🧬 This Week in Immunology: robotic ESD milestone, pediatric AD access, in vivo CAR T, Treg Nobel, and more
From the bioartificial liver survival benefit, LEO Pharma’s spesolimab acquisition, and early biologic impact in polyarticular JIA—this episode’s signals are robust. We span liver disease, dermatology, rheumatology, pediatrics, and more, with regulatory approvals and clinical advances across Immunology, underscoring pipeline expansion and translational innovation.
In Today’s Newsletter
🧫 Bioartificial liver shows preclinical survival benefit [1] [02 Oct 2025]
Context: Japan-led team with Cincinnati Children’s reports UTOpiA device using gene-edited liver organoids in rodent ACLF; 2-hour extracorporeal treatment.
Key point: UTOpiA-treated rodents had markedly higher short-term survival vs controls in ACLF model (endpoint specifics as reported; rodent model).
Implication: Could inform practice and payer discussions; interpretation depends on study design and confounding control.
🧪 Onco3R completes first cohort for SIK3 inhibitor O3R-5671, presents at UEGW [2] [EU • 06 Oct 2025]
https://uk.finance.yahoo.com/news/onco3r-therapeutics-announces-completion-first-070000132.html
Context: Phase 1, cohort 1, 5 mg dose in 6 subjects; PK and safety supported dose escalation; late-breaker in Berlin.
Key point: No clinically significant safety findings reported in cohort 1; cohort 2 enrolling at higher dose.
Implication: May influence prescriber choice and payer reviews pending full data.
🔬 Merck broadens tulisokibart program to HS, r-axSpA, RA Phase 2b trials [3] [US • 06 Oct 2025]
https://finance.yahoo.com/news/merck-expands-tulisokibart-clinical-development-104500324.html
Context: TL1A mAb MK-7240 adds three Phase 2b studies; ongoing Phase 3 in UC and CD, plus Phase 2 in SSc-ILD.
Key point: >640 patients targeted across new trials, expanding beyond IBD into dermatology and rheumatology.
Implication: Signals pipeline investment and modality expansion.
🧴 LEO Pharma closes spesolimab (Spevigo) acquisition for GPP [4] [03 Oct 2025]
https://www.dermatologytimes.com/view/leo-pharma-finalizes-acquisition-of-spesolimab-for-gpp
Context: Spesolimab, IL-36R blocker, approved for GPP flares in adults and ≥12 yrs in several regions; further dermatoses under study.
Key point: Transaction closed after regulatory approvals; near-term modest revenue impact, EBITDA margin headwind expected.
Implication: Signals pipeline investment and modality expansion.
🤧 EVO756 shows activity in refractory chronic inducible urticaria (Phase 2) [5] [02 Oct 2025]
https://www.dermatologytimes.com/view/phase-2-trial-highlights-efficacy-of-novel-urticaria-therapy
Context: Interview report on Phase 2 in refractory CIndU; Fric test and itch NRS referenced; duration not stated.
Key point: Up to 30% complete responses and ~70% with some improvement reported; safety favorable per source.
Implication: May influence prescriber choice and payer reviews pending full data.
🧒 FDA OKs roflumilast 0.05% for AD in children 2–5 years [6] [US • 06 Oct 2025]
https://www.dermatologytimes.com/view/fda-expands-roflumilast-access-to-children-as-young-as-age-2
Context: Approval supported by INTEGUMENT-PED and OLE; steroid-free, once-daily PDE4 inhibitor; formulation avoids common sensitizers.
Key point: Provides new pediatric AD option with rapid improvement reported in trials (percentages as cited by source).
Implication: May expand screening, initiation, and follow-up at scale.
🧫 FDA advises against immediate BLA for IO Biotech’s cylembio in melanoma [7] [US • 02 Oct 2025]
Context: Pre-BLA feedback following Phase 3 IOB-013/KN-D18 where PFS improvement narrowly missed statistical significance.
Key point: Company to discuss new registrational design; workforce reduction to extend runway; EU path under consideration.
