Pharmaceutical companies Eli Lilly and Baseline Therapeutics are advancing clinical trials to evaluate the effectiveness of GLP-1 receptor agonists in treating substance use disorders, according to a March 30 announcement. This effort comes amid growing interest in these diabetes and obesity drugs as potential therapies for conditions such as alcohol use disorder (AUD) and opioid dependence.
The move is significant because, despite nearly 30 million people affected by alcohol use disorder in the United States, only three medications have been approved by the Food and Drug Administration for its treatment. The potential expansion of GLP-1s into this area could address a gap where current options are limited compared to other diseases.
Nicholas Reville, head of strategy at Baseline Therapeutics, said these drugs could become “a new blockbuster class” for substance use conditions. Baseline plans to launch two Phase 3 trials this year testing its compound BT-001 in patients with AUD. Eli Lilly is leading larger efforts with ongoing studies of brenipatide—targeting both GLP-1 and GIP receptors—in Phase 3 trials for AUD and Phase 2 studies focused on tobacco relapse and opioid use disorder. These trials are expected to conclude between 2027 and 2028.
Other companies are also involved. Novo Nordisk completed a Phase 2 trial investigating semaglutide’s effects on liver damage and alcohol consumption earlier this year, though results have not yet been released. Previous research published in JAMA Psychiatry showed that semaglutide reduced alcohol intake after nine weeks but did not affect the number of drinking days.
Altimmune is testing pemvidutide—a drug targeting both GLP-1 and glucagon receptors—for reducing drinking among individuals with AUD who also have overweight or obesity, with study completion expected soon. Christophe Arbet-Engels, Altimmune's chief medical officer, said tolerability data so far has been compelling.
Recent observational studies add momentum to these efforts. A study published in TheBMJ found that U.S. veterans with diabetes taking GLP-1s had lower risks of developing substance use disorders or being hospitalized due to them compared to those on other medications; however, researchers caution that more large-scale clinical trials are needed before definitive conclusions can be drawn about causality.
Experts emphasize safety concerns must be addressed given the high-risk patient population but remain optimistic about ongoing research efforts. Joseph Schacht from the University of Colorado noted that "even a drug with potentially large effects on substance use will not be effective for everyone." Lorenzo Leggio from the National Institutes of Health added he is "very optimistic" while stressing more data is needed: “This is the part that we’re still missing.”
The increased focus on addiction treatments represents progress for an area often overlooked by pharmaceutical innovation.
