French Biotech Candidate for Cannabis Use Disorder Fails Phase 2B Clinical Trial, Indivior Will Not Exercise Option

Aelis Farma, a French biotechnology company specializing in brain disorders, has announced the failure of its Phase 2B clinical trial for AEF0117, a candidate drug for treating cannabis use disorder (CUD). The trial’s disappointing results have led Indivior, a global pharmaceutical company, to reconsider its $100 million option to license the drug. This setback highlights the challenges in developing effective treatments for CUD and raises questions about the future of AEF0117.

The Phase 2B trial involved 333 participants with moderate to severe CUD, who were randomized to receive one of three doses of AEF0117 or a placebo for 12 weeks. The primary goal was to reduce cannabis use to one day or less per week. Unfortunately, the trial failed to meet this endpoint, as AEF0117 was no more effective than the placebo.

Secondary endpoints, such as achieving complete abstinence or reducing use to two days per week, were also not met. The lack of significant improvement in these measures suggests that AEF0117 may not be effective in treating CUD. Despite these results, Aelis Farma noted that the drug was well-tolerated, with no significant safety concerns reported.

The failure of AEF0117 to demonstrate efficacy in this trial is a significant blow to Aelis Farma and its strategic partner, Indivior. The results challenge the hypothesis that selectively blocking the cannabinoid receptor 1 (CB1) can reduce cannabis use by inhibiting the signaling pathways that drive its intoxicating effects. This outcome underscores the complexity of developing pharmacological treatments for substance use disorders.

Indivior’s Decision and Future Prospects

Indivior had initially paid $30 million for an option to license AEF0117, with plans to make a final decision based on the Phase 2B data and feedback from the U.S. Food and Drug Administration (FDA). However, given the trial’s failure to meet its primary and secondary endpoints, Indivior has decided not to exercise its option at this time.

This decision reflects the company’s cautious approach to investing in new treatments, particularly those with uncertain efficacy. Indivior’s focus remains on developing and commercializing medicines for substance use disorders and serious mental illnesses. The setback with AEF0117 highlights the inherent risks in drug development and the importance of rigorous clinical testing.

Aelis Farma is still reviewing the trial results and considering its next steps. The company has indicated that it may continue to explore the potential of AEF0117, particularly in subgroups of patients with moderate CUD, where some positive trends were observed. However, the path forward remains uncertain, and further research will be needed to determine the viability of this candidate.

Broader Implications for Cannabis Use Disorder Treatment

The failure of AEF0117’s Phase 2B trial has broader implications for the field of CUD treatment. Cannabis use disorder is a growing public health concern, with increasing rates of cannabis use and dependence worldwide. Effective treatments are urgently needed to help individuals reduce or quit their cannabis use.

The challenges faced by Aelis Farma and Indivior highlight the complexity of developing pharmacological interventions for CUD. The endocannabinoid system, which is targeted by drugs like AEF0117, plays a crucial role in regulating mood, appetite, and cognition. Modulating this system without causing adverse effects is a significant scientific challenge.

Despite this setback, the search for effective CUD treatments continues. Researchers are exploring various approaches, including behavioral therapies, combination treatments, and novel pharmacological targets. The lessons learned from the AEF0117 trial will inform future research and development efforts, ultimately contributing to the advancement of CUD treatment options.