Thierry Passeron, MD, PhD, is a professor and chair of the Department of Dermatology at the Université Côte d'Azur in Nice, France.

Upadacitinib Safe, Effective Over 48 Weeks for Non-Segmental Vitiligo, With Thierry Passeron, MD, PhD

Upadacitinib is both safe and effective over 48 weeks among patients with 

, new findings suggest, with no new safety signals or major adverse cardiovascular events (MACE) identified.

These late-breaking data, the result of the Viti-Up-1 and Viti-Up-2 studies, were released during the 

2026 American Academy of Dermatology (AAD) Annual Meeting

 editorial team spoke on-site with trial investigator Thierry Passeron, MD, PhD, professor and chair of the Department of Dermatology at the Université Côte d'Azur in Nice, France.

“Unfortunately, so far, there is no systemic treatment approved for vitiligo, and there is still a high unmet need for the patients suffering from vitiligo,” Passeron explained.

Non-segmental vitiligo, an autoimmune inflammatory disease impacting the skin, is characterized by patients’ loss of melanocytes. This can lead to unpredictable skin depigmentation. Passeron stressed the lack of systemic medications approved for the condition, describing additional options as necessary.

Passeron and colleagues reported in this study on the efficacy and safety of upadacitinib, a selective once-daily Janus kinase (JAK) 1 inhibitor, 15 mg in adults and adolescents with non-segmental vitiligo. It was studied across 48 weeks in this pair of identical, pivotal phase 3 analyses.

In his interview, Passeron noted most of the patients were Fitzpatrick skin types 3 and 4, further describing those treated with upadacitinib in the study as being a severe disease population. The subjects were both adults and adolescents aged ≥ 12 years with this skin disease involving the face and body.

They all had a Facial Vitiligo Scoring Index (F-VASI) ≥ 0·5 and a Total Vitiligo Area Scoring Index (T-VASI) ≥ 5. The subjects were randomized 2:1 to be treated either with upadacitinib 15 mg or placebo once daily. There were 614 individuals involved across Viti-Up-1 (n=308) and Viti-Up-2 (n=306) in total.

“Two-thirds of the population were active, and three-fourths had T-VASI above 10…and the mean duration of the vitiligo was about 15 years,” Basseron said. “Most of them had vitiligo that had already been treated by topical treatment of phototherapy.”

In this poster at AAD, the treatment with upadacitinib was described as providing clinically meaningful repigmentation among adult and adolescent patients with non-segmental vitiligo.

 Passeron and coauthors’ safety data through week 48 were described as consistent with the overall profile for upadacitinib.

Specifically, they noted a lack of reports of adjudicated MACE, adjudicated gastrointestinal (GI) perforations, adjudicated venous thromboembolic events (VTE), lymphoma, active tuberculosis, non-melanoma skin cancer, or opportunistic infections outside of herpes zoster in either analysis.

 Among the most often reported TEAEs, examples included acne, upper respiratory infection, nasopharyngitis, and headache. There were 4 serious infections reported among patients in the Viti-Up-1 upadacitinib cohort, though none led to treatment cessation.

For any additional information on the new data from the Viti-Up-1 and 2 trials, view the full video with Passeron posted above.

Passeron is a consultant for AbbVie, Almirall, Amgen, Bristol Myers Squibb, Calypso, Galderma, Incyte Corporation, Janssen, Eli Lilly, Novartis, Pfizer, Roivant, UCB, and VYNE Therapeutics. He has received grants and/or honoraria from AbbVie, ACM Pharma, Almirall, Amgen, Astellas, Bristol Myers Squibb, Calypso, Celgene, Galderma, Genzyme/Sanofi, GlaxoSmithKline, Incyte Corporation, Janssen, LEO Pharma, Eli Lilly, Novartis, Pfizer, Roivant, Sun Pharmaceuticals, Takeda, UCB, and VYNE Therapeutics. He is the cofounder of NIKAIA Pharmaceuticals and founder of SUNLUTION, and has patents on WNT agonists or GSK3b antagonist for repigmentation of vitiligo and the use of CXCR3B blockers in vitiligo.

Passeron T, Prajapati V, Seneschal J, et al. Efficacy and Safety of Upadacitinib in Adolescents and Adults for Treatment of Non-Segmental Vitiligo: Results of Two Phase 3 Studies (Viti-Up). Presented at: 2026 American Academy of Dermatology Annual Meeting; March 27-31, 2026; Denver, CO.

Smith T. Upadacitinib Regulatory Applications Submitted for Adults, Adolescents with Vitiligo. HCPLive. February 3, 2026. Accessed March 30, 2026. 

https://www.hcplive.com/view/upadacitinib-regulatory-applications-submitted-adults-adolescents-vitiligo

Videos

Patients with non-segmental vitiligo (NSV) on upadacitinib versus placebo showed continuous improvement over 48 weeks with no plateau.

