Telitacicept Demonstrates Sustained Efficacy and Favorable Safety Profile in 48-Week China Phase 3 Open-Label Extension Generalized Myasthenia Gravis Data
Vor Bio (Nasdaq: VOR) collaborator RemeGen presented 48-week open-label extension data for telitacicept in generalized myasthenia gravis on Oct 29, 2025.
Key efficacy: continuous-treatment patients had a mean -7.5 MG-ADL change and crossover patients -6.3 at week 48; continuous patients had mean -9.8 QMG change and crossover -9.3. 96.2% of continuous and 90.2% of crossover patients reached ≥3-point MG-ADL improvement; most adverse events were mild‑to‑moderate with no new safety signals.
Vor Bio (Nasdaq: VOR) collaboratore RemeGen ha presentato dati di estensione aperta di 48 settimane per telitacicept nella miastenia gravis generalizzata il 29 ottobre 2025.
Efficienza chiave: i pazienti in trattamento continuo hanno avuto in media una variazione MG-ADL di -7,5 e i pazienti in crossover -6,3 alla settimana 48; i pazienti in continuo hanno avuto in media una variazione QMG di -9,8 e i crossover -9,3. Il 96,2% dei pazienti in continuo e il 90,2% dei crossover hanno raggiunto un miglioramento MG-ADL di ≥3 punti; la maggior parte degli eventi avversi era lieve o moderata e non sono stati rilevati nuovi segnali di sicurezza.
Vor Bio (Nasdaq: VOR) colaborador RemeGen presentó datos de extensión abierta de 48 semanas para telitacicept en la gravedad miasténica generalizada el 29 de octubre de 2025.
Eficacia clave: los pacientes en tratamiento continuo tuvieron una media de cambio MG-ADL de -7,5 y los de cruce -6,3 en la semana 48; los pacientes en continuo tuvieron una media de cambio QMG de -9,8 y los de cruce -9,3. El 96,2% de los pacientes en continuo y el 90,2% de los de cruce alcanzaron una mejora MG-ADL de ≥3 puntos; la mayoría de los eventos adversos fueron leves a moderados sin nuevas señales de seguridad.
Vor Bio (나스닥: VOR)와의 협력사인 RemeGen은 2025년 10월 29일 일반화된 중증 근무력증에서 telitacicept에 대한 48주 개방형 연장 데이터를 발표했습니다.
주요 효능: 지속 치료 환자는 MG-ADL 변화가 평균 -7.5이며 교차 환자는 주차 48에서 -6.3 이다; 지속 환자는 평균 -9.8의 QMG 변화, 교차는 -9.3이다. 지속 치료 환자의 96.2%, 교차 환자의 90.2%가 ≥3점 MG-ADL 개선을 달성했고; 대부분의 이상반응은 경증~중등도였으며 새로운 안전 신호는 없었다.
Vor Bio (Nasdaq: VOR) partenaire de RemeGen a présenté des données d’extension ouverte de 48 semaines pour telitacicept dans la myasthénie grave généralisée le 29 octobre 2025.
Efficacité clé : les patients sous traitement continu ont présenté une moyenne de changement MG-ADL de -7,5 et les patients en croisement -6,3 à la semaine 48 ; les patients en continu ont une moyenne de changement QMG de -9,8 et les croisés -9,3. 96,2% des patients en continu et 90,2% des croisés ont atteint une amélioration MG-ADL ≥3 points ; la plupart des événements indésirables étaient légers à modérés et aucun nouveau signal de sécurité n’a été détecté.
Vor Bio (Nasdaq: VOR) Partner RemeGen präsentierte am 29. Oktober 2025 48-wöchige, offene Verlängerungsdaten zu Telitacicept bei generalisierter Myasthenia gravis.
Wichtige Wirksamkeit: Patienten mit fortlaufender Behandlung wiesen eine mittlere MG-ADL-Änderung von -7,5 auf, Cross-over-Patienten -6,3 in Woche 48; fortlaufende Patienten wiesen eine mittlere QMG-Änderung von -9,8 und Cross-over -9,3 auf. 96,2% der kontinuierlichen und 90,2% der Cross-over-Patienten erreichten eine MG-ADL-Verbesserung von ≥3 Punkten; die meisten unerwünschten Ereignisse waren leicht bis moderat und es gab keine neuen Sicherheitszeichen.
Vor Bio (نازداك: VOR) الشريك ريميجم قدّم بيانات امتداد مفتوح لمدة 48 أسبوعاً لـ telitacicept في الوهن العضلي الوبيل العام في 29 أكتوبر 2025.
