PleoPharma Inc. has announced positive results from its Phase 2b clinical trial of PP-01, an investigational treatment for Cannabis Withdrawal Syndrome (CWS) in patients with Cannabis Use Disorder (CUD). The data, presented at the 2025 annual meeting of The College on Problems of Drug Dependence in New Orleans, demonstrates significant efficacy for what could become the first FDA-approved medication for this indication.
Phase 2b Trial Results Show Statistical Significance
The CAN-002 study, a multicenter, randomized, double-blind, multi-dose, placebo-controlled trial, evaluated PP-01's safety and effectiveness in mitigating cannabis withdrawal symptoms. PP-01 significantly reduced Cannabis Withdrawal Scores compared to placebo with a P value of less than 0.02. The investigational agent is a novel once-daily, oral, dual-mechanism treatment containing tapering and titrating doses of nabilone and gabapentin.
Adult treatment-seeking participants in the study reported average baseline cannabis use of 4.6 grams per day and a mean age of first use of approximately 16.5 years, with a range from 7 to 46 years. According to DSM-5 criteria, 84% of participants had severe CUD while 16% had moderate CUD.
Superior Abstinence Rates Observed
A key secondary endpoint evaluated cannabis abstinence, where PP-01 demonstrated 5-fold greater abstinence two weeks post-treatment compared to placebo. The study data revealed that reduction of cannabis withdrawal symptoms during the first week of treatment was predictive of abstinence, providing insights into treatment response patterns.
"We are excited to be developing PP-01, which was granted Fast Track designation by the FDA, and has the potential to be the first FDA approved product to help people suffering from CWS with CUD, a rapidly growing patient population in need of treatment," stated Shelli Graham, PhD, Senior Vice-President of Medical/Clinical Research at PleoPharma.
Safety Profile and Tolerability
PP-01 demonstrated a favorable safety profile throughout the trial. The treatment was well tolerated, with the most common adverse events being mild in nature, including headache, somnolence, fatigue, nausea, and dizziness. Notably, there were no serious adverse events reported during the study period.
Addressing a Significant Unmet Medical Need
The clinical development of PP-01 addresses a substantial healthcare gap. According to US Government data from 2023, approximately 19.2 million Americans had CUD, with 1.64 million people receiving treatment, including approximately 500,000 inpatient and 1.1 million outpatient cases. The number of individuals receiving treatment for cannabis-related health problems has grown by an average of 28% annually from 2018 to 2023.
Currently, there are no FDA-approved medications for the treatment of CWS or CUD. People with CUD commonly experience significant withdrawal symptoms that can make it extremely difficult to reduce or discontinue cannabis use, highlighting the critical need for effective therapeutic interventions.
Path Forward to Phase 3
Following these positive Phase 2b results, PleoPharma is currently preparing for PP-01 to enter Phase 3 clinical trials. The company's development program has already received FDA Fast Track designation, which is designed to facilitate the development and expedite the review of drugs to address unmet medical needs in serious conditions.
PleoPharma presented their findings through both a poster presentation titled "Predictors of Cannabis Abstinence in a Clinical Trial" on June 17, 2025, and an oral presentation "PP-01 Significantly Mitigated Cannabis Withdrawal Symptoms with a Decrease in Cannabis Use" on June 18, 2025, at the scientific meeting.
