Leo Pharma’s Timber reported Phase III failure

A year after investing in Timber Pharmaceuticals' pipeline, Leo Pharma has reported a Phase III failure for TMB-001, a topical isotretinoin ointment. The ASCEND trial (NCT05295732) for moderate to severe congenital ichthyosis did not meet its primary or secondary endpoints, showing no significant improvement over a vehicle treatment. Ichthyosis, characterized by dry, scaly skin, currently lacks an FDA-approved treatment.

The trial included 209 patients with a three-week induction, nine-week treatment, and 12-week open-label maintenance phase. Although TMB-001 previously met its Phase IIb endpoint with a 50% reduction in ichthyosis severity, the latest results do not support US regulatory submission.

Leo Pharma's $36 million acquisition of Timber in August 2023 included a $14 million upfront payment. Despite this setback, Leo Pharma's financial outlook for 2024 remains unaffected, supported by the recent FDA approval of its Adbry for atopic dermatitis. Timber also has TMB-003 in preclinical testing.