Ionis announces positive olezarsen topline results from Phase 3 study in people with familial chylomicronemia syndrome
- Olezarsen met the primary endpoint with a statistically significant reduction in triglyceride levels versus placebo
- First study to show an absolute reduction in acute pancreatitis in
FCSpatients, with olezarsen 80 mg monthly dose reducing events by 100 percent
- Olezarsen demonstrated a favorable safety and tolerability profile
- Ionis plans to file a New Drug Application with
U.S.FDA in preparation for first potential independent launch
- Phase 3 studies continue evaluating olezarsen in broader patient population with severe hypertriglyceridemia (SHTG)
- Ionis to host webcast today at
11 a.m. ET
Treatment with olezarsen 80 mg resulted in a >75% reduction in apoC-III, a protein produced in the liver that regulates TG metabolism in the blood. In addition to the 80 mg monthly dose, the study also evaluated a lower 50 mg monthly dose. Olezarsen demonstrated a dose-dependent effect, with both study doses showing a substantial reduction in pancreatitis. The lower 50 mg dose did not reach statistical significance at six months on the primary endpoint of triglyceride lowering (p=0.0775).
Ionis plans to file a New Drug Application in early 2024 with the
"These positive olezarsen topline results represent an important advance for people with
Olezarsen demonstrated a favorable safety and tolerability profile in the study. There were more adverse events in the placebo group compared to the olezarsen groups, primarily due to pancreatitis events. The majority of adverse events in the olezarsen groups were mild in severity. There was a low incidence of injection site reactions. No hepatic or renal toxicity events occurred and there were no clinically meaningful platelet reductions. One death was reported in the study, which was deemed as not related to study drug.
"Today is a proud moment in our company's evolution, as olezarsen is poised to be a long-awaited advance for
Ionis will conduct a webcast today at 11:00 a.m. Eastern time to discuss this announcement. Interested parties may access the webcast here. A webcast replay will be available for a limited time at the same address. Visit www.ionispharma.com for more information and to register.
About the Balance Study
The global, multicenter, randomized, double-blind, placebo-controlled Phase 3 Balance study (NCT04568434) enrolled 66 patients aged 18 and older with confirmed
Currently, there are no FDA-approved therapies for the treatment of
Olezarsen is an investigational LIgand Conjugated Antisense (LICA) medicine being evaluated for people at risk of disease due to elevated triglyceride (TG) levels including those with familial chylomicronemia syndrome, or
For more than 30 years, Ionis has been a leader in RNA-targeted therapy, pioneering new markets and changing standards of care. Ionis currently has four marketed medicines and a promising late-stage pipeline highlighted by cardiovascular and neurological franchises. Our scientific innovation began and continues with the knowledge that sick people depend on us, which fuels our vision to become the leader in genetic medicine, utilizing a multi-platform approach to discover, develop and deliver life-transforming therapies.
To learn more about Ionis visit www.ionispharma.com and follow us on Twitter @ionispharma.
Ionis' Forward-looking Statements
This press release includes forward-looking statements regarding Ionis' business and the therapeutic and commercial potential of olezarsen, Ionis' technologies and other products in development. Any statement describing Ionis' goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties including those related to our commercial products and the medicines in our pipeline, and particularly those inherent in the process of discovering, developing and commercializing medicines that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such medicines. Ionis' forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Ionis' forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Ionis. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Ionis' programs are described in additional detail in Ionis' annual report on Form 10-K for the year ended
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