Development of JR-220 (4-Chlorobenzylidenamino-guanidine hydrochloride) as a medication for alcohol dependence

Abstract The aim of phase I of the parent cooperative agreement (U44AA026126) was to generate sufficient data on the pharmacokinetics (PK) and toxicology (Tox) of JR-220 to support an application for a pre-IND meeting with the FDA. This aim has been successfully accomplished. The aim of phase II of the parent cooperative agreement is to expand the phase I studies with rat and a second species (canine) based upon the feedback of the FDA’s pre-IND meeting response to create a dossier that will be submitted to the FDA in support of IND status for JR-220 as a medication for treatment of alcohol use disorder (AUD). Neither of these aims has changed from the original funded application, but the cost to support several GLP/GMP tasks related to these aims have significantly increased for reasons outside the control of the company. All of these tasks support the potential value of JR-220 as a medication for AUD and are required for IND submission. This administrative supplemental proposal is a request for additional funds to cover the unanticipated increased cost of several key GLP/GMP tasks to be completed that are required by the FDA to submit the IND application. This administrative supplement proposal includes tasks previously proposed and accepted by reviewers and NIAAA. The three key tasks with increased costs are 1) GLP CNS safety study (Neurotoxicity) in rat specifically requested by the FDA in the pre-IND meeting response, 2) cGMP manufacturing of JR-220 (drug substance), and 3) cGMP manufacturing of JR-220 capsules (drug product) for clinical trial. This administrative supplemental request for additional funding under $100K to help cover the incremental unanticipated cost increase from 2016 to completed these original scope tasks is important for the successful completion of the overall goal of the parent cooperative agreement to submit the IND in support of JR-220 as medication for treatment for AUD.