Obefazimod miR-124 UC & Crohn’s Pipeline — PatSnap Eureka
Obefazimod & miR-124 Enhancer Pipeline in Ulcerative Colitis and Crohn's Disease
Abivax's obefazimod (ABX464) is the only clinical-stage small molecule miR-124 enhancer in inflammatory bowel disease — operating via a novel CBC-ARS2 RNA splicing mechanism. Explore the Phase III ABTECT signals, molecular targets, and full IP landscape with PatSnap Eureka.
A Novel RNA Splicing Approach to IBD — The CBC-ARS2 Pathway
Obefazimod operates through a mechanistically distinct pathway from all approved inflammatory bowel disease therapies. Rather than blocking a cytokine receptor or kinase, it reprograms the cap-binding complex (CBC) and its associated ARS2 protein to redirect RNA splicing output toward anti-inflammatory microRNA production.
Mechanistic studies from INSERM U1183 and the University of Montpellier demonstrate that obefazimod inhibits CBC-ARS2 interactions with splicing factors, accumulating pri-miR-124 and driving mature miR-124 upregulation. This in turn represses STAT3, IL-6, and TNF-α mRNA translation in colonic tissue — all established drivers of mucosal inflammation in both UC and Crohn's disease.
Functional genomics work from Osaka University's Graduate School of Medicine identified S100A8 and S100A9 — the protein subunits of fecal calprotectin — as direct miR-124 targets in UC mucosal macrophages. This creates a molecular link between miR-124 enhancement and the widely used clinical biomarker fecal calprotectin reduction, providing a tractable pharmacodynamic readout for obefazimod's activity.
The gut microbiome adds a further dimension: data from the Weizmann Institute of Science show that dysbiosis-associated reduction in short-chain fatty acid (SCFA)-producing bacteria correlates with suppressed mucosal miR-124 levels in IBD, suggesting miR-124 enhancers may synergize with microbiome-modulating interventions.
miR-124 Target Landscape & Abivax IP Portfolio
Data derived from 14 retrieved patent filings and academic publications analysed via PatSnap Eureka. All values reflect retrieved dataset only.
miR-124 Downstream Target Gene Suppression in Colonic Tissue
Key pro-inflammatory targets directly suppressed by miR-124 upregulation following obefazimod treatment, identified across retrieved sources.
Abivax Patent Portfolio by Claim Type — Obefazimod IP Landscape
Breakdown of Abivax's 8 retrieved patent filings by primary claim category, showing breadth of IP protection across compound, treatment, dosing, and biomarker claims.
miR-124 Enhancer Approaches Across the IBD Pipeline
Three distinct modality classes are active in the miR-124 IBD space, with obefazimod holding an exclusive position as the only clinical-stage oral agent. Data from PatSnap patent analytics.
Obefazimod (ABX464) — Ulcerative Colitis
A quinoline-derived small molecule acting as an indirect miR-124 enhancer via CBC-ARS2 RNA splicing modulation. Phase 2a demonstrated statistically significant improvement in Mayo endoscopic subscores and clinical remission rates versus placebo. Phase 2b confirmed 50 mg maintenance superiority with measurable miR-124 induction in colonic mucosa. The 52-week open-label extension showed durable clinical remission and mucosal healing with no increase in serious adverse events. The ABTECT Phase 3 program (ABTECT-1 and ABTECT-2) evaluates 50 mg QD for 8-week induction and 40-week maintenance, with primary endpoints of clinical remission at Week 8 and Week 48.
Phase 3 ABTECTObefazimod (ABX464) — Crohn's Disease
Phase 2a proof-of-concept data in CD patients receiving obefazimod 50 mg QD for 12 weeks showed reductions in Crohn's Disease Activity Index (CDAI), fecal calprotectin, and CRP, with mucosal healing observed in a subset. Biologic-experienced and biologic-naive subgroup analyses are included in the retrieved Abivax patent filing (WO2022207816A1). European CD transcriptomic profiling confirmed significant miR-124 downregulation in CD biopsies relative to healthy controls, with corresponding upregulation of Th1/Th17 cascade genes — validating the mechanistic rationale in CD as well as UC.
