Ionis Down, Glaxo Dumps IONIS-TTRRx Phase III Study Plans

Ionis Pharmaceuticals, Inc.'s (NASDAQ:IONS) shares touched a 52-week low of $21.09 on May 26 after the company announced that its partner GlaxoSmithKline plc (NYSE:GSK) has decided against starting a phase III outcome study – CARDIO-TTR – that was planned to evaluate IONIS-TTRRx in patients with transthyretin (TTR) amyloid cardiomyopathy. Ionis' shares plunged 39.4% on the news.

We note that IONIS-TTRRx is being developed by Ionis in collaboration with Glaxo for all forms of TTR amyloidosis. Per the terms of the agreement, Glaxo has an option to exclusively license the IONIS-TTRRx program.

The recent development came in the wake of the FDA placing a clinical hold on CARDIO-TTR due to safety concerns that were raised in the ongoing phase III study, NEURO-TTR, early last month. This study is evaluating IONIS-TTRRx for the treatment of patients with TTR familial amyloid polyneuropathy as well as TTR-related cardiomyopathy using a cardiac sub-study to evaluate patients who have cardiac involvement in addition to their polyneuropathy. Data from the NEURO-TTR study is anticipated in the first half of 2017.

Also, Dr. Merrill Benson is assessing IONIS-TTRRx in an investigator-initiated open-label phase II study in patients with TTR-related amyloid cardiomyopathy. Ionis intends to present updated data from Dr. Benson's study at the International Symposium on Amyloidosis this July.

Data from both these studies are expected to offer essential information on patients with TTR amyloid cardiomyopathy, which will help design a phase III study.

Per Ionis’ press release, the company is working with Glaxo on filing a new drug application for IONIS-TTRRx. The press release also noted that Glaxo remains dedicated toward the development of drugs for the treatment of amyloidosis as well as to its collaboration with Ionis for the development of IONIS-TTRRx for the treatment of TTR amyloidosis.

Glaxo intends to wait for data from the NEURO-TTR study before moving ahead with its plans for TTR amyloid cardiomyopathy.

Meanwhile, Alnylam Pharmaceuticals, Inc.’s (NASDAQ:ALNY) shares jumped 11.1% on the news. Alnylam also has a phase III candidate, revusiran, for familial amyloidosis cardiomyopathy. The company plans to report data in mid-2018.

Ionis is a Zacks Rank #3 (Hold) stock. Bristol-Myers Squibb Company (NYSE:BMY) is a better-ranked stock in the health care sector carrying a Zacks Rank #1 (Strong Buy).