Immunomodulatory Peptide Gets Orphan Drug Status for Eosinophilic Esophagitis

The Food and Drug Administration (FDA) has granted Orphan Drug designation to ‘1104 for the treatment of eosinophilic esophagitis (EoE).

‘1104 is a first-in-class immunomodulatory peptide derived from Mycobacterium tuberculosis Chaperonin 60.1. The designation is supported by data from the double-blind, placebo-controlled, phase 2a RVLO 121-04 study (ClinicalTrials.gov Identifier: NCT05084963), which evaluated the efficacy and safety of repeat doses of ‘1104 in adults with active EoE.

Study participants were administered 3 intravenous doses of ‘1104 or placebo over 2 weeks. The primary endpoint was the change from baseline in the peak esophageal intraepithelial eosinophil count at week 4.

Results showed treatment with ‘1104 led to a statistically significant reduction in eosinophils, CD4+, and CD8+ cells from baseline compared with placebo. Findings also demonstrated a statistically significant increase in T regulatory cells (Tregs) in esophageal tissue and B regulatory cells (Bregs) in the blood. Compared with placebo, a statistically significant improvement in clinical dysphagia (based on the absolute change in Dysphagia Symptom Questionnaire score from baseline) was reported in ‘1104-treated patients.

The Company is expected to start a phase 2b trial evaluating ‘1104 in adults with active EoE in 2024.