Timber Pharmaceuticals, Inc. announced the online publication of a sub-analysis of the Phase 2b CONTROL study in Clinical and Experimental Dermatology (CED), the educational journal of the British Association of Dermatologists. The CONTROL study evaluated TMB-001, a topical isotretinoin formulated using the Company's patented IPEG™ delivery system, in subjects nine years of age and older with moderate to severe congenital ichthyosis (CI). 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.

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.