Phase 3 Data Presented at International Symposium on Amyloidosis

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Data presented at the 16th International Symposium on Amyloidosis in Japan were presented this morning, showing that inotersen-treated patients with hATTR amyloidosis continued to exhibit sustained benefit.

Data presented at the 16th International Symposium on Amyloidosis (ISA) in Kumamoto, Japan were presented this morning, showing that inotersen (IONIS-TTRx)-treated patients with hereditary ATTR (hATTR) amyloidosis continued to exhibit sustained benefit in quality of life (QOL) and neuropathy measures.

The Phase 3 data was presented by Ionis Pharmaceuticals, Inc. and Akcea Therapeutics, Inc., and was from the NEURO-TTR and open-label extension (OLE) studies.

The NEURO-TTR study was a randomized (2:1), double-blind, placebo-controlled, international study in 172 patients with polyneuropathy due to hATTR. Data from the study highlighted the improvement in QOL and functional capacity experienced by 50% of patients treated with the investigational antisense drug. Throughout the study, significant benefit was demonstrated in comparison to placebo in patients with cardiac disease at baseline in both primary endpoints (Norfolk QOL-DN, p=0.036 and mNIS+7, p<0.001) and in the SF-36 Health Survey endpoint (p=0.025) at 15 months.

It was shown that half of patients receiving IONIS-TTRx were more capable of completing daily activities than they were at the start of the study, and these benefits were significant, early and sustained across patient types.

The NEURO-TTR OLE is an ongoing study for patients who completed the NEURO-TTR study and is designed to assess the long-term efficacy and safety profile of IONIS-TTRx.

In the OLE, the benefit observed with treatment continued with up to 27 months of total treatment (15 months in NEURO-TTR and up to 12 months in OLE). Additionally, the OLE results demonstrate that patients who initiated IONIS-TTRx treatment 15 months earlier experienced greater benefit than those who received placebo treatment in the NEURO-TTR study and progressed in their disease, and then initiated treatment with the drug in the OLE.

Patients receiving placebo in the NEURO-TTR study experienced a rapid onset of effect following IONIS-TTRx treatment that was sustained for up to 12 months in the OLE, including: improvements in QOL and activities of daily living, and decreased rate of disease progression.

During the NEURO-TTR study, 2 safety issues arose: thrombocytopenia and serious renal adverse events (AEs). These complications aside, the most common AEs included nausea, chills, vomiting, and anemia. No new safety concerns were identified in the OLE.

"I'm encouraged that inotersen-treated patients in the OLE continue to experience robust and sustained benefits in quality of life and measures of neuropathy impairment," said John L. Berk, M.D., associate professor of medicine at Boston University School of Medicine and a principal investigator in the NEURO-TTR study in a press release. "The rapid and sustained benefits observed with inotersen in the NEURO-TTR and OLE studies illustrate the substantial potential of inotersen to change the course of this devastating disease."

IONIS-TTRx has been developed to reduce the production of transthyretin (TTR). In January, it was announced that Priority Review had been granted to Ionis from the U.S. Food and Drug Administration (FDA).

Inotersen was also previously granted Orphan Drug Designation and Fast Track Status by the FDA.

Additionally, results from an ongoing investigator-sponsored Phase 2 study in cardiomyopathy patients with hATTR amyloidosis and wild-type ATTR (wtATTR) amyloidosis treated with inotersen further support the cardiac benefit observed in NEURO-TTR.

"The clinical benefits observed with inotersen treatment, coupled with the flexibility offered by a once-weekly, self-administered subcutaneous injection, can restore greater independence that many hATTR amyloidosis patients have lost to their disease," said Sarah Boyce, chief business officer at Ionis. "We believe that inotersen has the potential to transform the lives of patients with hATTR amyloidosis, and we are committed to rapidly bringing this important new treatment to patients following marketing approval."

For more from the ISA and other meetings pertaining to the rare disease community, follow Rare Disease Report on Facebook and Twitter.

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