Ionis announces initiation of the global Phase 3 BALANCE study for AKCEA-APOCIII-LRX in patients with familial chylomicronemia syndrome

CARLSBAD, CA, USA I December 1, 2020 I 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.

"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."

The 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.

AKCEA-APOCIII-LRx is one of 20 potentially transformative antisense programs in the growing Ionis-owned pipeline that the company is prioritizing and preparing for commercialization. Learn more about some of these programs at Ionis' Virtual Investor Day on Dec. 7, 2020 at 11 a.m. EST.

About FCS
FCS is an ultra-rare disease caused by impaired function of the enzyme lipoprotein lipase (LPL) and characterized by severe hypertriglyceridemia (>880mg/dL or 10mmol/L) and a risk of unpredictable and potentially fatal acute pancreatitis. Because of limited LPL production or function, people with FCS cannot breakdown chylomicrons, lipoprotein particles that are 90 percent triglycerides. In addition to pancreatitis, FCS patients are at risk of chronic complications due to permanent organ damage, including chronic pancreatitis and pancreatogenic (type 3c) diabetes. They can experience daily symptoms including abdominal pain, generalized fatigue and impaired cognition that affect their ability to work. People with FCS also report major emotional and psychosocial effects including anxiety, social withdrawal, depression and brain fog. Additional information on FCS is available at www.fcsfocus.com, through Action FCS at https://www.actionfcs.org/ and through The FCS Foundation at http://www.livingwithfcs.org. For a full list of organizations supporting the FCS community worldwide, please click here.

ABOUT AKCEA-APOCIII-LRx
AKCEA-APOCIII-LRx is an investigational antisense medicine designed to reduce the production of apolipoprotein C-III, or apoC-III. ApoC-III is a protein produced in the liver that plays a central role in the regulation of serum triglycerides. Genetically reduced levels of apoC-III are correlated to lower levels of triglycerides and lower risk of cardiovascular disease whereas elevated levels of apoC-III correlate with high triglyceride levels that have been associated with multiple metabolic abnormalities, such as insulin resistance and/or metabolic syndrome as well as elevated cardiovascular event risk. AKCEA-APOCIII-LRx was developed using Ionis' advanced LIgand Conjugated Antisense (LICA) technology platform. AKCEA-APOCIII-LRx is in Phase 3 development for patients with FCS, with plans to evaluate AKCEA-APOCIII-LRx in patients with other hypertriglyceridemia disorders. AKCEA-APOCIII-LRx was discovered and developed by Ionis.

ABOUT IONIS PHARMACEUTICALS, INC.
As the leader in RNA-targeted drug discovery and development, Ionis has created an efficient, broadly applicable, drug discovery platform called antisense technology that can treat diseases where no other therapeutic approaches have proven effective. Our drug discovery platform has served as a springboard for actionable promise and realized hope for patients with unmet needs. We created the first and only approved treatment for children and adults with spinal muscular atrophy as well as the world's first RNA-targeted therapeutic approved for the treatment of polyneuropathy in adults with hereditary transthyretin amyloidosis. Our sights are set on all the patients we have yet to reach with a pipeline of more than 40 novel medicines designed to potentially treat a broad range of diseases, including neurological, cardio-renal, metabolic, infectious, and pulmonary diseases. To learn more about Ionis visit www.ionispharma.com and follow us on Twitter @ionispharma.

SOURCE: Blueprint Pharmaceuticals

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