Timber Pharmaceuticals Inc. (NYSEAMERICAN: TMBR) has announced promising top-line data from the phase 2b CONTROL study evaluating TMB-001 in treating moderate to severe congenital ichthyosis (CI). TMB-001 is experimental topical isotretinoin formulated using the company’s proprietary IPEG delivery system.
No approved treatment available for CI
CI refers to a set of rare hereditary keratinization disorders that cause the skin to become dry, thickened, and scaling. Limited range of motion, inability to sweat, constant itching, and a high risk of secondary infections are all symptoms of CI. Currently, there are no approved drugs for CI. Hence the current standard of care for this chronic condition is to employ both topical and systemic therapies to reduce scaling and improve skin lubrication.
Timber CEO John Koconis said, “We are extremely pleased with the encouraging data generated by the Phase 2b CONTROL study of TMB-001. The study was not powered or intended to show statistical significance, but we demonstrated clinically meaningful efficacy with a favorable safety profile that support the continued development of our lead product candidate. We look forward to additional study data and plan to present the full data set at scientific congresses or peer-reviewed journal publication in the near future.”
TMB-001 is a potential CI treatment
Chief Medical Officer Alan Mendelsohn said, “These top-line results demonstrate the potential of TMB-001 to be an important option for the treatment of CI where no FDA-approved treatments are currently available and the standard of care is limited to the use of emollients or keratinolytics to reduce scaling and dryness. We are committed to fulfilling the unmet need of CI patients and continuing to develop TMB-001 as quickly as possible. I would like to thank all of those who helped us to rapidly advance the CONTROL trial during the pandemic including the outstanding team of investigators and clinical sites and particularly the patients who participated in the study.”