Timber Gets Second Tranche of $500K from FDA Trials Grant for Rare Skin Disease Therapy

Rare Daily Staff

Photo: John Koconis, CEO of Timber

The U.S. Food and Drug Administration Office of Orphan Products Development has awarded Timber Pharmaceuticals the second tranche of $500,000 of the $1.5 million grant from its Orphan Products Clinical Trials Grants Program after reaching certain clinical milestones in the development of its experimental therapy for a rare skin condition.

The FDA’s Orphan Products Clinical Trials Grants Program is designed to support the clinical development of products for use in rare diseases where no current therapy exists or where the proposed product will be superior to the existing therapy.

In 2018, Timber was one of four biopharmaceutical companies to be awarded the grant in addition to hospitals and academic institutions. The total grant of $1.5 million is non-dilutive and supports the phase 2a and phase 2b studies evaluating the company’s experimental therapy TMB-001 in congenital ichthyosis (CI), a rare genetic keratinization disorder that leads to dry, thickened, and scaling skin.

TMB-001 (topical isotretinoin) is being developed for the treatment of moderate to severe subtypes of CI. In a phase 2a study, treatment with TMB-001 was shown to be well tolerated with minimal evidence of systemic absorption of isotretinoin. There was a favorable signal of efficacy in the study with patients showing a reduction in scaling after eight weeks. The company is currently enrolling patients in the phase 2b CONTROL Study focused on moderate to severe subtypes of CI that affect about 80,000 people in the U.S.

“We believe this is a clear sign that we are making progress in the development of TMB-001 and we look forward to advancing through the late stages of clinical research,” said John Koconis, CEO of Timber.