Sobi UK: NICE issues final draft guidance recommending - Tryngolza (olezarsen) as an option to treat genetically confirmed familial chylomicronaemia syndrome (FCS) in adults in England and Wales

Ionis Pharmaceuticals and Roche reported positive Phase 3 interim data for sefaxersen in treating primary IgA nephropathy, meeting proteinuria endpoints. The company is still loss-making, with a net loss of $565.2 million on $874.1 million revenue. Analysts project $2.2 billion revenue and $261.6 million earnings by 2029, assuming 36.3% annual growth. Success depends on execution, pricing, and partner coordination.

The FDA approved several neurology drugs in September 2026. Ionis Pharmaceuticals' Zilganersen (Zanvastro) was approved for Alexander disease, Scholar Rock's apitegromab-mstn (Isembyld) for SMA, Ultragenyx's rebisufligene etisparvovec-hopf (Fayuvi) for Sanfilippo A, IntraBio's levacetylleucine (Aqneursa) for ataxia-telangiectasia, and AbbVie's Tavapadon (Juvmo) for Parkinson's. Azurity Pharmaceuticals' Lamlique, a liquid lamotrigine, was also approved for epilepsy.

Ionis Pharmaceuticals (IONS) and partners reported positive Phase 3 results for two RNA-targeted medicines, ulefnersen and sefaxersen, meeting primary endpoints. The company's revenue was US$874.1 million with a net loss of US$565.2 million. Analysts project US$2.3 billion in revenue and US$222.2 million in earnings by 2029, with a 37.1% annual growth rate. The stock is down 42.4% year-to-date as of late September 2026.

Ionis Pharmaceuticals (IONS) reported positive Phase 3 trial results for two RNA-targeted drugs: sefaxersen, partnered with Roche, met its primary endpoint in IgA nephropathy, and ulefnersen, developed with Otsuka, achieved its main goal in FUS-ALS. These results could expand Ionis's market share and revenue, but pricing and regulatory risks remain.

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.