LEO's Timber bankruptcy asset punt derailed by late-stage study failure

Timber Pharmaceuticals' late-stage study of TMB-001, aimed at treating moderate-to-severe congenital ichthyosis, has failed to meet its primary and key secondary endpoints. This setback significantly impacts its parent company, LEO Pharma. TMB-001 is an investigational topical ointment formulation of isotretinoin. LEO Pharma acquired it earlier this year through Timber’s asset acquisition, following Timber's chapter 11 bankruptcy filing. As a result of the acquisition, Timber was reorganized as a fully owned subsidiary of LEO Pharma.

The Phase III ASCEND trial was designed to evaluate the efficacy of TMB-001 in approximately 200 patients aged six years or older who were diagnosed with either autosomal recessive congenital ichthyosis (ARCI) or X-linked recessive ichthyosis (XLRI). Unfortunately, the trial did not demonstrate a statistically significant difference in response between patients treated with TMB-001 and those treated with the vehicle after a 12-week period. This lack of significant difference was a major reason the trial did not meet its primary and key secondary endpoints.

John Koconis, CEO of Timber, expressed his disappointment over the trial's outcomes. He stated, "We are disappointed and saddened by the results," noting that the high hopes set by encouraging Phase IIb results were dashed by an unexpectedly high vehicle response in the ASCEND trial. This unexpectedly high vehicle response skewed the results, making it difficult to ascertain the efficacy of TMB-001.

Despite the disappointing outcomes, the company did provide some reassuring information about the safety profile of TMB-001. According to Timber, the majority of adverse events observed during the study were non-serious localized skin reactions, which were mostly of mild or moderate severity. This suggests that while TMB-001 may not have met its efficacy endpoints, it was generally well-tolerated by the patients who participated in the trial.

The failure of the ASCEND trial is a significant blow to LEO Pharma, especially considering the optimism generated by earlier phases of research. The inability to demonstrate a statistically significant improvement with TMB-001 raises questions about the future development of this investigational drug. The outcomes of this study are likely to influence the strategic decisions LEO Pharma will make regarding its newly acquired subsidiary, Timber Pharmaceuticals, and its broader portfolio of dermatological treatments.

Given the rigorous nature of clinical trials and the high stakes involved, the failure to meet primary and key secondary endpoints in a Phase III trial can be a major setback for any pharmaceutical company. This is particularly true for LEO Pharma, which invested in Timber's assets with the expectation of expanding its treatment offerings in dermatology. The unexpectedly high vehicle response adds another layer of complexity, underscoring the unpredictable nature of drug development.

In summary, Timber Pharmaceuticals' late-stage study of TMB-001 did not achieve the desired outcomes in treating moderate-to-severe congenital ichthyosis. This failure has important implications for both Timber and its parent company, LEO Pharma. While the safety profile of the drug was acceptable, the efficacy results were not conclusive, putting the future of TMB-001 in jeopardy.

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