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Ionis Announces Submission of Marketing Authorization Application for Inotersen to the European Medicines Agency

Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced today that the company submitted a marketing authorization application (MAA) to the European Medicines Agency (EMA) for inotersen, based on the phase 3 NEURO-TTR study in patients with hereditary TTR amyloidosis (hATTR). The MAA for inotersen will be reviewed under the EMA's Accelerated Assessment program, which is intended to expedite access to drugs that the EMA considers to be of major therapeutic interest.

"The submission of the inotersen MAA to the EMA represents a critical milestone for Ionis and for European patients suffering from ATTR. We are pleased that we met the last deadline for Accelerated Assessment in the EU for 2017. Next week, we also plan to submit the NDA to the FDA. Today's submission to the EMA puts us one step closer to our goal of fundamentally changing the treatment landscape for patients and families globally who are facing this devastating, progressive, fatal disease, and for the healthcare professionals who treat them," said Sarah Boyce, chief business officer of Ionis Pharmaceuticals. "In the Phase 3 NEURO-TTR study, half of inotersen-treated patients saw improvement in their quality of life. Combined with significant efficacy and superior convenience offered with inotersen, we believe inotersen will be the treatment of choice for this patient population. We are making substantial progress in advancing inotersen to market, and we are in advanced discussions with potential co-commercialization partners. We believe the right partner can maximize the commercial success of inotersen."

"Patients with ATTR experience TTR build up in various tissues and major organs, including peripheral nerves, heart, intestinal tract, kidney and bladder, which causes debilitating symptoms, such as severe extremity pain and gastrointestinal manifestations, and eventually results in death. As their disease progresses, these patients lose the ability to perform even simple activities, such as feeding and dressing themselves, significantly limiting their independence," said Teresa Coelho, MD, neurologist and neurophysiologist at Santo António Hospital, Porto, Portugal. "Therapeutic options for the treatment of hATTR are very limited and include liver transplant for early-stage patients. I am encouraged by the results from the Phase 3 NEURO-TTR study with inotersen, which demonstrated substantial benefit in both quality of life and neurological function for inotersen-treated patients compared to placebo-treated patients."

"TTR amyloidosis is devastating, not only for patients but also for caregivers and family members. We are grateful that today we are closer than ever to having an effective treatment to fight this disease. Ionis has demonstrated great commitment to hATTR and these patients, and we look forward to collaborating further with the Ionis team as we work towards potential regulatory and subsequ