SKYRAN BIOLOGICS, INC. — Department of Health and Human Services STTR Phase I: NEI

Description

AbstractThe overall goal of Skyran Biologics is to develop a powerful topical therapeutic to combat diabetic retinopathyDRa leading cause of vision loss and total blindness globallyOur research team was one of the first to show that a novel proteinPigment Epithelium Derived FactorPEDFis expressed in the eye and provides significant protection to the injured retinaAfter identifying amer bioactive region of PEDFwe generated several structurally different analogs to this regionscreened these for bioactivityand identified Spxas a lead compound with improved activity in the retina over the native fragmentThese proof of concept studies showed that topical Spxcan safely and effectively restore multiple markers of DR to near control levels when applied topically to the Ins Akita mouse model of DRIn both the mouse model and preliminary experiments on non human primateswe have shown that topical Spxleads to therapeutic concentrations of the peptide in the vitreousWe will use two specific aims to address three major scientific issuesThe first is to confirm the proof of concept for Spxand to show that its efficacy is specific to neither species nor mechanism of inducing DRThe second is to confirm that Spximproves visual acuityThe third is to verify that Spxis available in the vitreous and or retina via drop form in eyes where there is substantial back to front vitreal flowSkyran Biologics Inccan significantly increase the value of Spxby definitively demonstrating that the drop formulation of Spxresults in therapeutic doses of the peptide in the vitreous and retinaOur first aim in this project is to validate efficacy of Spxusing pharmacologicallySTZinduced diabetes in Long Evans ratsThis model has been widely validated in preclinical DR studies and used to show proof of concept for improvement in visionthrough visual acuity and contrast sensitivity measurementsWe will also assess efficacy of Spxto reduce retinal cell death and inflammationIn Aimwe will analyze biodistribution of Spxwhen given topically to adult rabbits to show that topical administration will be effective as a route of deliveryWe will deliver radioactively labeled Spxto the eye topically at two different doses and measure distribution of labeled peptide in aqueousvitreous and retina to confirm that the data already demonstrated using drop form can be replicated in a larger eyeThe milestones achieved by the successful completion of these aims will position us to propose a set of key Phase II studies including systemic toxicity screeningdosingformulationbiostability and the development of GMP grade peptide for clinical translationThe Phase II work will move us towards essential IND enabling studies for FDA approval and development of clinical phase translational strategies NarrativePigment Epithelium Derived FactorPEDFis expressed in the eye and provides significant protection to the injured retinaSkyran and its academic partner are developing a small protein therapeuticderived from PEDFto treat the retinal complications from diabetic retinopathyDRThis novel drug will significantly simplify the treatment of DR through the use of an eye drop formulated therapeutic that has the potential to significantly reduce or halt the degeneration of the retinathereby reducing or eliminating loss of sight caused by DR