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Oct 15Thu
  1. N Engl J Med77

    Phase 2 Trial of Icotrokinra for Patients with Ulcerative Colitis.

    Once-daily oral icotrokinra, a peptide that blocks the interleukin-23 receptor, beat placebo for clinical response at week 12 in adults with moderately to severely active ulcerative colitis: 63% at 400 mg, 58% at 200 mg and 55% at 100 mg versus 27% on placebo (P<0.001 for all). The 400-mg group also showed more clinical remission (30% vs. 11%), symptomatic remission (46% vs. 19%) and endoscopic improvement (37% vs. 14%). It offers an oral alternative to injected interleukin-23-targeted antibodies.

    Why it matters: An oral interleukin-23 receptor blocker matched injected antibodies' mechanism with positive phase 2 results.

  2. nature.com83

    Engineered TYK2 dependence enables myeloid-selective IL-10 receptor agonism

    IL-10's therapeutic use is limited because its myeloid-suppressive and T-cell-activating effects cannot be separated. Using the JAK1 inhibitor VVD-118313 in bone-marrow-derived macrophages, the authors show that JAK1-JAK1 transphosphorylation only partially reduces IL-10-induced STAT3 phosphorylation, with TYK2-mediated transphosphorylation compensating; in TYK2-deficient BMDMs, VVD-118313 fully abrogated STAT3 phosphorylation and IL-10 function. In human monocytes, the TYK2 inhibitor zasocitinib similarly partially reduced IL-10-driven STAT3 phosphorylation, revealing differential TYK2 dependence that may enable myeloid-selective IL-10 receptor agonism.

    Why it matters: It maps a signalling route that could separate IL-10's anti-inflammatory benefit from its toxic T-cell activation.

Oct 14Wed
  1. Nat Biotechnol82

    In vivo engineering of CAR natural killer cells for immunotherapy using ligand-directed lipid nanoparticles.

    Ligand-directed lipid nanoparticles (NtLNPs) carrying mRNA transfected up to ~80% of NK cells in vivo in mice with minimal off-target effects, generating CAR NK cells without ex vivo engineering. CD19-targeted CAR NKs completely depleted circulating B cells in healthy mice, CD3-targeted CAR NKs eliminated nearly all autoreactive T cells in autoimmune hepatitis, and NKG2A-targeting versions improved solid tumor efficacy. Human-optimized NtLNPs produced CAR NK cells that regressed two humanized hematologic malignancy models. The work replaces viral, ex vivo CAR manufacturing with a modular in vivo mRNA delivery platform.

    Why it matters: It shows nonviral in vivo CAR NK generation, a route around ex vivo viral engineering of cell therapies.

Oct 13Tue
  1. biopharmadive.com40

    Bausch + Lomb swaps CEOs; Merck reveals anticipated eye drug data

    Bausch + Lomb named board chair Thomas Appio interim CEO after he and Brent Saunders mutually agreed to Saunders' exit, and raised its 2026 earnings outlook. Merck disclosed that proliferative diabetic retinopathy adverse events occurred in 6.7% and 6.1% of remigromig patients versus 0.9% on Lucentis, with the drug not superior on secondary measures. Amgen and AstraZeneca reported nearly 70% of eosinophilic esophagitis patients on Tezspire's highest dose were in remission at 24 weeks; the FDA also approved Asahi Kasei's Hovilpri.

    Why it matters: CEO change at Bausch + Lomb plus pivotal safety and efficacy data on remigromig, Tezspire and a new HSV approval.

Oct 10Sat
  1. doi.org49

    JAK-inhibitor treatment induces rapid yet incomplete immune reconstitution in STAT1 gain-of-function

    JAK inhibitors produced rapid but incomplete immune reconstitution in two STAT1 gain-of-function patients carrying p.R321S and p.T385M mutations. Changes were seen as early as 1 week after treatment: T cells rebalanced toward a homeostatic status, TCR repertoire diversity increased quickly, and transcriptional dysregulation in monocytes improved, though defects such as natural killer deficiency persisted in one patient. The work defines both the therapeutic reach and the limits of JAK inhibition in STAT1 GOF.

Oct 9Fri
  1. Sci Adv57

    Kupffer cells are key mediators of antigen-specific immune tolerance by peptide-coupled red blood cells.

