Ionis partner Novartis announces Lp(a)HORIZON Phase 3 topline results for pelacarsen in patients with elevated Lp(a) and established cardiovascular disease (CVD)

Amgen shares fell 5% after Novartis' pelacarsen failed to meet its primary endpoint in a phase III study, raising concerns about Lp(a)-lowering therapies. Amgen's olpasiran, also targeting Lp(a), is under investigation. Novartis and Ionis shares also declined. Year-to-date, Amgen shares are up 36.3%.

The FDA approved Ionis Pharmaceuticals' Zanvastro (zilganersen) for Alexander disease, a rare neurological disorder. The first treatment for the condition, it targets the protein buildup causing the disease. Approval was based on a small clinical trial showing improved walking speed and motor function. Side effects include vomiting, back pain, and meningitis. The drug will be available in the U.S. soon, with pricing undisclosed. Ionis licensed rights outside the U.S. to Recordati.

Novartis and Ionis' RNA drug, pelacarsen, failed to reduce heart attack, stroke, and death risks in a Phase 3 trial, despite lowering Lp(a) levels. Novartis stated the results were disappointing but may inform future cardiovascular approaches. Analysts noted the failure raises questions about similar therapies and potential impacts on ongoing trials. The drug was expected to generate significant revenue if successful.

Novartis and Ionis Pharmaceuticals reported that their experimental heart drug, pelacarsen, failed to reduce the risk of major heart attacks and strokes in a late-stage trial. The drug, targeting genetically inherited cholesterol Lp(a), lowered Lp(a) levels but did not meet its primary endpoint. Novartis shares fell 5%, and Ionis shares dropped 12% in aftermarket trading. The companies plan to present full trial data at an upcoming medical congress.

Ionis Pharmaceuticals (IONS) reported mixed results: Novartis' Phase 3 trial of pelacarsen failed to meet its primary endpoint, but the FDA approved Ionis' ZANVASTRO for Alexander disease. The company projects $2.3B revenue and $222.2M earnings by 2029, a 51% upside from its current price.

Ionis Pharmaceuticals (IONS) shares rose 4% after-hours following FDA approval for Tryngolza in severe hypertriglyceridemia. Goldman Sachs raised its price target to $75, maintaining a 'Neutral' rating. The drug showed significant triglyceride reduction and pancreatitis risk reduction in trials. Ionis plans a July U.S. launch. Analysts' average price target is $104.91, suggesting 37% upside from Wednesday's close.