TMB-001, a topical isotretinoin formulated using Timber Pharmaceuticals’ patented IPEG™ delivery system, shows benefit in the treatment of congenital ichthyosis (CI), regardless of the subtype. Findings are being presented in a late-breaking presentation of a sub-analysis of the Phase 2b CONTROL study presented by Christopher Bunick, MD, PhD at the American Academy of Dermatology (AAD) 2022 Annual Meeting in Boston.

“We know that the treatment of CI is a clear unmet need in dermatology as there are currently limited options available,” says Dr. Bunick. “Our goal with this analysis of the previously completed Phase 2b CONTROL study was to assess whether primary efficacy results for TMB-001 differed by CI subtype, and the results are encouraging for people who are living with even the most severe forms of the disease.”

Timber completed the Phase 2b CONTROL study evaluating TMB-001 in moderate to severe congenital ichthyosis (CI) in September 2021 and announced that data demonstrated clinically meaningful efficacy with a favorable safety profile. The primary efficacy endpoint was the proportion of patients with Visual Index for Ichthyosis Severity (VIIS)-scaling treatment success (VIIS-50 or a 50% reduction in the VIIS score versus baseline). The key secondary efficacy endpoint was the proportion of patients who achieved Investigator Global Assessment (IGA) treatment success (≥2-grade reduction in scaling and fissuring severity over all treated areas of the body).

The subtype analysis presented at the AAD Annual Meeting found that TMB-001 0.05% demonstrated a substantially greater proportion of patients achieving VIIS-50 and ≥2-grade IGA improvement compared with vehicle regardless of subtype. Among enrolled patients (TMB-001 0.05% [n=11], 0.1% [n=10], and vehicle [n=12]), 55% had ARCI-LI and 45% XLRI subtypes. The per-protocol analyses will be presented with the following results:

·All patients with XLRI who received TMB-001 0.05% achieved VIIS-50 compared to 75% receiving vehicle and all patients with ARCI-LI who received TMB-001 0.05% achieved VIIS-50 compared to 17% receiving vehicle.

·Improvement of ≥2-grade IGA score was observed in all patients with XLRI who received TMB-001 0.05% compared to 25% receiving vehicle, and in all patients with ARCI-LI who received TMB-001 0.05% compared to no patients receiving vehicle.

·For patients with XLRI who received TMB-001 0.05%, the median time to achieving VIIS-50 was 28 days compared to 50 days for patients who received vehicle. For patients with ARCI-LI who received TMB-001 0.05%, the median time to achieving VIIS-50 was 32 days compared to 86 days for patients who received vehicle.

·As a Phase 2b study, CONTROL was not powered to show statistical significance.

·No significant systemic adverse events (AEs) were identified. Most AEs reported were application site reactions and were similarly distributed among patients with both XLRI and ARCI-LI who received TMB-001 0.05%.

Based on FDA feedback at a completed End-of-Phase 2 meeting, Timber intends to initiate a pivotal Phase 3 study of TMB-001 in mid-2022.

“The acceptance of this late-breaking analysis of our Phase 2b CONTROL study for presentation at one of the most prestigious dermatology meetings in the world is further validation that we are advancing truly groundbreaking research that may disrupt how CI is treated in the years ahead,” says John Koconis, Chairman and Chief Executive Officer of Timber. “We believe that TMB-001 may be a promising topical alternative to oral retinoids and look forward to launching our Phase 3 investigation.”