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Ionis announces initiation of the global Phase 3 BALANCE study for AKCEA-APOCIII-LRX in patients with familial chylomicronemia syndrome
CARLSBAD, Calif., Dec. 1, 2020 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) today announced the initiation of the Phase 3 BALANCE study for
About this update from Ionis Pharmaceuticals, Inc.
[{"type":"text","content":"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.\n\n \n \n \n \n \n \n\n \n\"Initiation of the Phase 3 BALANCE study is a significant milestone for the FCS patient community and for our company. This study also reflects our continuing commitment to develop novel treatment options for patients with unmet medical needs such as those living with FCS,\" said Richard S. Geary, Ph.D., executive vice president of development. \"AKCEA-APOCIII-LRx is the second Ionis antisense medicine that we are developing with FCS patients in mind. We are hopeful about the prospect of bringing forward a new, safe, and effective treatment since these patients have limited available options.\"\nThe Phase 3 BALANCE study is a global, multi-center, randomized, double-blind, placebo-controlled study enrolling up to 60 patients (age 18 and over) with FCS. Participants will be randomized in a 2:1 ratio to receive AKCEA-APOCIII-LRx or placebo via subcutaneous injection once every four weeks for a total 53-week treatment period. The primary endpoint is percent change from baseline in fasting triglyceride levels at six months compared to placebo. Following the treatment period, eligible patients will have the option of enrolling in an open-label extension study. In addition to FCS, Ionis is evaluating additional indications for AKCEA-APOCIII-LRx development. More information on the BALANCE study is available at www.clinicaltrials.gov/NCT04568434.\nAKCEA-APOCIII-LRx is one of 20 potentially transformative antisense programs in the growing Ionis-owned pipeline t...