Vitiligo

Vitiligo condition center page is a comprehensive resource for clinical news and insights on the dermatologic condition. This page consists of interviews, articles, podcasts, and videos on the research, treatment and development of therapies for the affected skin of vitiligo patients.

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clinical page is a resource for content on every specialty, disease, and topic available on our medical news site.

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 conference coverage page features articles, videos, and expert-led live coverage from major medical meetings throughout the year.

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The HCPLive dermatology page is a resource for medical news and expert insights on skin disease. This page features expert-led coverage, articles, videos and research on the therapies and development of treatments for dermatologic conditions, and more.

Dominic Sisti, PhD, is an associate professor in the Department of Medical Ethics & Health Policy and the director of the Scattergood Program for the Applied Ethics of Behavioral Health Care. Sisti is also the host of 

, our monthly multimedia series on medical ethics in modern healthcare. 

Richard Miller, MD, staff psychiatrist at Elwyn Adult Behavioral Health in Cranston, Rhode Island.

Your Patient's New Therapist Is an AI. Now What?

Watch the full expert dynamic feature above.

In your clinic, you may hear patients say, “Chat told me this” or “Chat told me that.” Numerous individuals use ChatGPT, colloquially referred to as “Chat,” to get quick, convenient 

advice. This can be dangerous, depending on what the chatbot says, potentially providing patients with advice you would never, in a million years, give.

“More often, it's actually deleterious where information is cherry-picked, and the context is not always understood,” said Richard Miller, MD, a staff psychiatrist at Elwyn Adult Behavioral Health in Cranston, RI. “Patients are kind of going on an extended trip…to get the information that they're looking for…. maybe [a] particular answer. It's not always accurate.”

Despite the danger, AI can also be helpful in the psychiatry field. AI can provide a new perspective and can augment, not replace, treatment.

 Editorial Team put together a feature examining the benefits, dangers, and ethics surrounding AI use in psychiatry. Millions of Americans struggle to access mental health care, with waitlists sometimes lasting 3 to 6 months.

These individuals, desperate for mental health advice, turn to AI. As Darlene King, MD, from the University of Texas Southwest Medical Center, said, using AI as a therapist is like getting mental health advice from a friend, who can very well give bad advice.

Dominic Sisti, PhD, from Penn Medicine, discussed the ethical concerns of AI in psychiatry. Vulnerable populations may be susceptible to abusing AI and can even go as far as asking ChatGPT ways to self-harm. “How would these AI platforms [respond] to a patient who is indicating they have suicidal ideation or behavior?” he asked. “How might these platforms recognize elevated risk for self-harm or harm to others?

Sisti stressed the need for guardrails on age restrictions, redirection protocols, and HIPAA compliance standards. He also said there is a need for platforms to distinguish between an investigator asking about suicide methods versus someone in crisis.

Other than mental health advice, AI can assist in screening. In 2024, a study showed the promise of Sonde Health’s mental fitness vocal biomarker tool to 

identify depression from the sound of a voice

 Another study showed the potential of MoodCapture, a smartphone application using AI to 

detect the onset of depression based on facial cues 

More recently, a study showed AI can predict treatment response in major depressive disorder (MDD). In the 

phase 2b OLIVE trial assessing BH-200 (nelivaptan)

, a selective vasopressin V1b receptor antagonist, a proprietary genetic tool stratified patients by vasopressin-related biomarkers.

 Patients with lower peripheral but higher central vasopressin activity had a stronger antidepressant response, supporting biomarker-guided patient selection.

AI can also help with psychiatrists’ day-to-day responsibilities. For instance, Suki AI, Heidi, and DeepScribe, among others, are designed for clinician documentation.

As AI becomes more commonplace, experts recommend asking about its use non-judgmentally during routine history-taking. Although potentially dangerous, AI can be beneficial if used with a “human in the loop,” according to Sisti.

 Staff psychiatrist at Elwyn Adult Behavioral Health in Cranston, RI.

Associate professor of medical ethics & health policy, director of the scattergood program for applied ethics in behavioral health care, associate professor of psychiatry (secondary), associate professor of philosophy (secondary) at the University of Pennsylvania.

Assistant professor in the department of psychiatry at University of Texas Southwest Medical Center who specializes in general adult psychiatry, including women’s health, addiction, and trauma.

A psychiatrist and chief medical officer at HMNC Brain Health.

