In a news release from late May 2021, biopharmaceutical company Timber Pharmaceuticals, Inc. (“Timber”) shared that all patients have been enrolled in the Phase 2b CONTROL trial. During the trial, researchers will evaluate TMB-001 (topical isotretinoin) for patients with moderate-to-severe congenital ichthyosis (CI).
Altogether, TMB-001 fits into Timber’s goal of providing unique therapies for rare or orphan dermatological conditions. In 2018, Timber received a $1.5M grant from the FDA to evaluate TMB-001 in a Phase 2a and 2b clinical trial program. According to Timber:
In a Phase 2a study, treatment with TMB-001 was shown to be well tolerated with no evidence of systemic absorption of isotretinoin. There was also a clear signal of efficacy in the study with patients showing a reduction in scaling after eight weeks.
Initially, 45 patients were meant to enroll in the Phase 2b CONTROL study. However, based on the positive results thus far, Timber ended enrollment early with 35 total patients. Ultimately, Timber now seeks to move forward and plan a Phase 3 clinical trial. Data from this trial should be available by the end of 2021.
Overall, ichthyosis stands as an umbrella term for a group of rare genetic skin disorders. There are many subtypes of ichthyosis, such as X-linked ichthyosis, ichthyosis vulgarisms, and lamellar ichthyosis. In many cases, ichthyosis is congenital, meaning symptoms are present at birth. In ichthyosis, genetic mutations cause dry, scaly, and thickened skin, often over large areas of the body. This is because skin cells do not separate normally or shed quickly, or because skin cells are overproduced. In some cases, babies with ichthyosis are even born covered in a parchment-like membrane. However, different forms of ichthyosis may cause varying levels of scaliness, skin redness, or other symptoms. Symptoms include:
- Blocked pores
- Dry, scaly skin
- Note: The scaling associated with ichthyosis often appears on the buttocks, legs, scalp, trunk, and stomach. Additionally, scales could be fine and white, or dark and cracked.
- Chronic itching
- Impaired hearing or vision
- Ectropion (difficulty closing eyes due to surrounding skin tightness)
- Skin abrasions
- Reduced range of motion
- Red skin
- Frequent or increased risk of infections
Learn more about ichthyosis.