Ionis Shares Soar After TRYNGOLZA Receives FDA Approval

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Ionis Shares Soar After TRYNGOLZA Receives FDA Approval

Ionis Pharmaceuticals shares rose 6% in pre-market trading on Friday after announcing that the drug TRYNGOLZA™ (olezarsen) has been approved by the U.S. Food and Drug Administration (FDA) for the treatment of familial chylomicronemia syndrome (FCS), a rare and life-threatening disease. This marks an important milestone for the company, as TRYNGOLZA is the first treatment approved for FCS in the U.S., and Ionis expects this to be the first of four product launches over the next three years.

TRYNGOLZA, to be used alongside a low-fat diet, has significantly reduced triglycerides in adults and considerably lowered the risk of acute pancreatitis events. The approval is based on data from the Phase 3 Balance clinical trial, which showed a statistically significant reduction in triglyceride levels and a clinically meaningful decrease in acute pancreatitis events. The treatment, which has a favorable safety profile, is self-administered once a month via an automatic injector.

Ionis CEO Brett P. Monia, Ph.D., characterized the FDA approval as a transformative moment for FCS patients and their families, highlighting the potential of the treatment to change the course of the disease. This approval underscores Ionis’s transformation into a fully integrated, commercial-stage biotechnology company, with a rich pipeline of drugs to follow.

The FDA's decision was influenced by the positive results from the Phase 3 Balance study, previously published in The New England Journal of Medicine. Dr. Alan Brown, a researcher of the study, expressed his excitement about having an effective prescription option for FCS patients.

FCS is characterized by extremely high triglyceride levels and the risk of potentially fatal acute pancreatitis. In the U.S., it is estimated that FCS affects about 3,000 individuals, with most cases remaining undiagnosed. TRYNGOLZA is expected to be available in the U.S. before the end of the year, and Ionis is committed to providing support services to facilitate patients' access to treatment.

TD Cowen analyst Yaron Werber, who shares the company's views, commented on the significance of the approval: "Today’s FDA approval of Tryngolza (olezarsen; QM SC ApoCIII ASO) for familial chylomicronemia syndrome (FCS) represents a significant step in Ionis’s commercial evolution and marks the first approval of a product that is entirely their own. We believe Tryngolza is now the first and only approved treatment for FCS and its label is nearly the best-case scenario."

The approval also includes Priority Review and previously granted Fast Track, Orphan Drug, and Breakthrough Therapy designations. Additionally, olezarsen is under review in the European Union, and regulatory submissions are planned in other countries. Ionis is also evaluating olezarsen for the treatment of severe hypertriglyceridemia (sHTG) in three Phase 3 clinical trials.