Leo Pharma’s Dermatology Asset TMB-001 Fails Phase III Trial, Halting Plans for FDA Submission

Leo Pharma has disclosed a Phase III failure for the primary asset it purchased from Timber Pharmaceuticals, a year after it hedged its bets on the dermatological pipeline of the company.In the late-stage trial for individuals with moderate to severe congenital ichthyosis, TMB-001, a topical ointment version of isotretinoin, did not meet the expected outcome. The general name for congenital skin disorders that result in dry, scaly skin is ichthyosis.Leo reported that the treatment did not show a statistically meaningful benefit in patients compared to those treated with a vehicle in the Phase III ASCEND study (NCT05295732). This indicated a failure to meet the primary and important secondary objectives.According to a news statement issued by the privately held Danish business on August 21, the results do not support the US regulatory submission.

The US Food and Drug Administration (FDA) has not yet authorised a therapy for ichthyosis. Instead, using creams and lotions to hydrate the skin helps treat the problem.The ASCEND study included a three-week induction, a nine-week treatment phase, and a 12-week open-label maintenance regimen for patients who qualified. The research included 209 participants in total who were six years of age or older."We are disappointed and saddened by the results of the Phase III trial," stated John Koconis, CEO of Timber. In this research, we saw an atypically strong vehicle reaction following positive phase 2b findings.

The primary aim of a prior Phase IIb trial assessing the ointment was met—a 50% decrease in the Visual Index for Ichthyosis Severity scale score relative to baseline.In August 2023, Leo Pharma paid $36 million, including an advance payment of $14 million, to purchase the insolvent Timber.  The FDA had given Timber breakthrough, orphan, and fast-track designations at the time. After the purchase, the business became a wholly owned subsidiary of Leo Pharma.Chief development officer Kreesten Meldgaard Madsen of Leo Pharma stated that while the business is still dedicated to treating dermatological conditions, "not all trials are successful as is the case for all pharmaceutical research."

Leo Pharma declared that the trial's outcomes will not affect its financial performance in 2024. In June of this year, the FDA approved Adbry (tralokinumab-ldrm), the company's single-dose autoinjector for atopic dermatitis, giving it another regulatory victory in the dermatology field.Timber also has TMB-003 (sitaxentan), a topical ET-A receptor antagonist in preclinical testing for sclerotic skin conditions, even while the clinical journey of TMB-001 stutters.