Failure of Novartis’ Pelacarsen Could Reshape the Lp(a) Race for CRISPR and Ionis

Novartis won a lawsuit alleging it improperly listed a patent to delay generic competition for Entresto, its heart-failure drug. Judge Liman dismissed the case, agreeing the patent was properly listed. Entresto sales fell 42% to $1.31B in Q1 2026 after patent expiration and generic entry, accounting for 14% of Novartis's prior-year sales.

Gene-editing stocks fell Wednesday after Anthropic's AI model, Claude, identified a phage enzyme system with traits similar to CRISPR. CRISPR Therapeutics (CRSP) dropped 6%, Intellia (NTLA) 3%, Beam (BEAM) 6%, Prime Medicine (PRME) 12%, and Editas Medicine (EDIT) 8%. The discovery, called ART, links an RNA-to-DNA enzyme with repeating DNA and a mystery protein in bacteria-infecting viruses. CRISPR pioneer Feng Zhang called the finding intriguing but unproven.

Ionis Pharmaceuticals announced that its Phase 3 IMAgINATION trial for sefaxersen met its primary endpoint, showing a statistically significant and clinically meaningful reduction in urine protein at week 37 compared to placebo. The trial, conducted with Roche, remains blinded for a week-105 comparison of kidney function. Ionis may receive milestones and royalties if sefaxersen advances and sells.

Ionis Pharmaceuticals (IONS) stock hit a 52-week low of $44.20, trading at $44.24 with a $7.39B market cap. The stock has fallen 30.81% over the past year and 42% year-to-date. Analysts have mixed views, with price targets ranging from $54 to $105. Recent clinical trial results showed positive outcomes for Ulefnersen in FUS-ALS, but some analysts lowered price targets due to concerns about drug launches and trial results.

BMO Capital reiterated a Market Perform rating and $54 price target for Ionis Pharmaceuticals (IONS) after positive ALS drug trial results. The stock is near its 52-week low, down 42% YTD. The study met primary endpoints, showing functional improvement and survival benefits. Analysts have mixed outlooks, with some lowering price targets due to recent setbacks.

Roche and Ionis Pharmaceuticals reported positive Phase 3 trial results for sefaxersen, their RNA therapy for kidney disease, meeting the primary endpoint of reducing proteinuria. The drug showed a statistically significant improvement in urine protein-to-creatinine ratio compared to placebo, suggesting potential for accelerated FDA approval. Ionis' CEO indicated a regulatory filing would follow soon.