Roivant and Priovant Announce Positive Phase 3 VALOR Study Results for Brepocitinib in 52-Week Placebo-Controlled Trial in Dermatomyositis (DM)
Roivant (Nasdaq: ROIV) and Priovant Therapeutics announced breakthrough results from their Phase 3 VALOR study of brepocitinib in treating dermatomyositis (DM). The once-daily oral 30mg dose demonstrated significant superiority over placebo, achieving a week 52 mean Total Improvement Score (TIS) of 46.5 versus 31.2 (p=0.0006).
Key highlights include: 62% of brepocitinib patients reduced steroid use to ≤2.5 mg/day (vs 34% placebo), over two-thirds achieved moderate response (TIS≥40), and nearly half showed major response (TIS≥60). The drug demonstrated rapid onset, with significant improvements visible by week 4. Among patients with moderate-to-severe skin disease, 44% achieved cutaneous clinical remission by week 52 (vs 21% placebo).
The safety profile aligned with previous trials, and Priovant plans an NDA filing in the first half of 2026.
Roivant (Nasdaq: ROIV) e Priovant Therapeutics hanno annunciato risultati rivoluzionari dal loro studio di fase 3 VALOR su brepocitinib per il trattamento della dermatomiosite (DM). la dose orale una volta al giorno di 30 mg ha dimostrato una chiara superiorità rispetto al placebo, ottenendo una week 52 mean Total Improvement Score (TIS) di 46,5 contro 31,2 (p=0,0006).
Principali evidenze: 62% dei pazienti trattati con brepocitinib hanno ridotto l’uso di steroidi a ≤2,5 mg/giorno (rispetto al 34% del placebo), oltre due terzi hanno raggiunto una risposta moderata (TIS≥40) e quasi la metà una grande risposta (TIS≥60). Il farmaco ha mostrato un inizio rapido, con miglioramenti significativi visibili già dalla settimana 4. Tra i pazienti con malattia cutanea moderata-severa, il 44% ha raggiunto la remissione cutanea entro la settimana 52 (contro il 21% placebo).
Il profilo di sicurezza si è allineato a quello degli studi precedenti e Priovant prevede una presentazione NDA nella prima metà del 2026.
Roivant (Nasdaq: ROIV) y Priovant Therapeutics anunciaron resultados innovadores de su estudio de fase 3 VALOR con brepocitinib para el tratamiento de la dermatomiositis (DM). La dosis oral de 30 mg una vez al día demostró una superioridad significativa frente al placebo, logrando una puntuación total de mejora (TIS) media en la semana 52 de 46,5 frente a 31,2 (p=0,0006).
Entre los aspectos clave: 62% de los pacientes con brepocitinib redujeron el uso de esteroides a ≤2,5 mg/día (vs 34% placebo), más de dos tercios lograron una respuesta moderada (TIS≥40) y casi la mitad mostró una respuesta mayor (TIS≥60). El fármaco mostró un inicio rápido, con mejoras significativas visibles desde la semana 4. Entre los pacientes con enfermedad cutánea moderada a severa, el 44% alcanzó la remisión clínica cutánea en la semana 52 (vs 21% placebo).
El perfil de seguridad se mantuvo acorde con ensayos previos y Priovant planea presentar una NDA en la primera mitad de 2026.
Roivant (Nasdaq: ROIV)와 Priovant Therapeutics는 피부근육염(DM) 치료를 위한 3상 VALOR 연구에서 brepocitinib의 획기적인 결과를 발표했습니다. 하루 한 번 복용하는 30mg 용량이 위약보다 현저한 우수성을 보였으며, 52주 평균 총 개선 지수(TIS)는 46.5 대 31.2로 나타났습니다(p=0.0006).
주요 하이라이트로는 62%의 brepocitinib 환자가 스테로이드 사용을 1일 2.5mg 이하로 감소시키는 반면 위약은 34%였고, 3분의 2 이상이 중등도 반응(TIS≥40)을 달성했으며 거의 절반이 대규모 반응(TIS≥60)을 보였습니다. 약물은 빠른 발현을 보였고 4주차부터 유의한 개선이 관찰되었습니다. 중등도에서 중중도 피부 질환이 있는 환자 중 44%가 52주 차 피부 임상적 관해를 달성했습니다(위약 21%와 비교).
