Phase 3 study of TMB-001 for treatment of congenital ichthyosis fails to meet endpoints

Key takeaways:

• TMB-001 is an investigational topical reformulation of isotretinoin for the treatment of congenital ichthyosis.
• The phase 3 program evaluating TMB-001 did not meet primary and secondary endpoints.

Timber Pharmaceutical’s study investigating the use of a new topical ointment formulation of isotretinoin for moderate to severe congenital ichthyosis did not achieve its endpoints, LEO Pharma announced in a press release.

As Healio previously reported, Timber Pharmaceuticals filed for chapter 11 bankruptcy in November 2023. Since then, LEO Pharma acquired the company including its orphan, fast track and breakthrough designated asset, TMB-001.

TMB-001 is an investigational topical reformulation of isotretinoin that has been evaluated in a phase 3, randomized, double-blind, 12-week study called ASCEND for the treatment of patients aged 6 years or older with either the autosomal recessive congenital ichthyosis or X-linked recessive ichthyosis subtypes.

While the drug initially produced encouraging results in congenital ichthyosis, the company has announced that the current trial did not show a statistically significant difference between the proportion of patients that responded to treatment with TMB-001 vs. placebo after 12 weeks.

“We are disappointed and saddened by the results of the phase 3 trial,” John Koconis, CEO of Timber Pharmaceuticals, said in the release. “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.”

According to the release, these results will not have a material impact on the 2024 financial results for LEO Pharma. Full study results will be released at a later date.