XenoPort, Inc., a biopharmaceutical company focused on developing and commercializing a portfolio of internally discovered product candidates for the potential treatment of neurological disorders, has submitted an Investigational New Drug (IND) application to the US Food and Drug Administration (FDA) to begin clinical studies of XP23829 as a potential treatment for relapsing-remitting multiple sclerosis (RRMS).
Following clearance of the IND by the FDA, the first phase 1 clinical trial to be conducted in healthy subjects will commence. This study is intended to examine the safety, tolerability and pharmacokinetics of XP23829, including confirmation of its conversion to monomethyl fumarate (MMF), and the performance of four novel formulations of XP23829 designed to have different drug release mechanisms and/or time profiles.
Ronald W. Barrett, Ph.D., chief executive officer of XenoPort, stated, "We are pleased to take this step in advancing the development of XP23829. In addition to our filing the IND to initiate human clinical trials for the potential treatment of RRMS, we also recently demonstrated that XP23829 and related compounds were effective in an animal model of psoriasis. We are hopeful that our first Phase 1 clinical trial will provide evidence of safety and tolerability and also provide pharmacokinetic data that might support once-a-day dosing of XP23829. Our long-term goal is to create a best-in-class fumaric acid ester-based medicine for the potential treatment of RRMS and/or psoriasis."
XP23829 is a prodrug of MMF (also known as methyl hydrogen fumarate). In cell- and animal-based models, MMF has been shown to exhibit immuno-modulatory properties and inhibit damage from oxidative stress. In XenoPort's preclinical animal studies that compared molar equivalent doses of XP23829 to dimethyl fumarate (DMF), another prodrug of MMF, XP23829 demonstrated a greater degree of efficacy in animal models of both multiple sclerosis and psoriasis. Toxicology studies conducted in two species showed that XP23829 caused less stomach irritation compared to DMF.
XenoPort was awarded US Patent 8,148,414 for "Prodrugs of Methyl Hydrogen Fumarate, Pharmaceutical Compositions Thereof, and Methods of Use." The patent is directed to the XP23829 compound and analogs and formulations thereof. The term of the patent runs until 2029, subject to potential patent term extensions under the Hatch-Waxman Act.