Generalized Myasthenia Gravis

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Advancing Care for Patients with 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.

Despite available therapies, many patients continue to experience significant disease burden, and there remains an unmet need for additional treatment option. There are approximately 90,000 people in the United States, 140,000 in Europe, and 29,000 in Japan living with the disease.

Telitacicept is an investigational drug. It has not been approved by the U.S. Food and Drug Administration (FDA). The safety and efficacy of telitacicept have not been established.

 

Clinical Trials
UPSTREAM MG

Telitacicept in Generalized Myasthenia Gravis (gMG)

The UPSTREAM MG Phase 3 global study is evaluating the efficacy, safety, and tolerability of telitacicept in patients with generalized myasthenia gravis (gMG).

The ongoing Phase 3 global trial is a randomized, double-blind, placebo-controlled study with an open-label extension.

Objective

To assess the impact of telitacicept on MG-ADL and QMG scores in patients with AChR+ or MuSK+ gMG.

Endpoints

  • Primary – MG-ADL change from baseline at Week 24
  • Secondary – QMG, MG-QOL15r, responder rates, minimal symptom expression.

Enrollment

~180 patients across North America, Europe, Asia-Pacific.

If you have questions about our Phase 3 study in gMG, please email mgstudy@vorbio.com.