RARE Daily

NFlection Therapeutics Launches with $20 Million to Advance Therapy for Neurofibromatosis

June 10, 2020

Rare Daily Staff

NFlection Therapeutics launched with $20 million in a series A financing from venBio Partners and F-Prime Capital that it will use to discover and develop therapies for rare diseases.

NFlection’s focus is rare disorders known as RASopathies through the development of first-in-class soft MEK (mitogen-activated protein kinase) inhibitors as topical treatments to mitigate or treat cutaneous neurofibromas in neurofibromatosis type 1, congenital birthmarks, and immunosuppressant-mediated squamous cell carcinoma. The topical MEK inhibitors are designed to degrade rapidly in circulation to avoid systemic side effects.

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. It is less common to see this type of neurofibroma in young children with neurofibromatosis type 1 (NF1), a rare autosomal dominant disorder caused by mutations in the NF1 gene. Individuals with NF1 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, cutaneous neurofibromas may cause significant problems, such as depression and a sense of isolation, and may require surgical removal.

NFlection has started the first clinical trial of its lead experimental product, NFX-179 Gel, a topically applied therapy for the treatment of cutaneous neurofibromatosis type 1.

The phase 2a study will be a safety, tolerability and pharmacokinetic/pharmacodynamic study of NFX-179 Gel in adult patients with cutaneous NF-1. The study will evaluate three concentrations of NFX-179 Gel compared with placebo in a randomized, double-blind, placebo-controlled, clinical trial of 48 patients with cutaneous NF1. The study will be conducted at five investigational centers across the United States.

“This study allows us to determine the drug’s ability to suppress key biomarkers involved in the progression of neurofibromas and will aid in the selection of doses for a larger phase 2b study,” said Christopher Powala, president and chief executive officer of NFlection. “There are no FDA-approved products for this condition, and NFX-179 Gel has the potential to address this unmet medical need.”

Photo: Christopher Powala, president and chief executive officer of NFlection.


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