(Dupixent), indicate results from the phase 2 LIBERTY-PN PRIME trial.
The research was presented at the annual Congress of the European Academy of Dermatology and Venereology.
More than 150 patients with severe PN whose quality of life was impaired were randomly assigned to receive dupilumab (Dupixent) or placebo for 24 weeks. Use of the monoclonal antibody was associated with significant improvements in itch scores.
The researchers also found that the percentage of patients who had no or few PN lesions increased substantially with use of dupilumab, and there were no new safety signals, confirming results from previous studies. Dupilumab, an interleukin-4 receptor alpha antagonist administered by injection, was initially approved by the U.S. Food and Drug Administration for treating atopic dermatitis in 2022.
Study presenter Gil Yosipovitch, MD, professor of dermatology at the University of Miami, emphasized that the improvements in itch and skin lesions seen in these patients were “clinically meaningful.”
Dr. Gil Yosipovitch
In the discussion after the presentation, Dr. Yosipovitch was asked whether the presence or absence of atopy had any bearing on the results.
He replied that although there were too few patients with atopy in the current study to answer that question, other data indicate that there is no overall difference between patients with atopy and those without atopy.
Asked whether dupilumab should be used for only 24 weeks, Dr. Yosipovitch said his that “impression” is that there can be a “honeymoon period” during which the medication is stopped and the treating clinician sees “what happens.”
“It would be interesting in the future” to find out, he added, but he noted that whatever the result, patients would need treatment “for the rest of their life.”
Dr. Yosipovitch, director of the Miami Itch Center and the study’s principal investigator, began his presentation by noting that currently, no systemic therapies have been approved by the FDA or the European Medicines Agency for PN.
Although treatments such as topical medications, ultraviolet light therapy, immunosuppressive agents, and systemic neuromodulators are used off label, for many patients with moderate to severe PN, disease control is inadequate, and the patients are “miserable.”
Recently, the phase 3 LIBERTY-PN PRIME2 trial showed that dupilumab significantly reduced itch and skin lesions for patients with PN, and the safety profile was consistent with that seen in approved indications for the drug.
Dr. Yosipovitch explained that LIBERTY-PN PRIME was a phase 2 study in which, after a screening period, patients with PN were randomly assigned in a 1:1 ratio to receive dupilumab as a 600-mg loading dose followed by 300 mg twice weekly or a matched placebo. Treatment was given for 24 weeks, after which there was a post treatment 12-week follow-up period.
Participants were aged 18-80 years and had been diagnosed with PN for a period of at least 3 months. To be included in the trial, patients had to have an average Worst Itch Numerical Rating Scale (WI-NRS) score of at least 7 and at least 20 lesions, among other criteria. (Patients were allowed to continue treatment with mid- to low-potency topical steroids or topical calcineurin inhibitors if they had been taking them at baseline.)
Among 151 patients in the study, the mean age was 50.1 years, and 66.2% were women. The majority (53.0%) were White; 7.3% were Black; and 35.8% were Asian; 40.4% of patients had a history of atopy. The mean WI-NRS was 8.5, and the mean skin pain score on a 10-point scale was 7.2.
The Investigator’s Global Assessment for PN stage of disease (IGA PN-S) was also employed in the trial. That measure uses a 5-point scale to assess disease severity, with 0 indicating no lesions and 4 indicating more than 100 lesions. At baseline, 28.7% of patients had a score of 4, and the remainder had a score of 3, indicating the presence of 20-100 PN lesions.
Dr. Yosipovitch said that quality of life for these patients was “low” and that scores on the Hospital Anxiety and Depression scale indicated that the participants, many of whom had previously received topical and systemic medications for their PN, indicated they were depressed.
He showed that at week 24, the proportion of patients who had experienced an improvement in the WI-NRS score of greater than or equal to 4 (the study’s primary endpoint) was significantly greater with dupilumab, at 60.0% versus 18.4% among patients given placebo (P < .0001).
Moreover, the proportion of patients at week 24 with an IGA PN-S score of 0 or 1 (the secondary endpoint) was 48.0% in the active treatment group, versus 18.4% with placebo (P =.0004).
With regard to safety, rates of any treatment-emergent adverse events were similar between the groups, at 70.7% for dupilumab and 62.7% for placebo, as were rates for severe treatment-emergent adverse events, at 6.7% and 10.7%, respectively.
Rates of treatment-emergent adverse events of interest, such as skin infections, conjunctivitis, herpes viral infections, and injection site reactions, also suggested that there was no increased risk with active treatment.
Dupilumab is currently under review at the FDA and in Europe for the treatment of PN, according to dupilumab manufacturers Regeneron and Sanofi.
The study was sponsored by Sanofi in collaboration with Regeneron Pharmaceuticals. Dr. Yosipovitch has relationships with Arcutis Biotherapeutics, Bellus Health, Eli Lilly, Galderma, GSK, Kiniksa Pharmaceuticals, LEO Pharma, Novartis, Pfizer, Regeneron Pharmaceuticals, Sanofi, and Trevi Therapeutics.
A version of this article first appeared on Medscape.com.