Ionis Pharmaceuticals, Inc. (Ionis), formerly Isis Pharmaceuticals, is a commercial-stage biotechnology company founded in 1989 and headquartered in Carlsbad, California. The company pioneers RNA-targeted medicines, specializing in antisense oligonucleotides (ASOs) that inhibit disease-causing proteins by binding to specific RNA sequences.
Ionis has developed and commercialized several therapies for serious diseases, including SPINRAZA (nusinersen) for spinal muscular atrophy, TEGSEDI (inotersen) and QALSODY (tofersen) for hereditary transthyretin-mediated amyloidosis and ALS, respectively, WAYLIVRA (volanesorsen) for familial chylomicronemia syndrome, and WAINUA (eplontersen) for ATTRv-PN. Its pipeline targets neurology, cardiometabolic disorders, and rare diseases, with candidates like bepirovirsen (licensed to GSK for chronic hepatitis B) in late-stage development.
With over 1,400 employees, Ionis advances antisense technology alongside RNA interference and gene editing approaches through strategic partnerships with Biogen, GSK, AstraZeneca, Novartis, and Roche.
Also known as Isis Pharmaceuticals.
Interim analysis shows significant, clinically meaningful 24‑hour UPCR reduction at 37 weeks; blinded study continues to the eGFR endpoint at week 105.
Phase III interim data show significant proteinuria reduction at 37 weeks; drug targets liver-derived factor B via monthly subcutaneous dosing; blinded follow-up continues to week 105 eGFR.
Japan approves the first functional cure for chronic hepatitis B: 6‑month ASO therapy yields 19% functional cure in patients with HBsAg ≤3000 IU/ml; approval fast‑tracked by SENKU.
Pooled Phase III trials: 26% functional cure in patients with ≤1000 IU/mL HBsAg; 49% reached qHBsAg ≤100 IU/mL at 1 year; acceptable safety; regulatory reviews and anticipated Q3 2026 decisions.
Potential first-in-class CHB treatment under priority review in China; deal provides access to 5,000+ medical centres at launch, local import/distribution, hospital access and initial 5.5-yr supply.
Phase III B-Well trials showed significantly higher functional cure rates and acceptable safety in chronic hepatitis B; drug is an antisense oligonucleotide; PDUFA date 26 Oct 2026
China NMPA accepted NDA for bepirovirsen after Phase III B-Well trials showed significant functional cure rates in treated CHB patients; granted Breakthrough Therapy; acceptable safety.
EMA accepted bepirovirsen MAA for chronic hepatitis B after Phase III B‑Well 1/2 showed higher functional cure rates vs standard of care; safety profile acceptable.
