Neurofibromatosis Type 1
NFlection is developing a topical gel containing NFX-179, a proprietary “soft” (metabolically labile) MEK inhibitor for the topical treatment of persistently developing cutaneous neurofibromas (cNF) associated with neurofibromatosis type 1 (NF1).
In a mouse model of NF1, mice given MEK inhibitors for two months showed significant decreases in neurofibroma tumor volume. Additionally, an oral MEK inhibitor is approved for the treatment of plexiform neurofibromas in NF1 patients. Despite promising data demonstrating that oral MEK inhibitors may be efficacious in treating cutaneous neurofibromas, systemic exposure from oral MEK inhibitors commonly leads to significant adverse effects such as severe acneiform rash, diarrhea, nausea, vomiting, edema, fatigue, high blood pressure, cardiomyopathy, and retinal detachment.