On Monday, shares of Ionis Pharmaceuticals Inc. (NASDAQ:IONS) are falling, down about 10% in morning trading after the company announced results from its 15-month long Phase 3 NEURO-TTR Study of its drug Inotersen (IONIS-TTRrx).
Inotersen is being tested for the treatment of the inherited disorder familial amyloid polyneuropathy (FAP). While the study met both primary efficacy endpoints that demonstrated statistically valid treatment benefits in comparison to the placebo, there were some concerning side effects.
Three study participants developed thrombocytopenia, or low blood platelets; two recovered, but one died from intracranial hemorrhage, and another patient stopped taking Inotersen due to non-serious thrombocytopenia. There were also four patients that stopped the treatment due to kidney problems, one from a renal observation, two from meeting a predefined renal stopping rule, and one from chronic renal insufficiency.
Ionis said that all five of these instances happened before enhanced monitoring was completely implemented, and that a review of the safety data is ongoing.
“We are grateful to the patients who participated in the NEURO-TTR study, along with their families, the investigators and the broader TTR amyloidosis community, for their dedication, commitment and support. We share the collective sense of urgency to bring a new treatment to patients and their families facing this devastating disease," said Brett P. Monia, senior vice president of drug discovery and franchise leader for oncology and rare diseases at Ionis Pharmaceuticals.
Currently, IONS is a #3 (Hold) on the Zacks Rank, with a VGM score of ‘B.’
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Ionis Pharmaceuticals, Inc. (IONS): Free Stock Analysis Report
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