Andrew. How Long Is the Wait to See a Psychiatrist? What to Expect and How to Shorten It. Brain Health USA. Published September 30, 2025. 

https://brainhealthusa.com/how-long-is-the-wait-to-see-a-psychiatrist/

Derman C. A Voice Detecting Depression? Lindsey Venesky, PhD, Discusses New Data. HCPLive. March 14, 2024. Accessed on March 30, 2026. 

https://www.hcplive.com/view/a-voice-detecting-depression-lindsey-venesky-phd-discusses-new-data

Derman, C. Smartphone App with AI Detects Depression Onset from Facial Expressions. HCPLive. February 28, 2024. Accessed March 30, 2026. 

https://www.hcplive.com/view/smartphone-app-ai-detects-depression-onset-facial-expressions

Eriksson H. New Genetic Tool Shows Promise for Targeted MDD Treatment, With Hans Eriksson, PhD, MD. HCPLive. Published December 5, 2026. Accessed March 30, 2026. 

https://www.hcplive.com/view/new-genetic-tool-shows-promise-targeted-mdd-treatment-hans-eriksson-md-phd

Palm E, Manikantan A, Mahal H, Belwadi SS, Pepin ME. Assessing the quality of AI-generated clinical notes: validated evaluation of a large language model ambient scribe. 

. 2025;8:1691499. Published 2025 Oct 22. 

Mess SA, Mackey AJ, Yarowsky DE. Artificial Intelligence Scribe and Large Language Model Technology in Healthcare Documentation: Advantages, Limitations, and Recommendations. 

. 2025;13(1):e6450. Published 2025 Jan 16. 

DeepScribe. DeepScribe Solidifies Ambient AI Leadership in Oncology with New Study and Accelerated Growth. Prnewswire.com. Published September 16, 2025. Accessed March 30, 2026. 

https://www.prnewswire.com/news-releases/deepscribe-solidifies-ambient-ai-leadership-in-oncology-with-new-study-and-accelerated-growth-302557045.html

As patients increasingly turn to ChatGPT for mental health support, psychiatrists and ethicists weigh the clinical risks, ethical concerns, and what clinicians should do next.

Psychiatry

Psychiatry condition center page is a comprehensive resource for clinical news and insights on mental illnesses. This page consists of interviews, articles, podcasts, and videos on the research, treatment and development of therapies for depression, schizophrenia, bipolar disorder, anxiety, and more.

Major Depressive Disorder

Major Depressive Disorder condition center page, resources on the topics of medical news and expert insight into depression can be found. Content includes articles, interviews, videos, podcasts, and breaking news on MDD research, treatment, and drug development.

Schizophrenia

The HCPLive schizophrenia page is a resource for medical news and expert insights on the mental disorder. This page features expert-led coverage, articles, videos and research on the therapies and development of treatments for schizophrenia, and more.

Patrick Campbell is the editorial director of HCPLive. Patrick has spent years spearheading coverage surrounding cardiometabolic health and rheumatic disease for MJH Life Sciences. Before joining MJH Life Sciences in 2019, he spent time as a beat reporter and/or multimedia specialist with the Pocono Record, Star News Group, and NJ Advance Media. He is the executive producer for multiple HCPLive podcasts, including Diabetes Dialogue, Don't Miss a Beat, Kidney Compass, Medical Ethics Unpacked, The Medical Sisterhood, and Skin of Color Savvy. 


Follow him on Twitter @RealPatCampbell or reach him via email at PCampbell@mjhlifesciences.com.

Joseph Rossano, MD
Credit: American College of Cardiology

A phase 3 randomized trial found mavacamten (Camzyos) significantly reduced left ventricular outflow tract (LVOT) obstruction in adolescents with symptomatic 

obstructive hypertrophic cardiomyopathy (oHCM)

The findings from the SCOUT-HCM trial were presented as a late-breaking clinical trial at the 

American College of Cardiology's Annual Scientific Session (ACC.26)

 and, according to Bristol Myers Squibb, represent the first prospective phase 3 evidence for a cardiac myosin inhibitor in a pediatric oHCM population.

“Pediatric HCM is a rare cardiac disorder that is associated with severe, sometimes life-threatening, symptoms,” said principal investigator Joseph Rossano, MD, chief of the Division of Cardiology at Children’s Hospital of Philadelphia.

 “With no approved therapies for pediatric patients with oHCM and current recommendations for pharmacological therapy primarily extrapolated from evidence obtained from adult studies, the positive results of this trial represent a significant advance in the field of pediatric cardiology and the potential for a meaningful new therapy for adolescent patients if approved by the FDA.”

