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Ionis Pharmaceuticals, Inc. (IONS) announced on Tuesday that its medicine, Olezarsen, reduced triglycerides and acute pancreatitis events in late-stage trials evaluating the drug in individuals with severe hypertriglyceridemia.
Severe hypertriglyceridemia (sHTG) is defined by severely high triglycerides reaching into the thousands. It is characterized by an increased risk of acute pancreatitis and other morbidities. According to Ionis, approximately three million people are living with sHTG in the U.S., including more than one million who are considered high risk.
In two studies, Olezarsen demonstrated a highly statistically significant placebo-adjusted mean reduction in fasting triglycerides of up to 72% at six months, as well as a highly statistically significant 85% reduction in acute pancreatitis events, with favorable safety and tolerability profiles. The two trials cumulatively enrolled nearly 1,100 patients who were required to be on standard-of-care lipid-lowering therapy throughout the treatment period.
IONS stock rocketed by 34% by Tuesday afternoon, reaching its February 2021 highs. On Stocktwits, retail sentiment around IONS jumped from ‘bearish’ to ‘extremely bullish’ over the past 24 hours, while message volume rose from ‘low’ to ‘extremely high’ levels.
A Stocktwits user expressed optimism about the company’s future.
Sam Tsimikas, senior vice president of global cardiovascular development at Ionis, termed the data “groundbreaking” and noted that the data reinforce the company’s confidence that the medicine has the potential to change how sHTG is treated. According to the firm, current standard of care therapies for sHTG and lifestyle modifications do not sufficiently or consistently lower triglyceride levels or reduce the risks of sHTG in all patients.
Ionis plans to submit a supplemental new drug application (sNDA) to the U.S. Food and Drug Administration for approval of Olezarsen in sHTG by the end of the year. The drug is already approved in the U.S. as Tryngolza as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia (FCS).
Several analysts hiked their price target on the stock following the news:
IONS' stock is up 64% this year and approximately 21% over the past 12 months.
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