Sjögren-Larsson Syndrome

August 15, 2016

Aldeyra Therapeutics released results of a randomized, parallel-group, double-blind, vehicle-controlled, multicenter trial of topical dermatologic NS2 for the treatment of the dermal manifestations of Sjögren-Larsson Syndrome (SLS). Twelve subjects with SLS and moderate to severe ichthyosis were randomized equally to receive NS2 1.0% dermatologic formulation or vehicle formulation administered once daily (QD) on a 4x10 inch area of skin for two months. Ichthyosis was graded by a central review of digital photographs, as well as by clinical exam, using the Ichthyosis Severity Score, which is comprised of assessments of global impression, scaling, erythema (redness), lichenification (thickness) and excoriation (abrasion). In addition, dermal tissue from subjects was analyzed for biomarkers relevant to fatty aldehyde dehydrogenase deficiency. NS2 consistently produced clinically meaningful effects in reducing the severity of ichthyosis. As assessed by central review, five of six subjects (83%) treated with NS2 achieved a rating of “almost clear” or “mild” on global assessment. Six of six (100%) subjects treated with NS2 improved over the course of therapy as assessed by central review, and the improvement was greater than that observed with vehicle-treated patients (p<0.05). For NS2-treated subjects, mean reductions in ichthyosis severity were greater after eight weeks of therapy than after four weeks of therapy, suggesting a disease modifying effect of NS2. Consistent with the clinical activity of NS2, significant dermal biomarker changes were noted in NS2-treated patients but not vehicle-treated patients (p<0.004). Cholesterol, which is significantly elevated in the skin of SLS patients, was reduced in NS2-treated patients more than in vehicle-treated patients (p<0.001). NS2 was observed to be generally well-tolerated and there were no significant adverse events, serious adverse events or discontinuations in the trial.