A year after hedging its bets on Timber Pharmaceuticals’ dermatology pipeline, Leo Pharma has reported a Phase III failure for the lead asset it acquired at the time.
TMB-001, a topical ointment formulation of isotretinoin, came up short in the late-stage trial for patients with moderate to severe congenital ichthyosis. Ichthyosis is an umbrella term for congenital skin conditions that cause dry and scaly skin.
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In the Phase III ASCEND trial (NCT05295732), Leo stated the therapy did not demonstrate a statistically significant improvement in patients compared to those treated with a vehicle. This meant the primary and key secondary endpoints were not met.
The results do not support US regulatory submission, the Danish privately owned company stated in a 21 August press release.
There is currently no treatment approved for ichthyosis by the US Food and Drug Administration (FDA). Instead, the condition is managed by hydrating the skin with creams and lotions.
The ASCEND trial had a three-week induction, a nine-week treatment period, and – for eligible patients – a 12-week open-label maintenance regimen. A total of 209 patients aged six years and over were enrolled in the study.
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By GlobalDataTimber’s CEO John Koconis said: “We are disappointed and saddened by the results of the Phase III trial. After encouraging phase 2b results, we observed an unexpectedly high vehicle response in this trial.”
A previous Phase IIb trial evaluating the ointment achieved its primary endpoint of a 50% reduction in the Visual Index for Ichthyosis Severity scale score versus baseline.
Leo Pharma spent $36m to acquire a bankrupt Timber in August 2023, including a $14m upfront payment. At the time, Timber had received orphan, fast-track, and breakthrough designations from the FDA. Following the acquisition, the company became a fully owned subsidiary of Leo Pharma.
Leo Pharma’s chief development officer Kreesten Meldgaard Madsen said the company remains committed to dermatologic diseases but added “as is the case for all pharmaceutical research, not all trials are successful”.
Leo Pharma stated the trial results will not impact its 2024 financial results. The company had a regulatory win elsewhere in the dermatology space in June this year, when its single-dose autoinjector for atopic dermatitis Adbry (tralokinumab-ldrm) received FDA approval.
Whilst the clinical journey of TMB-001 stutters, Timber also has TMB-003 (sitaxentan) – a topical ET-A receptor antagonist in preclinical testing for sclerotic skin diseases.
