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Ionis lab gets FDA warning

May 12, 2017

Shares of Ionis Pharmaceuticals (IONS) plunged Wednesday after the U.S. Food and Drug Administration issued a warning letter to a researcher conducting clinical trials for the company that raised concerns over “objectionable conditions” found during an inspection.

The letter was sent to Dr. Merrill Benson of Indiana University who is conducting a TTR investigator-initiated cardiomyopathy study. In its letter, the FDA said the researchers failed to ensure that the investigation was conducted according to the investigational plan.

Benson’s lab has been conducting a Phase II study in patients with TTR amyloid cardiomyopathy, which includes patients with familial amyloid cardiomyopathy (FAC) and patients with wild-type transthyretin amyloidosis (wt-TTR amyloidosis).

The FDA said the trial protocol requires the researchers to perform certain study procedures at specific times, such as platelet count measurements, clinical chemistry panel tests, and urinalysis laboratory tests. Additionally, the FDA said the protocol requires the researchers to not enroll subjects who meet exclusion criteria.

In particular, the FDA said researchers had inadequate platelet monitoring and drug dosing protocols in the trial. Over a two-week period from May to June 2016, the researchers did not conduct platelet tests. The FDA criticized the researchers, saying that in their own reports the researchers said it was important to monitor platelets “in light of the reports concerning life-threatening thrombocytopenia in subjects treated.”

“You failed to adhere to these requirements,” the FDA said in its letter. “…frequent monitoring of platelets and strict adherence to protocol-specific stopping rules are essential to protect the rights, safety, and welfare of subjects participating in the study. Failure to adhere to protocol-required safety monitoring and stopping rules, particularly those related to platelet count results, jeopardizes subject safety and welfare.”

Joshua Schimmer, an analyst with Piper Jaffray, said the FDA’s warning letter “exposes a number of relevant concerns regarding the thrombocytopenia risk that will likely impact commercial outlook for a number of earlier generation antisense products in the portfolio, while also exposing some of the strategic fallacies which we see at the company,” according to a blog post on Barron’s.

One year ago, Ionis’ cardiomyopathy research hit a snag when GlaxoSmithKline (GSK) 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.

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.

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