TMB-001 Granted FDA Breakthrough Therapy Designation

Timber Pharmaceuticals announced that TMB-001, a topical isotretinoin formulation using Timer Pharmaceutical’s IPEG delivery system, has received a breakthrough therapy designation from the FDA for the treatment of congenital ichthyosis (CI). CI is a rare genetic keratinization disorder, according to the press release, that can lead to dry, thickened, and scaling skin. Patients with CI may have a litany of issues such as limited range of motion, chronic pruritis, an inability to sweat, and a high risk of secondary infections.

As of now, there are no FDA approved treatments for CI. “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,” said John Koconis, chairman and CEO of Timber, in the press release. “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 Through half of FDA’s 2022 fiscal year, more breakthrough applications have been rejected by FDA or withdrawn (16), than have been granted (9).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.”

The designation was created to speed up the development and review of treatments that are intended to treat serious or life-threatening conditions. To recieve the designation, the FDA does require preliminary clinical evidence of the drug's efficacy on a clinically significant endpoint. This data was acquired from the the phase 2b CONTROL (NCT04154293) trial evaluating TMB-001 in moderate-to-severe CI which was completed in September of 2021. Results of which can be found here.

TMB-001 was created 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), the press release explains.

The phase 3 ASCEND (NCT05295732) clinical trial, a study the company plans to start dosing patients in June 2022, will further investigate the safety and efficacy of TMB-001.

Reference

Timber pharmaceuticals receives FDA breakthrough therapy designation for TMB-001 for the treatment of congenital ichthyosis. Published May 31, 2022. Accessed May 31, 2022. https://www.yahoo.com/now/timber-pharmaceuticals-receives-fda-breakthrough-120000350.html