What Is Nipocalimab?
Nipocalimab is an investigational drug currently being studied in clinical trials. It is a type of drug developed by Janssen Research & Development, LLC. The available trial descriptions indicate that Nipocalimab is administered in different ways, including intravenously (into a vein) and subcutaneously (under the skin). As an investigational drug, Nipocalimab is undergoing research to determine its safety and effectiveness for various medical conditions.
There have been 20 clinical trials involving Nipocalimab to date, with a total enrollment of 2,773 participants. The first trial began on 2021-04-19, and the latest trial is expected to conclude on 2026-02-27. Currently, 8 trials are actively recruiting participants, and 8 trials have been completed.
Uses and Conditions Under Study
Nipocalimab is being investigated for its potential to treat a range of conditions, primarily focusing on autoimmune and immune-mediated disorders. These conditions often involve the body's immune system mistakenly attacking its own tissues.
- Autoimmune Neurological and Musculoskeletal Conditions: Nipocalimab is being studied for conditions like Myasthenia Gravis, which affects muscle strength (3 trials), and Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP), a neurological disorder (1 trial). It is also being investigated for Myositis, an inflammatory muscle disease (1 trial). The drug aims to modulate the immune response that contributes to these conditions.
- Systemic Autoimmune Diseases: Research includes conditions such as Rheumatoid Arthritis, an inflammatory joint disease (2 trials), and Sjogren's Syndrome, which causes dry eyes and mouth (2 trials). Systemic Lupus Erythematosus (SLE), another chronic autoimmune disease, is also being studied (1 trial). Nipocalimab may help reduce the inflammation and immune activity characteristic of these disorders.
- Autoimmune Blood Disorders: Nipocalimab is under investigation for Warm Autoimmune Hemolytic Anemia (WAIHA), where the immune system destroys red blood cells (1 trial). It is also being studied for Neonatal Alloimmune Thrombocytopenia (NAIT), a rare condition affecting newborns where maternal antibodies attack fetal platelets (2 trials).
- Healthy Participants: In addition to specific conditions, Nipocalimab is also being studied in 4 trials involving healthy participants. These studies typically focus on understanding how the drug is absorbed, distributed, metabolized, and excreted in the body, as well as its safety profile in individuals without the target disease.
Dosing
Nipocalimab is being studied in various dosage forms and administration routes in clinical trials. The primary routes of administration observed are intravenous (IV) and subcutaneous (SC). Intravenous administration involves injecting the drug directly into a vein, while subcutaneous administration involves injecting it under the skin.
Clinical trials are exploring different dosing regimens, including single-dose cohorts and multiple-dose cohorts. The data indicates that various doses, such as "Nipocalimab Dose 1" and "Nipocalimab Dose 2," are being evaluated to determine the most effective and safest concentrations for different conditions. In some studies, Nipocalimab is being investigated as a standalone treatment, while in others, it is being studied in combination with other medications. For example, some trials are comparing Nipocalimab to or combining it with treatments like Efgartigimod, Intravenous Immunoglobulins (IVIG), Certolizumab, Etanercept, or Hydroxychloroquine (HCQ).
Specific strengths (e.g., in milligrams) and the frequency of administration (e.g., once daily, weekly) are determined within each trial protocol and are not detailed in the general drug descriptions. The research aims to establish optimal dosing strategies for the various conditions under investigation.
Side Effects
In clinical trials, the most frequently reported adverse events for Nipocalimab included conditions like rheumatoid arthritis and myasthenia gravis, as well as more general symptoms. These events were observed across various studies involving a total of up to 300 patients in the drug arm.
- Rheumatoid arthritis was reported in 13.7% of patients taking Nipocalimab, compared to 9.8% on placebo (from trials with n=95 in the drug arm).
- Myasthenia gravis was reported in 10.2% of patients taking Nipocalimab, compared to 7.5% on placebo (from trials with n=205 in the drug arm).
- Muscle spasms occurred in 9.8% of patients receiving Nipocalimab, compared to 3.2% on placebo (n=205).
- Headache was experienced by 9.7% of patients on Nipocalimab, while 12.6% of those on placebo reported it (n=300).
- Nasopharyngitis (common cold) affected 9.3% of patients taking Nipocalimab, compared to 9.7% on placebo (n=300).
- Back pain was reported by 8.8% of patients on Nipocalimab, versus 4.8% on placebo (n=205).
- Upper respiratory tract infection occurred in 8.7% of patients on Nipocalimab, compared to 7.7% on placebo (n=300).
- Urinary tract infection was reported in 7.1% of patients taking Nipocalimab, compared to 4.4% on placebo (from trials with n=267 in the drug arm).
Other reported side effects, though less common, included pyrexia (fever), peripheral edema (swelling), diarrhea, and pain in extremities.
Clinical Trial Results
Clinical studies have evaluated Nipocalimab for its effectiveness in treating generalized myasthenia gravis and rheumatoid arthritis, both as a standalone therapy and in combination with other treatments.
Generalized Myasthenia Gravis
In a double-blind study (NCT04951622) involving adults with generalized myasthenia gravis, Nipocalimab demonstrated a greater improvement in patients' ability to perform daily activities. Patients treated with Nipocalimab experienced an average reduction of 4.70 points in their Myasthenia Gravis - Activities of Daily Living (MG-ADL) total score over weeks 22, 23, and 24, indicating improved function. In comparison, patients receiving placebo saw an average reduction of 3.25 points.
