Purpose of Study

The purpose of this study is to test the safety and effectiveness of 2 concentrations of NFX-179 gel compared with placebo gel in reducing the appearance of cutaneous neurofibromas (cNFs) in subjects with Neurofibromatosis Type 1 (NF1).

The Study Drug

  • NFX-179 Gel
  • NFX-179 Gel is a topical, first-in-class, “soft” MEK inhibitor that is currently being evaluated in Phase 2 clinical trials in patients with neurofibromatosis type 1

The Study

  • At Visit 1, the study doctor assesses patient eligibility and identifies 10 cutaneous neurofibromas for treatment with study medication
  • Patients must have 10 cutaneous neurofibromas: 8 on upper body and 2 on face OR 9 on upper body and 1 on face
  • The topical study medication will be applied once daily on the 10 cutaneous neurofibromas for 6 months
  • Patients who qualify may be reimbursed for time and travel

Study Expectations

  • There is NO cost to participate
  • Participation in the study will last approximately 6 months
  • The patient will apply the topical study medication once daily to the 10 cutaneous neurofibromas
  • Study doctor visits will occur at least 9 times over 6 months

Will Everyone Receive the Active Concentration of NFX-179 Topical Gel?

  • 1/3 patients will receive study medication with an active concentration of NFX-179 Gel (0.5%)
  • 1/3 patients will receive study medication with an active concentration of NFX-179 Gel (1.5%)
  • 1/3 of patients will receive study medication with placebo concentration of NFX-179 Gel (0.0%)
  • The study doctor and patient will not know which patient receives an active or placebo concentration of NFX-179
  • Active or placebo concentrations NFX-179 Gel are randomly selected

Study Patients Must Meet The Following Criteria

  • Ages 18+
  • Diagnosed with neurofibromatosis type 1
  • Have 10 clinically diagnosed cNFs
  • At least 1 cutaneous neurofibroma on face

About NFX-179 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 potentially significantly reducing side effects compared to systemically available MEK inhibitors.

NFlection Therapeutics, Inc. has recently completed a successful Phase 2 study. Read more here.

About Cutaneous
Neurofibromatosis Type 1

Cutaneous neurofibromas are tumors that grow from small nerves in the skin or just under the skin and appear as small or larger bumps typically beginning around the time of puberty. Individuals with neurofibromatosis type 1, commonly develop more cutaneous neurofibromas as they get older. They do not become malignant, but they may be disfiguring, itchy or painful when bumped. Despite their benign nature, they may cause significant problems (e.g., depression, isolation, etc.), and may require surgical removal.

About NFlection
Therapeutics, Inc.

NFlection Therapeutics focuses on the discovery and development of effective, targeted therapies for rare disorders. NFlection is chiefly concerned with rare disorders known as RASopathies, which are driven by the aberrant activation of the Ras/Raf/MEK/ERK pathway. NFlection is working to address RASopathies through the development of first-in-class soft MEK (mitogen-activated protein kinase kinase) inhibitors as topical treatments to mitigate or treat cutaneous neurofibromas in neurofibromatosis type 1. NFlection’s topical MEK inhibitors are designed to degrade rapidly in circulation to avoid systemic side effects. To learn more about the company, please visit www.nflectionrx.com.