A phase 1 study of PF-07242813, an anti-CD1a antibody, in healthy participants and in patients with atopic dermatitis
In brief
CD1a antibody shows no clinical benefit for atopic dermatitis
In this phase 1 study, PF-07242813 was generally well tolerated, with mostly mild adverse events among 122 participants, but a single dose showed no clinical benefit in 25 patients with moderate-to-severe atopic dermatitis. The result suggests blocking CD1a alone is not enough to improve the disease; whether the pathway has a role in other settings remains open.
- Journal
- JID innovations : skin science from molecules to population health (Q1)
- Published
- 10 August 2026
- Study design
- Phase 1 (first-in-human) trial
- Evidence level
- Level 4, Very Low (CEBM 4)
- Authors
- Spencer I Danto, Ruolun Qiu, Yifan Zhou, Vivek Pradhan, Natalie Rath, Karen M Page, et al.
- PMID
- 42921257
- DOI
- 10.1016/j.xjidi.2026.100523
Why clinicians should know about it
- Picked for Dermatology (paper of the day, 28 October 2026): Early clinical study of targeted therapy in atopic dermatitis
Abstract
BACKGROUND AND AIM: PF-07242813 (anti-CD1a) was designed to inhibit T-cell proinflammatory signaling. This analysis evaluated the safety and pharmacokinetics of single ascending doses and multiple ascending doses (MADs) of PF-07242813 in healthy participants and the pharmacodynamics of a single dose in patients with atopic dermatitis (AD). METHODS: In part 1, healthy participants (aged 18-55 years) received PF-07242813 intravenously (single ascending doses), subcutaneously (MADs) or intravenously (MADs), or placebo. In part 2, patients with moderate-to-severe AD (aged 18-65 years) were randomized (2:1) to receive 1000 mg PF-07242813 intravenously or placebo. Endpoints included safety and pharmacokinetic outcomes in healthy participants and efficacy endpoints in patients with AD. RESULTS: Of the 122 participants (single ascending doses: n = 57; MADs: n = 40; AD: n = 25), 41 reported treatment-emergent adverse events (mostly mild); headache (4.9%) and alanine transaminase increase (4.9%) were the most common. Peak serum PF-07242813 concentrations occurred at 0.6-2 hours (intravenously, single ascending doses) and 73-169 hours (subcutaneously, MADs). Overall, PF-07242813 did not demonstrate clinical benefit in patients with AD. CONCLUSIONS: CD1a blockade is not sufficient to produce a clinical effect in patients with AD. PF-07242813 was well tolerated and may be useful for CD1a pathway analyses. This study is registered at ClinicalTrials.gov (identifier NCT04668066).
Abstract as published, via PubMed.
For healthcare professionals. The summary is generated by AI from the published abstract, and the evidence level is assigned automatically from the study design on the Oxford CEBM hierarchy. Neither is medical advice. Read the full paper before changing practice.