Ionis Pharmaceuticals Shares Drop After GASK Decided Not To Initiate Its Phase Trial Outcome On TTRRx

Ionis Pharmaceuticals, Inc.

provided an update on its IONIS-TTRRx program. The company is evaluating IONIS-TTRRx in an ongoing Phase 3 study currently, NEURO-TTR, in patients with transthyretin (TTR) familial amyloid polyneuropathy. According to Ionis, GSK, which has an option to license IONIS-TTRx, has decided not to initiate the stage three outcome study, CARDIO-TTR that was planned to assess IONIS-TTRx in patients with TTR armyloid cardiomyopthy. The company said that Merrill Benson is also evaluating IONIS-TTRRx in an investigator-initiated stage two open-label study in patients with TTR-related amyloid cardiomyopathy. The company pointed out that the FDA had placed this trial on clinical hold as a result of safety findings in the ongoing NEURO-TTR study as announced in April 2016. The company added that GSK would consider options for TTR amyloid cardiomyopathy once more clinical data are available from the ongoing studies. Its SVP, Brett Monia, commented on the developments, saying, "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." The SVP added, "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." GSK's TTR amyloidosis program's Medicine Development leader, Helen Merianos, commented that "Medicine Development Leader for GSK's TTR amyloidosis program, said: "GSK remains committed to developing innovative medicines for the treatment of amyloidosis and to our ongoing collaboration with Ionis to develop IONIS-TTRRx for TTR amyloidosis. We will assess the results from the NEURO-TTR study to inform our next steps for TTR amyloid cardiomyopathy." The stock plunged 34.86 percent to trade at $22.88 on Thursday.