CASE STUDY

Dermal drug development for SLS associated ichthyosis

Aldeyra Therapeutics

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Sjögren-Larsson Syndrome (SLS) is a rare inborn error of aldehyde metabolism characterized by severe skin thickening (ichthyosis), retinal disease, and neurological dysfunction. As of today, no therapy that addresses SLS has been approved. Aldeyra Therapeutics has conducted a Phase 2 randomized, parallel-group, double-blind, vehicle-controlled, multi-center clinical trial of topical dermal reproxalap for the treatment of ichthyosis associated with SLS. The results of the trial demonstrated clinically meaningful and statistically significant improvements of SLS skin symptoms of patients after only eight weeks of treatment. A multi-center Phase 3 clinical trial is ongoing.

Lipotype Shotgun Skin Lipidomics provided Aldeyra Therapeutics with lipid analysis of skin samples for identification of relevant lipid biomarkers in the Phase 2 clinical trial. Results of the skin lipidomics analysis delivered evidence for a correlation between clinical effects and those skin lipid biomarkers. Consistent with the clinical activity of reproxalap, significant dermal biomarker changes were noted in reproxalap-treated patients but not vehicle-treated patients. Cholesterol, which is significantly elevated in the skin of SLS patients, was reduced in reproxalap-treated patients more than in vehicle-treated patients.

Aldeyra Therapeutics
Aldeyra Therapeutics is a biotechnology company devoted to improving lives by inventing, developing, and commercializing next-generation medicines that treat immune-mediated and metabolic diseases. Aldeyra’s lead product candidate, reproxalap (formerly called NS2), is a small molecule RASP (Reactive Aldehyde Species) inhibitor in Phase 3 clinical development for the treatment of dry eye disease, allergic conjunctivitis, noninfectious anterior uveitis, and Sjögren-Larsson syndrome. With its focus on a carefully selected range of diseases, Aldeyra Therapeutics is devoted to improving lives through outstanding innovation.

Reference
Aldeyra therapeutics announces positive results from a randomized, double-blind, vehicle-controlled clinical trial of topical dermatologic nS2 in patients with Sjögren-Larsson syndrome – Aldeyra Therapeutics, 2016