With a focus on neurological disorders, NFlection is a biopharmaceutical R&D firm addressing treatment of growth of tumors on nerves engaging novel therapies involving neurofibromatosis type 1, immunosuppressant-mediated squamous cell carcinoma, and congenital birthmarks such as keratinocytic epidermal nevi and nevus sebaceous. These rare disorders - also referred to as RASopathies (driven by the aberrant activation of the Ras/Raf/MEK/ERK pathway,) the firm is developing first-in-class MEK (mitogen-activated protein kinase kinase) inhibitors as topical treatments. NFlection had been founded to tackle developing proprietary, novel compounds that are highly selective mitogen-activated protein kinase kinase (MEK) inhibitors to mitigate or treat cutaneous neurofibromas in neurofibromatosis type 1, congenital birthmarks, and immunosuppressant-mediated squamous cell carcinoma. The firm's first proprietary MEK inhibitor, NFX-179, is formulated in a cosmetically elegant gel that is applied to the skin, enabling local suppression of the Ras/Raf/MEK/ERK pathway that drives the pathology of rare cutaneous RASopathies. NFX-179 is a metabolically labile âsoft drugâ that is rapidly broken down in the bloodstream so it no longer has the ability to inhibit MEK systemically. Designing the firm's compounds as soft drugs are an effort to eliminate significant systemic adverse events observed with orally administered MEK inhibitors, such as rash, diarrhea, nausea, vomiting, edema, fatigue, high blood pressure, cardiomyopathy, and retinal detachment. NFlection management plan to submit an Investigational New Drug Application with the Food and Drug Administration for NFX-179 and begin human studies in patients with neurofibromatosis type 1 and congenital birthmarks in 2020 and further to evaluate NFX-179 in patients with immunosuppressant-mediated squamous cell carcinoma in 2021. Facilities indicated both in PA and