SCOUT-HCM was a double-blind, randomized, placebo-controlled phase 3 trial enrolling adolescents aged 12 to less than 18 years with NYHA class II or III oHCM across 28 sites in 9 countries. Eligible patients had a Valsalva LVOT gradient of 30 mm Hg or greater, a maximal LVOT gradient of 50 mm Hg or greater, and a left ventricular ejection fraction (LVEF) of 60% or greater. Patients with HCM phenocopies such as Noonan syndrome or Fabry disease were excluded.

Of 65 patients screened, 44 were randomized 1:1 to mavacamten or placebo for 28 weeks. The 23 patients in the mavacamten group and 21 in the placebo group had a mean age of approximately 14.7 years, 83% to 86% were on background beta-blocker therapy, and approximately half carried a pathogenic or likely pathogenic HCM mutation.

Investigators pointed out the mean baseline Valsalva LVOT gradient was 78.4 mmHg in the mavacamten group and 80.8 mmHg in the placebo group. Additionally, mavacamten was initiated at 5 mg once daily for most patients, with dose adjustment permitted based on echocardiographic assessment.

The primary endpoint was the change from baseline to week 28 in Valsalva LVOT gradient. Secondary outcomes of interest included the change from baseline to week 28 in resting and postexercise LVOT gradients, the maximal left ventricular wall thickness, and the ratio of early mitral inflow velocity to mitral annular early diastolic velocity (E/e′ ratio).

48 mmHg Reduction in Valsalva LVOT Gradient

At week 28, the least-squares mean change in Valsalva LVOT gradient was -48.5 mmHg in the mavacamten group versus -0.5 mm Hg in the placebo group, a between-group difference of -48.0 mmHg (95% CI, -67.7 to -28.3; 

Analysis of secondary outcomes of interest also favored mavacamten, including reductions in resting LVOT gradient (difference, -47.0 mm Hg; 95% CI, -62.7 to -31.4), postexercise LVOT gradient (difference, -41.7 mm Hg; 95% CI, -59.7 to -23.7), maximal LV wall thickness (difference, -1.8 mm; 95% CI, -3.4 to -0.2), and average E/e' ratio (difference, -3.4; 95% CI, -5.1 to -1.6). Investigators also called attention to exploratory analyses pointing to reductions in NT-proBNP and high-sensitivity cardiac troponin levels with mavacamten.

Overall adverse event rates were similar between groups (78% mavacamten vs 81% placebo). Serious adverse events occurred in 2 patients per arm. A single patient in the mavacamten group had 2 episodes of syncope deemed related to study drug and another had an inappropriate ICD shock considered unrelated. No patient in either group died or experienced an LVEF below 50%, and no cases of atrial fibrillation or symptomatic heart failure were reported.

Investigators highlighted several key limitations in their study to consider. These included the small sample size, restriction to ages 12 and older, a predominantly White study population, and the 28-week placebo-controlled follow-up period.

“The SCOUT-HCM results underscore the potential for [mavacamten] to become the first CMI for adolescents, reinforcing our leadership in the CMI space and our role in reshaping the scientific understanding of oHCM and how the disease is diagnosed, evaluated and potentially treated,” said Cristian Massacesi, MD, executive vice president, chief medical officer, and head of development at Bristol Myers Squibb.

 “With these meaningful safety and efficacy data, we are excited about the potential to provide a paradigm-changing treatment for adolescents and their families.”

Rossano JW, Canter C, Wolf CM, et al. Mavacamten in Adolescents with Obstructive Hypertrophic Cardiomyopathy. 

. Published online March 29, 2026. Accessed March 30, 2026. doi:

Bristol Myers Squibb. Bristol Myers Squibb Presents Positive Results from Phase 3 SCOUT-HCM Trial Demonstrating Efficacy and Safety of Camzyos (mavacamten) in Adolescents with Symptomatic Obstructive Hypertrophic Cardiomyopathy (oHCM). Bms.com. Published March 29, 2026. Accessed March 30, 2026. 

https://news.bms.com/news/corporate-financial/2026/Bristol-Myers-Squibb-Presents-Positive-Results-from-Phase-3-SCOUT-HCM-Trial-Demonstrating-Efficacy-and-Safety-of-Camzyos-mavacamten-in-Adolescents-with-Symptomatic-Obstructive-Hypertrophic-Cardiomyopathy-oHCM/default.aspx

Articles

New information from the phase 3 SCOUT-HCM trial indicate the benefits of mavacamten may extend to adolescents as well as adults. 

Cardiomyopathy

Cardiology

Cardiology condition center page is a comprehensive resource for clinical news and insights on cardiovascular and cardiometabolic diseases. This page consists of interviews, articles, podcasts, and videos on the research, treatment and development of therapies for heart disease and cardiovascular events, as well as associated diabetes, renal failure, and more...

