The mental health treatment landscape is evolving rapidly, driven by new and innovative therapies. Recent approvals like KarXT (from Karuna Therapeutics, now part of Bristol Myers Squibb) for schizophrenia, as well as many promising pipeline drugs, such as MindMed’s MM-120 for generalized anxiety disorder, highlight this momentum.
Amid this progress, alcohol use disorder (AUD) remains an area of significant unmet need. Many individuals with AUD also experience co-occurring mental health conditions such as depression, PTSD, anxiety, schizophrenia, and bipolar disorder—underscoring the complexity of treatment. With growing attention from researchers and biopharma alike, this AUD therapeutic area is anticipated to grow in the years ahead.
Underlying Causes of Alcohol Use Disorder
According to the National Survey on Drug Use Health (NSDUH), 27.9 million Americans aged 12 and older experienced AUD in 2024. This medical condition is characterized by excessive and uncontrollable drinking that has an detrimental impact on a person’s life.
A family history of alcohol abuse is a significant risk factor for developing AUD, and research indicates that inheritable genes involved in how the body metabolizes and responds to alcohol are present in 40% to 60% of individuals with AUD. Environmental factors and mental health conditions also play a significant role in this disorder.
Many individuals may develop substance abuse problems in an effort to escape reality, as they may be unwilling or unable to deal with underlying issues such as trauma. Substance abuse may also contribute to the development and exacerbation of mental health disorders such as schizophrenia or bipolar disorders.
When substance abuse and psychiatric disorders coexist, it makes diagnosis more difficult, as it’s hard to determine which disorder came first. People with such disorders are also far less likely to seek assistance, and more likely to become isolated from others. The consequences of this isolation can be severe.
Significant Unmet Need in Standard of Care for AUD
There are currently three FDA approved drugs on the market for the treatment of AUD. In 1949, Wyeth-Ayerst Laboratories’ Antabuse (disulfiram) became the first drug — and for more than 40 years, the only drug — to treat alcoholism.
Although the Antabuse brand is no more, generic disulfiram is now available in the U.S. via multiple companies. This drug interferes with the body’s ability to metabolize alcohol, thereby creating a surfeit of acetaldehyde. Whenever a person drinks alcohol while taking disulfiram, they typically have several uncomfortable symptoms such as nausea, vomiting, sweating, flushing and increased heart rate.
For years, there has been significant unmet need in this therapeutic area, as disulfiram was the only treatment available. DuPont’s Revia/Vivitrol (naltrexone) was approved in 1994 as an oral medication to treat AUD, and in 2006 as an extended-release injectable, which blocks opioid receptors that involve the euphoric sensation associated with drinking.
In 2004, a decade after naltrexone hit the market, Forest Pharmaceutical’s Campral (acamprosate), which eases the negative side effects of quitting drinking, was also approved by the FDA.
High Cost of Care Associated With Members With AUD
In a recent MMIT Biologics & Injectables Index survey on Mental Health Access Management, payers reported several unmet needs in AUD, including duration of response, response rate, and onset of action. Some payers also noted the need to address the stigma associated with seeking treatment for AUD, and expressed the need for a better, more easily tolerated drug.
Although more than half of surveyed payers did report covering all three FDA-approved medications for AUD, the study highlights payers’ AUD-related budgetary concerns. Payers noted that recurring relapses, frequent ER visits, and multiple diagnoses (such as liver disease or injuries) routinely drive up utilization and costs for their members with AUD.
Slightly more than half of surveyed payers (51%) indicate that AUD places a high burden on impacted individuals, employers, and caregivers. In 2024, 4.1 million ER visits were alcohol-related, and approximately 178,307 deaths were alcohol-related.
New AUD Therapies on the Horizon
After a long period of inactivity, three exciting new therapies are currently in development.
Adial Pharmaceuticals is developing the first genetically targeted therapeutic for AUD. Known as drug AD04, this twice-a-day oral therapy is thought to reduce alcohol craving. This serotonin-3 receptor antagonist acts on the 5HT3 pathway that releases dopamine in response to alcohol, and represents a novel mechanism of action (MoA) for this therapeutic area.
Adial has also developed a companion diagnostic test, intended to be commercially available at the time of launch, to identify the genotypes that benefit from AD04. In August 2025, AD04 had a successful End of Phase 2 meeting and has proceeded into Phase 3 of its clinical trial.
Clearmind Medicine is currently in clinical trials for a breakthrough AUD treatment, known as CMND-100. This novel, psychedelic-derived drug is reported to modulate reward mechanisms associated with addictive behavior. The drug is meant to work by innervating neural pathways such as 5-HT1A, which leads to decreased impulsivity in binge drinkers.
And finally, BioCorRx Pharmaceuticals is developing BICX104, an implantable, long-acting naltrexone pellet for treatment of AUD, opioid use disorder (OUD), and methamphetamine use disorder (MUD). Implantation is a 15-minute, in-office procedure, and the effects last for three months. BICX104 can potentially help patients with these disorders reduce the rate of non-compliance and relapse.
Off-Label Treatments Show Promising Results in Patients With AUD
While those three treatments are specifically for AUD, other therapies that were originally created for other indications have recently proven useful for AUD as well. In September, Imbrium Therapeutics announced positive preliminary results from its Phase 1b clinical study of sunobinop in patients with interstitial cystitis/bladder pain syndrome (IC/BPS). Currently in Phase 2, Imbrium is also separately evaluating sunobinop as a potential treatment for AUD.
In the meantime, the wildly popular GLP-1 agonist drugs — Eli Lilly’s tirzepatide and Novo Nordisk’s semaglutide and liraglutide — are showing promising results in reducing alcohol use. Although these therapies are primarily used for diabetes and weight loss, they have already been approved to reduce the risk of cardiovascular events and to treat other conditions, such as obstructive sleep apnea.
A study published by Diabetes, Obesity, Metabolism notes that tirzepatide demonstrates the strongest association of reducing AUD. More research is needed in establishing safety and efficacy data for using GLP-1 medications to treat AUD.
This is an exciting time for the mental health landscape, as manufacturers are working to fill this much-needed therapeutic gap. Awareness of AUD is growing, and payers are clearly ready for more tolerable and effective options for their members. A combination of mental health awareness, increased research, and the shared stories of individuals impacted by alcohol abuse will decrease the stigmatization for those suffering from AUD and substance addiction.
As new therapies reach the market, learn how payers are perceiving specific brands and their competitors with MMIT’s Message Monitor.
