Ionis Continues FAP Therapy Development Alone as GSK Reconsiders Rare Diseases Field

GlaxoSmithKline (GSK) has backed away from the chance to license two Ionis Pharmaceuticals treatments for familial amyloid polyneuropathy (FAP) — also known as TTR amyloidosis (ATTR) — just as Ionis prepares to file for U.S. and European approval of inotersen, one of the two drugs.

The move, which also included IONIS-FB-LRx, came as the British drug conglomerate opted to move away from rare diseases in a restructuring of the GSK pipeline. Yet this doesn’t mean inotersen will be stranded, as Ionis will continue the process of bringing both  medications to market.

“We are pleased to move forward these two important drugs ourselves,” B. Lynne Parshall, chief operating officer of Ionis, said in a press release. “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.”

Inotersen is a so-called RNA antisense drug that aims to stop production of the TTR protein, which aggregates into amyloid deposits in people with FAP and other types of TTR amyloidosis. Ionis hopes to use Inotersen to treat polyneuropathy caused by FAP.

“We want to thank our collaboration team at GSK for their support and commitment to patients with TTR amyloidosis, and their efforts to work closely with us to ensure a smooth transition so that this important medicine can be available to patients as planned,” said Parshall.

The recently completed NEURO-TTR Phase 3 trial (NCT01737398) of inotersen showed the drug improved neurological symptoms and quality of life in FAP patients. But FAP does not only affect nerves, and Ionis is also focusing its efforts on heart disease in FAP.

“We are also accelerating the expansion of our TTR program for patients with cardiomyopathy due to TTR amyloidosis and the development of our LICA follow-on drug,” said Stanley T. Crooke, chairman and CEO of Ionis, which is based in Carlsbad, California.

LICA refers to “ligand conjugated antisense” — the scientific term for the other Ionis drug affected by the GSK dropout, IONIS-FB-LRx.

“Our experience in the completed Phase 3 NEURO-TTR study provides important information to aid in design of a study in patients with cardiomyopathy due to TTR amyloidosis,” said Crooke. “We have already identified a more potent and convenient LICA follow-on and we expect development of the LICA drug to also proceed rapidly.”

The European Medicines Agency and the U.S. Food and Drug Administration have both granted inotersen an Orphan Drug Designation, with the FDA also giving the treatment fast-track status in an effort to speed up its clinical development for FAP.