Roche-Ionis’ RNA therapy reduces kidney disease marker, opening path to swift FDA nod

Novartis' Phase 3 trial for Pelacarsen showed it lowered lipoprotein(a) but failed to reduce major cardiovascular events. Citi suggests this may influence CRISPR Therapeutics' focus on CTX321 over CTX320, while Ionis Pharmaceuticals may see limited impact. Citi maintains Buy ratings for both companies with price targets of $88 for CRISPR and $100 for Ionis.

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.

Ionis Pharmaceuticals (IONS) and Roche (RHHBY) reported positive late-stage trial results for their kidney drug sefaxersen, meeting its primary endpoint based on interim data. The therapy is designed for patients with primary IgA nephropathy, a kidney disorder.

Ionis and Otsuka reported positive Phase 3 trial results for ulefnersen, an antisense therapy for FUS-ALS, a rare genetic form of ALS. The trial met its primary endpoint, showing significant improvements in function and survival. The companies plan to discuss expedited approval with regulators. Ionis' stock has seen volatility, but the neurology portfolio shows momentum with recent approvals and trial successes.