안전성 프로필은 이전 임상과 일치했으며 Priovant는 2026년 상반기에 NDA를 제출할 계획입니다.
Roivant (Nasdaq: ROIV) et Priovant Therapeutics annoncent des résultats révolutionnaires de leur étude de phase 3 VALOR sur le brepocitinib pour le traitement de la dermatomyosite (DM). La dose orale de 30 mg une fois par jour a démontré une supériorité significative par rapport au placebo, avec une score moyen de l’amélioration totale (TIS) à la semaine 52 de 46,5 contre 31,2 (p=0,0006).
Points clés : 62% des patients sous brepocitinib ont réduit l’usage des corticoïdes à ≤2,5 mg/jour (contre 34% sous placebo), plus des deux tiers ont obtenu une réponse modérée (TIS≥40) et près de la moitié une grande réponse (TIS≥60). Le médicament a montré un démarrage rapide, avec des améliorations significatives visibles dès la semaine 4. Parmi les patients présentant une maladie cutanée modérée à sévère, 44% ont atteint une rémission cutanée clinique à la semaine 52 (contre 21% placebo).
Le profil de sécurité est conforme aux essais précédents et Priovant prévoit de déposer une NDA dans la première moitié de 2026.
Roivant (Nasdaq: ROIV) und Priovant Therapeutics gaben bahnbrechende Ergebnisse ihrer Phase-3-VALOR-Studie mit brepocitinib zur Behandlung der Dermatomyositis (DM) bekannt. Die einmal täglich eingenommene 30 mg-Dosis zeigte eine signifikante Überlegenheit gegenüber Placebo und erreichte eine Woche-52-Mittelwert-TIS von 46,5 vs. 31,2 (p=0,0006).
Wichtige Highlights: 62% der Brepocitinib-Patienten reduzierten die Steroidnutzung auf ≤2,5 mg/Tag (vs 34% Placebo), über zwei Drittel erreichten eine moderate Antwort (TIS≥40) und fast die Hälfte zeigte eine große Reaktion (TIS≥60). Das Medikament zeigte einen schnellen Wirkeintritt, mit signifikanten Verbesserungen bereits in Woche 4. Bei Patienten mit mäßiger bis schwerer Hauterkrankung erreichten 44% in Woche 52 eine kutane klinische Remission (vs 21% Placebo).
Das Sicherheitsprofil entsprach dem früherer Studien, und Priovant plant die Einreichung einer NDA in der ersten Hälfte von 2026.
رويفانت (ناسداك: ROIV) و Priovant Therapeutics أعلنتا عن نتائج رائدة من تجربتهما من المرحلة 3 VALOR لدواء brepocitinib في علاج التهاب العضلات والجلد (DM). الجرعة الفموية اليومية 30 ملغ أظهرت تفوقاً كبيراً على الدواء الوهمي، محققة متوسط درجة التحسن الإجمالي في الأسبوع 52 (TIS) 46.5 مقابل 31.2 (p=0.0006).
النقاط الرئيسية تشمل: 62% من مرضى Brepocitinib خفَّضوا استخدام الستيرويدات إلى ≤2.5 ملغ/اليوم (مقابل 34% في الدواء الوهمي)، وأكثر من ثلاثة أرباع حققوا استجابة متوسطة (TIS≥40)، وبحوالي نصفهم أظهروا استجابة كبيرة (TIS≥60). أظهر الدواء بداية سريعة، مع تحسنات ملحوظة منذ الأسبوع 4. بين المرضى الذين يعانون من جلد متوسط إلى شديد، وصل 44% إلى remission جلدي سريري في الأسبوع 52 (مقابل 21% دواء وهمي).
ملف السلامة كان متسقاً مع التجارب السابقة وتخطط Priovant لتقديم NDA في النصف الأول من عام 2026.
