MISSOULA, Montana, Nov. 20, 2018 – DermaXon LLC, Montana, an early stage biotechnology company, focused on skin disorders and pain, has been awarded a $1,500,000 Phase II Small Business Innovative Research Grant (SBIR) from The National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), part of the National Institutes of Health (NIH), to further develop their proprietary lead CYP26 inhibitor candidate for the treatment of ichthyosis.
The NIH SBIR program is a highly competitive program aimed at translating cutting edge research with health, economic and societal benefits into the commercialization of innovative drug products. This new two-year grant follows the successful completion of a previous Phase I SBIR award granted to DermaXon by NIH, covering the selection of a CYP26 selective inhibitor development candidate for pre-clinical studies.
DermaXon’s project goal is to develop efficacious and highly selective inhibitors of CYP26s, the enzymes responsible or retinoic acid (RA) metabolism in the epidermis, for the topical treatment of ichthyosis. This approach will provide a therapeutic advantage in ichthyosis without the adverse effects mediated by RA and the marketed retinoid derivatives resulting in poor health-related quality of life and patient compliance.
DX308, is a potent inhibitor of RA clearance, targeting cytochrome p450 26 retinoic acid hydroxylase enzymes. This candidate compound has a unique design based on RA structure. DermaXon’s, novel CYP26 inhibitor candidate, DX308 has already shown efficacy in different human three dimensional models of ichthyosis without showing any toxicity in in vivo models. DermaXon has already conducted a series of studies to evaluate initial toxicity, pharmacology and metabolic properties of DX308 to establish initial safety parameters and dosing strategies. This inhibitor by only increasing atRA endogenous concentration without interacting with the RA receptors, will have the therapeutic efficacy of retinoids, without inducing the adverse effects associated with exogenously administered retinoids.
Congenital ichthyosis is a family of rare and neglected genetic disorders of keratinization characterized by dry and scaling skin, resulting in itching and pain, The disease makes patients prone to numerous other complications like dehydration, infections and chronic blistering. Due to the appearance of chronically shedding skin, many patients experience accompanying psychological issues such as depression, low self-esteem and even psychosocial disruption, greatly impacting the quality of life of patients and their family members.
“There is no cure for ichthyosis. Current treatments only relieve the symptoms, but no cure has been found to date” said Philippe Diaz, PhD, CSO and co-founder of DermaXon and professor at the University of Montana. Available (off-label) treatments do not adequately address patient needs, presenting significant efficacy or tolerability concerns. “This phase II award will allow DermaXon to initiate IND-enabling studies for our CYP26 inhibitor candidate, DX308, and offer a future treatment option for ichthyotic patients and patients suffering from other keratinization disorders in general” further added Pr. Diaz.
“We are very pleased for this SBIR funding as it will support continued pre-clinical advancement of DX308 and will allow DermaXon to progress more rapidly toward a treatment of keratinization disorders and set us on a path toward a cure for ichthyosis” said Fanny Astruc Diaz, COO and co-founder of DermaXon.
Supported by the data generated during this phase II award, our DX308 compound is expected to enter clinical testing in 2020.
The estimated annual cost for congenital ichthyoses is estimated to $37MM per year (excluding ichthyosis vulgaris).The estimated out-of-pocket costs for off-label drugs associated with ichthyosis is estimated to $17 MM per year. The availability of a prescription drug that will be reimbursed will transform the patient’s quality of life by decreasing their medication costs.