Dermatology biotech Timber Pharmaceuticals’ Phase 3 trial investigating its treatment in a rare inherited skin disorder missed all primary and secondary endpoints, the company announced Wednesday.
In the 12-week ASCEND trial with Timber’s topical ointment formulation of isotretinoin, dubbed TMB-001, there was not a statistically significant difference between the proportion of patients with moderate to severe congenital ichthyosis that responded to treatment with TMB-001 compared to placebo.
The primary endpoint of the trial was a two or more point change in an assessment used to measure fissuring of the skin. The secondary endpoints were the proportion of patients who saw 50% reduction in ichthyosis severity and proportion of patients experiencing local skin reactions.
“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,” Timber CEO John Koconis said in a statement. “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.”
Leo Pharma acquired Timber and TMB-001 in January after Timber filed for bankruptcy. As part of the agreement, Timber would have received $22 million in milestone payments for the candidate. According to the company, this trial failure won’t have a “material impact” on the 2024 finances for Leo.
More detailed data from the trial will be submitted to a publication at a later date, and Timber said that the results don’t support its original plan to submit an NDA for the FDA. The company previously hoped to have FDA approval by Oct. 1, 2025.
According to Timber’s website, the biotech has just two assets in its pipeline. Its other candidate, TMB-003 (sitaxsentan), is a topical ET-A receptor antagonist for the treatment of sclerotic skin diseases, but it’s still preclinical.