May 26, 2016
By Alex Keown, BioSpace.com Breaking News Staff
CARLSBAD, Calif. – Shares of Ionis Pharmaceuticals are down more than 35 percent this morning after its partner GlaxoSmithKline opted to not initiate a Phase III study of its IONIS-TTR drug program for the treatment of patients with TTR amyloidosis.
GSK’s move follows an April decision by the U.S. Food and Drug Administration to place an Ionis study of IONIS-TTR in patients with transthyretin (TTR) familial amyloid polyneuropathy (FAP) on clinical hold as a result of safety findings. Although the initial Phase III trial is not going to be performed soon, GSK said it will consider options for TTR amyloid cardiomyopathy once additional clinical data are available from the ongoing studies. There are an estimated 10,000 FAP patients worldwide.
“GSK remains committed to developing innovative medicines for the treatment of amyloidosis and to our ongoing collaboration with Ionis to develop IONIS-TTR for TTR amyloidosis. We will assess the results from the NEURO-TTR study to inform our next steps for TTR amyloid cardiomyopathy,” Helen Merianos, Medicine Development Leader for GSK’s TTR amyloidosis program, said in a statement.
In addition to the trial for patients with TTR amyloidosis, Ionis is also conducting a small Phase II trial of IONIS-TTR in patients with TTR-related amyloid cardiomyopathy.
Brett Monia, senior vice president of Ionis antisense drug discovery, said Ionis is committed to advancing IONIS-TTR for the treatment of 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 a broad range of tissues and organs, including peripheral nerves, heart, intestinal tract, eyes, kidneys, central nervous system, thyroid and bone. The progressive accumulation of TTR amyloid deposits in these tissues and organs leads to organ failure and eventually death. Therapeutic options for the treatment of patients with TTR amyloidosis are very limited.
Ionis is trading at $23.95 this morning, down from its Wednesday closing price of $35.26 per share. Ionis Pharmaceuticals is an RNA-targeted drug discovery and development company focused on developing drugs for patients who have the highest unmet medical needs. In addition to its work with GSK, Ionis, formerly known as Isis, has a partnership with Merck in the development of therapies for hepatitis C treatments. Ionis is set to share in a percentage of a jury award to Merck over a patent violation dispute with California-based Gilead Sciences .
