Ionis Pharmaceuticals scored a "home run" Tuesday after its experimental drug topped the placebo in a study of patients with severely high triglycerides.
The company tested its drug, olezarsen, in patients with severe hypertriglyceridemia, or sHTG. In this condition, very high triglycerides leave patients at risk of pancreatitis, cardiovascular disease and liver damage.
After half a year of monthly doses, patients who received olezarsen showed statistically significant reductions in triglycerides, up to 72% better, on average, than the placebo group. Olezarsen recipients also had an average 85% reduction in pancreatitis events compared to placebo recipients.
Sam Tsimikas, senior vice president of global cardiovascular development for Ionis, described the results as "groundbreaking" for the Core and Core2 studies. Leerink Partners analyst Mani Foroohar said Ionis had hit a "blue-sky" scenario while RBC Capital Markets analyst Luca Issi called the results a "home run."
"We view these data as the best-case scenario for Ionis and substantially exceeding our expectations," William Blair analyst Myles Minter said in a report titled "Core Hits Bull Case." Ionis "set a high competitive bar as potentially the first therapy for sHTG to significantly reduce acute pancreatitis events, if approved with the data on label."
On the stock market today, Ionis Pharmaceuticals stock rocketed nearly 34.8% to 57.49. Shares hit their highest point since February 2021.
Ionis To Seek Approval This Year
Ionis is now planning to ask the Food and Drug Administration to approve olezarsen as a treatment for sHTG before the end of the year. Olezarsen already sells as a branded drug called Tryngolza to reduce triglycerides in patients with familial chylomicronemia syndrome, or FCS.
RBC's Issi says the results easily beat investors' expectations.
"This is an absolute best-case scenario for IONS," he said in a client note. "Statistically significant reduction in triglycerides (primary endpoint) was largely anticipated, but most investors thought statistically significant reduction in acute pancreatitis (key for access/reimbursement) was a coin toss."
The 85% reduction, on average, in acute pancreatitis cases was well ahead of a minimum bar for 20% to 30%. In FCS, patients experienced a roughly 30% to 40% reduction in triglycerides with olezarsen.
The much higher result in sHTG is "splashier than expected," he said. This is likely because the drug works on the lipoprotein lipase pathway, which is disrupted in FCS but remains intact in sHTG.
"We think this is an absolute home run scenario for IONS (with also positive readthrough to ARWR as this validates the class) and we are buyers into strength as we think this is a key milestone in the ongoing journey to become a fully integrated Biotech company," Issi said.
Arrowhead Pharmaceuticals stock also jumped nearly 10% to 24.23, though that was partly related to a licensing deal worth up to $2.2 billion with Novartis.
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