Ionis intends to form a supplemental page to further submit its potential by the end of the year to extend Tryngolza into an extreme hypertriglyceridemia. If accepted, then William Blair will strengthen its hope, claiming the antisense drug to be known as a transformational one for this indication. Ionis Pharmaceuticals' antisense oligonucleotide Tryngolza prominently reduces triglyceride levels and improves outcomes in patients with acute pancreatitis in ongoing late-stage studies for extreme hypertriglyceridemia. This will support California biotech in building a case for label extension into this indication.
William Blair named Ionis’ readout as a ‘game changer’ move that will act as a transformational treatment for sHTG (severe hypertriglyceridemia) throughout its vast area of patients on the FDA’s approval for Tryngolza. As of now, Tryngolza is only considered for familial chylomicronemia syndrome (FCS) patients, which is a rare genetic disease that causes increased blood triglyceride levels.
At the 2025 Scientific Sessions of the American Heart Association on Saturday, the Ionis presentation highlighted Tryngolza's 72% reduction capacity of fasting triglyceride levels in 6 months and an impact that was survived via 12 months of follow-up. Alongside, Ionis’ antisense oligonucleotide also mitigated the acute pancreatitis scenario by 85%. The 89% patient group living on 50 mg Tryngolza experienced a drop in triglyceride levels by 880 mg/dL, this almost in generally linked with the greatest risk of acute pancreatitis.
The same dose observed 34% of patients below 150 mg/dL, as it’s a standard bar for common triglyceride levels. William Blair named the data as groundbreaking with a precise demonstration of mitigated pancreatitis risk in the patient group study and throughout the major subgroups, especially those who were at the highest risk of this extreme. Analysts added, “Tryngolza was observed with near-immediate shield from AP (acute pancreatitis) events.”
The company’s late-stage achievement has been consistently accelerating in a winning game for the biotech in these months. The company received an FDA approval for Dawnzera, the company’s first-ever RNA-focused therapy, in August. This therapy proved to be a prophylactic alternative for hereditary angioedema (HAE), suitable for patients 12 years and older patient group.
William added, “Ionis will cover all diversified aspects to run a commercial business with the introduction of Tryngolza in FCS and Dawnzera in HAE prophylaxis.” Tryngolza’s valued extension into sHTG under a group ‘de-risked’ will also contribute to the business profile and accelerate the company’s portfolio effectively.
Holding M.Pharm in Pharmaceutical Chemistry, Chandni crafts cutting-edge, research-driven healthcare news for Towards Healthcare, combining scientific depth with innovative storytelling to simplify complex topics for global readers.
