Ionis to Independently Advance Inotersen and IONIS-FB-L Rx
- Category: DNA RNA and Cells
- Published on Saturday, 12 August 2017 10:38
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Inotersen on Track for Marketing Authorization Filings This Year
CARLSBAD, CA, USA I August 11, 2017 I Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) today reported that the Company has retained all rights to inotersen and IONIS-FB-LRx. As part of a reprioritization of its pipeline and strategic review of its Rare Diseases business, GSK declined its options on both drugs. Ionis plans to file for marketing authorization for inotersen this year to support a commercial launch of inotersen in 2018. Inotersen is a drug designed to treat patients with TTR amyloidosis (ATTR). The first indication Ionis is pursuing for inotersen is to treat patients with polyneuropathy due to hereditary TTR amyloidosis (hATTR-PN).
"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," 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 3 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 3 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 chief executive officer 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 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 initiate 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.
"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 2 studies for both drugs.
Ionis has successfully completed the Phase 3 NEURO-TTR study with inotersen in patients with polyneuropathy due to hereditary TTR amyloidosis (hATTR-PN). Ionis plans to file for marketing approval of inotersen in the U.S. and EU in the second half of 2017. The Company also plans to present results from the NEURO-TTR study at an upcoming medical meeting and to submit results from the study for publication in a peer-reviewed medical journal.
The U.S. Food and Drug Administration has granted Orphan Drug Designation and Fast Track Status to inotersen for the treatment of patients with familial amyloid polyneuropathy. The European Medicines Agency has granted Orphan Drug Designation to inotersen for the treatment of patients with TTR amyloidosis.
ABOUT TTR AMYLOIDOSIS
One key manifestation of ATTR results when TTR amyloid fibrils deposit in peripheral nerves, which causes nerve damage throughout the patient's body resulting in the progressive loss of motor functions, such as walking. When this occurs, patients are diagnosed with polyneuropathy due to hereditary TTR amyloidosis (hATTR-PN). Another major manifestation of ATTR occurs when TTR amyloid fibrils build up in heart tissue, which results in cardiomyopathy due to TTR amyloidosis (ATTR-CM). In all manifestations of ATTR, TTR accumulates in major organs, progressively impacts organ function and eventually leads to death. Therapeutic options for the treatment of ATTR are very limited and there are currently no drugs approved for the treatment of ATTR in the United States.
Ionis has completed a Phase 1 study evaluating IONIS-FB-LRx in healthy volunteers. In the Phase 1 study, IONIS-FB-LRx achieved dose-dependent reductions in FB and demonstrated a safety and tolerability profile that supports further clinical development. Ionis plans to initiate the first Phase 2 study with IONIS-FB-LRx in patients with dry AMD later this year, and studies in other indications in 2018.
ABOUT IONIS PHARMACEUTICALS, INC.
SOURCE: Ionins Pharmaceuticals