FDA Grants Breakthrough Therapy Designation to Timber’s Treatment for Congenital Ichthyosis

The U.S. Food and Drug Administration has granted Breakthrough Therapy designation to Timber Pharmaceuticals’ TMB-001, a topical isotretinoin formulated using the company’s patented IPEG delivery system, for the treatment of congenital ichthyosis.

Photo: John Koconis, chairman and CEO of Timber

“This is a significant moment for people who are living with congenital ichthyosis (CI), their families and caregivers, and clinicians and researchers who have been working for years to find new treatment options for this debilitating condition,” said John Koconis, chairman and CEO 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 Through half of FDA’s 2022 fiscal year, more breakthrough applications have been rejected by FDA or withdrawn than have been granted. 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.”

Breakthrough Therapy designation is a process designed to expedite the development and review of drugs that are intended to treat serious or life-threatening conditions. Preliminary clinical evidence must indicate that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint.

Congenital ichthyosis (CI) is a group of rare genetic keratinization disorders that lead to dry, thickened, and scaling skin. In patients with XRI and ARCI-LI, cutaneous manifestations include large, dark scaling throughout the body.

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 2022 Annual Meeting showed patients achieved treatment success with TMB-001 regardless of the subtype of CI.

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 United States, 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.

Author: Rare Daily Staff