DENVER — Clearance of lesions, lowered disease severity, and improved itch ratings were demonstrated with nemolizumab in children 2-11 years old with moderate-to-severe atopic dermatitis (AD) in a phase 2 open-label study.

The FDA approved nemolizumab in December 2024 to treat moderate-to-severe AD in adults and adolescents 12 years or older. In the current study, researchers explored the interleukin-31 receptor antagonist in a younger population, including the optimal dosing.

“I’m thrilled to talk about a really important topic, and that’s looking at another biologic agent in our younger atopic dermatitis patients,” Linda Stein Gold, MD, said during a late breaking research session here at the American Academy of Dermatology 2026 Annual Meeting. “We found that weight-based nemolizumab dosing was well tolerated and it was effective,” she added.

“The bottom line is these findings support the potential for pediatric use,” said Stein Gold, director of Dermatology Clinical Research for the Henry Ford Health System in Detroit and division head of Dermatology for the Henry Ford Health System in West Bloomfield, Michigan.

Methodology

Stein Gold and co-investigators for the 52-week multicenter study enrolled 109 children with moderate-to-severe AD with an Eczema Area Severity Index (EASI) score of at least 16 and an Investigator Global Assessment (IGA) score of at least 3 at baseline. Participants also had at least 10% affected body surface area and a Peak Pruritus-Numerical Rating Scale (PP-NRS) score ≥ 4.

The mean itch score was around 7, or in the severe itching range. “We see that it had a fairly significant impact on the overall quality of life,” Stein Gold said. All participants had previous, inadequate responses to topical therapy.

Children were enrolled in three staggered cohorts. Cohort 1 included 36 children for whom the pharmacokinetics of nemolizumab were higher than levels seen in adult and adolescent trials. Therefore, investigators cut the initial doses in half so the pharmacokinetics would better match those seen in adults and adolescent research.

Cohort 1.1 included 37 participants aged 7-11 years treated with the lower dosing. They received nemolizumab 5 mg, 10 mg, 15 mg, 20 mg, or 30 mg every 4 weeks, depending on body weight, after a baseline loading dose. Cohort 2, with 36 children aged 2-6 years, received the same dosing regimen as Cohort 1.1.

The majority of participants were White individuals and were enrolled in Europe. Participants could use concomitant topical medications such as corticosteroids or calcineurin inhibitors during the study. Exclusion criteria included a body weight less than 10 kg; an asthma hospitalization in the prior year, uncontrolled asthma in the prior three months, or a history of chronic bronchitis.

Double Dose Did Not Double Efficacy

By week 16, 41% of the Cohort 1.1 patients and 47% of the Cohort 2 patients achieved IGA success, defined as clear or almost clear skin with a two point or greater improvement. At the same time, 73% of children in Cohort 1.1 and 69% of children in Cohort 2 achieved EASI 75 responses by week 16.

“It’s interesting to note that that cohort that used the double dose didn’t necessarily have a higher efficacy than those patients who used the half dose,” Stein Gold said. “But we see a nice, very rapid onset of action, and we see a maintenance of effect over the course of 52 weeks.”

Session co-moderator Amy S. Paller, MD, said, “It’s fascinating that the higher dose did not even give as good results, but that’s reminiscent of what we saw in adult studies where there was that sweet spot for the best results, which was not the highest dose.” Paller is professor of dermatology and pediatrics, chair of dermatology, and director of the Skin Disease Research Center, at Northwestern University, Chicago.

Successful itch improvement, defined as a 4 point or greater reduction in PP-NRS, was achieved by 60% of Cohort 1.1 patients (aged 7-11 years) and 72% of Cohort 2 patients (aged 2-6 years) at week 16.

Clinically meaningful reductions in itch were noted as early as week 1 on descriptive analysis and sustained up to week 52, Stein Gold said. “As we would expect when we look at itch, we saw a rapid onset of action, and the youngest age cohort did have a better response with an earlier kick-in.”

Two Severe Adverse Events Reported

One patient in Cohort 1 discontinued the study because of a respiratory tract infection.

There were no serious adverse events. There were two severe adverse events, an exacerbation of AD in Cohort 1, and eosinophilia associated with exacerbation of disease that later resolved in Cohort 2.

“We don't see any adverse events that would be unexpected for this patient population,” Stein Gold added.

A meeting attendee asked why the pediatric patient population saw greater improvements in EASI 75 and itch scores than adults treated with nemolizumab.

“With a lot of our studies, the kids tend to outperform the adults. Again, it was an open-label study, and as with results in the adolescents, they could use concomitant topical corticosteroids,” Stein Gold replied. “At week 16, we see even about a 20% difference between the adolescents and adults versus these pediatric patients. But as you go farther out to week 52, the numbers are fairly similar.”

“This is very positive phase 2 data for nemolizumab in the treatment of children ages 2-11 with moderate to severe atopic dermatitis,” Christopher G. Bunick, MD, PhD, associate professor of dermatology, Yale University, New Haven, Connecticut, told Medscape Medical News. Among the highlights of the study is the data on every 4-week dosing, he noted. “We do not have any FDA-approved therapies on-label for [once every 4 weeks] dosing in pediatric patients, and parents, children, as well as providers will appreciate less frequent injections,” he said.

The efficacy rates for skin clearance were good, added Bunick, who was not affiliated with the study and was asked to comment on the results. In addition, almost half of treated children reached an optimal treatment target according to the Aiming High in Eczema/AD treat-to-target framework, Bunick added, with the caveat that participants could use other medications, so this was not a nemolizumab monotherapy trial.

The data reported also goes beyond week 16, to week 52, showing strong maintenance of response in the pediatric age group, he pointed out. Durability of response in the children is important, and the data suggests nemolizumab achieves that endpoint. In addition, “the lack of new safety signals is encouraging, as clinicians and parents want to see that biologics used in adults are as safe or safer in children,” Bunick said.

This study was funded by Galderma. Stein Gold disclosed being on the advisory board for AbbVie, Almirall, Galderma Laboratories L.P., Incyte Corporation, Kymera Therapeutics, La Roche-Posay Laboratoire Pharmaceutique, LEO Pharma US, Lilly ICOS LLC, Pfizer Inc., Promius Pharmaceuticals, Sanofi/Regeneron, Takeda Pharmaceuticals USA Inc., Taro Pharm, and Valeant Pharmaceuticals International. She reported also being a consultant for AnaptysBio, Botanix Pharmaceuticals, BMS, Cutera Inc., Incyte, Janssen Scientific Affairs LLC, Taro, and UCB; investigator for AbbVie, Alumis, AnaptysBio, Galderma, Journey Medical Corporation, Leo Pharma Inc., Novartis Pharmaceuticals Corp., Oruka Therapeutics, Pfizer, and Valeant; a speaker for Actavis, BMS, and Sun Pharmaceutical Industries Ltd; and a speaker/faculty educator for Almirall, Pfizer, and Sanofi/Regeneron. Bunick reported serving as a consultant for Galderma, Sanofi, and Regeneron.

Damian McNamara is a freelance contributor to Medscape Medical News. He worked full-time for Medscape and WebMD from 2018 to 2024. McNamara has a BA in chemistry and an MA in science, health and environmental reporting/journalism.