IMMUNOLOGY2026™ Conference Recordings For Attendees-Preliminary immunologic and clinical Phase 1b study outcomes from patients with ROSAH syndrome treated with the first-in-class ALPK1 inhibitor DF-003

Preliminary immunologic and clinical Phase 1b study outcomes from patients with ROSAH syndrome treated with the first-in-class ALPK1 inhibitor DF-003

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Video Transcription

Video Summary

The transcript describes ALPK1 as a kinase involved in NF-kappa-B activation and autosomal dominant autoinflammatory disease, especially ROSAH syndrome, which includes retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis, and headache. It then summarizes clinical observations and early trial data for DF003, an ALPK1 inhibitor being developed to treat ROSAH syndrome. The treatment appeared safe, showed a favorable pharmacokinetic profile, and produced promising clinical responses. A question-and-answer segment discussed whether biomarkers like CRP and other measures had normalized and mentioned plans for additional splenic biopsies to better understand treatment effects.

Meta Tag

Date April 17, 2026 12:45 PM - 1:00 PM

Room 205

Speaker Neil Solomons

Track Translational and Interventional Immunology (TI)

Year 2026

Keywords

ALPK1

ROSAH syndrome

DF003

autoinflammatory disease

biomarkers

April 17, 2026 12:45 PM - 1:00 PM

Neil Solomons

Translational and Interventional Immunology (TI)

205

2026