Roivant (纳斯达克：ROIV) 与 Priovant Therapeutics 公布了其 Phase 3 VALOR 研究中 brepocitinib 用于治疗皮肌炎（DM）的突破性结果。每日一次口服 30 mg 的剂量显示出显著优于安慰剂，达到 第52周总改善分数（TIS）均值 46.5 对 31.2（p=0.0006）。
要点包括：62% 的 brepocitinib 患者将类固醇使用降至 ≤2.5 mg/日（对照组 34%），超过三分之二实现了中度反应（TIS≥40），近一半显示出重大反应（TIS≥60）。药物显示出快速起效，在第 4 周便可观察到显著改善。在中度至重度皮肤病变患者中，44% 在第 52 周达到皮肤临床缓解（对照组 21%）。
安全性特征与之前的试验一致，Priovant 计划在 2026 年上半年提交 NDA。
- First ever positive 52-week placebo-controlled trial in dermatomyositis
- Statistically significant improvement on primary endpoint and all nine key secondary endpoints
- 62% of patients achieved reduced steroid use (≤2.5 mg/day) compared to 34% for placebo
- 44% of patients with moderate-to-severe skin disease achieved clinical remission
- Rapid onset of action with significant improvements visible by week 4
- Safety profile consistent with previous trials, with no increased AESI frequency versus placebo
- NDA filing not planned until first half of 2026, indicating a lengthy timeline to potential commercialization
Insights
Roivant's brepocitinib showed remarkable efficacy in dermatomyositis with significant improvements across all endpoints and strong steroid-sparing effects.
The Phase 3 VALOR study results for brepocitinib represent a significant clinical breakthrough in dermatomyositis (DM) treatment. The once-daily oral 30mg dose achieved a week 52 mean Total Improvement Score (TIS) of 46.5 versus 31.2 for placebo (p=0.0006), demonstrating robust statistical significance. What makes these results particularly impressive is the consistent efficacy across all nine secondary endpoints, covering skin disease, muscle strength, and patient-reported outcomes.
The strong steroid-sparing effect is especially noteworthy - 42% of patients on brepocitinib 30mg eliminated steroid use entirely (versus 23% for placebo), and 62% reduced to minimal doses (≤2.5mg/day). This steroid reduction typically makes showing efficacy more challenging, yet brepocitinib still demonstrated superiority, highlighting its potent disease-modifying potential.
The rapid onset of action (statistically significant separation from placebo at week 4) combined with sustained efficacy through 52 weeks addresses a critical unmet need in DM. The clear dose-response relationship between the 15mg and 30mg arms provides strong validation of the selected optimal dose.
The clean safety profile, with adverse events of special interest occurring at rates similar to placebo, is particularly encouraging for a TYK2/JAK1 inhibitor. This positions brepocitinib favorably against other JAK inhibitors that carry black box warnings.
As the first successful 52-week placebo-controlled trial and first positive registrational trial for a targeted therapy in DM, these results potentially establish brepocitinib as a first-in-class therapy for this indication, with NDA submission planned for 1H 2026.
These brepocitinib results represent a landmark achievement in autoimmune disease treatment. Dermatomyositis has been notoriously difficult to treat effectively, with patients suffering from both debilitating skin manifestations and muscle weakness. The dual TYK2/JAK1 inhibition mechanism of brepocitinib addresses the complex immunopathology driving both cutaneous and muscular symptoms.
The data shows impressive response rates across multiple measures: nearly half of patients achieved major responses (TIS≥60), and 44% of patients with moderate-to-severe skin disease reached cutaneous clinical remission versus 21% on placebo. These outcomes suggest brepocitinib effectively interrupts the inflammatory cascade driven by type I interferons and other cytokine pathways central to dermatomyositis pathogenesis.
Particularly compelling is the improvement in objective muscle strength (MMT-8) combined with better functional outcomes (HAQ-Disability Index), indicating the therapy addresses both the molecular pathways and the practical disability aspects affecting patients' daily lives.
The steroid-sparing effect cannot be overstated from an immunological perspective. Corticosteroids, while effective, cause significant immunosuppression and metabolic complications with long-term use. Brepocitinib's ability to allow 42% of patients to discontinue steroids entirely while maintaining disease control represents a significant advance in treatment paradigms.
