Ionis Pharmaceuticals and AstraZeneca announced further positive topline data from a Phase III NEURO-TTRansform trial investigating eplontersen in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN).
The 66-week analysis demonstrated that patients treated with eplontersen continued to show a statistically significant and clinically meaningful change from baseline, versus an external placebo group on the co-primary endpoints.
The co-primary endpoints measured modified Neuropathy Impairment Score +7 (mNIS+7) and Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN). The drug also met the trial’s third co-primary endpoint that measured the percentage change from baseline in serum transthyretin (TTR) concentration.
Eplontersen continued to show consistency in its safety and tolerability profile, as well as TTR reductions, as seen in the 35-week data analysis reported in June 2022. Data from both 35-week and 66-week analyses will be presented at the American Academy of Neurology (AAN) Annual Meeting in April.
The open-label trial (NCT04136184) enrolled 168 participants with ATTRv-PN. Patients received subcutaneous injections of either eplontersen once every four weeks or Tegsedi (inotersen) once a week. Participants in the control group received the active comparator for 34 weeks and then converted to the experimental arm until the end of the study.
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ATTRv-PN is a rare and fatal disease caused by gene coding mutations that lead to the dysfunction of different organs and tissues.
AstraZeneca BioPharmaceuticals R&D executive vice-president Mene Pangalos said: “With limited treatment options currently available, there is an urgent unmet medical need for new therapies and earlier, accurate diagnosis across the different types of this systemic, progressive and fatal condition.”
Indeed, the clinical trial activity is low in familial amyloid neuropathies (FAN). According to GlobalData’s Clinical Trial Database, only 67 clinical trials in FAN were initiated since 2006. While 53 of them were completed, there are only five ongoing and one planned Phase I-IV trials. GlobalData is the parent company of Clinical Trials Arena.
As part of their global commercialisation and development agreement, Ionis and AstraZeneca are seeking regulatory approval for the treatment of ATTRv-PN in the US.
In March 2023, the FDA accepted a New Drug Application (NDA) for eplontersen with a Prescription Drug User Fee Act (PDUFA) action date of 22 December 2023. The two pharma companies plan to receive regulatory approval in Europe and other global regions.
Eplontersen is currently being investigated in a Phase III trial (NCT04136171) for transthyretin amyloid cardiomyopathy (ATTR-CM), a systemic, progressive and fatal condition that typically leads to progressive heart failure and often death within three to five years from disease onset.