Timber Pharmaceuticals, Inc., a LEO Pharma company, provides an update on the development program for TMB 001 in congenital ichthyosis.

ASCEND is the phase III clinical trial of TMB 001, an investigational topical ointment formulation of isotretinoin for the potential treatment of patients with moderate to severe congenital ichthyosis. The trial evaluated the efficacy and safety of TMB-001 in the treatment of congenital ichthyosis in patients (aged 6 years or older) with either the autosomal recessive congenital ichthyosis (ARCI) or X-linked recessive ichthyosis (XLRI) subtypes.

LEO Pharma acquired TMB 001 from Timber Pharmaceuticals in January 2024 following Timber’s Chapter 11 bankruptcy filing. Timber Pharmaceuticals was a clinical-stage biopharmaceutical rare disease dermatology company focused on the development of treatments for rare and orphan dermatologic diseases, and TMB 001 had received orphan, fast-track, and break-through designation by the FDA. As a consequence of the acquisition, Timber Pharmaceuticals was reformed as a fully owned subsidiary of LEO Pharma.

“There are currently no topical treatments specifically developed and approved for the treatment of congenital ichthyosis in the United States, making it a challenging disease to treat. We are disappointed and saddened by the results of the phase III trial. After encouraging phase IIb results, we observed an unexpectedly high vehicle response in this trial. We had hoped that TMB 001 could have been a new potential treatment to help children and adults suffering from moderate to severe congenital ichthyosis and to allow them to live a quality life they had previously not known,” said John Koconis, Chief Executive Officer of Timber Pharmaceuticals.

The results of the analysis will not have material impact on the 2024 financial results for the LEO Pharma group.

About ASCEND: ASCEND is the pivotal phase III clinical trial of TMB 001, an investigational topical ointment formulation of isotretinoin for the potential treatment of patients with moderate to severe congenital ichthyosis. The trial program represents the first vehicle-controlled Phase III trial program of any topical retinoid in this condition. The phase III trial evaluated the efficacy and safety of TMB 001 in the treatment of congenital ichthyosis in patients aged greater than 6 years with either the autosomal recessive congenital ichthyosis (ARCI) or X-linked recessive ichthyosis (XLRI) subtypes.