    Red blood cells coupled with multiple sclerosis immunodominant peptides prevented experimental autoimmune encephalomyelitis when given prophylactically and reduced symptoms when given therapeutically, with tolerance dependent on antigen, cell dose and peptide dose. Kupffer cells were the main phagocytes of the peptide-coupled RBCs, took them up without complement component 3, SIRPα, or Stabilin 1 and 2, and were indispensable for tolerance, showing PD-L1 up-regulation and a tolerogenic transcriptional shift. The work positions Kupffer cells as the central mediators of this tolerogenic strategy for autoimmune disease.

  2. biospace.com80

    How Abogen went from COVID hype to a $7.8B Novartis deal

    Novartis has taken an exclusive worldwide license to ABO2203, Abogen Biosciences' mRNA-encoded CD19xCD3 T cell engager for B-cell malignancies and autoimmune diseases, paying $575 million upfront with up to roughly $7.2 billion in potential option, milestone and royalty payments. Early data in relapsed or refractory B-cell non-Hodgkin lymphoma showed overall response rates of 33%, 67% and 100% across dose cohorts, with no CRS or ICANS reported. The deal, which also gives Novartis options on further Abogen RNA platform programs, marks Abogen's shift beyond its faded COVID vaccine business.

    Why it matters: Large upfront payment signals big-pharma validation of in vivo mRNA T cell engagers and Abogen's post-COVID pivot.

  3. biopharmadive.com61

    TRex Bio prices $117M IPO to support immune drug work

    TRex Bio priced a $117 million IPO, selling 8,333,334 shares at $14 each, and begins trading Friday on Nasdaq under "TRXB." The company is developing Treg-activating drugs for immunological conditions, including TRB-061, in a Phase 1 trial with topline data expected in 2027, and TRB-071 for inflammatory bowel disease, which could enter testing next year. Eli Lilly, TRex's largest equity holder at about 24% pre-IPO, and Johnson & Johnson have licensed or optioned treatments discovered by TRex, which has raised $220 million in venture financing to date.

    Why it matters: IPO shows continued investor appetite for immune-focused biotechs, with six of 23 drug developers going public in 2026 working on immune diseases.

Oct 8Thu
  1. biospace.com66

    Big Pharma–backed Matchpoint scores $150M series B, advancing autoimmune asset

    Preclinical Matchpoint Therapeutics has raised a $150 million series B co-led by Nextech Invest and Norwest, with participation from Sanofi Ventures, Atlas Venture, Access Biotechnology and the T1D Fund. The money will advance MPT-062, an oral T cell modulator for autoimmune and inflammatory conditions, into human trials starting next year. Matchpoint also has a July 2025 deal with Novartis, which paid $60 million upfront for its ACE platform and could owe up to $1 billion in milestones plus royalties.

    Why it matters: A large series B funds a first-in-class oral autoimmune program before it reaches human trials, against a market trend favouring later-stage assets.

Oct 7Wed
  1. Mol Cell57

    DNASE1L3 maintains homeostatic levels of mitochondrial DNA on the mammalian cell surface.

    Mitochondrial DNA is tethered to the surface of select mammalian cells through cell surface proteins and heparan sulfate proteoglycans, accumulating in large clusters that partially overlap with RNA binding protein domains. The secreted nuclease DNASE1L3 clears this surface DNA from human and murine B cells. Patients carrying a DNASE1L3 missense variant linked to autoimmune disease risk show increased surface DNA on B and T cells, connecting cell-surface molecules to DNA targeting in autoimmunity.

  2. fda.gov40

    Rare Disease Drug Approvals

    FDA's CDER lists recent rare disease drug approvals on its Accelerating Rare Disease Cures Program page. In 2026 alone these include Camzyos for an inherited heart condition in children, Emcitate for MCT8 deficiency, Isembyld for muscle loss in spinal muscular atrophy, and Zanvastro for Alexander Disease, among others. The list spans 2021 to 2026 and links to the underlying FDA news releases.

  3. nature.com40

    Next-generation immune cell engagers for B cell depletion in autoimmune diseases

    Nature Reviews Drug Discovery has published a review of immune cell engagers as a strategy for deep B cell depletion in autoimmune diseases. It covers their mechanisms, clinical development and next-generation designs, including T cell, natural killer cell and gamma-delta T cell-engaging formats. It also draws lessons from haematological malignancies to improve the efficacy, safety and durability of immune modulation.

