– Ionis Earns $5 Million Milestone Payment from Janssen

CARLSBAD, CA, USA I December 21, 2017 I Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) today announced that it has earned a $5 million milestone payment associated with initiation and enrollment in a newly initiated Phase 1 clinical study of IONIS-JBI1-2.5Rx, an orally delivered antisense drug designed to treat patients with a gastrointestinal (GI) immune disorder by reducing the production of an undisclosed target locally in the GI tract. Janssen Biotech, Inc. (Janssen), which is responsible for global development, regulatory and commercialization activities, is conducting the Phase 1 study.

“This study represents an important milestone for us and our antisense technology because IONIS-JBI1-2.5Rx is our first orally delivered antisense drug for the treatment of GI disorders to advance into the clinic. We are pleased with the success of our collaboration with Janssen. In under two years, we have advanced a drug into clinical trials and identified and initiated IND-enabling studies for a second drug development candidate, IONIS-JBI2-2.5Rx. Our collaboration with Janssen combines Ionis’ broad applicability of our antisense technology with their expertise in GI autoimmune disorders and therapeutic formulation,” said C. Frank Bennett, senior vice president of research at Ionis Pharmaceuticals.

Under its global collaboration agreement with Janssen, Ionis is eligible to receive nearly $585 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 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