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Isis Pharmaceuticals Reports an Update on ISIS-TTR Rx, Including Positive Data from Multiple Clinical Studies, Presented Today at the EC-ATTR Meeting

CARLSBAD, Calif., Nov. 3, 2015 /PRNewswire/ -- Isis Pharmaceuticals, Inc. (ISIS) announced today positive preliminary results from an ongoing investigator sponsored, open-label Phase 2 study in patients with TTR amyloid cardiomyopathy, which included patients with familial amyloid cardiomyopathy (FAC) and patients with wild-type transthyretin amyloidosis (wt-TTR amyloidosis, previously referred to as senile systemic amyloidosis, or SSA). In this study, patients receive one injection of ISIS-TTRRx once a week. The study will continue for 24 months. Data from patients treated for 12 months were presented today (abstract # PM14) by the lead investigator, Merrill D. Benson, M.D., at the First European Congress on Hereditary ATTR amyloidosis in Paris, France. Isis will review these data and the overall development plan for ISIS-TTRRx in a webcast scheduled for Tuesday, Nov. 3 at 8:00 a.m. Eastern Time.

"Data from our study is encouraging. The study is designed to monitor progression of cardiomyopathy in patients treated with ISIS-TTRRx. The MRI data measured left ventricular mass for the first three patients to complete 12 months on therapy. These data show evidence of disease stabilization when compared to baseline. These observations compare favorably to those from our previously published natural history data. In the natural history study1, using the same measurements, we observed disease progression at 12 months," said Merrill D. Benson, M.D., professor of pathology and lab medicine, medical and molecular genetics at Indiana University School of Medicine. "Our previously reported echocardiographic measurements in these patients also showed disease stabilization after six months of ISIS-TTRRx treatment. The MRI data from longer term treatment I reported today support our earlier observations."

  • Patients in Dr. Benson's previously published natural history study with an interventricular septum thickness (IVS) >1.3cm (mean =1.9) at study entry had a mean increase of 14 percent in left ventricular mass as measured by MRI at 12 months.
  • Patients entering the Phase 2 ISIS-TTRRx study had baseline IVS > 1.3cm (mean =2.03). The first three patients treated with ISIS-TTRRx had a mean decrease of 1.9 percent in left ventricular mass from baseline as measured by MRI at 12 months.
  • No discontinuations and injection site reactions occurring in less than two percent of all injections, which were predominantly mild. The safety and tolerability profile of ISIS-TTRRx supports continued development.

Isis Pharmaceuticals also announced new data from the ongoing open-label extension (OLE) study of ISIS-TTRRx in patients with familial amyloid polyneuropathy (FAP) who have completed the Phase 3 study. An analysis conducted on the first 38 patients to reach at least three months of treatment in the OLE study showed a maximum reduction in TTR protein levels of up to 92 percent with a mean maximum (nadir) reduction of 76 percent as compared to patients' baseline TTR levels at entry into the Phase 3 study. Patients continue to be enrolled in the OLE as they complete the Phase 3 study. In addition, a blinded analysis of the Phase 3 study showed injection site reactions occurring in approximately one percent of all injections, which were predominantly mild. The Phase 3 study is expected to complete enrollment by the end of the year.

"We are encouraged by the substantial reductions in TTR protein we have observed in patients who have a variety of variants of the TTR gene. We believe that the observed tolerability profile and the convenience of dosing ISIS-TTRRx (one low volume, (1.5 mL) once weekly, subcutaneous injection that enables at home administration) contribute significantly to the high rate of patient...