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Effect of NFX-179 MEK inhibitor on cutaneous neurofibromas in persons with neurofibromatosis type 1. Science advances Sarin, K. Y., Bradshaw, M., O'Mara, C., Shahryari, J., Kincaid, J., Kempers, S., Tu, J. H., Dhawan, S., DuBois, J., Wilson, D., Horwath, P., de Souza, M. P., Powala, C., Kochendoerfer, G. G., Plotkin, S. R., Webster, G. F., Le, L. Q. 2024; 10 (18): eadk4946

Abstract

This phase 2a trial investigated the efficacy of NFX-179 Topical Gel, a metabolically labile MEK inhibitor, in the treatment of cutaneous neurofibromas (cNFs) in neurofibromatosis type 1. Forty-eight participants were randomized to four treatment arms: NFX-179 Topical Gel 0.05%, 0.15%, and 0.5% or vehicle applied once daily to five target cNFs for 28 days. Treatment with NFX-179 Topical Gel resulted in a dose-dependent reduction in p-ERK levels in cNFs at day 28, with a 47% decrease in the 0.5% NFX-179 group compared to the vehicle (P = 0.0001). No local or systemic toxicities were observed during the treatment period, and systemic concentrations of NFX-179 remained below 1 ng/ml. In addition, 20% of cNFs treated with 0.5% NFX-179 Topical Gel showed a =50% reduction in volume compared to 6% in the vehicle group by ruler measurement with calculated volume (P = 0.021). Thus, NFX-179 Topical Gel demonstrated significant inhibition of MEK in cNF with excellent safety and potential therapeutic benefit.

View details for DOI 10.1126/sciadv.adk4946

View details for PubMedID 38691597