ONL Therapeutics, Inc. — Department of Health and Human Services SBIR Phase II: NEI

Description

PROJECT SUMMARYABSTRACTRetinal degenerative disease and injury are among the leading causes of irreversible vision loss and blindness in the United States and the developed worldaffecting hundreds of thousands of people every year and resulting in billions of dollars in added healthcare costs and lost productivityPhotoreceptor cell death is a consequence of many of these acute and chronic retinal diseasesleading to vision lossand helping photoreceptors survive during the period of disease would significantly improve the visual outcomes for patientsA problem that limits the efficacy of current treatments is the lack of interventions that can specifically prevent photoreceptor cell deaththereforea safe and potent photoreceptor protectant would provide a significant visual benefit to hundreds of thousands of people every yearONLis a novelsmall peptide inhibitor of Fas mediated photoreceptors cell death in development for peri operative adjunctive use in patients with acute retinal detachmentan orphan indication for which ONLhas received an orphan drug designationwith the goal of preventing photoreceptor cell death until definitive surgical repair can occurThis acute indication provides a rapid development pathway that lays the groundwork for future expansion of ONLdevelopment for chronic indicationssuch as age related macular degenerationThe product for acute retinal detachment will be a prescription drug administered by intravitreal injectionCompletion of the proposed studies in this grant will help ONL Therapeutics address final investor questions and attract the significant financial investment required for IND submission and clinical development of ONLSpecificallyin this grantwe willdetermine the minimal dose of ONLrequired for photoreceptor protection and preservation of function in large eye animal models of retinal detachmentanddemonstrate the GLP safety and tolerability of a single intravitreal injection of ONLat clinically meaningful dosesThe studies proposed in this application are necessary for development and commercialization of ONLwill de risk the programand enable significant financial investmentallowing ONL Therapeutics to evaluate clinical POC for ONLin retinal detachment PROJECT NARRATIVEDiseases of the retina are a leading cause of irreversible blindness in the United States and result in tremendous social and economic impact on patients and societyONL TherapeuticsIncis dedicated to developing innovative therapies that will prevent retinal cell death and improve visual outcomes for patients with retinal diseaseSuccessful development of our lead productONLa small peptide inhibitor of Fasmediated cell deathwould result in the first photoreceptor neuroprotective agent available to physicians to improve visual outcomes and reduce blindness in patients