About NS

Netherton syndrome is a serious, rare, lifelong genetic disorder affecting the skin, hair, and immune system, caused by lack of normal function of a natural inhibitor of KLK⁵. People with Netherton syndrome often have red, scaly, inflamed skin, fragile hair, and are more likely to develop skin infections, allergies, asthma, and eczema. Netherton syndrome can be life-threatening, especially during infancy when patients are vulnerable to dehydration and recurrent infections.¹ Currently, there are no approved treatments for Netherton syndrome.²

We are developing BCX17725, a potential best-in-class protein therapeutic that is designed to treat the underlying protein deficiency that causes Netherton syndrome by inhibiting KLK5, a serine protease in the skin that is unregulated in people with Netherton syndrome. Data from nonclinical studies have shown the potential for BCX17725 to achieve the potency, specificity, and convenient dosing needed to treat patients with Netherton syndrome. Initial results from a Phase 1 clinical trial evaluating BCX17725 are expected in 2025.