Johnson & Johnson (NYSE:JNJ) has announced positive findings from its Phase 2/3 ENERGY clinical trial, with IMAAVY (nipocalimab-aahu) demonstrating statistically significant improvements in hemoglobin levels among patients with warm autoimmune hemolytic anemia compared with placebo.
The results provide further support for the therapy as the company seeks regulatory approval for a condition that currently has no FDA-approved treatment options.
Trial Meets Primary Endpoint
The randomized, placebo-controlled study showed that patients receiving the 30 mg/kg dose of IMAAVY achieved sustained hemoglobin improvements at roughly three times the rate observed in the placebo group after 24 weeks of treatment.
Patients treated with the therapy also experienced an average hemoglobin increase of at least 1 g/dL as early as the first week of treatment.
The trial’s primary endpoint focused on durable hemoglobin improvement, defined as an increase of at least 2 g/dL from baseline and a hemoglobin concentration of at least 10 g/dL across three visits over a 28-day period beginning by Week 16, without the need for rescue therapy or adjustments to background medications.
Significant Improvement in Patient Outcomes
According to the study results, nearly two-thirds of patients receiving the 30 mg/kg dose achieved both a hemoglobin level of at least 10 g/dL and an increase of at least 2 g/dL from baseline by Week 24.
Beyond improvements in hemoglobin levels, the treatment was also associated with reduced fatigue and lower reliance on steroid therapy, both important measures for patients living with the disease.
The findings suggest that IMAAVY could provide meaningful clinical benefits for individuals affected by the rare autoimmune condition.
Safety Profile Remains Consistent
Johnson & Johnson reported that IMAAVY’s safety profile in the study was consistent with its approved use in patients with generalized myasthenia gravis.
Among patients with warm autoimmune hemolytic anemia, the most frequently reported adverse events were peripheral edema, diarrhea and fever, each occurring in at least 10% of treated participants.
The company indicated that no new safety concerns were identified during the trial.
Regulatory Review Underway
Warm autoimmune hemolytic anemia is a rare and potentially life-threatening disease in which the immune system destroys red blood cells, leading to anemia and other serious complications.
Currently, there are no FDA-approved therapies specifically indicated for the condition.
Johnson & Johnson has already submitted a supplemental Biologics License Application for IMAAVY, and the treatment has been granted Priority Review status by the U.S. Food and Drug Administration.
Data Presented at Major Hematology Conference
The results from the ENERGY study were presented at the European Hematology Association 2026 Congress, according to the company.
The positive data strengthen Johnson & Johnson’s position in rare disease treatment and could support the expansion of IMAAVY into an additional therapeutic indication if regulatory approval is secured.