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Ionis Closer To First Wholly-Owned Drug Launch With Olezarsen Phase III Success

Drug Targets apoC-III To Lower Triglycerides

 
• By Mandy Jackson

Ionis will seek US and EU approvals to treat familial chylomicronemia syndrome in the first half of 2024. Phase III data in the larger severe hypertriglyceridemia indication are due in late 2024 or early 2025.

Anatomical model of pancreas on doctor table over background gastroenterologist consulting woman patient
A high dose of olezarsen eliminated risk of acute pancreatitis in FCS • Source: Shutterstock

Ionis Pharmaceuticals, Inc. took another step forward on its path to bringing its first wholly-owned drug to market without a partner on 26 September when it reported positive topline results from the Phase III BALANCE clinical trial of olezarsen in the treatment of familial chylomicronemia syndrome (FCS). The company plans to file for US and EU approvals during the first half of 2024 and, if granted priority review, expects US Food and Drug Administration approval before the end of next year and a launch soon after.

Key Takeaways

  • Ionis will file for US and EU approvals of olezarsen in FCS during the first half of 2024 based on positive results from the Phase III BALANCE study and expects FDA approval, assuming priority review due to a prior fast track designation, before the end of 2024.

  • FCS is a rare, genetic disease with as few as 1,000 patients in the US, but if triglyceride lowering observed in BALANCE is seen in the much larger SHTG indication of 3 million US patients late next year or in early 2025, olezarsen could be a blockbuster product for Ionis

The positive BALANCE readout also bodes well for olezarsen in the much larger indication of severe hypertriglyceridemia (SHTG), which encompasses about 3 million patients in the US versus 1,000-5,000 US patients with the rare, genetic disease FCS, according to William Blair analyst Myles Minter

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