الفعالية الأساسية: مرضى العلاج المستمر أظهروا تغيّراً متوسطاً في MG-ADL قدره -7,5، والمرضى المتبدلون -6,3 في الأسبوع 48؛ المرضى المستمرون أظهروا تغيّراً متوسطاً في QMG قدره -9,8 والمتبدلون -9,3. بلغ 96,2% من مرضى الاستمرار و90,2% من المتبدلين تحقيق تحسن MG-ADL بمقدار ≥3 نقاط؛ أغلب الأحداث الضائرة كانت خفيفة إلى معتدلة ولم تظهر إشارات أمان جديدة.
Vor Bio（纳斯达克：VOR） 的合作伙伴 RemeGen 于 2025 年 10 月 29 日公布了 telitacicept 在广义重症肌无力中的 48 周开放标签扩展数据。
关键有效性：持续治疗的患者在第 48 周的 MG-ADL 变化平均为 -7.5，转换组为 -6.3；持续治疗的患者在 QMG 变化方面平均为 -9.8，转换组为 -9.3。持续组有 96.2% 的患者和转换组有 90.2% 的患者达到 MG-ADL 提升 ≥3 分；大多数不良事件为轻度至中度，未观察到新的安全信号。
- 100% achieved ≥2-point MG-ADL improvement over 48 weeks
- Mean MG-ADL change of -7.5 in continuous telitacicept patients
- Mean QMG change of -9.8 in continuous telitacicept patients
- 96.2% of continuous patients reached ≥3-point MG-ADL improvement
- No new safety signals observed through 48-week OLE
- Placebo crossover patients had smaller mean MG-ADL change (-6.3)
- Injection site reactions occurred in crossover patients (mild, self-resolving)
Insights
Telitacicept shows robust 48-week efficacy and a benign safety profile in a China Phase 3 OLE, supporting its global Phase 3 program.
Telitacicept produced large, sustained symptom improvements in generalized myasthenia gravis through week
The safety signal was described as favorable and stable: no new safety signals, mostly mild–moderate adverse events, no injection-site reactions in continuous patients, and only mild, self-resolving reactions in crossover patients. The dataset comes from a randomized, double-blind 24-week core followed by an open-label extension, so observed durability across
Key dependencies and risks are explicit in the disclosed facts: confirmatory global results from the ongoing Phase 3 program across 14 countries and regulatory review processes will determine broader impact. Watch for full peer-reviewed datasets and regulatory-grade endpoints, the global Phase 3 enrollment progress, and any emerging safety signals beyond week
Telitacicept delivered consistent quality-of-life improvement across both treatment and placebo crossover arms
Sustained efficacy and favorable safety extension data support potential global best-in-disease profile in generalized myasthenia gravis (gMG)
BOSTON, Oct. 29, 2025 (GLOBE NEWSWIRE) -- Vor Bio (Nasdaq: VOR), a clinical-stage biotechnology company transforming the treatment of autoimmune diseases, today announced that its collaborator, RemeGen Co., Ltd (HKEX: 9995, SHA: 688331), presented 48-week open-label extension (OLE) data from its Phase 3 study in China evaluating telitacicept in patients with gMG. The results will be presented in a late-breaking session on October 29 at 10:50am PT at the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) Annual Meeting.
“The strength and consistency of these results with telitacicept in China mark a major step forward as we look to redefine long-term disease control for patients living with generalized myasthenia gravis. Achieving sustained and meaningful improvements across both treatment and crossover groups sets a new standard of care expectation globally, especially with nearly eighty-seven percent of patients reaching six-point or greater gains in MG-ADL and over seventy-one percent reaching eight-point or greater gains in QMG,” said Jean-Paul Kress, M.D., Chief Executive Officer and Chairman of the Board. “With our global Phase 3 trial now enrolling across 14 countries, we are excited to build on this momentum and work toward delivering the same transformative benefits to patients worldwide.”
The Phase 3 trial in China was a randomized, double-blind, placebo-controlled study in patients with AChR-Ab or MuSK-Ab positive gMG. Following the 24-week double-blind period, all patients entered the OLE, with those previously on placebo crossing over to telitacicept 240mg.
The primary endpoint of the study was change from baseline in MG-ADL at 24 weeks, with secondary endpoints including changes in MG-ADL and QMG (Quantitative Myasthenia Gravis) at 12, 24, 36, and 48 weeks, as well as the proportion of patients achieving clinically meaningful improvements (≥3-point decrease in MG-ADL and ≥5-point decrease in QMG) at 24 and 48 weeks. The initial 24-week double-blind treatment stage data were presented at the American Academy of Neurology (AAN) Annual Meeting 2025.
