Timber Pharmaceuticals Inc. (NYSEAMERICAN: TMBR) has announced the publication of findings from its previously finalized Phase 2b CONTROL trial in the Journal of the American Academy of Dermatology.
The study evaluated TMB-001 in moderate to severe CI
The study evaluated TMB-001 in the treatment of moderate-to-severe congenital ichthyosis (CI). TMB-001 is the company’s topical isotretinoin formula that employs its novel IPEG delivery system.
The results have been published in a paper titled, “The CONTROL study: A randomized, double-blind vehicle-controlled Phase 2b study of novel topical isotretinoin formulation demonstrates improvement in recessive X-linked and autosomal recessive lamellar congenital ichthyosis.” In addition, the paper highlights findings the company initially released on September 2021, showing TMB-001’s clinically significant efficacy and favorable safety profile.
Chief Medical Officer Alan Mendelsohn said, “The opportunity to publish these data in a world-renowned journal reviewed by our peers in rare and orphan dermatologic diseases exemplifies the significance of our clinical development program and the potential we have to change the treatment landscape for people living with CI.”
The first patients have already been enrolled in the newly commenced Phase 3 ASCEND clinical study, intended to generate the key data needed to traverse the regulatory approval process based on the success of Phase 2b CONTROL study. Phase 2b CONTROL Study was randomized, double-blind, parallel, vehicle-controlled trials designed to assess the safety and efficacy of two TMB-001 concentrations in autosomal recessive lamellar or X-linked recessive subtypes o CI affecting close to 80,000 individuals in the US.
The primary goal was vehicle-treated patients who attained successful treatment
The main efficacy goal was the percentage of TMB-001- and vehicle-treated individuals who achieved successful therapy according to the Visual Index for Ichthyosis Severity (VIIS) scale. The secondary efficacy goal was the number of individuals who obtained Investigator Global Assessment (IGA) treatment effectiveness (2-grade drop in scale and fracture severity throughout all treated body parts) when contrasting TMB-001 to control.
Lead study author Joyce M.C. Teng said, “Hyperkeratosis and widespread scaling are characteristic findings among patients with XLRI or ARCI-LI subtypes of CI, and current management predominantly includes emollients, keratolytics, and off-label retinoids.”