- Treatment with TMB-001 demonstrated greater proportions of participants achieving primary and key secondary endpoints versus vehicle regardless of the subtype of congenital ichthyosis -
CI is a group of rare genetic keratinization disorders that lead to dry, thickened, and scaling skin. CI subtypes display variable responses to oral retinoids. The sub-analysis was designed to assess whether the efficacy and safety of TMB-001 differed between subjects with autosomal recessive congenital ichthyosis (ARCI) and X-linked recessive ichthyosis (XLRI). Results show that treatment with TMB-001 demonstrated greater proportions of participants achieving the primary and key secondary endpoints versus vehicle regardless of the subtype of CI.
“Patients with severe CI must often combine bathing, mechanical scale removal, and topical over-the-counter emollient therapy with the possible addition of systemic retinoid therapy throughout their lifetimes to address their disease, sometimes without significant improvement,” said
A total of 33 participants in the CONTROL study were randomized to TMB-001 0.05%, TMB-001 0.1%, or vehicle twice daily, stratified by CI subtype, for 12 weeks. The primary endpoint was the proportion of participants with a 50% or greater reduction versus baseline in Visual Index for Ichthyosis Severity (VIIS) scaling. The key secondary endpoint was a two-grade or greater reduction in Investigator Global Assessment (IGA) scaling score versus baseline. Adverse events (AEs) were monitored. Among enrolled participants (TMB-001 0.05% [n = 11], TMB-001 0.1% [n = 10], and vehicle [n = 12]), 52% had ARCI and 48% had XLRI subtypes. The intent-to-treat (ITT) population consisted of all randomized participants who received one or more doses of the study medication. The per-protocol (PP) population included all participants who met all inclusion criteria, were treatment compliant (≥80%–120% of study drug applied), had a VIIS scaling measurement at the end of Week 12, and had no major protocol violations.
- In the ITT population, 33%/50%/17% of participants with ARCI and 100%/33%/75% of participants with XLRI who received TMB-001 0.05%/TMB-001 0.1%/vehicle, respectively, achieved VIIS-50.
- In the PP population, 100%/33%/17% of participants with ARCI and 100%/50%/75% of participants with XLRI who received TMB-001 0.05%/TMB-001 0.1%/vehicle, respectively, achieved VIIS-50.
- In the ITT population, improvement of ≥2-grade IGA score was observed in 33%/50%/0% of participants with ARCI and 83%/33%/25% of participants with XLRI who received TMB-001 0.05%/TMB-001 0.1%/vehicle, respectively.
- In the PP population, improvement of ≥2-grade IGA score was observed in 100%/67%/0% of participants with ARCI and 100%/50%/25% of participants with XLRI who received TMB-001 0.05%/TMB-001 0.1%/vehicle, respectively.
- Most AEs reported were application site reactions and were similarly distributed among both subgroups.
“ARCI and XLRI are severe subtypes of CI that can lead to cutaneous manifestations including large, dark scaling throughout the body and reduced quality of life,” said
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