• Results from the randomized, double-blind period of the phase 3 clinical trial with TMB-001 – the ASCEND trial – show that the primary and key secondary endpoints were not met.
• The trial investigated TMB-001 in patients 6 years or older with moderate to severe congenital ichthyosis.
• The majority of adverse events observed were non-serious localized skin reactions of mild or moderate severity.
• The trial results do not support submission of a New Drug Application to the US FDA.

NOT FOR UK USE – NOT INTENDED FOR UK MEDIA

Timber Pharmaceuticals Inc., a LEO Pharma company, today announced the results for the randomized, double-blind 12-week period of the ASCEND trial. The trial did not show a statistically significant difference between the proportion of patients treated with TMB-001 responding to treatment after 12 weeks compared with patients treated with vehicle.

ASCEND is the phase 3 clinical trial of TMB-001, an investigational topical ointment formulation of isotretinoin for the potential treatment of patients with moderate to severe congenital ichthyosis. The trial evaluated the efficacy and safety of TMB-001 in the treatment of congenital ichthyosis in patients (aged 6 years or older) with either the autosomal recessive congenital ichthyosis (ARCI) or X-linked recessive ichthyosis (XLRI) subtypes.

LEO Pharma acquired TMB-001 from Timber Pharmaceuticals in January 2024 following Timber’s Chapter 11 bankruptcy filing. Timber Pharmaceuticals was a clinical-stage biopharmaceutical rare disease dermatology company focused on the development of treatments for rare and orphan dermatologic diseases, and TMB-001 had received orphan, fast-track, and break-through designation by the FDA. As a consequence of the acquisition, Timber Pharmaceuticals was reformed as a fully owned subsidiary of LEO Pharma.

“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. 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. 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,” said John Koconis, Chief Executive Officer of Timber Pharmaceuticals.

Kreesten Meldgaard Madsen, Chief Development Officer, LEO Pharma added: "LEO Pharma remains fully committed to innovative research in dermatologic diseases with a high unmet need as we strive to make a difference for people living with skin diseases and their families. This means investing in trials to explore potential new treatments that can address the high unmet need these people face every day. Unfortunately, as is the case for all pharmaceutical research, not all trials are successful.”

The results of the analysis will not have material impact on the 2024 financial results for the LEO Pharma group.

Detailed results from ASCEND are planned to be submitted for scientific publication at a later date.

About ASCEND

ASCEND is the pivotal phase 3 clinical trial of TMB-001, an investigational topical ointment formulation of isotretinoin for the potential treatment of patients with moderate to severe congenital ichthyosis. The trial program represents the first vehicle-controlled Phase 3 trial program of any topical retinoid in this condition. The phase 3 trial evaluated the efficacy and safety of TMB-001 in the treatment of congenital ichthyosis in patients aged ≥6 years with either the autosomal recessive congenital ichthyosis (ARCI) or X-linked recessive ichthyosis (XLRI) subtypes. For more information on the ASCEND trial (NCT05295732) go to clinicaltrials.gov.

About LEO Pharma

LEO Pharma is a global company dedicated to advancing the standard of care for the benefit of people with skin conditions, their families and society. Founded in 1908 and majority owned by the LEO Foundation, LEO Pharma has devoted decades of research and development to advance the science of dermatology, and today, the company offers a wide range of therapies for all disease severities. LEO Pharma is headquartered in Denmark with a global team of 4,200 people, serving millions of patients across the world. In 2023, the company generated net sales of DKK 11.4 billion. For more information, please visit www.leo-pharma.com.

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