Significant Progress in Timber Pharmaceuticals’ Phase 3 ASCEND Study
Timber Pharmaceuticals (NYSE American: TMBR), a clinical-stage biopharmaceutical company specializing in novel therapies for rare and orphan dermatologic diseases, recently announced that 70% enrollment has been achieved in their pivotal Phase 3 ASCEND study. This study focuses on the investigation of TMB-001 (topical isotretinoin) as a potential treatment for moderate to severe congenital ichthyosis (CI), a rare genetic skin disorder.
FDA Grants CARC Waiver to TMB-001, Accelerating its Development
In a major breakthrough, the U.S. Food and Drug Administration (FDA) has granted a dermal carcinogenicity (CARC) waiver for TMB-001, which is formulated using Timber’s patented IPEG™ delivery system. This waiver comes on the back of a 39-week repeat dose dermal toxicity study, which exhibited no evidence of skin or organ carcinogenicity from chronic applications of TMB-001 in rodents. This CARC waiver not only eliminates the need for a 2-year dermal rodent carcinogenicity study but also expedites the development process of this promising therapy.
Timber Pharmaceuticals’ Commitment to Advancing CI Treatment
“High-dose and chronic oral therapy cannot be tolerated by most patients due to systemic toxicity,” explained John Koconis, Chairman and Chief Executive Officer of Timber. He further stressed Timber’s commitment to providing a new topical treatment for people living with moderate to severe CI. Achieving the CARC waiver will aid in circumventing a costly and prolonged non-clinical study, facilitating swift and efficient progress with the TMB-001 program.
The Potential Impact of TMB-001 on CI
CI is characterized by dry, thickened, and scaling skin. The condition affects around 80,000 people in the U.S., with TMB-001 currently being evaluated as a treatment for its moderate to severe subtypes, including lamellar ichthyosis and X-linked ichthyosis.
In 2018, the FDA acknowledged the potential of TMB-001 by awarding an Orphan Products Grant to support clinical trials, including the Phase 3 ASCEND study. Furthermore, Timber received both Breakthrough Therapy Designation and Fast Track Status from the FDA for TMB-001, attesting to the treatment’s potential to address a significant unmet medical need.
TMBR Market Response
Shares of Timber Pharmaceuticals are rallying 40% through early trading on Monday, June 5, 2023. Over the past three months, the biotech company has seen average daily volume of 61,460 shares. However, volume of 16.11 million shares or dollar volume of around $35.54 million, has already exchanged hands through early trading. As of this writing, shares of TMBR has returned 15.41% year-to-date.