- First Presentation of Data From Phase 3 ASCEND Study Will Feature Pharmacokinetic Results That Indicate Minimal Systemic Absorption of Isotretinoin -
- Data to be Presented at the Society for Pediatric Dermatology’s Annual Meeting -
“Many dermatologists are familiar with oral isotretinoin, but high-dose and chronic oral isotretinoin therapy required in the treatment of CI cannot be tolerated by many patients due to systemic toxicity,” said
The Phase 3 ASCEND study (https://ichthyosistrial.com/about_ascend_study.htm) is an ongoing double-blind, vehicle-controlled randomized 12-week study followed by 12 weeks of open label treatment of patients who are greater than 6 years of age with moderate to severe X-linked recessive CI (XLRI) or autosomal recessive CI (ARCI). The primary endpoint is the difference in proportions of treated patients with 2-point Investigator's Global Assessment (IGA) scaling/fissuring score improvement from baseline at 12 weeks. A subset of patients is also participating in a 12-week open label Maximal Use Study (MuST) that includes the collection of PK data as well as safety and efficacy data.
The interim PK analyses include the first nine patients who completed 15 days of the MuST study. The results indicate minimal absorption of isotretinoin when TMB-001 0.05% is applied to 75-90% of the body surface area. The 9 subjects (7 ARCI, 2 XLRI; ages 12-62) demonstrated mean plasma concentrations (ng/mL) of isotretinoin at Day 14 of 4.83 (range: 1.46-16.59), 4-oxo-isotretinoin of 13.71 (6.52-47.86), tretinoin of 0.08 (0-0.66) and 4-oxo-tretinoin of 0.
“The dry, thickened, and scaling skin that people living with these rare genetic keratinization disorders must deal with can be seriously debilitating, especially in the most severe cases of XLRI and ARCI,” said
Timber has received both Breakthrough Therapy Designation and Fast Track Status from the
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