OptiquelT, based on Enzo's immune regulation technology platform, is an oral formulation designed as a potential treatment for autoimmune uveitis, an intraocular inflammatory condition that reduces visual acuity and can lead to blindness. The active ingredient in Optiquel,T B27PD, is a small peptide that has been shown to mimic the retinal S-antigen, the offending protein antigen responsible for the ocular inflammation. Intraocular uveitis appears to result from an autoimmune reaction to antigens in the eye, specifically the S-antigen and the interphotoreceptor-retinoid-binding-protein (IRBP). OptiquelT has been designed to down regulate this undesired autoimmune response by inducing a specific immune tolerance to the retinal S-antigen. Reduction of intraocular inflammation by down-regulation of the autoimmune response has the key advantage of avoiding the side effects associated with treatment by systemic steroids and immunosuppressive agents.
Pre-clinical testing revealed no potential for adverse reactions, and the drug was well tolerated and no adverse effects were observed in an investigator-initiated pilot clinical trial of 9 autoimmune uveitis patients. The results of this pilot study demonstrated the potential of OptiquelT to ameliorate intraocular inflammation with the added benefit of a reduction of the required corticosteroid dosage.
OptiquelT has been granted orphan status in Europe and we will apply for the same in the U.S. since orphan status designation can confer both financial and marketing benefits.
Based on favorable preclinical animal studies and the pilot clinical trial, a CRADA between Enzo and the National Eye Institute was established to perform a proof-of-concept clinical trial. The trial is being led by Dr. Robert Nussenblatt, one of the world's foremost authorities on uveitis.