Timber Pharmaceuticals, Inc. (“Timber” or the “Company”) (NYSE American: TMBR), a clinical-stage biopharmaceutical company focused on the development and commercialization of treatments for rare and orphan dermatologic diseases, today announced that the first four patients have been enrolled in the pivotal Phase 3 ASCEND clinical trial.
ASCEND will evaluate the efficacy, pharmacokinetics and safety of TMB-001 0.05%, a topical isotretinoin formulated using the Company’s patented IPEG™ delivery system, for the treatment of moderate to severe forms of congenital ichthyosis (CI).
The U.S. Food & Drug Administration (FDA) awarded a $1.5 million grant through its Orphan Products Clinical Trials Grant program to support completed Phase 2a and Phase 2b clinical trials that evaluated TMB-001. The FDA also granted Breakthrough Therapy and Fast Track designations to TMB-001. Leading research centers in the U.S., Canada, Italy, France, and Germany are participating in the ASCEND study. The first patients were enrolled by Kenneth Dawes, M.D., and his research team at the Dawes Fretzin Dermatology Group in Indianapolis, Indiana.
“We are pleased to launch the pivotal ASCEND study and enroll the first patients, which brings us one step closer to delivering an important new treatment option for people who are burdened by the life-long endeavor of managing CI,” said John Koconis, Chairman and Chief Executive Officer of Timber Pharmaceuticals. “Many dermatologists may be familiar with oral isotretinoin and its effectiveness as a treatment for CI, but they are also aware of the systemic toxicity associated with oral therapy. We believe that TMB-001 has the potential to deliver significant efficacy and relief to patients while minimizing systemic absorption, thereby lowering the rates of side effects seen with the oral compounds. Our patented topical formulation might allow this therapy to be used chronically and over larger areas of the body.”
CI is a group of rare genetic keratinization disorders that lead to dry, thickened, and scaling skin. The randomized, parallel, double-blind, vehicle-controlled ASCEND study is designed to enroll 142 participants 6 years of age or older with moderate to severe CI including recessive X-linked ichthyosis (RXLI) and autosomal recessive congenital ichthyosis (ARCI). These subtypes affect about 80,000 people in the U.S. and lead to cutaneous manifestations that include large, dark scaling throughout the body. Timber is providing genetic testing as part of the study for patients whose subtype of CI is not already genetically confirmed.
Timber Pharmaceuticals notes participants in the ASCEND study are randomized 2:1 to TMB-001 or vehicle control ointment (two participants on TMB-001 for every one participant on vehicle) for 12 weeks, at which point eligible participants in both arms of the study are randomized again to either once-a-day or twice-a-day TMB-001 treatment for an additional 12 weeks to provide valuable information on longer term treatment with the compound.
