialized. IONIS-JBI1-2.5Rx, the first collaboration target, was licensed to Janssen last year and Janssen has assumed all global development, regulatory and commercialization responsibilities related to this drug.

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