Mavacamten Shows Benefit for Pediatric Patients in SCOUT-HCM Trial

REZOLVE-AD: Rezpegaldesleukin Shows Consistent Efficacy Across Atopic Dermatitis Severity, With Raj Chovatiya, MD, PhD

Rezpegaldesleukin (Nektar Therapeutics), novel IL-2 receptor agonist targeting regulatory T cells has demonstrated dose-dependent, statistically significant improvements in atopic dermatitis outcomes across both moderate and severe disease — with consistent responses observed regardless of baseline severity, geographic region, or asthma comorbidity status.

These findings are from the phase 2b REZOLVE-AD study (NCT06136741) presented at the 

 held in Denver, Colorado, from March 27-31, by Raj Chovatiya, MD, PhD, MSCI, FAAD, Associate Professor at Rosalind Franklin University of Medicine and Science Chicago Medical School and Founder and Director of the Center for Medical Dermatology and Immunology Research in Chicago. 

spoke with Chovatiya to learn more about REZOLVE-AD, which enrolled 393 patients with moderate-to-severe atopic dermatitis not previously treated with a JAK inhibitor or biologic. Participants were randomized 3:3:3:2 to rezpegaldesleukin 24 µg/kg every 2 weeks (n = 104), 18 µg/kg every 2 weeks (n = 106), 24 µg/kg every 4 weeks (n = 110), or placebo every 2 weeks (n = 73) over a 16-week induction period. Chovatiya described the mechanism as fundamentally distinct from existing approved therapies — rather than blocking active inflammation downstream, rezpegaldesleukin expands and activates Tregs via preferential binding to the IL-2 receptor complex, targeting the resolution phase of immune dysregulation.

On the primary endpoint of mean percent EASI reduction at week 16, the 24 µg/kg q2w arm achieved 61% improvement versus 31% for placebo (

 <.001), with the 18 µg/kg q2w and 24 µg/kg q4w arms achieving 58% (

 <.001), respectively — demonstrating clear dose-dependence. The finding that most engaged Chovatiya was the consistency across baseline disease severity: in patients with moderate disease (vIGA = 3), EASI improvements were 59%, 58%, and 54% across the three active arms versus 32% for placebo; in patients with severe disease (vIGA = 4), improvements were 63%, 58%, and 50% versus 29% — nearly identical trends suggesting baseline severity did not meaningfully modulate treatment response. EASI-75 and EASI-90 response rates showed the same pattern across both moderate and severe subgroups. A forest plot analysis of EASI-75 response for the 24 µg/kg q2w arm further confirmed consistent treatment effect across baseline EASI above and below 21, geographic region, and presence or absence of asthma comorbidity — all favoring rezpegaldesleukin over placebo.

On safety, injection site reactions occurred in 69.7% of all rezpegaldesleukin-treated patients versus 4.1% for placebo, nearly all mild-to-moderate; other TEAEs occurring at ≥5% frequency included eosinophilia (7.8% vs. 2.7%), pyrexia (6.3% vs. 2.7%), headache (6.3% vs. 4.1%), upper respiratory tract infections (5.9% vs. 5.5%), and arthralgia (5.0% vs. 1.4%), with no increased risk of conjunctivitis, oral ulcers, malignancy, or infections including oral herpes. The phase 3 ZENITH-AD program is in final planning with initiation expected in Q2 2026, with Chovatiya noting that the Treg-modulating mechanism raises the prospect of extended dosing intervals or treatment breaks — a meaningfully different proposition from the continuous cytokine inhibition model that defines the current standard of care.

“One of the exciting things about rezpeg is that it may potentially allow for... extended dosing intervals [and] potentially breaks off therapy, given that it's modulating a regulatory T cell, as opposed to inhibiting an activated T cell. So this is really what next phase of studies is going to be designed to test,” Chovatiya said.

Nektar Therapeutics also presented late breaking data on rezpegaldesleukin in alopecia areata, from the phase 2b REZOLVE-AA study (NCT06340360).

also caught up with that presenter David Rosmarin, MD, Chair of the Department of Dermatology and Associate Professor of Dermatology at Indiana University School of Medicine in Indianapolis, during the meeting. 

Chovatiya previously reported serving as an advisor, consultant, speaker, and/or investigator for AbbVie, Amgen, Apogee Therapeutics, Arcutis, Argenx, ASLAN Pharmaceuticals, Beiersdorf, Boehringer Ingelheim, Bristol Myers Squibb, Cara Therapeutics, Dermavant, Eli Lilly and Company, FIDE, Formation Bio, Galderma, Genentech, GSK, Incyte, LEO Pharma, L’Oréal, Nektar Therapeutics, Novartis, Opsidio, Pfizer Inc., Regeneron, RAPT, Sanofi, Sitryx, and UCB.

