Drugs / IONIS-FB-LRx
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IONIS-FB-LRx

also known as RO7434656 · sefaxersen

Antisense oligonucleotide targets CFB via inhibition

Developed for
diffuse secondary choroid atrophy · IgA glomerulonephritis · age-related macular degeneration · macular degeneration
Investigated by
Ionis Pharmaceuticals, Inc. · Hoffmann-La Roche

Trials 4

PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 23 trials · 1 met primary
Phase 2 NCT04014335 Dec 2019 → Feb 2024 IgA glomerulonephritis Ionis Pharmaceuticals, Inc. Completed Met primary
Phase 2 NCT03815825 Mar 2019 → Apr 2024 diffuse secondary choroid atrophy, macular degeneration Ionis Pharmaceuticals, Inc. Completed No outcome recorded
Phase 2 NCT03446144 Mar → Oct 2018 age-related macular degeneration, diffuse secondary choroid atrophy Ionis Pharmaceuticals, Inc. Withdrawn No outcome recorded Stop: Business
Phase 31 trial
Phase 3 NCT05797610 Aug 2023 → Aug 2026 overdue IgA glomerulonephritis Hoffmann-La Roche Recruiting No outcome recorded

News releases announcing trial results or a regulatory action · 1

DateIssuerRelease
2022-11-07 Ionis Pharmaceuticals Results Ionis presents positive Phase 2 data in patients with IgA Nephropathy at American Society of Nephrology's Kidney Week 2022 ionis.com ↗
Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today presented positive results from a Phase 2 clinical study of IONIS-FB-LRx in patients with immunoglobulin A nephropathy (IgAN).

Evidence & citations 7 cited values

Every value below carries the sentence it was read from. 5 sources stand behind the page.

FieldValueCited text
Known as IONIS-FB-Lrx ClinicalTrials.gov intervention name — accepted as the source's own label NCT03446144 ↗
2

NCT03815825 ↗

NCT04014335 ↗

Known as RO7434656 “Patients received sefaxersen 70 mg (RO7434656) subcutaneously monthly for 24 weeks followed by voluntary treatment extension.” PMID 41443406 ↗ Dec 2025
1

“Sefaxersen (RO7434656) will be administered as SC injection per schedule as specified.” NCT05797610 ↗

Known as sefaxersen “Patients received sefaxersen 70 mg (RO7434656) subcutaneously monthly for 24 weeks followed by voluntary treatment extension.” PMID 41443406 ↗ Dec 2025
1

NCT05797610 ↗

Action Inhibit “an Antisense Inhibitor of Complement Factor B” NCT03446144 ↗
Modality Antisense oligonucleotide “an Antisense Inhibitor of Complement Factor B” NCT03446144 ↗
Route Subcutaneous “administered subcutaneously every 4 weeks” NCT03815825 ↗
Target CFB “complement factor B messenger ribonucleic acid (CFB mRNA)” NCT04014335 ↗