▎WuXiEdited by Kant Content Team

Johnson & Johnson recently announced that the U.S. FDA has granted Breakthrough Therapy Designation (BTD) to its investigational antibody therapy nipocalimab, which targets the neonatal Fc receptor (FcRn), for the treatment of adult patients with moderate to severe Sjögren’s disease (SjD).According to the press release, nipocalimab is the first therapy to receive BTD in the SjD field.It is worth mentioning that,This therapy was listed by the industry media Evaluate earlier this year.Top 10 Potential Blockbuster Therapies in DevelopmentOne of.

SjD is one of the most common autoimmune antibody-driven diseases, and there is currently no approved therapy that targets the root cause of the disease.It is a chronic autoimmune disease that is estimated to affect approximately four million people worldwide, with the incidence rate in women being nine times that of men. SjD is characterized by the production of autoantibodies, chronic inflammation, and lymphocytic infiltration of the exocrine gland system. Most patients suffer from mucosal dryness (eyes, mouth, vagina), joint pain, and fatigue. Patients with SjD have a high risk of developing various related conditions, with the risk of B-cell lymphoma being up to 20 times higher compared to the general population.

The granting of this BTD was primarily based on data from the Phase 2 DAHLIAS study. This study evaluated the efficacy and safety of nipocalimab in adult patients with moderate to severe SjD. The trial enrolled a total of 163 SjD patients (5 mg/kg nipocalimab group, n=53; 15 mg/kg nipocalimab group, n=54; placebo group, n=56), with similar baseline characteristics across groups. The primary endpoint, ClinESSDAI, is a comprehensive scale specifically designed for SjD, assessing disease activity across 11 organ system domains, including skin, lungs, kidneys, joints, muscles, peripheral nervous system (PNS), central nervous system (CNS), blood, glands, lymphadenopathy, and lymphoma components, with higher scores indicating more severe symptoms.

The analysis shows,The 15 mg/kg group met the primary endpoint, with a least squares mean difference in ClinESSDAI score of –2.65 (90% CI: –4.03, –1.28; p=0.002) compared to the placebo group.; –0.34 for the 5 mg/kg group (90% CI: –1.71, 1.03; p=0.681).Starting from Week 4,15 mg/kgThe group of patients began to experience relief and continued to increase throughout the 24-week treatment period.At Week 24, compared with placebo,Subjects in the 15 mg/kg nipocalimab group showedAverageMore than 70% improvement in systemic disease activity compared to placebo.

▲Summary of Efficacy Results from the DAHLIAS Study (Image Source: Reference [2])

In most secondary/exploratory endpoints, patients in the 15 mg/kg nipocalimab group also showed improvement relative to placebo., including the physician's global assessment (PhGA) of disease severity at week 24, remission according to the Sjögren's Syndrome Treatment Response Assessment Tool (STAR), Composite Response Endpoint in Sjögren’s Syndrome (CRESS), and Disease Activity Level (DAL) response, etc. Patient-reported outcomes also showed numerical improvements in the 15 mg/kg nipocalimab group compared to placebo, with important Sjögren's disease (SjD) symptoms such as dry mouth, dry eyes, and vaginal dryness showing a trend toward improvement. The safety and tolerability results of the investigational drug were consistent with previous clinical trials.

▲Summary of DAHLIAS Study Safety Results (Image Source: Reference [2])

The press release noted that these data represent the first positive clinical trial results for nipocalimab in the treatment of SjD, supporting the potential of the investigational FcRn blocker as a targeted therapy for SjD.Positive results from the DAHLIAS trial support the ongoing continuation of the Phase 3 study.

Nipocalimab is a potential "best-in-class" antibody therapy targeting FcRn.It binds to FcRn, causing the autoantibodies taken up by monocytes and endothelial cells to be degraded within the cells rather than being released back into the bloodstream.Studies have shown that it can reduce IgG levels, including pathogenic autoantibodies in the bloodstream, by more than 75%.This antibody therapy has the potential to treat various autoimmune antibody-mediated immune diseases. Previously, it has receivedBreakthrough Therapy Designation, used to treat pregnant women at high risk of severe hemolytic disease of the fetus and newborn (HDFN). Nipocalimab has also achieved positive topline results in the pivotal Phase 3 clinical trial, VIVACITY, for the treatment of adult patients with generalized myasthenia gravis (gMG).

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References:

[1] Nipocalimab is the first and only investigational therapy granted U.S. FDA Breakthrough Therapy Designation for the treatment of adults living with moderate-to-severe Sjögren's disease. Retrieved November 12, 2024 from https://www.prnewswire.com/news-releases/nipocalimab-is-the-first-and-only-investigational-therapy-granted-us-fda-breakthrough-therapy-designation-for-the-treatment-of-adults-living-with-moderate-to-severe-sjogrens-disease-302301655.html

[2] LBA0010 EFFICACY AND SAFETY OF NIPOCALIMAB, AN ANTI-FcRn MONOCLONAL ANTIBODY, IN PRIMARY SJOGREN’S DISEASE: RESULTS FROM A PHASE 2, MULTICENTER, RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE-BLIND STUDY (DAHLIAS). Retrieved November 12, 2024 from https://ard.bmj.com/content/83/Suppl_1/240.2

[3] Late-breaking results show nipocalimab significantly improves Sjögren’s disease activity in a Phase 2 study. Retrieved June 17, 2024, from https://www.jnj.com/media-center/press-releases/late-breaking-results-show-nipocalimab-significantly-improves-sjogrens-disease-activity-in-a-phase-2-study

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