Ionis Partner Licenses Rare Kidney Disease Treatment and Will Advance into Phase 3 Clinical Study
July 11, 2022 at 07:06 am EDT
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Ionis Pharmaceuticals, Inc. announced that its long-standing partner, Roche, will license and advance IONIS-FB-L(Rx), an investigational antisense medicine, into a Phase 3 clinical study in patients with immunoglobulin A nephropathy (IgAN). IgAN is a rare and serious condition that often leads to chronic kidney disease and renal failure. Roche's decision to advance the program comes after positive data from a Phase 2 clinical study in which IONIS-FB-L(Rx) met its primary endpoint of change in 24-hour urinary protein at 29 weeks compared to baseline. In the Phase 2 study (NCT04014335), IONIS-FB-L(Rx) demonstrated a favorable safety and tolerability profile. The study data are consistent with the clinical profile seen across Ionis' other LICA programs, further validating how
advancements in the company's LIgand-Conjugated Antisense technology platform position Ionis to deliver potentially transformative treatments for a range of unmet medical needs. Data from the Phase 2 study of IONIS-FB-L(Rx) in patients with IgAN has been submitted for presentation at an upcoming medical meeting. IgAN occurs when too much IgA protein accumulates in the kidneys, causing inflammation and tissue damage, which is the
root cause of the disease. IONIS-FB-L(Rx) was designed by Ionis to reduce the production of complement factor B (FB), which is associated with the development of several complement-mediated diseases, including IgAN.
Ionis Pharmaceuticals, Inc. is engaged in discovering and developing ribonucleic acid (RNA)-targeted therapeutics. The Company is primarily focused on cardiovascular and neurology franchises. The Company's products include SPINRAZA, TEGSEDI and WAYLIVRA. SPINRAZA is for the treatment of patients with spinal muscular atrophy (SMA), a progressive, debilitating and often fatal genetic disease. TEGSEDI is for the treatment of patients with polyneuropathy caused by hereditary transthyretin (TTR), amyloidosis (hATTR), a debilitating, progressive, and fatal disease. WAYLIVRA is an antisense medicine indicated as an adjunct to diet in adult patients. The Company has over seven medicines in Phase III studies for nine indications, which include Eplontersen (TTR), Olezarsen (ApoC-III), Donidalorsen (PKK), ION363 (FUS), Pelacarsen (Apo(a)), Tofersen (SOD1) and Bepirovirsen. Its cardiovascular medicine in development includes Eplontersen, Olezarsen, Pelacarsen, Fesomersen, ION904 and IONIS-AGT-LRx.