Ionis announces initiation of the global Phase 3 BALANCE study for AKCEA-APOCIII-LRX in patients with familial chylomicronemia syndrome
2020-12-01 · Ionis Pharmaceuticals · original ionis.com ↗
CARLSBAD, Calif., Dec. 1, 2020 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) today announced the initiation of the Phase 3 BALANCE study for AKCEA-APOCIII-LRx in adult patients with familial chylomicronemia syndrome (FCS). FCS is a debilitating genetic disease characterized by severely high plasma levels of triglycerides and a risk of unpredictable and potentially fatal acute pancreatitis. In addition to acute pancreatitis, FCS patients are at risk of chronic complications due to permanent organ damage, including chronic pancreatitis and pancreatogenic diabetes. AKCEA-APOCIII-LRx is designed using Ionis' proprietary Ligand Conjugated Antisense (LICA) technology platform and is designed to inhibit production of apolipoprotein C-III (apoC-III), a protein produced in the liver that plays a central role in the regulation of serum triglycerides.
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