Rheumatoid Arthritis
A proof-of-concept study (NCT04991753) investigated Nipocalimab as a monotherapy for participants with active rheumatoid arthritis. At Week 12, patients on Nipocalimab showed a greater reduction in disease activity, with an average decrease of 1.03 points in the DAS28-CRP score, compared to a 0.58-point decrease for those on placebo. Additionally, 45.5% of patients treated with Nipocalimab achieved an ACR 20 response (a 20% improvement in symptoms), while 20.0% of placebo patients achieved this. More significant improvements were also observed, with 15.2% of Nipocalimab patients achieving ACR 50 (compared to 5.0% on placebo) and 12.1% achieving ACR 70 (compared to 0% on placebo).
Another study (NCT06028438) explored Nipocalimab in combination with certolizumab for active rheumatoid arthritis. At Week 12, the combination therapy led to higher rates of significant improvement compared to certolizumab alone. For instance, 40.32% of patients receiving Nipocalimab plus certolizumab achieved an ACR 50 response, compared to 31.71% of those on certolizumab plus placebo. Furthermore, 43.55% of patients on combination therapy achieved DAS28-CRP Low Disease Activity, a notable increase from 26.83% in the certolizumab plus placebo group. Remission rates (DAS28-CRP) were also higher with combination therapy, at 25.81% compared to 14.63% for certolizumab plus placebo.
Currently Recruiting Trials
Nipocalimab is currently being investigated in several clinical trials for various autoimmune conditions. These studies aim to understand how well nipocalimab works and its safety profile for patients.
One ongoing Phase 3 study, NCT07438496, is evaluating nipocalimab in adults with moderate to severe Systemic Lupus Erythematosus (SLE). SLE is a long-term disease where the immune system mistakenly attacks healthy tissues. This study plans to enroll about 600 participants to compare nipocalimab against a placebo.
For individuals with generalized myasthenia gravis, a condition causing muscle weakness due to immune system attacks on nerve-muscle connections, there are two recruiting studies. A Phase 3 trial, NCT07217587, is comparing the effectiveness of nipocalimab to another treatment, efgartigimod, in 115 participants. Another Phase 3 study, NCT04951622, is assessing nipocalimab's efficacy and safety against placebo in 199 adults with generalized myasthenia gravis, including a subcutaneous administration substudy. Additionally, a Phase 2/3 study, NCT05265273, is focusing on children aged 2 to less than 18 years with generalized myasthenia gravis, aiming to enroll 12 participants to determine the effect of nipocalimab on total serum immunoglobulin G (IgG) and its safety.
Nipocalimab is also being studied for its potential to reduce the risk of severe fetal and neonatal alloimmune thrombocytopenia (FNAIT), a condition affecting newborns. Two Phase 3 trials are recruiting for this. Study NCT06533098 is comparing nipocalimab to intravenous immunoglobulin (IVIG) in 50 pregnancies at risk of FNAIT. A separate Phase 3 study, NCT06449651, is evaluating nipocalimab against placebo in 39 pregnancies at risk of FNAIT.
Another important area of research is Hemolytic Disease of the Fetus and Newborn (HDFN), where a baby's red blood cell volume falls below normal levels in the womb. The Phase 3 study NCT05912517 is recruiting 120 pregnant participants at risk for severe HDFN to assess nipocalimab's effectiveness compared to placebo in decreasing the risk of fetal anemia.
Finally, for adults with chronic inflammatory demyelinating polyneuropathy (CIDP), a Phase 2/3 study, NCT05327114, is evaluating the safety and efficacy of nipocalimab compared to placebo in delaying relapse. This study aims to enroll 201 participants.
Where to Participate
Clinical trials for nipocalimab are being conducted across a wide geographic area, with study sites located in 92 locations across 71 cities and 29 states. This broad reach aims to make participation accessible to a diverse group of patients.
Some of the cities with multiple active trial sites include:
- Aurora, Colorado (4 sites)
- Philadelphia, Pennsylvania (3 sites)
- Cincinnati, Ohio (3 sites)
- Tampa, Florida (3 sites)
- Los Angeles, California (3 sites)
- Miami, Florida (2 sites)
- Galveston, Texas (2 sites)
- Cleveland, Ohio (2 sites)
- Portland, Oregon (2 sites)
- Charlotte, North Carolina (2 sites)
Eligibility for these studies generally includes individuals between the ages of 2 and 75 years, regardless of gender. Healthy volunteers are not being recruited for these specific nipocalimab trials, as the focus is on patients living with the targeted conditions, and children are eligible for some studies.
Development Timeline
The journey of nipocalimab began with its first clinical trial on April 19, 2021, spearheaded by Janssen Research & Development, LLC, who has sponsored all 20 trials to date. Since its inception, the development program has expanded significantly, enrolling a total of 2,773 participants across various studies.
Initially, nipocalimab's development explored conditions such as Irritable Bowel Syndrome with Constipation (IBS-C) and hyperphosphatemia. However, the focus quickly broadened to a range of autoimmune and alloimmune disorders. The pipeline expanded to include conditions like Thrombocytopenia, Neonatal Alloimmune, Rheumatoid Arthritis, Myositis, Chronic Inflammatory Demyelinating Polyneuropathy, Sjogren's Syndrome, Systemic Lupus Erythematosus, Warm Autoimmune Hemolytic Anemia, Hemolytic Disease of the Fetus and Newborn, and Lupus Nephritis.
Nipocalimab's development has progressed through various stages, with 7 trials reaching Phase 3, 6 in Phase 2, and 4 in Phase 1. Two studies are in a combined Phase 2/3 stage, indicating a mature and active development program. The latest trial is projected to conclude by February 27, 2026, reflecting the ongoing commitment to understanding nipocalimab's full potential for patients with significant unmet medical needs.