Ionis Touts Phase 3 Win, But Safety Worries Cloud Nerve Drug Study

▤ Summary

Patients on the drug for 15 months had a statistically significant improvement, compared to placebo, on two tests that measure their symptoms: the “modified neuropathy impairment score,” or mNIS+7, a measure of symptoms like muscle weakness and reflexes; and the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy, which evaluates how the disease is impacting patients’ lives.Ionis, however, didn’t disclose the magnitude of the benefit on either test, or mention how the drug fared on secondary measures, like a change in body mass index—full details from the study will be presented at a future medical meeting.In a research note, Leerink Partners analyst Paul Matteis wrote the drug’s safety “remains a major question,” along with the possible “frequency/necessity of dose reductions.” For its part, Ionis said it has since changed how it tests for low platelet counts and kidney problems and that all of the serious side effects occurred before these measures were implemented.FAP is a form of transthyretin amyloidosis, an inherited condition in which amyloid proteins build up in tissues of the body and cause damage to the nerves, heart, and other organs.Matteis wrote that given the similarities between the two drugs, Ionis’s data “virtually de-risks patisiran, barring an unexpected safety concern.” On the call, Monia made a case for inotersen’s superiority.

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