NFlection Therapeutics Announces Publication of Clinical Data Demonstrating the Effect of NFX-179 Topical MEK Inhibitor on Cutaneous Neurofibromas in Persons with Neurofibromatosis Type 1

NFlection Therapeutics Announces Publication of Clinical Data Demonstrating the Effect of NFX-179 Topical MEK Inhibitor on Cutaneous Neurofibromas in Persons with Neurofibromatosis Type 1

  • Preclinical data and results of Phase 2a clinical study published in Science Advances
  • First-in-human study demonstrated an excellent safety profile and potential therapeutic benefit for cutaneous neurofibromas based on biomarker and early clinical endpoints
  • NFlection’s lead candidate NFX-179 recently completed a successful Phase 2b study demonstrating meaning cutaneous neurofibroma size reduction after six months of daily therapy

BOSTON, MA, May 1, 2024 – NFlection Therapeutics Inc., a company developing topical MEK inhibitors for RAS-mediated skin conditions, today announced the publication of the results of preclinical studies and a Phase 2a clinical study in Science Advances.

In a randomized, double-blind, vehicle-controlled Phase 2a trial (NCT04435665) of NFX-179 Topical Gel in persons with neurofibromatosis type 1 (NF1) across six centers in the United States, NFX-179 Gel 0.05%, 0.15%, and 0.5% (w/w) or vehicle was applied once daily to 5 target cutaneous neurofibroma (cNF) tumors per participant for 28 days. Forty-seven participants completed treatment.  Treatment with NFX-179 Topical Gel led to a dose-related reduction in the established phosphorylated ERK (pERK) biomarker in treated cNFs at Day 28 with a 47% reduction in pERK levels in the 0.5% NFX-179 Topical Gel group compared with vehicle (p= 0.0001, ANOVA). No treatment-related local or systemic toxicities were observed. Twenty percent of cNF tumors treated with 0.5% NFX-179 Topical Gel had a ≥50% reduction in volume that is considered clinically meaningful, versus 6% in the vehicle group (P = 0.021, ANOVA). Kavita Sarin, MD PhD, lead author of the publication stated “This first-in human study of NFX-179 Topical gel demonstrates proof-of concept for NFX-179 Gel, and its potential as the first topical therapy for the treatment of cNFs in people with NF1.”

Reference: Kavita Y. Sarin et al., Effect of NFX-179 MEK inhibitor on cutaneous neurofibromas in persons with neurofibromatosis type 1. Sci. Adv. 10, eadk4946 (2024). DOI: 10.1126/sciadv.adk4946

About NFX179 Topical Gel

NFX‑179 is an investigational mitogen-activated protein kinase kinase (MEK) inhibitor. NFX‑179 is a “soft” (metabolically labile) drug, which, when formulated as NFX‑179 Gel for topical application, is designed to concentrate at the dermal site of action but degrade in systemic circulation, thereby significantly reducing side effects routinely seen with systemically available MEK inhibitors. NFlection is developing NFX‑179 Gel for the treatment of cutaneous neurofibromas in people with neurofibromatosis type 1 and has received Orphan Designation in the United States and European Union for this indication. NFlection recently announced the results from the successful completion of a large Phase 2b study (NCT05005845) demonstrating once-daily topical treatment of target cNFs with NFX‑179 Gel 1.5% achieved statistically significant reduction in size.

About NFlection Therapeutics Inc.

NFlection is a clinical-stage biopharmaceutical company focused on the development of novel therapies to address the needs of patients with neurofibromatosis type 1, immunosuppressant-mediated squamous cell carcinoma, and congenital birthmarks such as keratinocytic epidermal nevi and nevi sebacei. To address these RAS-mediated disorders driven by the aberrant activation of the Ras/Raf/MEK/ERK pathway, we are developing first-in-class MEK (mitogen-activated protein kinase kinase) inhibitors for topical treatment of these conditions. To learn more about the company, please visit

For more information about NFlection Therapeutics please contact:

To reach Kavita Sarin, MD PhD, please contact:
Tel: +1 (650) 804-2899