Timber Pharmaceuticals Fails Phase 3 Trial for Congenital Ichthyosis Drug Candidate

LEO Pharma's subsidiary, Timber Pharmaceuticals, failed a late-stage trial for its investigational topical ointment formulation of isotretinoin for treating moderate to severe congenital ichthyosis.

The phase 3 ASCEND trial was 12-week randomized, double-blind clinical study evaluating TMB-001, an investigational topical ointment formulation of isotretinoin. The trial did not demonstrate a statistically significant difference between the proportion of patients treated with TMB-001 and those treated with a vehicle after 12 weeks.

ASCEND focused on assessing the efficacy and safety of TMB-001 in patients aged 6 years or older with either autosomal recessive congenital ichthyosis (ARCI) or X-linked recessive ichthyosis (XLRI), two subtypes of congenital ichthyosis. Despite promising phase 2b results, the phase 3 trial encountered an unexpectedly high vehicle response, leading to the lack of a statistically significant outcome in the comparison between TMB-001 and the vehicle.

“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 3 trial. After encouraging phase 2b 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," John Koconis, Chief Executive Officer of Timber Pharmaceuticals, said in a company news release.

TMB-001 was acquired by LEO Pharma in January 2024 after Timber Pharmaceuticals filed for Chapter 11 bankruptcy. TMB-001 had been granted orphan, fast-track, and breakthrough therapy designations by the FDA. Following the acquisition, Timber Pharmaceuticals was reformed as a fully owned subsidiary of LEO Pharma.

The ASCEND trial results are not expected to have a material impact on LEO Pharma’s financial results for 2024. The detailed findings from the trial are planned to be submitted for scientific publication at a later date.