The safety profile appears favorable, with no increased incidence of typical JAK inhibitor-associated adverse events like thromboembolic events or malignancies compared to placebo, suggesting brepocitinib's selectivity may offer improved risk-benefit over less selective JAK inhibitors.
- Once-daily oral brepocitinib 30 mg demonstrated clinically meaningful and statistically significant improvement compared to placebo on the primary endpoint and all nine key secondary endpoints, including measurements of skin disease, muscle disease, steroid-sparing effect, and rapidity of onset
- On the primary endpoint, brepocitinib 30 mg achieved a week 52 mean Total Improvement Score (TIS) of 46.5 compared to 31.2 for placebo (p=0.0006), even with nearly twice as many patients coming off background steroids on brepocitinib 30 mg compared to placebo
- More than two thirds of brepocitinib 30 mg patients experienced at least a moderate response (TIS≥40), and nearly half experienced a major response (TIS≥60)
- Consistent dose response was seen between 30 mg and 15 mg dose arms, establishing 30 mg dose as optimal in this setting
- Safety profile was consistent with previous clinical trials of brepocitinib and NDA filing is planned for calendar 1H 2026
- Roivant will host an investor call to discuss these updates today, September 17, 2025, at 8:00 a.m. EDT
BASEL, Switzerland and LONDON and NEW YORK and DURHAM, N.C., Sept. 17, 2025 (GLOBE NEWSWIRE) -- Roivant (Nasdaq: ROIV) and Priovant Therapeutics today announced positive results from the Phase 3 VALOR study evaluating brepocitinib in dermatomyositis (DM).
On the primary endpoint, brepocitinib 30 mg achieved a week 52 mean TIS of 46.5 compared to 31.2 for placebo (p=0.0006). A statistically significant difference between brepocitinib 30 mg and placebo on mean TIS was seen at all time points, including as early as week 4. This result represents the first ever positive outcome for a 52-week placebo-controlled trial in DM, and the first ever positive registrational trial for a targeted therapy in DM.
“Dermatomyositis is an incredibly debilitating autoimmune disease for patients, and we urgently need novel, approved efficacious therapies,” said Dr. Ruth Ann Vleugels, M.D., M.P.H., M.B.A., Heidi and Scott C. Schuster Distinguished Chair in Dermatology, Founding Director of the Autoimmune Skin Disease Center and Connective Tissue Disease Clinics at Brigham and Women's Hospital and Program Director for the Dermatology-Rheumatology Fellowship at Harvard Medical School. “The VALOR study’s success represents a groundbreaking moment for the dermatomyositis field, and the results reinforce brepocitinib’s potential to serve as a deeply impactful treatment option for a substantial number of dermatomyositis patients once approved.”
Brepocitinib also demonstrated clinically meaningful and statistically significant improvement over placebo on all nine key secondary endpoints. Dose-dependent response between brepocitinib 30 mg and brepocitinib 15 mg was seen consistently across the primary and secondary endpoints.
Approximately
Even against this backdrop, clinical improvement in the brepocitinib 30 mg arm was rapid, deep, lasting and broad, both in absolute terms and relative to placebo:
- Brepocitinib demonstrated clinically meaningful and statistically significant improvement relative to placebo on the CDASI (skin), MMT-8 (motor strength) and HAQ-Disability Index (patient questionnaire around daily living activities requiring functional muscle strength, like getting dressed or walking up five steps).
- More than two-thirds of brepocitinib 30 mg patients experienced at least a moderate response (TIS≥40), and nearly half experienced a major response (TIS≥60); of patients who entered the trial with moderate-to-severe skin disease,
44% on brepocitinib 30 mg achieved cutaneous clinical remission by week 52, compared to21% on placebo. - Both TIS and CDASI achieved statistically significant separation from placebo as early as week 4 and sustained that separation at every assessment out to one year; median time to a TIS≥40 response was approximately 8 weeks.
The observed brepocitinib 30 mg safety profile was consistent with previous brepocitinib clinical trials. Adverse events of special interest (AESIs), which included malignancy, cardiovascular events, and thromboembolic events, did not occur with greater frequency in the brepocitinib 30 mg arm than the placebo arm.
