Does Pelacarsen’s Phase 3 Miss And ZANVASTRO’s FDA Win Change The Bull Case For Ionis (IONS)?

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) 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.

Ionis Pharmaceuticals (IONS) lost $877 million in market value after its heart drug, pelacarsen, failed a Phase 3 trial. Shares dropped 9.1% to $52.81, and Novartis (NVS) also saw a 5.0% decline. The drug reduced Lp(a) but did not lower cardiovascular events. Ionis retains 75% of future royalties, with Royalty Pharma owning the rest.

Novartis (NVS) and Ionis (IONS) announced that their heart drug, pelacarsen, failed in Phase 3 trials. Both companies' stocks declined in after-hours trading. The drug targets Lp(a), a cardiovascular marker not significantly affected by diet or exercise.

Ionis Pharmaceuticals (IONS) received FDA approval for Zanvastro, the first treatment for Alexander disease, an ultra-rare neurological disorder. The approval came ahead of schedule, and the drug showed meaningful clinical benefits in trials. Ionis estimates peak sales of over $100 million, though patient identification and access will be key. The company also received a Rare Pediatric Disease Priority Review Voucher.