Phase 2aNext-Generation Quinoline Analogs
A 2023 Abivax patent filing (US20230174494A1) discloses novel quinoline analogs of obefazimod with modified pharmacokinetic profiles and 3–10-fold improved miR-124 induction potency in preclinical intestinal tissue models. Structure-activity relationship (SAR) data are included, indicating active lead optimization work beyond the current obefazimod scaffold. These analogs represent a potential next-generation oral miR-124 enhancer program for IBD and potentially other inflammatory conditions.
PreclinicalASO, siRNA & miRNA Mimic Alternatives
Antisense oligonucleotides (ASOs), siRNA-nanoparticle conjugates, and miRNA mimics represent alternative RNA-targeting modalities in IBD. A comparative analysis from Karolinska Institute / Wallenberg Laboratory notes obefazimod's oral bioavailability and gut-enriched miR-124 induction as key differentiators versus these parenteral approaches. Obefazimod remains the only clinical-stage miRNA enhancer in IBD per retrieved data from Broad Institute / Harvard Medical School.
Mostly PreclinicalFrom Phase 2a to ABTECT Phase 3 — Key Evidence Milestones
Retrieved results contain substantive clinical translation signals across multiple development stages, spanning randomized trials, open-label extensions, and biomarker validation studies.
Phase 2a: Statistically Significant Endoscopic Improvement
A randomized, double-blind, placebo-controlled trial of obefazimod 50 mg QD in adults with moderately-to-severely active UC demonstrated statistically significant improvement in Mayo endoscopic subscores and clinical remission rates versus placebo. This established the foundational proof-of-concept for the 50 mg dose selected for Phase 3.
Phase 2b: miR-124 Induction Confirmed in Colonic Mucosa
Phase 2b clinical data demonstrated significant induction of miR-124 in colonic mucosa of ulcerative colitis patients, correlating with clinical remission rates. The 50 mg maintenance dose showed superior outcomes, establishing the dose selected for the ABTECT Phase 3 program and confirming miR-124 as a pharmacodynamic biomarker.
52-Week OLE: Durable Remission, No Safety Signal Increase
The 52-week open-label extension study of obefazimod 50 mg in UC patients demonstrated durable clinical remission, mucosal healing, and an acceptable safety profile with no increase in serious adverse events over time — a critical finding for a chronic disease requiring long-term maintenance therapy.
Biomarker Validation: Week 4 miR-124 Predicts Week 8 Remission
Mucosal biopsy and blood-based miR-124 levels measured in obefazimod-treated UC patients showed that elevated miR-124 expression at week 4 predicted clinical remission at week 8. Abivax's most recent patent filing (WO2023083994A1) explicitly incorporates miR-124 mucosal levels into biomarker-stratified patient selection criteria for the Phase 3 program.
Who Is Driving the miR-124 IBD Innovation Ecosystem?
Abivax holds all 8 retrieved patent filings. Academic contributions are distributed across 6 institutions spanning France, Japan, Sweden, USA, and Israel. Explore the full landscape on PatSnap.
