– Trial designed to study 0.05%, 0.15% and 0.5% NFX-179 Gel in subjects with cutaneous neurofibromatosis type 1 (NF1)
– NFX-179 Gel was well tolerated, with a safety profile similar to placebo (vehicle)
– Primary endpoint achieved: NFX-179 Gel showed a dose-dependent suppression of phosphorylated ERK (p-ERK) in cutaneous neurofibroma (cNF) tumors, suppressing p-ERK by 47% (p = 0.0001; 0.5% dose) and 26% (p = 0.04; 0.15% dose) versus vehicle
– Data supports progression to Phase 2b, set to commence in the second half of 2021
WAYNE, Pa., May 26, 2021 /PRNewswire/ — NFlection Therapeutics, Inc., a clinical-stage biopharmaceutical company focused on the development of targeted therapies for rare disorders driven by aberrant activation of the RAS pathway (RASopathies), today announced positive results from a 28-day, Phase 2a, multicenter, randomized, double-blind, parallel-group, vehicle-controlled clinical trial investigating the safety, tolerability, pharmacokinetics and pharmacodynamics of NFX-179 Gel in subjects with NF1. Topical application of NFX-179 Gel is designed to deliver a proprietary “soft” (metabolically labile) MEK inhibitor to cNF tumors to suppress the overactivation of the Ras/Raf/MEK/ERK pathway in these tumors while avoiding the systemic toxicities of orally administered MEK inhibitors, which have not been approved for this indication.
In the trial, 48 subjects were randomized in a 1:1:1:1 ratio to receive once-daily NFX-179 Gel at 0.05%, 0.15%, or 0.5%, or placebo (vehicle), for 28 days. The primary endpoints were safety, tolerability and suppression of p-ERK, a key biomarker known to promote the growth of cNF tumors.