RARE Daily

J&J Reports Positive Results in GMG and Sjögren’s Disease

February 5, 2024

Rare Daily Staff    

Johnson & Johnson reported topline results from the pivotal phase 3 VIVACITY study of nipocalimab in adults living with generalized myasthenia gravis as well as the phase 2 DAHLIAS study of nipocalimab in adults with Sjögren’s disease.

Generalized myasthenia gravis (gMG), a chronic, life-long, rare, and highly debilitating autoantibody-driven neuromuscular disease characterized by fluctuating muscle weakness.

In myasthenia gravis, the immune system mistakenly attacks muscle receptors by producing anti-receptor antibodies (most commonly anti-acetylcholine receptor [AChR] or anti-muscle-specific kinase [MuSK] antibodies) that can block or destroy these muscle receptors, preventing signals from transferring from nerves to muscles. Symptoms include limb weakness, drooping eyelids, double vision, and difficulties with chewing, swallowing, speech, and breathing. Although gMG may be managed with current therapies, research is needed to develop new treatments for those who may not respond well enough to or tolerate current therapies.

In the phase 3 VIVACITY study, a randomized, double-blind, placebo-controlled study in adult patients with moderate to severe gMG with insufficient response to standard-of-care therapies,

nipocalimab met the primary endpoint, achieving statistically significant reduction in MG-ADLa score from baseline over weeks 22 to 24 compared with placebo.

Sjögren’s disease (SjD) is one of the most prevalent autoantibody driven diseases for which no therapies are currently approved that treat the underlying and systemic nature of the disease. It is a chronic autoimmune disease characterized by autoantibody production, chronic inflammation, and lymphocytic infiltration of exocrine glandular systems. Most patients are affected by mucosal dryness (eyes, mouth, vagina), joint pain, and fatigue. Patients with SjD have a high risk of developing numerous associated conditions, including up to 20 times higher risk of developing B-cell lymphomas when compared to the general population. Disease burden can be as high as that of rheumatoid arthritis or systemic lupus erythematosus. It is usually associated with impaired quality of life and functional capacity.

The phase 2 DAHLIAS study was a randomized, double-blind, placebo-controlled dose-ranging study in patients with SjD who had moderate to severe disease activity on standard of care. The primary endpoint was also met in the DAHLIAS study with a statistically significant reduction in clinESSDAIb score from baseline at week 24 compared with placebo. ClinESSDAI is an endpoint specific to SjD and is a composite scale that assesses organ disease activity across 11 organ system domains 9cutaneous, pulmonary, renal, articular, muscular, peripheral nervous system, central nervous system, hematological, glandular, constitutional, lymphadenopathy and lymphoma); a higher score indicates greater symptom severity.

These data represent the first positive results of an investigational anti-FcRn treatment in this chronic, debilitating autoantibody disease that is without approved advanced therapies.

Nipocalimab was well-tolerated by participants in both studies.

As next steps, Johnson & Johnson plans to present full results from the phase 3 VIVACITY study at an upcoming scientific medical congress and engage with global regulatory authorities about bringing nipocalimab to patients living with gMG. The results from the phase 2 DAHLIAS study support further clinical development of nipocalimab in SjD, and the full results from the study will be presented at a scientific medical congress this year.

Nipocalimab has demonstrated clinical effect in four autoantibody-driven diseases within the past year, including hemolytic disease of the fetus and newborn (HDFN) and rheumatoid arthritis, in addition to gMG and SjD.

Nipocalimab was granted Fast Track designation in HDFN and warm autoimmune hemolytic anemia (wAIHA) in July 2019 and gMG in December 2021, and was granted orphan drug status for wAIHA in December 2019, HDFN in June 2020, gMG in February 2021, chronic inflammatory demyelinating polyneuropathy (CIDP) in October 2021 and fetal and neonatal alloimmune thrombocytopenia (FNAIT) in December 2023 by the U.S. Food and Drug Administration. The treatment was also granted orphan medicinal product designation by the European Medicines Agency in October 2019 for HDFN. Nipocalimab is under development and not currently approved.

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