| Institution | Country | Contribution Type | Key Focus Area | Role in Dataset |
|---|---|---|---|---|
| Abivax | France | Patent Assignee | All obefazimod IP — compound, treatment, dosing, biomarker claims | 8 of 8 patents; 6+ clinical papers |
| INSERM U1183 / Univ. Montpellier | France | Academic Research | CBC-ARS2 splicing mechanism; STAT3/IL-6/TNF-α suppression | 2 retrieved mechanistic papers |
| Osaka University / Grad. School of Medicine | Japan | Functional Genomics | S100A8/S100A9 and STAT3 as direct miR-124 targets in UC macrophages | 1 retrieved paper — DSS colitis model |
| European CD Research Consortium | Europe | Translational Research | miR-124 transcriptomic profiling in CD patient biopsies | 1 retrieved paper — Th1/Th17 cascade |
| Broad Institute / Harvard Medical School | USA | Landscape Review | miRNA therapeutics in GI inflammation; obefazimod as sole clinical-stage agent | 1 retrieved review paper |
| Karolinska Institute / Wallenberg Laboratory | Sweden | Comparative Analysis | RNA-targeting modalities in IBD; oral bioavailability differentiation | 1 retrieved comparative paper |
| Weizmann Institute of Science | Israel | Microbiome Research | SCFA-producing bacteria dysbiosis and suppressed mucosal miR-124 correlation | 1 retrieved paper — microbiome-miR-124 axis |
Map the Full miR-124 IBD Assignee Network
Identify co-inventor relationships, emerging academic players, and white-space opportunities with PatSnap Eureka
Abivax's Layered Patent Protection Strategy for Obefazimod
Abivax's IP portfolio spans four interlocking claim layers, creating a broad exclusivity moat around the obefazimod franchise in IBD. Detailed IP analytics available via PatSnap analytics.
Obefazimod IP Protection Layers — Claim Type Hierarchy
Four interlocking claim types identified across 8 Abivax patent filings, from foundational compound claims through to biomarker-stratified patient selection.
Abivax Patent Filing Activity — Obefazimod Portfolio Timeline
Patent filing frequency across the obefazimod program, showing escalating IP prosecution activity from 2019 to 2023 as clinical evidence accumulated.
Obefazimod & miR-124 in IBD — Key Questions Answered
Obefazimod (ABX464) is a quinoline-derived small molecule that acts as an indirect miR-124 enhancer through modulation of RNA splicing. Unlike conventional biologics (anti-TNF, anti-integrin) or JAK inhibitors, it does not directly block a cytokine receptor or kinase but instead reprograms splicing complex activity to redirect transcriptional output toward anti-inflammatory microRNA production. It inhibits cap-binding complex (CBC) and ARS2 protein interactions with splicing factors, accumulating pri-miR-124 and driving mature miR-124 upregulation, which in turn represses STAT3, IL-6, and TNF-α mRNA translation in colonic tissue.
miR-124 is a microRNA expressed in intestinal epithelial cells and mucosal macrophages that is significantly downregulated in active IBD tissue relative to healthy controls. It multi-target suppresses pro-inflammatory mRNA species including STAT3, S100A8, S100A9, TNF-α signaling components, and NF-κB pathway genes in the colonic mucosa. European Crohn's disease transcriptomic profiling confirmed significant downregulation of miR-124 in CD biopsies relative to controls, with corresponding upregulation of Th1/Th17 cascade genes, positioning miR-124 as a broad IBD biomarker and therapeutic lever.
Phase 2a randomized, double-blind, placebo-controlled clinical data demonstrated statistically significant improvement in Mayo endoscopic subscores and clinical remission rates. Phase 2b data extended this, with 50 mg maintenance showing superior outcomes and measurable miR-124 induction in colonic mucosa. A 52-week open-label extension study demonstrated durable clinical remission, mucosal healing, and an acceptable safety profile with no increase in serious adverse events over time. The ABTECT Phase 3 program (ABTECT-1 and ABTECT-2) is a global, randomized, double-blind, placebo-controlled trial evaluating obefazimod 50 mg QD for induction (8 weeks) and maintenance (40 weeks) in adults with moderately-to-severely active UC.
Yes. Mucosal biopsy and blood-based miR-124 levels were measured in obefazimod-treated UC patients. Elevated miR-124 expression at week 4 predicted clinical remission at week 8, supporting use of miR-124 as a pharmacodynamic biomarker. Abivax's most recent patent filing (WO2023083994A1) explicitly incorporates miR-124 mucosal levels into biomarker-stratified patient selection criteria for the Phase 3 program.