1. Silverberg JI, Bieber T, Chovatiya R, et al. Novel regulatory T-cell enhancing biologic rezpegaldesleukin: phase 2b efficacy, safety, and baseline severity–dependent treatment response in moderate-to-severe atopic dermatitis. Presented at: American Academy of Dermatology Annual Meeting; March 27–31, 2026; Denver, CO. Poster #73858

Chovatiya discussed new data showing significant EASI improvements across moderate and severe atopic dermatitis.

Atopic Dermatitis

Atopic Dermatitis condition center page, audiences can find a variety of coverage mediums for medical news and updates, including articles, video interviews, podcasts, and breaking news on dermatitis research, treatment, and drug development.

Victoria Johnson joined MJH Life Sciences in 2020 and has written for NeurologyLive, CGTLive, and is now an assistant managing editor for HCPLive and RheumatologyLive. She doesn’t have enough time for her numerous hobbies. You can reach her at vjohnson@mjhlifesciences.com.

Brepocitinib, an investigational oral therapy targeting TYK2 and JAK1 produced rapid, sustained improvements in global disease activity, skin disease, itch, and physical function in adults with dermatomyositis who had failed multiple prior treatments, prompting the United States Food and Drug Administration (US FDA) to grant the therapy Priority Review with a Prescription Drug User Fee Act (PDUFA) target action date in the third quarter of 2026.

The new data, from the phase 3 VALOR trial, were published March 28 in the 

 and simultaneously presented as a late-breaking abstract at the 

 held in Denver, Colorado, from March 27-31.

Brepocitinib 30 mg (Priovant Therapeutics) achieved a statistically significant 15.3-point greater improvement in mean Total Improvement Score at week 52 compared with placebo — meeting the primary endpoint and all nine key secondary endpoints — while enabling nearly twice as many patients to meaningfully reduce background corticosteroid use, findings that position the drug as potentially the first targeted therapy approved for the disease.¹

Dermatomyositis is a rare systemic autoimmune disease characterized by proximal muscle weakness, recalcitrant cutaneous eruptions, disabling pruritus, and risks of interstitial lung disease and malignancy. Current standard-of-care relies on systemic glucocorticoids, immunosuppressants, and intravenous immunoglobulin — agents with limited efficacy, significant toxicity, and no targeted mechanism specific to dermatomyositis pathogenesis. Most patients require multiple therapies, often achieving only partial disease control while accumulating steroid-related morbidity.¹

Brepocitinib is a first-in-class oral selective inhibitor of TYK2 and JAK1. This dual-targeting profile suppresses type I and type II interferon signaling, IL-6, IL-12, and IL-23, all cytokine pathways implicated in dermatomyositis pathogenesis.⁴ Preclinical work demonstrated that type I interferon drives myotube damage and dermal microvascular injury in dermatomyositis, both of which were attenuated by brepocitinib at clinically relevant concentrations — providing the mechanistic rationale for the VALOR trial.⁴

VALOR (NCT05437263) was a phase 3, randomized, double-blind, placebo-controlled trial enrolling 241 adults with definite or probable dermatomyositis across 90 global sites. Patients were randomized 1:1:1 to brepocitinib 30 mg once daily, brepocitinib 15 mg once daily, or placebo for 52 weeks. Stable background therapy with a single antimalarial agent, a single DMARD, or both was permitted. Patients on glucocorticoids at baseline tapered to ≤20 mg prednisone-equivalent before randomization, with further protocol-guided tapering from weeks 12 to 36.¹

The population was heavily pretreated and refractory: mean age was 50.6 years, 77.6% were women, 81.3% had moderate-to-severe disease activity at baseline, and approximately 2/3 were receiving 2 or more background agents at enrollment.¹ The primary endpoint was mean Total Improvement Score (TIS) at week 52 — a validated composite myositis index scored 0 to 100, with higher scores reflecting greater clinical improvement across six core measures of disease activity.¹

Brepocitinib 30 mg met the primary endpoint with a mean TIS of 46.5 at week 52 versus 31.2 with placebo, a least-squares mean difference of 15.3 points (95% CI, 6.7–24.0; 

 <.001).¹ The 15 mg dose achieved a mean TIS of 37.5 — a 6.3-point difference versus placebo that did not reach statistical significance.¹

