Ionis to go it alone with Inotersen and IONIS-FB-L Rx after GSK decline

Inotersen on track for marketing authorization filings this year.

"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 US and EU this year," said B. Lynne Parshall, chief operating officer of Ionis Pharmaceuticals. "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."

Ionis completed the Phase III NEURO-TTR study of inotersen in which the drug demonstrated significant benefit on both primary clinical endpoints of neurological disease progression and quality of life in patients with hATTR-PN.

"Our goals for inotersen are to maximize its commercial success and optimize our commercial participation. To achieve these goals, we are actively considering forming a commercial subsidiary to commercialize or co-commercialize inotersen in North America, as well as other options. Our recent experience building a commercial subsidiary has prepared us for this opportunity. We have substantial interest from potential partners and are in discussions with several parties. We believe that, together with the right commercial partner, we can maximize the commercial success of the drug worldwide," said Sarah Boyce, chief business officer of Ionis Pharmaceuticals.

"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. Our experience in the completed Phase III NEURO-TTR study provides important information to aid in design of a study in patients with cardiomyopathy due to TTR amyloidosis. We have already identified a more potent and convenient LICA follow-on and we expect development of the LICA drug to also proceed rapidly," said Stanley T. Crooke, chairman and CEO of Ionis Pharmaceuticals. "We are deeply committed to the TTR amyloidosis patient community. Patients with TTR amyloidosis, their families and healthcare providers are desperately seeking improved therapeutic options for this devastating, progressive, fatal disease."

IONIS-FB-LRx is a ligand conjugated antisense (LICA) drug in development for the treatment of complement-mediated diseases. In a Phase I 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 initiate the first Phase II study with IONIS-FB-LRx in patients with dry age-related macular degeneration (AMD) later this year, and studies in other indications in 2018.

"IONIS-FB-LRx represents a unique opportunity to develop a treatment for underserved rare and broad patient populations affected by a variety of complement-mediated diseases," said Brett P. Monia, senior vice president of drug discovery and franchise leader for oncology and rare diseases at Ionis Pharmaceuticals. "IONIS-FB-LRx takes advantage of our LICA technology, which can potentially provide greater patient convenience by allowing for significantly lower doses and less frequent administration."

GSK, consistent with its focus on treatments for infectious diseases, continues to advance two drugs targeting hepatitis B virus (HBV) under its collaboration with Ionis: IONIS-HBVRx and IONIS-HBV-LRx. GSK is currently conducting Phase II studies for both drugs.