Implication: May influence prescriber choice and payer reviews pending full data.
🏅 Nobel for regulatory T-cell tolerance work (Brunkow, Ramsdell, Sakaguchi) [8] [EU • 07 Oct 2025]
Context: Recognition for FOXP3 and Treg biology underpinning immune tolerance; >200 human Treg trials ongoing per source.
Key point: Award highlights translational path toward autoimmune and oncology applications leveraging Tregs.
Implication: Could inform practice and payer discussions; interpretation depends on study design and confounding control.
🧒 Early biologic use improves polyarticular JIA outcomes in CARRA STOP-JIA [9] [03 Oct 2025]
Context: Observational comparative effectiveness using CARRA CTPs over 3 years, ~300 children.
Key point: Early combination (biologic + MTX) associated with more inactive disease time vs step-up; very early start (≤2 months) favored.
Implication: Could inform practice and payer discussions; interpretation depends on study design and confounding control.
💉 Celltrion launches AVTOZMA (tocilizumab-anoh) IV in the US [10] [US • 03 Oct 2025]
https://www.celltrion.com/en-us/company/media-center/press-release/4191
Context: FDA-approved Jan 2025; IV biosimilar to Actemra for RA, GCA, PJIA, SJIA, COVID-19 and CRS; multiple vial sizes.
Key point: US commercial availability announced, with support programs and portfolio expansion to IL-6 pathway.
Implication: Introduces competition that may affect pricing and formulary access.
Why it matters
- TL1A competition is heating up as big pharma pushes beyond IBD into derm-rheum, potentially reshaping standards in HS and axSpA.
- Pediatric access is expanding on two fronts, steroid-sparing topical PDE4 in AD and IL-6 biosimilar availability across JIA subtypes.
- Early, aggressive treatment strategies in pediatric rheum gain real-world support that could influence guidelines.
- Novel organoid-based devices signal a parallel track to cell-free regenerative support for acute organ failure.
- The Treg Nobel underscores a durable pivot toward tolerance-engineering approaches across autoimmunity and oncology.
📢 Stay Ahead in Immunology Research!
✅ Contact LucidQuest at info@lqventures.com for strategic guidance on Immunology innovation and breakthrough therapies.
FAQ
What is UTOpiA and how far along is it?
A bioartificial liver using gene-edited organoids for extracorporeal blood filtration, shown to extend short-term survival in rodent ACLF; human use will require further development in Japan per authors. [1]
Which diseases is Merck’s tulisokibart now targeting?
Beyond Phase 3 UC and CD, Merck initiated Phase 2b trials in hidradenitis suppurativa, radiographic axial spondyloarthritis and rheumatoid arthritis, plus an ongoing Phase 2 in SSc-ILD. [3]
What did Onco3R report from its first-in-human SIK3 program?
Completion of Phase 1 cohort 1 at 5 mg in 6 subjects with no clinically significant safety findings, and dose escalation underway; preclinical efficacy shown in IBD and psoriasis models. [2]
What does LEO Pharma’s spesolimab deal change for GPP care?
LEO now leads global commercialization and further development of the IL-36R antibody approved for GPP flares in multiple regions, aiming to expand access and indications. [4]
What exactly did the FDA approve for roflumilast?
Roflumilast cream 0.05% for mild-to-moderate atopic dermatitis in children 2–5 years, once daily, steroid-free, supported by INTEGUMENT studies with favorable tolerability. [6]
Why was IO Biotech advised against a BLA for cylembio?
Phase 3 showed a PFS trend but narrowly missed statistical significance; FDA recommended against filing now. The company plans further discussions and new registrational design. [7]
Entities / Keywords
UTOpia bioartificial liver; organoids; ACLF • Onco3R Therapeutics; O3R-5671; SIK3 inhibitor • Merck; tulisokibart; MK-7240; TL1A • LEO Pharma; spesolimab; Spevigo; IL-36R • Evommune; EVO756; CIndU • Arcutis; roflumilast; Zoryve; PDE4; pediatric AD • IO Biotech; cylembio; imsapepimut; etimupepimut; melanoma • Nobel Prize; FOXP3; regulatory T cells; tolerance • CARRA; STOP-JIA; early biologic; MTX • Celltrion; AVTOZMA; tocilizumab-anoh; IL-6; biosimilar.
