IONS
Ionis Pharmaceuticals develops RNA-targeted medicines to treat serious genetic and neurological diseases. The company has transitioned to a fully integrated commercial-stage biotech with seven marketed medicines, including SPINRAZA for spinal muscular atrophy, WAINUA for hereditary transthyretin amyloidosis, QALSODY and TEGSEDI for ALS, and DAWNZERA for hereditary angioedema, among others. Most of these medicines are already approved and being commercialized in the U.S. and international markets, either independently or through partnerships with larger pharmaceutical companies.
Pipeline
Donidalorsen is an ASO‑GalNAc conjugate that causes ribonuclease H1 (RNase H1)‑mediated degradation of PKK mRNA through binding to PKK mRNA, which results in reduced production of PKK protein.
Eplontersen is an antisense oligonucleotide-GalNAc conjugate that causes degradation of mutant and wild-type TTR mRNA through binding to the TTR mRNA, which results in a reduction of serum TTR protein and TTR protein deposits in tissues.
Olezarsen is an ASO-GalNAc 3 conjugate that binds to apoC-III mRNA leading to mRNA degradation and resulting in a reduction of serum apoC-III protein. Reduction of apoC-III protein leads to increased clearance of plasma TG and VLDL.
Olezarsen is an ASO-GalNAc 3 conjugate that binds to apoC-III mRNA leading to mRNA degradation and resulting in a reduction of serum apoC-III protein. Reduction of apoC-III protein leads to increased clearance of plasma TG and VLDL.
Catalyst Calendar
“GSK is developing bepirovirsen, including conducting the ongoing B-Well Phase 3 program in patients with HBV, which achieved full enrollment in June 2024 and for which we anticipate data in 2026.”
“We are currently conducting a Phase 3 study of ulefnersen in juvenile and adult patients with FUS-ALS for which we anticipate data in 2026.”
“In July 2023, we completed enrollment of the Phase 3 CARDIO-TTRansform study of WAINUA in patients with ATTR-CM with data expected in 2026.”
“Zilganersen is currently under Priority Review by the U.S. Food and Drug Administration with a Prescription Drug User Fee Act action date of September 22, 2026.”
“Biogen is evaluating QALSODY as a potential treatment for presymptomatic SOD1-ALS patients in the ongoing ATLAS study for which we anticipate data in 2028.”
“In June 2024, the FDA accepted our NDA for patients with FCS for Priority Review with a Prescription Drug User Fee Act, or PDUFA, date of December 19, 2024.”
“Novartis is developing pelacarsen, including conducting the ongoing Lp(a) HORIZON Phase 3 cardiovascular outcome study in patients with elevated Lp(a)-driven cardiovascular disease, which achieved full enrollment in July 2022 with more than 8,000 patients and for which we anticipate data in 2025.”
“In July 2024, we completed enrollment in the Phase 3 portion of the ongoing study for patients with AxD. We anticipate data from this study in 2025.”
“we are currently conducting a broad Phase 3 development program for olezarsen for the treatment of sHTG, including three Phase 3 studies supporting development (CORE, CORE2 and ESSENCE), which achieved full enrollment in 2024 and for which we expect data in 2025.”
“We plan to submit a supplemental new drug application (sNDA) to the U.S. Food and Drug Administration by end of year.”
“These data were presented during a late-breaking session at the American Heart Association Scientific Sessions, taking place November 7-10 in New Orleans, and simultaneously published in The New England Journal of Medicine.”
“We plan to submit a new drug application to the U.S. Food and Drug Administration in Q1 2026”
“These findings, which will be presented today at the 2026 American Academy of Neurology annual meeting, build on previously reported positive topline data and provide a more comprehensive view of treatment effect across multiple clinically meaningful domains.”