Key Findings from the 48-Week OLE:
- At week 48, patients on telitacicept throughout achieved a -7.5 mean MG-ADL change, while placebo crossover patients achieved -6.3;
96.2% of continuous patients and90.2% of crossover patients reached ≥3-point improvement. - At week 48, patients on telitacicept throughout achieved a -9.8 mean QMG change, while placebo crossover patients achieved -9.3;
94.2% of continuous patients and90.2% of crossover patients reached ≥5-point improvement. - Telitacicept demonstrated a favorable profile comparable to placebo and consistent with prior studies across other autoimmune indications, including systemic lupus erythematosus, rheumatoid arthritis, primary Sjögren's disease, and IgA nephropathy. No new safety signals were observed. Most adverse events were mild to moderate in severity.
- No injection site reactions were reported during the OLE in patients previously on telitacicept. Injection site reactions in placebo crossover patients were mild, self-resolving, and did not lead to discontinuation.
About Telitacicept
Telitacicept is a novel, investigational recombinant fusion protein designed to treat autoimmune diseases by selectively inhibiting BLyS (BAFF) and APRIL - two cytokines essential to B cell and plasma cell survival. This dual-target mechanism reduces autoreactive B cells and autoantibody production, key drivers of autoimmune pathology. In a Phase 3 clinical trial in generalized myasthenia gravis in China, telitacicept demonstrated a placebo adjusted 4.83-point improvement in MG-ADL (Myasthenia Gravis Activities of Daily Living scale) at 24 weeks, the primary endpoint of the trial.
Telitacicept is approved in China for systemic lupus erythematosus (SLE), rheumatoid arthritis (RA), and generalized myasthenia gravis (gMG). A global Phase 3 clinical trial in gMG is currently underway across the United States, Europe, South America, and Asia-Pacific to support potential approval in the United States, Europe, and Japan.
About Generalized Myasthenia Gravis (gMG)
gMG is a rare, chronic autoimmune neuromuscular disorder that disrupts communication between nerves and muscles, leading to muscle weakness that can impact mobility, vision, swallowing, and breathing. The disease is mediated by autoantibodies, most commonly targeting the acetylcholine receptor (AChR) or muscle-specific kinase (MuSK), which interfere with neuromuscular transmission. While several therapies are available, many patients continue to experience persistent symptoms or intolerable side effects. As a result, there remains a significant unmet need for new therapies that offer durable efficacy, a favorable safety profile, and convenient administration to improve the quality of life for people living with gMG. There are approximately 90,000 people in the United States, 140,000 in Europe, and 29,000 in Japan living with the disease.
About Vor Bio
Vor Bio is a clinical-stage biotechnology company transforming the treatment of autoimmune diseases. The Company is focused on rapidly advancing telitacicept, a novel dual-target fusion protein, through Phase 3 clinical development and potential commercialization to address serious autoantibody-driven conditions worldwide. For more information visit www.vorbio.com.
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. The words “aim,” “anticipate,” “can,” “continue,” “could,” “design,” “enable,” “expect,” “initiate,” “intend,” “may,” “on-track,” “ongoing,” “plan,” “potential,” “should,” “target,” “update,” “will,” “would,” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Forward-looking statements in this press release include Vor Bio’s statements regarding the potential of telitacicept to have a global best-in-disease profile in generalized myasthenia gravis (gMG) and to offer a disease modification rather than cyclical symptom management; telitacicept’s safety profile; our goal to redefine long-term disease control for patients living with gMG and deliver the same transformative benefits we see in China to patients worldwide; and telitacicept’s market opportunity.
Vor Bio may not actually achieve the plans, intentions, or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various factors, including the data for our product candidates may not be sufficient for obtaining regulatory approval to commercialize products; we may not be able to execute our business plans, including meeting our planned clinical and regulatory milestones and timelines, and possible limitations of financial and other resources. The results of the clinical trial described in this press release is based on information reported by RemeGen; Vor Bio has not independently verified this data. These and other risks are described in greater detail under the caption “Risk Factors” included in Vor Bio’s most recent annual or quarterly report and in other reports it has filed or may file with the Securities and Exchange Commission.
Any forward-looking statements contained in this press release speak only as of the date hereof, and Vor Bio expressly disclaims any obligation to update any forward-looking statements, whether because of new information, future events or otherwise, except as may be required by law.
Media & Investor Contacts:
Carl Mauch
cmauch@vorbio.com
Sarah Spencer
investors@vorbio.com
FAQ
What did Vor Bio (VOR) report about telitacicept efficacy at 48 weeks on Oct 29, 2025?
How many VOR patients reached clinically meaningful MG-ADL improvement at week 48?
What safety findings did Vor Bio (VOR) present for telitacicept in the 48-week OLE?
How did placebo crossover patients perform vs continuous telitacicept patients at week 48 (VOR)?
When and where were the telitacicept 48-week data for VOR presented?
Does the 48-week data for telitacicept suggest global development for VOR?