  4. medicalxpress.com55

    International consensus sets updated framework for CAR T-cell therapy in severe autoimmune diseases

    A group of 31 international experts has published updated consensus recommendations in The Lancet Rheumatology on using CAR T-cell therapy for severe, treatment-refractory autoimmune diseases. The 2026 recommendations, developed by EBMT, ISCT and JACIE with input from EULAR and ECTRIMS, update the first best-practice guidance from 2024 and cover patient selection, treatment in experienced FACT/JACIE-accredited centers, and reporting of patient data to the EBMT Registry. The panel calls for treatment within clinical trials where possible and lists larger controlled trials, biomarkers and novel CAR targets as research priorities.

    Why it matters: New international guidance shapes how clinicians select and manage CAR T-cell patients in autoimmune disease, a fast-growing off-cancer use.

Oct 6Tue
  1. N Engl J Med84

    Obexelimab for the Treatment of IgG4-Related Disease.

    Weekly subcutaneous obexelimab, 250 mg for 52 weeks, cut the risk of IgG4-related disease flare requiring rescue therapy by more than half in a phase 3 trial of 194 patients (hazard ratio 0.44; flares in 26.8% vs. 54.6% with placebo). Complete remission at week 52 was reached by 37.1% versus 19.6%, and cumulative glucocorticoid rescue dose was 329.5 mg versus 929.8 mg. The results position this non-depleting anti-CD19/FcγRIIb antibody as an alternative to glucocorticoid-dependent management.

    Why it matters: Phase 3 evidence that a non-depleting B-cell-targeted antibody reduces flares and steroid exposure in IgG4-related disease.

Oct 5Mon
  1. biospace.com62

    CSL strikes up to $1.6B deal with Alentis for autoimmune drug with broad potential

    CSL will pay Alentis Therapeutics $355 million upfront, with up to $1.2 billion more, to co-develop and co-promote the claudin-1-targeting antibody lixudebart. The drug is in the Phase 2 RENAL trial for AAV-RPGN, a rare autoimmune kidney disease, and is also planned for mid-stage development in FSGS and PSC, with CSL funding these studies and any approved product profits split 55/45 in CSL's favour. An FDA orphan drug designation for IPF, granted in May 2024, sits outside the deal.

Oct 3Sat
  1. biocentury.com67

    Novartis turns to mRNA-encoded engagers for B cell depletion

    Novartis is paying Suzhou Abogen Biosciences $575 million up front for exclusive worldwide rights to ABO2203, an mRNA-encoded CD19 x CD3 T cell engager for autoimmune indications, plus options on undisclosed next-generation assets from Abogen's RNA platform. Abogen could receive up to $7.2 billion in milestones if all options are exercised. Early clinical data support the mRNA-encoded T cell engager approach, which adds a new modality to B cell-depleting therapies for autoimmune diseases.

Oct 2Fri
  1. Nature79

    Population-scale immune multiome atlas reveals regulatory disease mechanisms.

    Simultaneous single-nucleus profiling of chromatin accessibility and gene expression in 10 million peripheral blood mononuclear cells from 1,108 Finnish individuals yields 51,083 cis-expression QTLs, 338,100 cis-chromatin accessibility QTLs and 593,765 peak-gene links. Variants completing chromatin-to-expression cascades show twice the disease colocalization of chromatin-only effects, and massively parallel reporter assays validate 10,428 fine-mapped QTLs. The atlas supplies testable hypotheses for over half of immune disease associations and explains why disease variants preferentially target evolutionarily constrained genes through regulatory buffering at the chromatin-to-expression interface.

    Why it matters: It converts non-coding immune disease variants into mechanistic chromatin-to-expression cascades for most immune disease associations.

  2. Nature73

    A two-hit mechanism triggers autoimmune hair loss.

    Autoimmune alopecia can be triggered by a two-hit mechanism: transient regulatory T cell depletion during the anagen phase of the hair cycle causes CD8+ T-cell-mediated destruction of hair follicle stem cells and hair loss, while telogen skin is resistant. Anagen susceptibility depends on sympathetic nerve activity, which drives noradrenaline-ADRB2 signalling, reactivation of endogenous retroviruses, and AIM2-dependent innate immune activation. Genetic ablation of Aim2 or pharmacological inhibition of ERV reverse transcription protects against disease; stress or optogenetic sympathetic activation renders telogen skin susceptible.

    Why it matters: It defines a mechanistic trigger for tissue-specific autoimmunity, linking neural activity, endogenous retroviruses and Treg cells, with druggable entry points.

