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Ionis Earns $10 Million Milestone Payment from Biogen for Advancing a New Program in its Neurology Collaboration

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Monday, July 17, 2017

Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced that it has earned a $10 million milestone payment from Biogen associated with the validation of an undisclosed neurological disease target. Ionis and Biogen have a broad strategic collaboration to develop drugs to treat patients with neurological disorders. Ionis will continue to evaluate this newest target with the goal of advancing this program into development.
Ionis Pharmaceuticals

"We continue to make substantial progress in validating new targets and developing new drugs for neurological diseases using our antisense technology. SPINRAZA, the first drug to come out of our neurology collaboration with Biogen, exemplifies the potential of our antisense technology in the neurological disease space. To date, our collaboration with Biogen has generated a combined total of nearly $650 million," said B. Lynne Parshall, chief operating officer at Ionis Pharmaceuticals. "As we continue to advance our neurological disease pipeline, we look forward to bringing additional important medicines to patients suffering from neurodegenerative diseases."

About IONIS' STRATEGIC COLLABORATION WITH BIOGEN

Ionis and Biogen have a broad strategic collaboration combining Biogen's expertise in neurology with Ionis' leadership in antisense technology to develop novel therapies to treat neurological disorders. Ionis is primarily responsible for drug discovery and early development of antisense therapies. Biogen has the option to license each antisense program at a particular stage in development. Under this collaboration, Ionis developed and licensed SPINRAZA to Biogen. SPINRAZA is approved in the U.S., EU, Japan and Canada for the treatment of patients with spinal muscular atrophy (SMA). Also under this collaboration, Ionis and Biogen are developing IONIS-SOD1Rx to treat amyotrophic lateral sclerosis, IONIS-MAPTRx to treat Alzheimer's Disease, and IONIS-BIIB5Rx, IONIS-BIIB6Rx and IONIS-BIIB7Rx to treat undisclosed neurodegenerative diseases. In addition to these six drugs, Ionis and Biogen have numerous opportunities to evaluate additional targets for the development of drugs to treat neurological disorders.

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) is a drug that has been approved in the U.S., Europe, Japan and Canada for the treatment of spinal muscular atrophy (SMA). Biogen is responsible for commercializing SPINRAZA. Drugs that have successfully completed Phase 3 studies include volanesorsen, a drug Ionis is developing and plans to commercialize through its subsidiary, Akcea Therapeutics, to treat patients with either familial chylomicronemia syndrome or familial partial lipodystrophy; and inotersen (IONIS-TTRRx), a drug Ionis is developing with GSK to treat patients with TTR amyloidosis. Both drugs are progressing toward regulatory filings in the second half of 2017. 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, Inc.
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