- Ionis Pharmaceuticals (NASDAQ:IONS) announces positive results from a Phase 3 clinical trial, NEURO-TTR, assessing inotersen (IONIS-TTRrx) for the treatment of the inherited disorder familial amyloid polyneuropathy (FAP). The study met both primary efficacy endpoints demonstrating a statistically valid treatment benefit compared to placebo.
- Shares are down 10% premarket on increased volume, however, as investors hit the sell buttons in response to the side effects of treatment. Three participants developed thrombocytopenia (low blood platelets), two recovered but one died from intracranial hemorrhage. Another patient discontinued treatment due to non-serious thrombocytopenia.
- Four patients receiving inotersen stopped treatment due to kidney events; one from a renal observation, two from meeting a predefined renal stopping rule and one from chronic renal insufficiency (one patient in the control group also met the renal stopping rule).
- The company says all five serious adverse events occurred before enhanced monitoring was fully implemented. A review of the safety data is ongoing.
- Inotersen is an antisense drug that targets a specific sequence of RNA. It is designed to reduce the production of a protein called transthyretin. It is administered once per week via injection.
- Now read: Akcea Therapeutics Provides Update To IPO Plans
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