Ionis shares surge after drug to lower blood fat shows promise in trials

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By Kamal Choudhury

(Reuters) -Ionis Pharmaceuticals’ shares surged on Tuesday after its drug significantly lowered the levels of a type of blood fat and the risk of a pancreatic disease in two late-stage studies, potentially paving the way for a billion-dollar opportunity.

Shares of the drugmaker jumped about 34% to $57.23.

Ionis was testing the drug, olezarsen, in two trials with nearly 1,100 patients combined who had severe hypertriglyceridemia, marked by high levels of a fat called triglycerides in the blood. The disorder raises the risk of heart disease and pancreatitis, caused by inflammation and scarring of the pancreas.

The drug cut triglyceride levels by up to 72% compared with placebo and reduced acute pancreatitis events by 85% after 12 months — the first treatment to achieve statistically significant reduction of risk in people with severely high triglycerides.

RBC Capital Markets analyst Luca Issi called the results “splashier-than-expected” and “an absolute home run scenario.” Most investors thought significant reduction in acute pancreatitis was a “coin toss,” he said.

The reduction in triglycerides alone is sufficient for approvals in the U.S. and EU, said Oppenheimer analyst Jay Olson, adding that reduced pancreas damage further sets the drug apart.

Jefferies analysts estimate peak annual sales at about $2.5 billion.

Ionis plans to seek expanded approval for the drug in the U.S. by year-end.

Olezarsen is already approved in the U.S. under the brand name Tryngolza to treat familial chylomicronemia syndrome, a rare genetic disorder leading to extremely high triglyceride levels.

Over 3 million people in the U.S. have severe hypertriglyceridemia with more than 1 million considered high risk, according to Ionis.

Patients are now dependent on omega-3 fatty acids and fibrates, drugs that help break down fats, to manage the condition.

(Reporting by Kamal Choudhury in Bengaluru; Editing by Sahal Muhammed)