Disease Area

Alcohol Use Disorder

PreclinicalPhase IPhase IIPhase IIINDAFDA ApprovalPartner
Preclinical
Phase I
Phase II
Phase III
NDA
FDA Approval

Background

About 17 million people in the United States suffer from some form of Alcohol Use Disorder (AUD).  The economic costs of AUD are staggering: roughly $250 billion annually are spent on alcohol-related healthcare, lost productivity, and criminal justice costs. AUD is the third leading preventable cause of death in the United States. More Americans die from alcohol-related causes each year (estimated at 88,000) than all other drug poisoning fatalities combined.

Our approach

Multiple medications are approved for the treatment of AUD, yet less than 5% of individuals with an AUD currently receive treatment. We are developing OPNT002, a rapid onset formulation of nasal naltrexone for the treatment of AUD. Alcohol is a potent trigger for the release of the brain’s naturally occurring opioids (endorphins). This release stimulates several distinct types of opioid receptors which produce an elevation of the brain’s “pleasure transmitter”, dopamine.

opioid antagonist treatment graphic

By blocking opioid receptors, opioid antagonists like naltrexone reduce the effects of endorphins released by alcohol. This blockade blunts the pleasurable (reinforcing) effects of alcohol. This can result in lower levels of drinking. Naltrexone, in both pill and injection, is already approved for use in AUD. The blood levels of naltrexone in these formulations result in an effective blockade of one type of opioid receptor (mu) in the brain, which is thought to contribute to the ability of naltrexone to reduce alcohol consumption and craving. However, the blockade of another opiate receptor (delta) that contributes to the pleasurable effects of alcohol is much lower and highly variable. 

Our programs and progress

OPNT002 is a very rapid-acting, intranasal formulation of naltrexone. The ability to swiftly elevate plasma naltrexone concentrations to levels higher than currently achieved by available naltrexone products may result in a more effective blockade of delta-opioid receptors, thereby increasing its therapeutic effects compared to oral or injectable naltrexone.

Because Phase 1 clinical data with OPNT002 demonstrated rapid intranasal absorption and a short duration of action compared to currently available products, OPNT002 can be taken intranasally on an ‘as needed/on-demand’ (targeted) basis to moderate alcohol intake when the patient craves alcohol, anticipates drinking or is in a “high risk” situation (e.g., at a party or restaurant).  Following the completion of a formulation selection study later this year, Opiant will initiate a double-blind, placebo-controlled Phase II study in patients diagnosed with AUD.