Data reported from NEURO-TTR study of Tegsedi in patients with hereditary ATTR amyloidosis

Akcea Therapeutics, Inc., an affiliate of Ionis Pharmaceuticals, Inc., and Ionis Pharmaceuticals, Inc. announced new long-term, open-label extension (OLE) data from the NEURO-TTR study of Tegsedi (inotersen) in patients with hereditary ATTR amyloidosis (hATTR) with polyneuropathy . The data was highlighted as an oral presentation at the 2019 American Academy of Neurology (AAN) Annual Meeting in Philadelphia.

Of the 139 patients who completed the NEURO-TTR phase III study, 97% of patients elected to continue in the OLE study. Initiation of Tegsedi, substantially reduced TTR protein levels 75% to 79% below baseline and sustained reductions throughout treatment. Patients who received Tegsedi through the NEURO-TTR and OLE studies (104 weeks) continued to show benefit throughout, and patients who switched from placebo to Tegsedi demonstrated an improvement in measures of neuropathy progression and QoL compared with a continued predicted worsening with placebo. Additionally, patients who initiated Tegsedi as part of the OLE demonstrated a stabilization of neurologic disease progression by mNIS+7 and Norfolk QoL-DN within six months, while greater benefit in mNIS+7 (17.1 points) and Norfolk QoL-DN (11.9 points) was observed in patients treated earlier with Tegsedi. No new safety signals were identified in the open-label extension study.

"In the OLE study, over 40% of patients initiating treatment with Tegsedi for the first-time experienced improvement in their quality of life and nearly 50% saw improvement in a measure of neurological disease progression," said Brett P. Monia, Ph.D., chief operating officer of Ionis Pharmaceuticals. .