Treatment effects with brepocitinib 30 mg were evident as early as week 4 and sustained at every visit through week 52. More than 2/3 of patients in the 30 mg arm achieved TIS ≥40, twice the minimum clinically important difference; more than half achieved TIS ≥40 while simultaneously reducing corticosteroid use to ≤2.5 mg/day prednisone-equivalent — a composite outcome reflecting both disease control and meaningful steroid-sparing benefit.²,³ Brepocitinib 30 mg also demonstrated statistically significant improvements on all nine key secondary endpoints, including muscle strength, CDASI-A skin disease activity, functional disability, and corticosteroid tapering, with nearly twice as many patients reducing background corticosteroids compared with placebo.¹,²,³

The AAD late-breaking presentation by Ruth Ann Vleugels, MD, MBA, MPH, of Mass General Brigham and Harvard Medical School, extended the VALOR dataset with skin-specific and patient-reported outcomes not fully captured in the 

Among patients with a baseline pruritus numeric rating scale (PP-NRS) score of ≥4, 54.0% achieved a ≥2-point improvement in itch at week 4 with brepocitinib 30 mg versus 9.5% with placebo. An 18.9% greater proportion achieved itch remission (PP-NRS ≤1) at week 4 (95% CI: 5.0–32.9), with benefits sustained through week 52.²

Skin-related quality of life, assessed by Skindex-16, showed a mean change of −12.9 in the brepocitinib 30 mg arm versus −0.9 with placebo at week 4, with continued improvements sustained across the trial.² Among the 64% of patients with moderate-to-severe skin disease at baseline — a population particularly refractory to existing therapies — brepocitinib 30 mg was associated with a 26.6% higher rate of functional skin remission by CDASI-A compared with placebo (95% CI: 7.6–45.5; 

 =.0060), and a 23.9% higher rate of Cutaneous Dermatomyositis Investigator Global Assessment "Clear" or "Almost Clear" status with ≥2-grade improvement (45.7% vs 21.8%).²

Serious infections occurred more frequently with brepocitinib 30 mg than with placebo (10% vs 1%), the most clinically meaningful safety signal in the trial. These events resolved with medical management and treatment was completed in most cases.¹ Adverse events leading to discontinuation occurred more frequently in the placebo arm, as did malignancies, cardiovascular events, and thromboembolic events — findings the 

 authors attribute to the elevated baseline disease burden and chronic immunosuppressive treatment carried by the placebo population rather than a protective drug effect.¹ The overall brepocitinib safety profile across more than 2,000 patients in the clinical development program was characterized as consistent with approved JAK and TYK2 inhibitors.³

"I anticipate that the VALOR trial results… will be practice-changing for patients with dermatomyositis," Vleugels said in a statement.

 "These findings underscore the need to move beyond the historical paradigm of suboptimal disease control and reliance on systemic corticosteroids toward a patient-centric model focused on rapid, sustained, steroid-sparing efficacy with a modern, targeted therapy."³

If approved, brepocitinib would be the first targeted therapy specifically approved for dermatomyositis, addressing a disease where the clinical trial landscape has been marked by repeated failures of immunosuppressants and biologic agents.

Vleugels RA, Paik JJ, Bauer Ventura I, et al. A phase 3 trial of brepocitinib in dermatomyositis. 

 Published March 28, 2026. doi:10.1056/NEJMoa2503531

Vleugels RA. Brepocitinib achieves rapid and sustained control of cutaneous disease activity, itch, and skin-related quality of life in dermatomyositis: results from the phase 3 VALOR trial. Late-breaking abstract 78745 presented at: 2026 American Academy of Dermatology Annual Meeting; March 27–31, 2026; Denver, CO.

Priovant Therapeutics. New England Journal of Medicine publishes positive phase 3 VALOR trial results of brepocitinib in dermatomyositis. News release. March 28, 2026. 

https://www.globenewswire.com/news-release/2026/03/28/3264202/34323/en/New-England-Journal-of-Medicine-Publishes-Positive-Phase-3-VALOR-Trial-Results-of-Brepocitinib-in-Dermatomyositis.html

Paik JJ, Vencovský J, Charles-Schoeman C, et al. Brepocitinib, a potent and selective TYK2/JAK1 inhibitor: scientific and clinical rationale for dermatomyositis. 

 2025;43(2):354–363. doi:10.55563/clinexprheumatol/eeglsa

Results from the phase 3 trial were published in NEJM and simultaneously presented as a late-breaking abstract at AAD 2026.

VALOR: Brepocitinib AAD Data Support Priority Review for Dermatomyositis

William Fearon, MD, is a professor of medicine and the director of interventional cardiology at Stanford University School of Medicine.

Fearon discusses results from the ALL-RISE trial, which demonstrated the FFRangio’s efficacy in assessing lesions for patients with coronary artery disease.

