Shares of Ionis Pharmaceuticals Inc (NASDAQ:IONS) are trading in red after the company announced that GlaxoSmithKline plc (ADR) (NYSE:GSK), which has an option to exclusively license IONIS-TTR Rx, will not initiate the phase 3 study, CARDIO-TTR. The study, designed to evaluate IONIS-TTR Rx in patients with transthyretin (TTR) amyloid cardiomyopathy, was put on a clinical hold by the U.S. Food and Drug Administration (FDA) in April due to safety findings in the ongoing NEURO-TTR study.
California-based Ionis Pharmaceuticals’ stock dropped by 36.88% in the today’s trading session. The company on Wednesday announced that it is evaluating the IONIS-TTR Rx program in the ongoing phase 3 study, NEURO-TTR, in patients with transthyretin (TTR) familial amyloid polyneuropathy. The IONIS-TTR Rx program is also being evaluated by Dr. Merrill Benson in an investigator-initiated phase 2 open-label study in patients with TTR-related amyloid cardiomyopathy.
GlaxoSmithKline plc (ADR) (NYSE:GSK) will consider options for TTR amyloid cardiomyopathy once additional clinical data are available from the ongoing studies.
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“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,” said Dr. Helen Merianos, medicine development leader for GSK’s TTR amyloidosis program.
What Is TTR Amyloidosis?
TTR amyloidosis is a severe, progressive and fatal disease with multiple overlapping clinical manifestations. There are three forms of TTR amyloidosis: familial amyloid polyneuropathy, FAP, familial amyloid cardiomyopathy, FAC, and wild type (wt)-TTR amyloidosis.
The disease is caused by the accumulation of misfolded TTR protein in tissues and organs, including peripheral nerves, heart, intestinal tract, eyes, kidneys, central nervous system, thyroid and bone. The disease can cause organ failure and eventually death.
Therapeutic options for the treatment of patients with TTR amyloidosis are very limited, according to Ionis Pharmaceuticals Inc (NASDAQ:IONS).
Ionis Pharmaceuticals: Study Results Are Encouraging
The company also said that the NEURO-TTR and Dr. Benson’s study will provide important data on patients with TTR amyloid cardiomyopathy, contributing to the design of an optimal phase 3 study.
In addition to Dr. 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 sub study to evaluate patients with cardiac involvement in addition to their polyneuropathy. Approximately half of the patients in the NEURO-TTR study also have TTR-related amyloid cardiomyopathy, according to the company.
Both NEURO-TTR study and Dr. Benson’s study are moving on the track, while the data from the NEURO-TTR study is expected in the first half of 2017. Updated data from Dr. Benson’s study will be presented at the International Symposium on Amyloidosis in July 2016, Ionis Pharmaceuticals said.
“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,” said Dr. Brett Monia, senior vice president of Ionis Pharmaceuticals antisense drug discovery.