PROJECT SUMMARY This Direct-to-Phase II application aims to complete the preclinical development activities required for the development of a novel treatment for moderate to severe traumatic brain injury (TBI), an area with a huge unmet need. TBI is characterized by an impact to the brain followed by a development of a secondary injury that results from processes initiated by the trauma. The secondary injury occurs in the hours and days following the primary injury and plays a large role in the brain damage and death that results from TBI. There are currently general management guidelines for patients with TBI with no specific neuroprotective interventions available. We propose to validate a novel therapeutic agent for treatment of moderate to severe TBI. AivoCode, Inc. has developed CAQK, a proprietary peptide, for the management of TBI. CAQK specifically targets both penetrating and contusion type of brain injuries and has a pluripotent effect when administered early on in the injury by limiting the secondary damage in TBI. This effect results in improved healing of the injuries as well functional recovery in rodent model of TBI. In this application, we will conduct efficacy studies in pig model of focal TBI and generate a pharmacokinetic profile in a gyrencephalic model of TBI. We will also complete pre-clinical studies that include CMC, formulation development and non-GLP toxicology studies needed for an IND (Investigational New Drug) application for the Food and Drug Administration (FDA) to initiate clinical trials of CAQK for treatment of TBI.

PROJECT NARRATIVE The goal of this proposal is to complete pharmacology studies and generate the requisite preclinical data package and regulatory dossiers to support the clinical development of a novel neuroprotective peptide as a treatment for moderate to severe traumatic brain injury. Our neuroprotective peptide specifically targets injured brain and reduces the secondary injury after the primary impact. This project addresses an underserved medical area, where there is a pressing need for new therapeutic advances.