  3. biorxiv.org45

    Genetic risk and inflammatory signaling converge on cell type-specific regulatory programs in type 1 diabetes

    A subset of type 1 diabetes-associated variants may drive beta-cell dysfunction by disrupting transcription factor binding at cell type-specific candidate cis-regulatory elements. The authors integrated epigenomic and single-cell multiomic data from human pancreatic islets with T1D GWAS variants, and used the deep learning model ChromBPNet plus TF footprinting to nominate several disease-associated SNPs predicted to alter chromatin accessibility at candidate TF binding sites. The work links noncoding variants to cell type-specific enhancer activity and provides candidates for future mechanistic studies.

  4. biopharmadive.com65

    Sanofi, Regeneron broaden alliance to find their next Dupixent

    Sanofi and Regeneron have expanded their alliance to co-develop and co-commercialize four long-acting, Regeneron-invented antibodies targeting IL-13, IL-4 or an IL-4 subunit, like Dupixent. The most advanced, REGN20423, is in an early-stage eczema trial; the other three should enter human testing next year. Regeneron receives $1 billion up front and as much as $7 billion in milestone payments, and can opt to add Sanofi's lunsekimig after certain late-stage studies. Dupixent, first cleared in 2017, brought in $10.9 billion in net sales in the first half of this year.

    Why it matters: Expanded Sanofi-Regeneron alliance with $1 billion up front and up to $7 billion in milestones aims to extend the Dupixent franchise.

Oct 1Thu
  2. statnews.com60

    STAT+: Sanofi, Regeneron expand partnership after Dupixent success

    Sanofi and Regeneron Pharmaceuticals, partners on Dupixent, have expanded their collaboration to develop a new series of antibodies for immunological diseases. Sanofi will pay Regeneron $1 billion upfront, with possible milestone payments of up to $7 billion more. Regeneron will lead research and development, Sanofi will lead commercialization, and profits will be split evenly.

  3. nature.com68

    A two-hit mechanism triggers autoimmune hair loss

    The paper reports a two-hit mechanism that triggers autoimmune hair loss, drawing on single-cell RNA-seq and bulk RNA-seq datasets generated in the study. The sequencing data have been deposited in the NCBI Gene Expression Omnibus under accession codes GSE343853 and GSE343613.

    Why it matters: It proposes a defined two-hit mechanism for autoimmune hair loss with underlying transcriptomic data now public.

  4. biopharmadive.com50

    FDA starts review of Roche’s multiple sclerosis pill

    The FDA has accepted Roche's approval application for fenebrutinib, an experimental multiple sclerosis pill, under a priority review with a six-month timeline, covering both relapsed and primary progressive forms of the disease. The application rests on three late-stage studies that met their main objectives, but approval chances could be complicated by an imbalance in deaths, eight among fenebrutinib recipients versus one on Aubagio in two Phase 3 trials, and a case of liver enzyme elevations meeting Hy's law. Roche did not say when a decision might come.

    Why it matters: Fenebrutinib could become the first BTK inhibitor approved for two major forms of MS despite unresolved safety concerns.

Sep 28Mon
  1. doi.org41

    Brief report: Effectiveness and safety of recombinant zoster vaccine in inflammatory bowel disease patients aged 18 years and older: A matched cohort and self-controlled case series study

    Two doses of recombinant zoster vaccine prevented herpes zoster in inflammatory bowel disease patients, with adjusted vaccine effectiveness of 65.4% (95% CI 40.6%-79.9%) overall, 62.7% in ulcerative colitis and 70.2% in Crohn's disease. In a self-controlled case series, the IBD flare rate ratio after vaccination was 0.80 (95% CI 0.58-1.12), indicating no increased flare risk. The results support RZV use in this higher-risk population.

Sep 25Fri
  1. nature.com56

    Single-cell analysis of chromatin accessibility in the human intestine identifies regulatory programs and clarifies genetic associations in Crohn’s disease

    Single-cell ATAC-seq was used to map chromatin accessibility in intestinal mucosa from patients with Crohn's disease and healthy controls. The work characterizes regulatory programs associated with inflammation and genetic effects, clarifying genetic associations in Crohn's disease.

    Why it matters: Links Crohn's disease genetic associations to specific cell-type regulatory programs, aiding interpretation of risk variants.

Sep 13Sun
  1. N Engl J Med63

    Obinutuzumab or Tacrolimus in Primary Membranous Nephropathy.

