Ionis Pharmaceuticals Inc. (NASDAQ: IONS) shares dropped severely after a less-than positive update on its IONIS-TTR Rx program. The company is currently evaluating IONIS-TTRRx in an ongoing Phase 3 study, NEURO-TTR, in patients with transthyretin (TTR) familial amyloid polyneuropathy. Dr. Merrill Benson is also evaluating IONIS-TTRRx in an investigator-initiated Phase 2 open-label study in patients with TTR-related amyloid cardiomyopathy.

Unfortunately the company decided not to initiate a Phase 3 outcome study, which was planned to evaluate IONIS-TTRRx in patients with TTR amyloid cardiomyopathy. As previously announced in April, the U.S. Food and Drug Administration (FDA) had placed this study on clinical hold as a result of safety findings in the ongoing NEURO-TTR study.

According to Ionis, both the NEURO-TTR and Dr. Benson’s study should provide important data on patients with TTR amyloid cardiomyopathy, which could contribute to the design of an optimal Phase 3 study. Additionally, Benson’s investigator-initiated study in patients with TTR-related amyloid cardiomyopathy, the NEURO-TTR study is also evaluating patients with TTR-related cardiomyopathy using a cardiac substudy to evaluate patients who have cardiac involvement in addition to their polyneuropathy.

About half of the patients in the NEURO-TTR study also have TTR-related amyloid cardiomyopathy. The NEURO-TTR study and Dr. Benson’s study are both proceeding on track, with the data from the NEURO-TTR study planned in the first half of 2017. In July updated data from Benson’s study will be presented at the International Symposium on Amyloidosis.

Dr. Brett Monia, senior vice president of Ionis antisense drug discovery, commented:

We are committed to advancing IONIS-TTRRx as a potentially first-in-class and best-in-class therapy for all patients suffering from TTR amyloidosis, a severe, progressive and fatal disease. We are encouraged by the strong retention we have observed in the NEURO-TTR study and the robust participation and the substantial reductions in TTR protein we are observing in the open-label extension study.  We are also encouraged by the results to date from Dr. Benson’s study in a small number of patients with the cardiac form of the disease as presented at the European Congress on Hereditary ATTR Amyloidosis meeting in Paris last year.

Shares of Ionis were last trading seen down over 30% at $23.80, with a consensus analyst price target of $52.56 and a 52-week trading range of $22.49 to $71.50.

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