Timber Pharmaceuticals Updates TMB-001 Development for Congenital Ichthyosis

Timber Pharmaceuticals Inc., now a subsidiary of LEO Pharma, recently shared the findings of the ASCEND trial, a phase 3 clinical study evaluating the investigational topical ointment TMB-001 for treating moderate to severe congenital ichthyosis. The study, which was randomized and double-blind over a 12-week period, did not demonstrate a statistically significant difference between patients treated with TMB-001 and those who received a placebo.

Congenital ichthyosis is a genetic skin disorder characterized by dry, scaly skin. The ASCEND trial focused on patients aged six and older with either autosomal recessive congenital ichthyosis (ARCI) or X-linked recessive ichthyosis (XLRI). TMB-001, a topical formulation of isotretinoin, was being tested for its efficacy and safety in this patient population.

Timber Pharmaceuticals, prior to its acquisition by LEO Pharma in January 2024, was a clinical-stage biopharmaceutical company specializing in rare dermatologic diseases. TMB-001 had previously received orphan, fast-track, and breakthrough designations from the FDA. Following Timber's Chapter 11 bankruptcy filing, LEO Pharma acquired TMB-001, and Timber Pharmaceuticals became a wholly owned subsidiary of the Danish company.

John Koconis, CEO of Timber Pharmaceuticals, expressed disappointment in the trial's outcome. He noted that despite promising results from a phase 2b study, the phase 3 trial saw an unexpectedly high placebo response. Koconis had hoped that TMB-001 could offer a new treatment option for children and adults suffering from moderate to severe congenital ichthyosis, significantly improving their quality of life.

Kreesten Meldgaard Madsen, Chief Development Officer of LEO Pharma, reiterated the company's commitment to dermatologic research, particularly for conditions with high unmet needs. He acknowledged that not all pharmaceutical trials yield positive outcomes but emphasized the importance of continuing to explore potential treatments.

The trial results are not expected to significantly impact LEO Pharma's financial performance for 2024. Detailed findings from the ASCEND trial will be submitted for future scientific publication.

The ASCEND trial represents the first vehicle-controlled phase 3 program of any topical retinoid for congenital ichthyosis. This trial aimed to provide comprehensive data on the efficacy and safety of TMB-001 in treating this challenging condition. The study enrolled patients aged six and above, focusing on those with ARCI or XLRI subtypes.

LEO Pharma, founded in 1908 and majority-owned by the LEO Foundation, is dedicated to improving care for individuals with skin conditions. The company has invested heavily in dermatology research and development over the decades, offering a broad range of therapies for various skin diseases. Headquartered in Denmark, LEO Pharma employs 4,200 people globally and serves millions of patients. In 2023, the company reported net sales of DKK 11.4 billion.

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