Aside from lowering triglyceride levels, Ionis’ olezarsen reduced acute pancreatitis events, an outcome that BMO Capital Markets said could help the asset deliver a “significant first-in-class commercial launch.”
Ionis Pharmaceuticals’ antisense oligonucleotide Tryngolza significantly lowered triglyceride levels and reduced acute pancreatitis events in two pivotal Phase III trials.
Topline data from the Phase III CORE and CORE2 studies showed that olezarsen, the active ingredient in Tryngolza, resulted in a 72% mean reduction of fasting triglycerides versus placebo in patients with severe hypertriglyceridemia (sHTG).
Notably, CORE and CORE2 additionally showed that olezarsen slashed acute pancreatitis events by 85%, an effect that Ionis called “highly statistically significant” in a news release issued Tuesday.
Shares of Ionis shot up nearly 35% to $57.49 at close on Tuesday.
Analysts at BMO Capital Markets wrote in a Tuesday note to investors that this particular finding could prove to be the drug’s market edge. “We anticipate olezarsen’s [acute pancreatitis] benefit to render olezarsen very attractive to patients/physicians/payers, fueling a significant first-in-class commercial launch.”
William Blair agreed, writing on Tuesday that by lowering acute pancreatitis events, olezarsen “addresses the main driver of sHTG-related hospitalization and mortality” and could make it the “first therapy in sHTG to address such events.”
“Taken together, we view these data as a best-case scenario for Ionis,” William Blair wrote.
With CORE and CORE2 data in hand, the biotech plans to file a supplemental application for olezarsen in sHTG by the end of the year. According to William Blair, this sets Ionis up for a potential approval in “late 2026.”
Designed to be delivered via self-injection, Tryngolza is a ligand-conjugated antisense oligonucleotide that targets mRNA coding for apoC-III, leading to an overall reduction in serum levels of the corresponding protein. ApoC-III is an apolipoprotein typically found in the liver and small intestine which helps prevent the breakdown and clearance of lipids. Tryngolza’s action, therefore, helps lower the levels of these fatty molecules.
Tryngolza was approved in December last year to lower triglycerides in adults with familial chylomicronemia syndrome, alongside diet. In its first full commercial quarter, Tryngolza brought in more than $6 million in sales, which ramped up to $19 million in the second quarter, according to the company’s business report in July.
Beyond Tryngolza, Ionis last month secured the FDA’s approval for Dawnzera, the U.S.’s first RNA-targeting therapy indicated as a prophylactic treatment for hereditary angioedema.
