Upcoming AWS Coverage on United Therapeutics Post-Earnings Results

LONDON, UK / ACCESSWIRE / May 16, 2017 / Active Wall St. blog coverage looks at the headline from Ionis Pharmaceutical, Inc. (NASDAQ: IONS) as the Company announced on May 16, 2017, that the Phase 3 NEURO-TTR study of Inotersen (IONIS-TTRRx) in patients with familial amyloid polyneuropathy (FAP) met both primary endpoints, however, there were some concerning side effects. Register with us now for your free membership and blog access at:

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One of Ionis Pharmaceutical's competitors within the Drug Manufacturers - Other space, United Therapeutics Corp. (NASDAQ: UTHR), reported on April 26, 2017, its financial results for Q1 ended March 31, 2017. AWS will be initiating a research report on United Therapeutics in the coming days.

Today, AWS is promoting its blog coverage on IONS; touching on UTHR. Get all of our free blog coverage and more by clicking on the link below:

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Inotersen is a generation 2.0+ antisense drug Ionis is developing for the treatment of TTR amyloidosis. The US Food and Drug Administration has granted Orphan Drug Designation and Fast Track Status to Inotersen for the treatment of patients with FAP.

The Phase III Study

Ionis Pharma's Inotersen was evaluated in a Phase 3 randomized (2:1), double-blind, placebo-controlled, international study in 172 patients with hATTR-PN, to support an application for marketing approval of Inotersen. The 15-month study measured the effects of Inotersen on neurological dysfunction and on quality-of-life by measuring the change from baseline in the modified Neuropathy Impairment Score +7 (mNIS+7) and in the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QOL-DN) total score.

Over the 15-month period of the study, Inotersen-treated patients demonstrated statistically significant benefit compared to placebo in mNIS+7 and the Norfolk QoL-DN (p<0.0001 and p=0.0006, respectively).

"We are grateful to the patients who participated in the NEURO-TTR study, along with their families, the investigators and the broader TTR amyloidosis community, for their dedication, commitment and support. We share the collective sense of urgency to bring a new treatment to patients and their families facing this devastating disease," said Brett P. Monia, Senior Vice President of drug discovery and franchise leader for oncology and rare diseases at Ionis Pharmaceuticals, "We are excited about the positive top-line results from the Phase 3 NEURO-TTR study. We observed a benefit in disease progression in patients treated with Inotersen, regardless of disease stage (Stage 1 and Stage 2) or TTR mutation (V30M and non-V30M). We believe these preliminary results suggest a favorable benefit-risk profile for Inotersen in patients with FAP."

Long-term safety and efficacy data with Inotersen are currently being collected in an open-label extension of the Phase 3 NEURO-TTR study. More than 80% of patients completed the NEURO-TTR study, and of these more than 95% participated in the open-label extension study.

Adverse Event Mar the Results

Ionis reported few adverse events considered related to treatment with Inotersen than placebo. The Company observed three serious adverse events of thrombocytopenia in Inotersen-treated patients, out of which two patients recovered, but one patient died due to intracranial haemorrhage. One additional Inotersen-treated patient discontinued treatment due to non-serious thrombocytopenia. Moreover, four Inotersen-treated patients discontinued treatment due to a renal observation; two patients met a predefined renal stopping rule and two experienced serious renal adverse events, one of whom experienced chronic renal insufficiency. Meanwhile, Ionis stated that since the Company discovered the problem and implemented enhanced monitoring procedures they have not observed any serious adverse events.

Review of the full data package from the NEURO-TTR study by Ionis and GSK is ongoing and detailed results from the study will be presented at an upcoming medical meeting and submitted for publication in a peer-reviewed medical journal.

What is TTR AMYLOIDOSIS ? FAP?

FAP, now referred to as hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN), is a progressive, debilitating, and fatal genetic disease in which patients experience TTR build up in major organs, including peripheral nerves, heart, intestinal tract, kidney, and bladder.

Therapeutic options for the treatment of hATTR-PN are very limited and there are currently no drugs approved for the treatment of hATTR-PN in the United States. There are an estimated 10,000 hATTR-PN patients worldwide.

Stock Performance

On Monday, May 15, 2017, following the results, the stock closed the trading session at $43.90, declining 6.75% from its previous closing price of $47.08. A total volume of 7.03 million shares have exchanged hands, which was higher than the 3-month average volume of 1.69 million shares. Ionis Pharma's stock price surged 3.86% in the last month, 7.20% in the past six months, and 39.72% in the previous twelve months. At Monday's closing price, the stock's net capitalization stands at $5.19 billion.

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