Zilganersen

Ionis Pharmaceuticals' shares rose 5% after FDA approved its RNA-targeted therapy, Tryngolza, for familial chylomicronemia syndrome (FCS), marking the first treatment for FCS in the U.S. The approval is supported by phase III BALANCE study results showing significant triglyceride-lowering and reduced acute pancreatitis attacks. Tryngolza is Ionis' first wholly-owned drug and independent commercial launch, expected by year-end. The company is also evaluating Tryngolza for severe hypertriglyceridemia and has collaborations with major drugmakers for various treatments.

Ionis Pharmaceuticals (IONS) reported a narrower-than-expected Q3 2024 loss of 95 cents per share, with total revenues of $134 million, beating estimates. The company's shares have declined 22.9% YTD. Ionis receives royalties from Biogen for Spinraza and co-markets Qalsody with Biogen. Wainua, co-marketed with AstraZeneca, was approved in the U.S. and the UK, generating $30 million in milestone payments. Commercial revenues were $76 million, missing estimates. Ionis maintained its 2024 financial guidance, expecting total revenues over $575 million and an adjusted operating loss of less than $475 million.

Ionis Pharmaceuticals reports Q3 2024 financial results, highlighting progress with WAINUA U.S. launch, olezarsen FDA action date in December 2024, and donidalorsen HAE PDUFA date in August 2025. The company is on track to achieve 2024 financial guidance and increased cash guidance to $2.2 billion. Key pipeline milestones include positive CHMP opinion for WAINUA in the UK, QALSODY approval in China, and ongoing pivotal development programs for olezarsen and donidalorsen.

Ionis Pharmaceuticals reports Q3 2024 financial results, highlighting successful U.S. launch of WAINUA, upcoming FDA action dates for olezarsen and donidalorsen, and reaffirmed 2024 P&L financial guidance with increased cash guidance to $2.2 billion.

Ionis Pharmaceuticals announced the FDA granted zilganersen Fast Track designation for treating Alexander disease, an ultra-rare neurological disorder. Zilganersen aims to address the underlying cause by reducing excess glial fibrillary acidic protein production. Topline data from the pivotal study is expected in 2025.