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Ionis: Success in Antisense Technology

06/16/2017 2:56 am EST


John McCamant

Editor, Medical Technology Stock Letter

Ionis Pharmaceuticals (IONS) has a broad pipeline that continues to advance; it ANGPTL3-L has significant potential as a new and affordable cardio drug with its novel and unique mechanism of action, explains biotech expert John McCamant, editor of The Medical Technology Stock Letter.

This exciting drug development candidate that is being developed to treat rare hyperlipidemias, non-alcoholic fatty liver disease and combined dyslipidemias to potentially prevent and treat cardiovascular disease.

Akcea Therapeutics, a wholly-owned subsidiary of Ionis, has announced the publication in The New England Journal of Medicine of key preclinical findings with angiopoietin-like 3-targeting drugs and Phase 1/2 clinical study results that showed substantial, dose-dependent reductions in multiple lipids and lipoproteins.

Dr. John Kastelein, the guru of new cholesterol drug development and eminent professor of medicine at the University of Amsterdam recently stated that adding drugs that target ANGPTL3 to the existing arsenal of statins and blood pressure medications “will drive the final nail in the coffin of heart disease.”

The drugs that dramatically lower triglycerides are evinacumab from  Regenron (REGN) and IONS’ ANGPTL3-L which both block the action of angiopoietin-like 3 (ANGPTL3).

The discovery of these drug development candidates came from identifying a genetic mutation in people who had no working copy of the gene that produces ANGPTL3, resulting in abnormally low triglyceride levels. These patients also had a greatly reduced risk of cardiovascular disease.

The theory is that blocking the effects of ANGPTL3 could mimic the genetically mutated state and reduce triglycerides to unprecedented levels.

In our view, the IONS drug would have a significant pricing advantage over REGN’s mAB, as they could charge 2-3K per year, the necessary value added price point to create a new blockbuster cardio drug.

Meanwhile, IONS and their partner Biogen (BIIB) have received marketing authorization for SPINRAZA from the European Commission (EC) for the treatment of 5q spinal muscular atrophy (SMA). 5q SMA is the most common form of the disease and represents approximately 95% of all SMA cases.

SPINRAZA is the first approved treatment in the European Union (EU) for SMA, a leading genetic cause of death in infants that is marked by progressive, debilitating muscle weakness. BIIB has already been working with health systems and government agencies across the EU to help patients secure access to SPINRAZA.

IONS continues to execute at a high level with the SPINRAZA approval in Europe and the publication in the The New England Journal of Medicine provide significant validation for the company’s antisense drug development platform.

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