NFlection Therapeutics Inc., a US-based company developing topical MEK inhibitors for RAS-mediated skin conditions, announced on Monday positive topline results from a randomised, double-blind, vehicle-controlled Phase 2b clinical trial (NCT05005845) assessing NFX-179 Gel as a treatment for cutaneous neurofibromas (cNFs) in subjects with neurofibromatosis type 1 (NF1), a rare genetic condition.

The Phase 2b study was conducted in 199 subjects to assess the safety and effectiveness of NFX-179 Gel 0.5% and 1.5% compared with vehicle in subjects with cNFs. The study was carried out at 24 investigational centres in the US.

NFX-179 Gel was applied topically to target cNF tumours for six months, enabling delivery of high concentrations of NFX-179 to the tumours. The company says that the highest concentration of NFX-179 tested, 1.5%, achieved the study's primary efficacy endpoint, with a statistically significant improvement over vehicle in the shrinkage of cNFs in people with NF1 that was highly correlated with patient-reported meaningful improvements in their cNFs. Consistent with its design as a soft, or metabolically labile, MEK inhibitor, NFX-179 Gel was generally well tolerated, and plasma drug concentrations were orders of magnitude lower than those typically observed for oral MEK inhibitors.