Clinical-stage biopharmaceutical company Vascarta Inc announced on Wednesday the successful completion of the sublingual arm of its Phase I clinical trial evaluating VAS-101 (Vasceptor; topical curcumin gel) for the treatment of sickle cell disease (SCD).

The study was conducted by the Foundation for Sickle Cell Disease Research (FSCDR) in Hollywood, Florida, and the fhe findings have been published in the Journal of Sickle Cell Disease.

Five patients with sickle cell disease were treated sublingually with a minimal dosing (0.1 mL) of VAS-101 twice per week for a total of 8 treatments. Their blood was drawn and analysed weekly. The duration of treatment was 29 days. According to the company, all participants demonstrated favourable safety and tolerability. Directional improvements were observed across several patient-reported outcomes.

Red blood cell mechanical fragility indices (MFI) were within normal range at baseline and remained stable over the course of the study. Baseline concentrations of standard inflammatory markers were and remained below the lower limit of detection for the initial three participants; consequently, further analytical testing was suspended.

According to the company, VAS-101 demonstrated safety and tolerability in the sublingual cohort. Improvements in integrated ePRO and RBC functional biomarkers were noted suggesting potential mechanistic and patient-centred benefits of VAS-101 in individuals with SCD. The resulting biomarkers shift at low dose supports advancement towards dose optimisation. Future studies evaluating higher dose and frequency may yield greater improvements in pain, ePRO measures, and laboratory biomarkers. Pre-selecting a cohort that exhibits a higher adhesive burden, indicative of more inflammation may show further improvements in RBC health normalisation.

VAS-101 is a topical curcumin formulation that employs a patented transdermal delivery technology designed to increase bioavailability. It is being clinically evaluated in several chronic inflammatory indications.