📢 Stay Ahead in Immunology Updates!
✅ Contact LucidQuest at info@lqventures.com for strategic guidance on immunology innovations and clinical research.
FAQ
What is UTOpiA and how far along is it?
A bioartificial liver using gene-edited organoids for extracorporeal blood filtration, shown to extend short-term survival in rodent ACLF; human use will require further development in Japan per authors. [1]
Which diseases is Merck’s tulisokibart now targeting?
Beyond Phase 3 UC and CD, Merck initiated Phase 2b trials in hidradenitis suppurativa, radiographic axial spondyloarthritis and rheumatoid arthritis, plus an ongoing Phase 2 in SSc-ILD. [3]
What did Onco3R report from its first-in-human SIK3 program?
Completion of Phase 1 cohort 1 at 5 mg in 6 subjects with no clinically significant safety findings, and dose escalation underway; preclinical efficacy shown in IBD and psoriasis models. [2]
What does LEO Pharma’s spesolimab deal change for GPP care?
LEO now leads global commercialization and further development of the IL-36R antibody approved for GPP flares in multiple regions, aiming to expand access and indications. [4]
What exactly did the FDA approve for roflumilast?
Roflumilast cream 0.05% for mild-to-moderate atopic dermatitis in children 2–5 years, once daily, steroid-free, supported by INTEGUMENT studies with favorable tolerability. [6]
Why was IO Biotech advised against a BLA for cylembio?
Phase 3 showed a PFS trend but narrowly missed statistical significance; FDA recommended against filing now. The company plans further discussions and new registrational design. [7]
Entities / Keywords
UTOpia bioartificial liver; organoids; ACLF • Onco3R Therapeutics; O3R-5671; SIK3 inhibitor • Merck; tulisokibart; MK-7240; TL1A • LEO Pharma; spesolimab; Spevigo; IL-36R • Evommune; EVO756; CIndU • Arcutis; roflumilast; Zoryve; PDE4; pediatric AD • IO Biotech; cylembio; imsapepimut; etimupepimut; melanoma • Nobel Prize; FOXP3; regulatory T cells; tolerance • CARRA; STOP-JIA; early biologic; MTX • Celltrion; AVTOZMA; tocilizumab-anoh; IL-6; biosimilar.
References
- https://scienceblog.cincinnatichildrens.org/novel-bioartificial-liver-extends-survival-in-preclinical-models-of-advanced-liver-failure/
- https://uk.finance.yahoo.com/news/onco3r-therapeutics-announces-completion-first-070000132.html
- https://finance.yahoo.com/news/merck-expands-tulisokibart-clinical-development-104500324.html
- https://www.dermatologytimes.com/view/leo-pharma-finalizes-acquisition-of-spesolimab-for-gpp
- https://www.dermatologytimes.com/view/phase-2-trial-highlights-efficacy-of-novel-urticaria-therapy
- https://www.dermatologytimes.com/view/fda-expands-roflumilast-access-to-children-as-young-as-age-2
- https://www.dermatologytimes.com/view/fda-recommends-against-immediate-bla-filing-for-melanoma-vaccine-candidate
- https://www.reuters.com/business/healthcare-pharmaceuticals/brunkow-ramsdell-sakaguchi-win-2025-nobel-medicine-prize-2025-10-06/
- https://www.prnewswire.com/news-releases/new-results-from-long-term-study-show-early-use-of-biologics-improves-outcomes-for-children-with-polyarticular-juvenile-idiopathic-arthritis-302574794.html
- https://www.celltrion.com/en-us/company/media-center/press-release/4191