Timber initiates phase 3 trial of topical isotretinoin for congenital ichthyosis

Timber Pharmaceuticals has enrolled its first four patients in the phase 3 trial of its topical isotretinoin formula for the treatment of congenital ichthyosis, the company announced in a press release.

TMB-001 0.05% is formulated using Timber’s patented IPEG delivery system and was granted an orphan drug designation by the FDA.

“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 lifelong endeavor of managing [congenital ichthyosis (CI)],” John Koconis, chairman and CEO of Timber, said in the release. “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.”

The randomized, parallel, double blind, vehicle-controlled phase 3 ASCEND trial is expected to enroll 140 patients with autosomal recessive CI or recessive X-linked ichthyosis.

Subjects will be randomly assigned 2:1 to receive TMB-001 or vehicle control ointment for 12 weeks.