Phase II Amount
$1,000,000
The development of safe and effective medications for the treatment of alcohol use disorders (AUDs) is apublic health priority. Stress and mood are significant components of relapse in addiction, including alcoholdependence. The kappa opioid receptor (KOR) system plays a role in relapse to substance use and KORantagonism has the potential to play an important role in the treatment of mood and substance use disorders.CERC-501 is an oral, once daily, highly specific and bioavailable KOR antagonist that reduces the EtOHpreference behaviors of P-preferring rats, alleviates nicotine withdrawal syndrome in mice, and reducesdepression- and anxiety- behaviors in animal models. CERC-501 was well tolerated in the three completedPhase 1 human studies (SAD, MAD and PET study).The primary objectives of the proposal are to enable pre-IND activities, file an IND for a new formulation andcomplete a pharmacokinetic and safety study of the new formulation. The pre-IND activities includedevelopment of new enteric coated formulation (CERC-501 EC) designed for maximal dispersion and releasein the small intestine, bypassing the stomach. Cerecor will conduct a repeat-dose range-finding study, 6-monthsafety study with CERC-501 EC in minipigs and file an IND with FDA's DAAAP for the treatment of AUDs.Cerecor will conduct a pharmacokinetic (PK) study in healthy humans that will assess the bioavailability andPK profile of the CERC-501 EC, compare systemic exposure of CERC-501 EC to the current formulation andassess if food impacts bioavailability. Lastly, Cerecor will conduct a 9-month toxicology study with CERC-501in dogs, so as to conclude the toxicology program that is required to support administration longer than 6months in patients.Cerecor plans to register CERC-501 EC as new pharmacotherapy for AUDs. CERC-501 EC will be indicatedfor the maintenance of abstinence from alcohol and reduction in heavy drinking days in patients who areabstinent at treatment initiation and ii) for the reduction of heavy drinking in patients who are actively drinkingat treatment initiation. After initial registration for treatment of AUD is completed clinical studies in co-occurringdisorders, AUD and depression will be conducted.
Public Health Relevance Statement: PROJECT NARRATIVE
The development of safe and effective medications for the treatment of alcohol use disorders (AUDs) is a
public health priority. The kappa opioid receptor (KOR) system plays a role in relapse to substance use and
CERC-501, a KOR antagonist, has the potential to play an important role in the treatment of AUDs. We
propose to conduct pre-IND activities, file an IND for a new formulation, complete a PK study of the new
formulation and ultimately register and commercialize CERC-501 EC as a new pharmacotherapy for AUDs.
Project Terms:
