US-based biotechnology company Aldeyra Therapeutics reported positive clinical trial results of topical dermatologic NS2 to treat Sjögren-Larsson Syndrome (SLS).
The NS2 is developed as an aldehyde trap, specifically targeting and reducing levels of toxic aldehydes that are toxic, as well as pro-inflammatory agents of numerous diseases.
The randomised, parallel-group, double-blind, vehicle-controlled, multi-centre trial involved 12 patients suffering from moderate to severe ichthyosis, which is a dermal manifestation of SLS.
The patients were randomised to receive NS2 1.0% dermatologic formulation or vehicle formulation on a once-daily regimen applied on 4in x 10in area of skin for two months.
Both the patients and investigators were blinded to treatment group and ichthyosis was examined by a blinded central review of digital photographs.
The clinical exam was reviewed on a blinded basis by the Ichthyosis Severity Score, which comprises assessments of global impression, scaling, erythema (redness), lichenification (thickness) and excoriation (abrasion).
Additionally, dermal tissue from subjects was examined for biomarkers relevant to fatty aldehyde dehydrogenase deficiency.
Results suggested that NS2 has reduced severity of ichthyosis on a consistent basis.
Exhibiting a tolerable profile, NS2 was also noted to have reduced cholesterol level in the skin of SLS patients.
Principal investigator of trial Dr William Rizzo said: "The data from this trial suggests that NS2, when applied topically to the skin, has the potential to help patients suffering from SLS by lowering toxic aldehyde levels and preventing the dermal dysfunction that causes ichthyosis.
“I am excited about these results, and I am particularly excited for SLS patients and their caregivers."
SLS is a rarely occurring inborn error of aldehyde metabolism caused due to mutations in fatty acid aldehyde dehydrogenase, leading to elevated toxic fatty aldehyde levels.
NS2 reduces the aldehyde levels, thereby inhibiting the inflammation of the toxins to trigger the disease.