    Obinutuzumab outperformed tacrolimus for inducing complete remission in primary membranous nephropathy in a phase 3 trial of 142 adults. At week 104, complete remission occurred in 26 of 71 patients given obinutuzumab versus 4 of 70 given tacrolimus (37% vs. 6%; adjusted difference, 31 percentage points; P<0.001). Grade 3 or higher adverse events were similar, at 22% and 19%. The finding positions obinutuzumab as an effective alternative to tacrolimus in this disease.

    Why it matters: Phase 3 data show obinutuzumab superior to tacrolimus for complete remission in primary membranous nephropathy.

Sep 10Thu
  1. Sci Transl Med57

    Monoclonal antibodies inhibiting MMP-9 activity alleviate peripheral neuropathy in diabetic mice.

    Systemically administered monoclonal antibodies that selectively inhibit MMP-9 relieved mechanical and cold pain and restored hind paw wound healing in diabetic mice. The antibodies reduced epidermal nerve fiber degeneration by improving nerve mitochondrial function and promoting skin angiogenesis. Human relevance is supported by increased MMP-9 expression in dorsal root ganglia of patients with diabetes and by associations of MMP9 variants with neuropathic pain phenotypes.

    Why it matters: It identifies a specific druggable target with antibody therapy for a diabetic complication lacking disease-modifying treatments.

  2. nature.com51

    A bispecific CD3×CD19 antibody for systemic lupus erythematosus: a phase 1 trial

    A next-generation CD3×CD19 bispecific T cell engager given intravenously was well tolerated in patients with active systemic lupus erythematosus in a phase 1 trial, and most patients showed improvements in disease activity scores. The findings support further testing of B cell-directed T cell engagers in systemic lupus erythematosus.

    Why it matters: First-in-class tolerability and disease activity signal for a CD3×CD19 bispecific antibody in active SLE.

  3. Nat Med53

    Antifungal therapy improves microbiome dynamics in inflammatory bowel disease.

    Fluconazole, but not nystatin, reduced intestinal Candida burden and reshaped gut fungal-community composition in 53 patients with mild-to-moderate ulcerative colitis or Crohn's disease. The gastrointestinal and oral fluconazole fungal targeting arm (n = 35) showed increased bacterial diversity, expansion of short-chain fatty-acid-producing taxa, restored anti-inflammatory metabolites, and improved disease activity indices with decreased risk of progression over 8 weeks. The work establishes a framework for antifungal cotherapy in fungal-associated IBD.

    Why it matters: It provides first evidence that targeted antifungal therapy can reshape the gut microbiome and improve disease activity in IBD.

Sep 8Tue
  1. Mol Cell64

    Selective decay of interferon mRNAs controls antiviral immunity and innate memory.

    The RNA-binding protein tristetraprolin (TTP) and the mRNA decay machinery selectively limit type I and type III interferon (IFN) mRNA levels at steady state and after stimulation. Loss of this pathway increases IFN mRNA poly(A) tail length, stability and translation, raising IFN expression enough to block SARS-CoV-2 infection in vitro and in vivo. A human TTP variant linked to autoimmunity shows reduced TTP levels and increased IFN signaling, and transient loss of mRNA decay primes innate memory for long-term antiviral protection.

    Why it matters: It identifies TTP-mediated IFN mRNA decay as a tunable switch controlling antiviral immunity and autoimmune risk.

  2. Nature64

    Intracellular complement factor H protects neurons during CNS inflammation.

    Intracellular complement factor H (CFH) protects neurons from inflammatory injury through a non-canonical role independent of complement C3. In single-nucleus RNA-sequencing of retinal ganglion cells from donors with multiple sclerosis and controls, CFH expression correlated strongly with resilience to degeneration. CFH was induced by inflammatory and oxidative stress, localized to the endoplasmic reticulum, limited reactive oxygen species accumulation and lipid peroxidation, and its protection depended on the C-terminal SCR20 domain in mice and humans.

    Why it matters: Identifies a complement-independent, intracellular neuroprotective mechanism relevant to MS therapy.

Sep 5Sat
  1. Nat Med61

    Inhaled siRNA therapy targeting RAGE for pulmonary inflammation: a first-in-human randomized trial.

