Ionis to file rare disease drug after GSK declines option

Antisense drug specialist Ionis Pharmaceuticals has said it plans to market rare disease drug inotersen on its own after GlaxoSmithKline declined options on two rare disease drugs.

GSK had options on inotersen for TTR amyloidosis and IONIS-FB-LRX for the treatment of complement-mediated diseases through an R&D partnership – but decided against taking either of them as part of a pipeline rethink under new CEO Emma Walmsley.

Inotersen, which has produced strong late-stage results in TTR amyloidosis, is closest to market and Ionis said it plans to file the drug with regulators in the US and Europe this year ahead of a potential launch next year.

The first indication Ionis is pursuing for inotersen is for polyneuropathy due to hereditary TTR amyloidosis.

In May, Ionis completed the phase 3 NEURO-TTR study of inotersen in which the drug demonstrated a significant benefit on both primary clinical endpoints of neurological disease progression and quality of life in patients with family amyloid polyneuropathy.

The trial met its two main goals of statistically significant benefit compared with placebo based on a neuropathy impairment score and a diabetic neuropathy score.

IONIS-FB-LRx is a ligand conjugated antisense (LICA) drug in development for the treatment of complement-mediated diseases.

In a phase 1 study completed earlier this year, IONIS-FB-LRX achieved dose-dependent reductions in plasma factor B (FB) and demonstrated a safety and tolerability profile that supports further clinical development.

Ionis plans to begin the first phase 2 study with IONIS-FB-LRX in patients with dry age-related macular degeneration (AMD) later this year, and studies in other indications in 2018.

GSK continues to focus on treatments for infectious diseases and continues to advance two hepatitis B virus drugs under its collaboration with Ionis.

The UK pharma is conducting phase 2 studies for IONIS-HBVRX and IONIS-HBV-LRX.

Ionis specialises in antisense drugs that selectively block RNA caused by rogue genes, preventing the body manufacturing rogue proteins that lead to disease.

It has partnerships with several other pharma companies, including Biogen, which led to the launch this year of Spinraza (nusinersen) for spinal muscular atrophy.