GSK jilts Ionis rare disease drug as FDA filing nears

GSK jilts Ionis rare disease drug as FDA filing nears | FierceBiotech

GlaxoSmithKline has jilted Ionis Pharmaceuticals just before the pair was due to file for approval of inotersen. The action furthers recently installed GSK CEO Emma Walmsley’s plan to purge the Big Pharma of its rare disease programs. 

Ionis recently brought the transthyretin-targeting antisense drug through a phase 3 program that teed it up to file for approval in polyneuropathy due to hereditary TTR amyloidosis this year. But it will now have to take the drug the final mile unpartnered after GSK declined its option to pick up it and another asset, IONIS-FB-LRx.

Management at Ionis put a positive spin on the news. 

"We are pleased to move forward these two important drugs ourselves. We are prepared to independently advance inotersen and remain on track to file for marketing approval of inotersen in the U.S. and EU this year," Ionis COO Lynne Parshall said in a statement. 

For GSK, the action represents another facet of the sweeping changes implemented by Walmsley since she took the reins at the Big Pharma. Walmsley signaled the GSK rare disease unit is on borrowed time when she unveiled plans to cull more than 30 programs from the company’s pipeline late last month. 

Inotersen and IONIS-FB-LRx were absent from the list shared at that time. But Ionis has now confirmed the drugs have joined the Johnson & Johnson-partnered rheumatoid arthritis drug sirukumab on the list of assets GSK thinks it can do without. 

GSK has spared the two other drugs it is working on with Ionis from the ax. Those programs, IONIS-HBVRx and IONIS-HBV-LRx, target hepatitis B. And, as such, still have a home within the infectious disease unit of GSK’s trimmed-down R&D organization. 

The Big Pharma will continue testing the hepatitis drugs in phase 2 studies while Ionis works on the other assets solo. Ionis plans to get inotersen in front of regulators in the U.S. and Europe this year with a view to bringing it to market in 2018.

Work on IONIS-FB-LRx is less advanced. The ligand-conjugated antisense drug came through a phase 1 trial earlier this year. With that early test out of the way, Ionis is now preparing to move into phase 2, initially in patients with dry age-related macular degeneration, later this year.