GlaxoSmithKline opts not to start Phase III outcome study for Ionis' experimental TTR amyloidosis therapy

Shares in Ionis Pharmaceuticals declined as much as 17 percent Thursday after the company said partner GlaxoSmithKline has decided against initiating the planned Phase III CARDIO-TTR outcome trial to evaluate the antisense drug IONIS-TTRRx, previously known as ISIS-TTRRx, in patients with transthyretin (TTR) amyloid cardiomyopathy. According to Ionis, the UK drugmaker "will consider options for TTR amyloid cardiomyopathy once additional clinical data are available from [other] ongoing studies."

In April, the FDA placed a clinical hold on the planned study "as a result of safety findings" in the ongoing late-stage NEURO-TTR trial, which Ionis is conducting in patients with TTR familial amyloid polyneuropathy. Meanwhile, an investigator-initiated Phase II study of IONIS-TTRRx is underway in the treatment of TTR-related amyloid cardiomyopathy, with Ionis noting that NEURO-TTR is also evaluating TTR-related cardiomyopathy via a cardiac substudy of patients who have cardiac involvement in addition to their polyneuropathy. The company indicated that about half the participants in NEURO-TTR also have TTR-related amyloid cardiomyopathy. 

According to Ionis, the NEURO-TTR trial and the ongoing Phase II study are proceeding as expected, with NEURO-TTR data anticipated in the first half of next year, while updated results from the investigator-initiated study will be presented at the International Symposium on Amyloidosis (ISA) in July. Ionis added that it is working with GlaxoSmithKline, which has an exclusive option to license IONIS-TTRRx, in "actively preparing" to submit a new drug application for the treatment. 

Helen Merianos, medicine development leader for GlaxoSmithKline's TTR amyloidosis programme, commented that the company 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." In 2010, GlaxoSmithKline entered into a partnership with Ionis, formerly known as Isis Pharmaceuticals, to use the latter's antisense drug discovery platform for the development of treatments for rare and serious diseases, including infectious diseases and some conditions causing blindness.



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