LEO Pharma, a parent company of Timber Pharmaceuticals, Inc., presents initial findings from the ASCEND trial in a late-breaking session at the 2024 American Academy of Dermatology (AAD) Annual Meeting. The study provides a preliminary assessment of safety, efficacy, and pharmacokinetic (PK) data from 17 adult and adolescent participants (aged 12-64 years) enrolled in the open-label maximum use arm of the phase 3 ASCEND trial.
This presentation is part of LEO Pharma’s broader data program at AAD, with a total of eight abstracts accepted, demonstrating a commitment to advancing medical dermatology research.
The ASCEND trial evaluates TMB-001, an investigational topical isotretinoin, for the treatment of moderate-to-severe X-linked and autosomal recessive congenital ichthyosis (CI), a rare genetic keratinization disorder resulting in dry, thickened, and scaling skin. Currently, there are no FDA-approved treatments for CI.
Participants in the open-label maximum use arm of the ASCEND trial, with 75-90% body surface area involvement and moderate-to-severe disease, were included in the analysis. The results showed significant improvements in the Investigator Global Assessment (IGA) scores, with a mean total IGA composite scaling and fissuring score decreasing from 3.2 to 1.6 from baseline to Week 12. Additionally, 59% of patients achieved a ≥2 point decrease in IGA scores, and 82% demonstrated a clinically significant (≥1 point) improvement in IGA scores from baseline.
No serious adverse events (SAEs) were reported, and most adverse events (AEs) were mild to moderate, with erythema, pruritus, and stinging/pain being the most common treatment-emergent AEs.
Kreesten Meldgaard Madsen, Chief Development Officer at LEO Pharma, expressed optimism about TMB-001’s potential to address the considerable unmet need in X-linked and autosomal recessive CI. Dr. Christopher Bunick, Lead Author and Associate Professor of Dermatology at Yale School of Medicine, emphasized the challenges faced by CI patients and the importance of advancing treatment options for this community.
The acquisition of Timber Pharmaceuticals by LEO Pharma in January 2024 incorporated TMB-001 into LEO Pharma’s pipeline, furthering its commitment to dermatologic research and development.
The ASCEND trial is ongoing and aims to evaluate the efficacy and safety of TMB-001 0.05% topical ointment in subjects aged ≥6 years with either the autosomal recessive congenital ichthyosis (ARCI) or X-linked recessive ichthyosis (XLRI) subtypes. The trial includes a randomized, double-blind, vehicle-controlled phase III study and an optional maximal use arm to assess systemic exposure of isotretinoin and metabolites.
Congenital ichthyosis (CI) is a group of rare genetic keratinization disorders characterized by dry, thickened, and scaling skin. It can lead to various symptoms, including limited range of motion, chronic itching, and an increased risk of secondary infections.
TMB-001, a topical isotretinoin in a patented polyethylene glycol (IPEG™) delivery system, is being developed for the treatment of moderate-to-severe subtypes of congenital ichthyosis. It received Breakthrough Therapy Designation and Fast Track Status from the FDA in Q1 2022, following positive Phase 2b results in Q4 2021.
LEO Pharma is a global company dedicated to advancing the standard of care for people with skin conditions. Founded in 1908 and majority-owned by the LEO Foundation, LEO Pharma has a long history of research and development in dermatology, offering a wide range of therapies for all disease severities. With a global team of 4,400 people, LEO Pharma serves millions of patients worldwide, generating net sales of DKK 10.6 billion in 2022.
