A Phase 2a Multicenter, Randomized, Vehicle-Controlled, Dose Escalating Study to Evaluate the Safety, Efficacy and Pharmacokinetics of CBT-001 Ophthalmic Solution in Pterygium Patients

Pterygium is an ocular surface disease with abnormal fibrovascular growth on the cornea that affects aboutmillion individuals in the USLater stage disease impairs vision and the current standard of care is surgical removal of lesion tissueHoweverlesions recur in aboutof patients after surgeryBecause there is no approved drug to treat pterygiumwe propose to address this unmet medical need with a topical ocular drugWe have formulated a topical eye drop nintedaniba small molecule multikinase inhibitorMKIthat targets growth factors implicated in pterygium pathogenesisNintedanib is the active pharmaceutical ingredient of Ofevan FDA approved oral treatment for idiopathic pulmonary fibrosisWe have shown that our formulated nintedanibCBTsuppresses cornea neovascularization in a rabbit corneal suture model and causes regression of human pterygium lesion tissue grown on the eyes of immune deficient miceThese results have enabled us to globally file the method of use patent of nintedanib for pterygium treatmentWe have successfully submitted our IND application to FDA and have been approved to proceed with a clinical trial in the USOur regulatory strategy takes thebpath by referencing existing human safetypharmacokinetics and non clinical study results of OfevOur private angel funding of $million enabled us to complete all the IND enabling pre clinical studies and will partially support the proposed clinical trialThe Phase I Aims of this Fast Track application are toEvaluate ocular and systemic safety and tolerability as well as effect on pterygium vascularity after a single topical ocular administration of CBTophthalmic solution in primary pterygium patientsDetermine systemic pharmacokinetics of CBTand select the highest safe dose after a single topical ocular administration of CBTophthalmic solution in primary pterygium patientsOur Phase II Aims are toEvaluate ocular and systemic safety of CBTin primary and recurrent pterygium patients during and after TIDday repeat ocular dosingAssess the efficacy of CBTin primary and recurrent pterygium patients during and after TIDday repeat ocular dosingWe will determine whether CBTcanireduce the key pterygium symptom of abnormal vascularity on cornea and conjunctiva by analyzing eye photos using a standardizedpoint Pterygium Hyperemia Grading Scaleiistabilize or regress lesion by measuring corneal lesion lengthwidth and area of pterygium in eye photosiiiimprove the quality of life of patientsandivimprove astigmatism caused by pterygium lesionThe Project Milestone for this two year Research Plan is to complete a Phasea multicenterrandomizedvehicle controlleddose escalating study to evaluate the safetyefficacy and pharmacokinetics of CBTophthalmic solution in pterygium patientsIf CBTproves to be clinically safe and efficacious and is approved for marketingit may significantly improve the treatment of pterygium patients by reducing the number of surgical interventions and lower the risk of post surgical disease recurrence Project Narrative Pterygium is an eye disease without any approved drug treatmentsurgery is the only option when vision is impacted and rapidly growing lesions recur in aboutof patients after surgeryThe proposed fast track project is to perform a Phasea clinical trial to evaluate the safety and efficacy of a novel pharmacological therapy that could change the standard of care of pterygium treatmentIf this therapy proves to be clinically safe and efficacious and is approved for marketingit may significantly improve the treatment of pterygium patients by reducing the number of surgical interventions and lower the risk of post surgical disease recurrence of patients