Potential Novel Anti-thrombotic Treatment by Ionis Pharmaceuticals

On July 28, Ionis Pharmaceuticals announced positive top-line results for their anti-thrombotic treatment, known as fesomersen, for patients with end-stage renal disease (ESRD) on hemodialysis. Fesomersen is not only designed to prevent thrombosis but is safe and well-tolerated by patients in treatments that lasted up to 48 weeks. 

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Thrombosis and its Current Medications

Thrombosis is a condition wherein blood clots form inside arteries and veins. When this occurs, these clots can block oxygen flow to tissues and organs throughout the body. These clots can break apart, and the fragments can travel to different parts of the body’s circulation. As a result, thrombosis is the leading cause of morbidity and mortality for the number of heart attacks and strokes it causes.

Nearly 15.3 million die a year due to heart attacks and strokes. Blood clots formed from thrombosis can cause this after they block blood vessels in the heart or brain.

Currently, anti-thrombotic treatments include anticoagulants, Factor Xa inhibitors, and thrombin inhibitors. However, though these established therapies effectively lower the risk of thrombosis, their downside is the risk of patients bleeding because they target factors required for normal blood clotting.

On these anti-thrombotic treatments, Sanjay Bhanot, M.D., Ph.D., senior vice president, chief medical officer, and metabolic and liver franchise leader at Ionis, said in a statement, “For decades, anticoagulants have been a therapeutic mainstay in the treatment and prevention of thrombosis. However, they can increase bleeding risk, leading to major, sometimes fatal, bleeding events.”

Fesomersen Can Help Prevent Heart Attacks and Strokes

Fesomersen is a new drug designed by Ionis to treat patients with ESRD. It does this by targeting and inhibiting Factor XI, a protein in the liver vital in coagulation. However, high levels of this protein can lead to thrombosis.

The RE-THINC ESRD Study, a double-blind, placebo-controlled study evaluating the efficacy of fesomersen, found that inhibition of Factor XI not only lowered the risk of developing thrombosis but also reduced the risk of an increase in bleeding as opposed to the placebo.

This novel inhibitor was safe and well tolerated at all of its monthly doses of 40mg, 80mg, and 120mg for up to 48 weeks. The results of this Phase 2b study consistently match the clinical profile seen across Ionis’ other Ligand-Conjugated Antisense (LICA) programs.

As a result of fesomersen achieving its primary outcome measure, an upcoming medical meeting will present data from the study. It is very likely that this medication, which could positively impact preventing heart attacks and strokes, will move onto a Phase 3 study in the future.

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Max Heirich