    Inhaled ARO-RAGE, a lung-epithelium-targeted siRNA against pulmonary RAGE mRNA, was safe and well tolerated in a first-in-human randomized trial in 58 healthy volunteers and 19 patients with asthma. Plasma levels were low, consistent with drug retention in the lung, and prolonged dose-responsive decreases in serum and bronchoalveolar lavage sRAGE indicated effective pulmonary target engagement. The results support continued clinical development of inhaled therapeutic siRNA for pulmonary inflammation.

    Why it matters: First-in-human data show an inhaled siRNA can safely engage a lung target with minimal systemic exposure.

Sep 1Tue
  1. gov.uk78

    MHRA concludes review of Avacopan Vifor following reassessment of benefit-risk balance

    The MHRA will suspend the use, supply and sale of Avacopan Vifor (previously Tavneos) to new patients from 1 September 2026, after concluding that data integrity problems in the pivotal trial mean the study can no longer demonstrate efficacy. The drug, authorised on 6 May 2022 with rituximab or cyclophosphamide for severe, active GPA or MPA in adults, will be supplied only to existing patients during a six-month managed withdrawal, with the UK marketing authorisation to be revoked on 1 March 2027. Clinicians should not start new patients on the medicine and should review current patients for alternative treatments.

    Why it matters: UK regulator suspends and plans to revoke a vasculitis drug's authorisation over unreliable pivotal trial data.

  3. biocentury.com55

    Lilly’s deal spree continues with Merida takeout: Deals Report

    Eli Lilly has agreed to acquire Merida Biosciences, a four-year-old Cambridge, Massachusetts autoimmunity and allergy biotech, in a deal valued at up to $2.88 billion. The acquisition gives Lilly MER511, a Phase I Fc therapeutic that binds anti-TSHR autoantibodies for Graves' disease and thyroid eye disease. Merida raised a $121 million series A in 2025 co-led by Bain Capital Life Sciences, BVF Partners and Third Rock Ventures.

    Why it matters: Lilly's thirteenth takeout expands its autoimmune pipeline into Graves' disease and thyroid eye disease.

  4. Lancet60

    Rezpegaldesleukin treatment of moderate-to-severe atopic dermatitis (REZOLVE-AD): final results from the 16-week induction period of an international, double-blind, placebo-controlled, randomised phase 2b study.

    Rezpegaldesleukin, an interleukin-2 receptor agonist that expands regulatory T cells, met its primary endpoint in adults with moderate-to-severe atopic dermatitis naive to biological treatment. At week 16, mean percentage change in EASI was -61% for 24 μg/kg every 2 weeks, -58% for 18 μg/kg every 2 weeks and -53% for 24 μg/kg every 4 weeks, versus -31% for placebo, with treatment differences of -30, -27 and -22 percentage points respectively (p<0·0001, p<0·0001, p=0·0002). Injection-site reactions occurred in 70% of rezpegaldesleukin-treated patients but were nearly all mild to moderate and resolved, with no deaths reported.

    Why it matters: Phase 2b data support a Treg-enhancing mechanism as a new biological approach in moderate-to-severe atopic dermatitis.

Aug 29Sat
  1. Cell70

    Genome-scale perturb-seq in primary human CD4+ T cells maps context-specific regulators of T cell programs and human immune traits.

    A genome-scale perturb-seq platform perturbed all expressed genes across 22 million primary human CD4+ T cells from four donors, measuring transcriptome effects at rest and after stimulation. The resulting maps identify regulators of immune pathways, including previously uncharacterized regulators of cytokine production, and show that active regulators and the programs they control change dramatically across stimulation conditions. The signatures nominate regulators of T cell polarization, age-related phenotypes, and context-specific pathways in autoimmune disease risk.

    Why it matters: It maps context-specific gene regulators in primary human CD4+ T cells and links them to immune traits and autoimmune disease risk.

Aug 28Fri
  1. Lancet70

    Efficacy and safety of upadacitinib in adults and adolescents for treatment of non-segmental vitiligo (Viti-Up): results of two phase 3 randomised controlled studies.

    Upadacitinib 15 mg once daily produced clinically meaningful repigmentation in non-segmental vitiligo, a disease with no approved systemic therapies. In two phase 3 placebo-controlled trials, T-VASI 50 at week 48 was reached by 19% and 21% of patients on upadacitinib versus 6% on placebo in each study, and F-VASI 75 by 25% and 23% versus 6% and 7%; both coprimary endpoints met p<0·001. Safety was consistent with the known upadacitinib profile, with no new signals.

    Why it matters: First phase 3 data supporting a systemic therapy for non-segmental vitiligo, an approved-therapy gap.

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