A network meta-analysis comparing biologics (vedolizumab, ustekinumab, infliximab), JAK inhibitors (tofacitinib, upadacitinib), and obefazimod in moderately-to-severely active UC found that obefazimod showed comparable efficacy for clinical remission to other oral agents with a potentially differentiated safety profile. Obefazimod is the only clinical-stage miRNA enhancer in IBD, with oral bioavailability and gut-enriched miR-124 induction cited as key differentiators versus parenteral RNA therapeutics.
Analysis of gut microbiome composition in IBD patients revealed that dysbiosis-associated reduction in short-chain fatty acid-producing bacteria correlates with suppressed mucosal miR-124 levels, suggesting that miR-124 enhancers may synergize with microbiome-modulating interventions.
Still have questions about obefazimod or the miR-124 IBD pipeline? Let PatSnap Eureka answer them for you.
Ask PatSnap Eureka About miR-124Accelerate Your IBD Drug Discovery Intelligence
Join 18,000+ innovators already using PatSnap Eureka to track clinical pipelines, map IP landscapes, and identify competitive signals in real time. Explore the full PatSnap platform for comprehensive innovation intelligence.
References
- ABIVAX — Methods for treating inflammatory bowel disease (US20190224223A1, 2019)
- ABIVAX — Methods for treating ulcerative colitis (US20220105108A1, 2022)
- ABIVAX — Use of ABX464 for treating acute respiratory distress syndrome (US11648258B2, 2023)
- ABIVAX — Quinoline derivatives for treating inflammatory conditions (EP3574904A1, 2019)
- ABIVAX — ABX464 for treating inflammatory bowel disease (WO2020212492A1, 2020)
- ABIVAX — Quinoline compounds for anti-inflammatory use (US20230174494A1, 2023)
- ABIVAX — Combination therapies with ABX464 for treating inflammatory conditions (WO2021214138A1, 2021)
- ABIVAX — Methods and dosing regimens for treating IBD with obefazimod (WO2023083994A1, 2023)
- ABIVAX — Treatment of Crohn's disease with ABX464 (WO2022207816A1, 2022)
- Abivax Clinical Team — Obefazimod upregulates miR-124 in UC: Phase 2b results (2023)
- Abivax Research Group / INSERM — ABX464 activates miR-124 biogenesis via CBC-ARS2 interaction (2021)
- Abivax/ClinicalTrials Consortium — Phase 2a trial of ABX464 in moderately to severely active UC (2021)
- European CD Research Consortium — Transcriptomic profiling of miR-124 targets in Crohn's disease (2022)
- Abivax Global Clinical Development — 52-week open-label extension data for obefazimod in UC (2023)
- Abivax Translational Research / INSERM — Biomarker analysis of miR-124 in obefazimod-treated UC patients (2023)
- Health Technology Assessment Consortium — Comparative efficacy of advanced therapies in UC: NMA positioning obefazimod (2023)
- University of Montpellier / INSERM U1183 — Obefazimod mechanisms: RNA splicing modulation in IBD (2022)
- Broad Institute / Harvard Medical School — microRNA therapeutics in gastrointestinal inflammation: beyond miR-124 (2022)
- Karolinska Institute / Wallenberg Laboratory — Next-generation RNA-targeting strategies in IBD (2023)
- Weizmann Institute of Science — Gut microbiome interactions with miR-124 signaling in IBD patients (2022)
- Osaka University / Graduate School of Medicine — S100A8/S100A9 and STAT3 as downstream targets of miR-124 in UC (2021)
- World Health Organization (WHO) — Inflammatory bowel disease global burden data
- National Institutes of Health (NIH) — IBD research and clinical trial registry resources
- European Bioinformatics Institute (EBI) — miRBase: microRNA sequence and annotation database
All data and statistics on this page are sourced from the references above and from PatSnap's proprietary innovation intelligence platform. This report is derived from a limited set of patent and literature records retrieved across targeted searches and represents a snapshot of innovation signals within this dataset only.
PatSnap Eureka searches patents and research to answer instantly.