TMB-001, a topical isotretinoin formulated using patented IPEG™ delivery system from Timber Pharmaceuticals, Inc. has been granted Breakthrough Therapy designation by FDA for the treatment of congenital ichthyosis (CI).

Timber is developing TMB-001 for the treatment of moderate to severe forms of CI, including X-linked recessive ichthyosis (XRI) and autosomal recessive congenital ichthyosis lamellar ichthyosis (ARCI-LI). In the Phase 2b CONTROL study, treatment with TMB-001 demonstrated a clinically meaningful reduction in targeted and overall severity of CI along with a favorable safety profile. A sub-analysis of the study presented at the American Academy of Dermatology (AAD) 2022 Annual Meeting showed patients achieved treatment success with TMB-001 regardless of the subtype of CI. Editor's note: Read more in the May edition of Practical Dermatology.

“This is a significant moment for people who are living with CI, their families and caregivers, and clinicians and researchers who have been working for years to find new treatment options for this debilitating condition,” says John Koconis, Chairman and Chief Executive Officer of Timber. “A Breakthrough Therapy designation is no small achievement. Through half of FDA’s 2022 fiscal year, more breakthrough applications have been rejected by FDA or withdrawn (16), than have been granted (9). I am proud of our team for demonstrating the potential of TMB-001 in our Phase 2b program, and we are rapidly pushing forward with a pivotal Phase 3 clinical trial.”

Timber has initiated the pivotal Phase 3 ASCEND clinical trial to further investigate the efficacy and safety of TMB-001 for the treatment of CI at leading research centers in the US, Canada, Italy, France, and Germany and is expecting to dose the first patients in June 2022. The ASCEND trial will evaluate the efficacy, pharmacokinetics, and safety of TMB-001 0.05% in 142 patients with moderate to severe CI.