Strategic Alliance Partnership

FFR Angiography Noninferior to Pressure-Wire Strategy in CAD Analysis, With William Fearon, MD

Stay updated with the latest healthcare breakthroughs, including late breaking data from 3 conferences, in this week’s essential news roundup. 

Nephrology

The HCPLive nephrology page is a resource for medical news and expert insights on kidney disease. This page features expert-led coverage, articles, videos and research on the therapies and development of treatments in nephrology, and more.

The HCPFive: Top News for Healthcare Providers from the Week of 03/22

Results of the MOMENTUM trial suggest the prevalence of hypercortisolism in resistant hypertension could be greater than previous estimates have indicated.

Hypertension

The HCPLive hypertension page is a resource for medical news and expert insights on high blood pressure. This page features expert-led coverage, articles, videos and research on the therapies and development of treatments for hypertension, and more.

Type 2 Diabetes

MOMENTUM: Hypercortisolism Present in 1-in-4 with Resistant Hypertension

Navin Kapur, MD, is the director of the Acute Circulatory Support Program and associate director of the Cardiac Catheterization Laboratory at Tufts Medical Center.

Although not statistically significant, the reduction represents a potential new pathway for treating patients with STEMI.

Structural Heart Disease

LV Unloading Before Delayed PCI Reduces Infarct Size by 1%, With Gregg Stone, MD, and Navin Kapur, MD

In this segment of his interview, Eichenfield highlights the safety and tolerability of roflumilast cream for children with atopic dermatitis in INTEGUMENT-OLE.

Roflumilast Cream 0.05% Well Tolerated in Children with Eczema, With Lawrence Eichenfield, MD

Swapnil Hiremath, MD, MPH, is a Staff Nephrologist at the Ottawa Hospital, a Professor in the Faculty of Medicine, cross appointed to the School of Epidemiology and Public Heath at the University of Ottawa, and also an Associate Scientist in the Clinical Epidemiology Programme at the Ottawa Hospital Research Institute.

Hypertension drives millions of preventable deaths, yet control lags globally. The HEARTS framework offers practical, scalable solutions to close this gap.

Bridging the Gap in Hypertension Care Delivery, With Swapnil Hiremath, MD, MPH

The IMMpactful direct comparison provides prospective data in a moderate psoriasis population that previous MAICs could not fully capture.

Plaque Psoriasis

The HCPLive plaque psoriasis page is a resource for medical news and expert insights on psoriasis. This page features expert-led coverage, articles, videos and research on the therapies and development of treatments for psoriatic disease, and more.

Risankizumab Outperforms Deucravacitinib on Psoriasis Clearance Across Patient Subgroups in Phase 4 Head-to-Head Trial

Podcasts

Vlado Perkovic, MBBS, PhD, discusses APPLAUSE-IgAN data showing iptacopan reduced eGFR decline and lowered kidney event risk in IgA nephropathy.

IgA Nephropathy

The HCPLive Berger disease page is a resource for medical news and expert insights on IgA nephropathy. This page features expert-led coverage, articles, videos and research on the therapies and development of treatments in Berger disease, and more.

podcasts page features a feed of medical news and interview podcast series, including DocTalk, Lungcast, Derm Discussions, and Heart Trials.

Kidney Compass: Navigating Clinical Trials

Hosted by Brendon Neuen, MBBS, PhD, and Shikha Wadhwani, MD, MS, Kidney Compass: Navigating Clinical Trials is a regular podcast exploring and breaking down the latest updates and nuances of clinical trials in nephrology. By listening to Kidney Compass, you'll get the guidance you need to navigate the changing landscape of clinical trials across the spectrum of kidney disease. 

Kidney Compass: New 2-Year APPLAUSE-IgAN Data for Iptacopan at WCN 2026

Hiddo Heerspink, MD, PhD, highlights ASSIST trial data showing atrasentan reduced proteinuria by 25% on top of SGLT2 inhibitors in IgA nephropathy.

Kidney Compass: ASSIST Trial Shows Atrasentan Benefit on Top of SGLT2 Inhibitors

CAR-T therapy shows potential for deep, durable remission in lupus nephritis, but questions remain on safety, durability, and patient selection.

Latest News

Upadacitinib Safe, Effective Over 48 Weeks for Non-Segmental Vitiligo, With Thierry Passeron, MD, PhD

Your Patient's New Therapist Is an AI. Now What?

Mavacamten Shows Benefit for Pediatric Patients in SCOUT-HCM Trial

REZOLVE-AD: Rezpegaldesleukin Shows Consistent Efficacy Across Atopic Dermatitis Severity, With Raj Chovatiya, MD, PhD

VALOR: Brepocitinib AAD Data Support Priority Review for Dermatomyositis