“We are thrilled with the results of the VALOR study, and I would like to thank all of the patients, investigators, and study site staff who contributed to this important research achievement,” said Ben Zimmer, Priovant CEO. “We are excited to continue working towards the rapid approval of brepocitinib in dermatomyositis and our broader goal of developing brepocitinib as a transformational therapy for multiple highly morbid autoimmune diseases where the need for novel efficacious therapies is greatest.”
“The VALOR study is a hallmark example of what Roivant does best: identify a high value program with a differentiated mechanism of action, focus on creative development plans in indications like DM with high unmet need, and deliver on clinical execution excellence,” said Mayukh Sukhatme, Roivant President and Chief Investment Officer. “I am extremely proud of Roivant’s continued track record with VALOR as the 12th consecutive positive Phase 3 study for the company – an unparalleled achievement and a testament to Roivant’s unrelenting focus on clinical strategy and execution.”
Priovant plans to file an NDA for brepocitinib in dermatomyositis in the first half of 2026.
About Dermatomyositis
Dermatomyositis (DM) is a multi-organ idiopathic inflammatory condition that affects approximately 50,000 adults in the United States. DM is characterized by debilitating muscle weakness and skin lesions. DM-related muscle weakness causes significant impairment to daily living activities, such as walking up stairs, carrying groceries, and getting dressed. Dermatomyositis rashes often affect large portions of a patient's body including the scalp and are often disfiguring and painful. No targeted therapies are approved for DM, and a majority of patients require high-dose chronic oral corticosteroids.
About the VALOR Study
VALOR was the longest and largest interventional DM study ever conducted, enrolling 241 subjects globally. Subjects were randomized 1:1:1 to brepocitinib 30 mg, brepocitinib 15 mg and placebo, with one-year of double-blind treatment. The primary endpoint was the difference at week 52 between brepocitinib and placebo in mean Total Improvement Score (TIS), a composite endpoint of multiple measures of DM disease activity.
Investor Conference Call Information
Roivant will host a live conference call and webcast at 8:00 a.m. EDT on Wednesday, September 17, 2025, to discuss the Phase 3 results for brepocitinib in dermatomyositis. To access the conference call by phone, please register online using this registration link. The presentation and webcast details are available under “Events & Presentations” in the Investors section of the Roivant website at https://investor.roivant.com/news-events/events. The archived webcast will be available on Roivant’s website after the conference call.
About Priovant
Priovant Therapeutics is a biotechnology company dedicated to developing novel therapies for autoimmune diseases with high morbidity and few available treatment options. The company's lead asset is brepocitinib, a dual selective inhibitor of TYK2 and JAK1. Through dual TYK2/JAK1 inhibition, brepocitinib distinctively suppresses key cytokines linked to autoimmunity—including type I IFN, type II IFN, IL-6, IL-12, and IL-23—with a single, targeted, once-daily oral therapy. Brepocitinib recently generated positive Phase 3 data in dermatomyositis, and an NDA submission is planned for the first half of 2026. Brepocitinib is also being evaluated in non-infectious uveitis (Phase 3) and cutaneous sarcoidosis (Phase 2).
About Roivant
Roivant (Nasdaq: ROIV) is a biopharmaceutical company that aims to improve the lives of patients by accelerating the development and commercialization of medicines that matter. Roivant’s pipeline includes brepocitinib, a potent small molecule inhibitor of TYK2 and JAK1 in development for the treatment of dermatomyositis, non-infectious uveitis and cutaneous sarcoidosis; IMVT-1402 and batoclimab, fully human monoclonal antibodies targeting FcRn in development across several IgG-mediated autoimmune indications; and mosliciguat, an inhaled sGC activator in development for pulmonary hypertension associated with interstitial lung disease. We advance our pipeline by creating nimble subsidiaries or “Vants” to develop and commercialize our medicines and technologies. Beyond therapeutics, Roivant also incubates discovery-stage companies and health technology startups complementary to its biopharmaceutical business. For more information, visit https://roivant.com.
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Contacts: Investors Keyur Parekh keyur.parekh@roivant.com Media Stephanie Lee stephanie.lee@roivant.com Priovant Daniel Herz Roiphe daniel.herz-roiphe@priovant.com
FAQ
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