Akcea Therapeutics Inc. (NASDAQ:AKCA), the majority-owned Ionis Pharmaceuticals Inc. (NASDAQ:IONS) affiliate, and Ionis Pharmaceuticals have announced the publications of its long-term data from the open-label extension NEURO-TTR TEGSEDI (inotersen) study.
The study evaluated the efficacy of TEGSEDI in treatment of polyneuropathy in hATTR
The company is conducting pivotal study of TEGSEDI in hereditary transthyretin (hATTR) amyloidosis patients, with polyneuropathy. The objective of the open-label study was to evaluate the tolerability and safety of long-term TEGSEDI dosing. The study’s secondary objective included discerning disease progression as per measures like modified Neuropathy Impairment Score +7. This also included the Norfolk Quality of Life Questionnaire-diabetic Neuropathy. Also, there was an exploratory objective of understanding the changes that happen with time in general health-related quality of life depending on the Short For 36 Health Survey.
The interim data was published in the European Journal of Neurology. The results show that TEGSEDI treatment did not demonstrate any more safety concerns or indications of increased toxicity in study participants that received treatment for up to five years. Equally, the results indicated that the treatment of participants with TEGSEDI leads to continued efficacy for two years in patients.
Interestingly the participants that began early treatment with TEGSEDI showed significant long-term stabilization of the disease. This is in comparison to those that switched to TEGSEDI from Placebo in the open-label study.
Results from the OLE study show positive benefit of TEGSEDI in patients
The company’s Chief Medical Officer, Louis O’Dea, indicated that the data validates the favorable risk-benefit profile witnessed in patients with hATTR that received TEGSEDI for polyneuropathy. O’Dea added that usually, hATTR amyloidosis patients face debilitating and progressive symptoms. Therefore the results of the OLE study show the benefits that the company observed in the initial 15-month period for the third phase study.
They observed improved outcomes in patients that were treated with TEGSEDI compared to placebo regarding the progression of neuropathy and as well as enhanced quality of life. The results demonstrated in the OLE study are important in having therapy for treating neuropathy in hATTR.