Timber Pharmaceuticals Reports Positive Top-line Results from Phase 2b CONTROL Study Evaluating TMB-001 in Moderate to Severe Congenital Ichthyosis

• Data demonstrate reduction in targeted and overall severity of CI in patients treated with topical IPEG TMB-001 (topical isotretinoin)
•  Company planning for end-of-Phase 2 meeting with FDA in the beginning of 2022
•  Phase 3 study is expected to begin in Q2 2022

Basking Ridge, NJ, Oct. 07, 2021 (GLOBE NEWSWIRE) -- via NewMediaWire -- Timber Pharmaceuticals, Inc. ("Timber" or the “Company”) (NYSE American: TMBR), a biopharmaceutical company focused on the development and commercialization of treatments for rare and orphan dermatologic diseases, today announced positive top-line data from its Phase 2b CONTROL study evaluating TMB-001, an investigational topical isotretinoin formulated using Timber’s patented IPEG delivery system, in patients with moderate to severe congenital ichthyosis (CI). 

CI is a group of rare, genetic keratinization disorders that lead to dry, thickened, and scaling skin. People living with CI may have limited range of motion, chronic itching, an inability to sweat, high risk of secondary infections. There are no approved medications for CI and the current common management of this chronic disease is focused on reducing scaling or improving skin lubrication with both systemic and topical treatments. 

The Phase 2b CONTROL study was a randomized, double-blind, vehicle-controlled study designed to assess the efficacy and safety of two concentrations of TMB-001 (0.05% and 0.1% isotretinoin) for the treatment of two distinct subtypes of moderate-to-severe CI (X-linked recessive and lamellar ichthyosis) in patients (n=33) nine years old or older. Subjects applied TMB-001 twice daily for 12 weeks. The primary endpoint was the reduction of targeted ichthyosis severity, determined by a 50 percent or greater reduction in the validated Visual Index for Ichthyosis Severity (VIIS) scaling score (or VIIS-50), a clinically meaningful change. Secondary endpoints included reduction in overall ichthyosis severity, as measured by a two-point improvement using the Investigator Global Assessment (IGA) scale, also considered to be a clinically relevant improvement. The study was not designed or powered for statistical analysis of the endpoints and was intended to provide information for future development.