Leo Pharma’s Timber bet falls flat following Phase III failure

Leo Pharma spent $36m to acquire Timber Pharmaceuticals last year, along with its lead dermatology asset TMB-001.

Robert Barrie August 22 2024

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.

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.

Timber’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.

Uncover your next opportunity with expert reports

Steer your business strategy with key data and insights from our latest market research reports and company profiles. Not ready to buy? Start small by downloading a sample report first.

Newsletters by sectors

close

Sign up to the newsletter: In Brief

Visit our Privacy Policy for more information about our services, how we may use, process and share your personal data, including information of your rights in respect of your personal data and how you can unsubscribe from future marketing communications. Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address.

Thank you for subscribing

View all newsletters from across the GlobalData Media network.

close