Artelo Biosciences Reports Promising Phase 2 Results for ART27.13 in Cancer Cachexia Treatment
Key Insights
Artelo Biosciences (search) announced positive interim Phase 2 results for ART27.13, showing a 6.38% mean weight gain at 12 weeks versus 5.42% weight loss on placebo in cancer cachexia patients.
The peripherally acting cannabinoid receptor agonist demonstrated significant improvements in lean body mass (+4.23% vs -3.15% placebo) and activity scores in the 18-patient interim analysis.
ART27.13 was well tolerated with mostly mild to moderate adverse events, supporting its safety profile for treating cancer anorexia-cachexia syndrome, which affects up to 80% of cancer patients.
Artelo Biosciences (search) has announced highly encouraging interim results from its Phase 2 Cancer Appetite Recovery Study (CAReS) trial, demonstrating significant efficacy for ART27.13 in treating cancer anorexia-cachexia syndrome (CACS). The peripherally acting cannabinoid receptor agonist showed compelling weight gain and improved physical activity in cancer patients, marking a potential breakthrough for a condition affecting up to 80% of people living with cancer.
Significant Weight Gain and Body Composition Improvements
In the interim analysis of 18 evaluable patients, those escalated to the 1300 microgram dose achieved a remarkable +6.38% mean weight gain after 12 weeks, compared to a -5.42% average loss on placebo. The maximum weight gain observed reached +18.5% on ART27.13 versus only +0.4% on placebo, while the maximum weight loss in the placebo arm was -17.4%, compared to just -3.0% in the ART27.13 group.
The drug also demonstrated significant benefits in lean body mass preservation. At one month, patients treated with ART27.13 experienced a +4.23% increase in lean body mass (SD 5.37), while placebo patients lost -3.15% (SD 4.89). These improvements in body composition are particularly significant given that muscle wasting is a hallmark of cancer cachexia.
Enhanced Physical Activity and Quality of Life
Beyond weight metrics, ART27.13 showed qualitative improvements in total and weekly activity scores. Patients receiving treatment demonstrated improvements in moderate and vigorous activity, the latter being an endpoint that may be required by regulators for drug approval. This enhancement in physical function represents a crucial therapeutic benefit for cancer patients struggling with debilitating weakness and fatigue.
Favorable Safety Profile Maintained
Safety results were consistent with prior Phase 1 findings, supporting ART27.13's overall favorable tolerability profile. Among the 32 participants enrolled in the CAReS Phase 2 trial to date, 7 patients (22%) experienced adverse events that may be related to ART27.13. All were mild or moderate, with the exception of a single case of severe malaise, and no drug-related serious adverse events were reported.
"ART27.13 was well tolerated. The most common adverse events were mild or moderate. No new safety signals were observed and interim safety results were consistent with Phase 1 of CAReS," the company reported.
Addressing Critical Unmet Medical Need
CACS represents a significant unmet medical need, marked by loss of appetite, weight loss, and breakdown of muscle and fat. This condition is a leading cause of death in cancer patients, yet currently has no FDA-approved treatment in the US, UK, or EU. The syndrome affects patients with advanced cancer and represents a greater than $3 billion addressable market.
Barry Laird, Professor of Palliative Medicine at Oslo University Hospital and Chief Investigator in CAReS, commented: "The findings from the CAReS Phase 2 interim analysis build on the strong safety profile and signal from the Phase 1 trial and provide encouraging data supporting efficacy of ART27.13 on weight and physical activity in patients with advanced cancer."
Strategic Implications and Next Steps
The positive results are catalyzing Artelo's partnering initiatives with pharmaceutical companies that have expressed interest in ART27.13. Steven Reich, MD, Chief Medical Officer of Artelo, stated: "These data represent a reassuring and convincing drug effect for the highest dose of ART27.13 tested. With these interim results, we are evaluating plans to advance the ART27.13 program by starting patients at the highest dose, which has the greatest treatment effect and acceptable safety."
Gregory D. Gorgas, President and CEO of Artelo, emphasized the strategic value: "Based on the strength of this data and the recently announced Notice of Allowance for the patent application covering our intended commercial formulation through 2041, we are rapidly accelerating our licensing strategy as the most value-accretive path forward."
Trial Design and Patient Population
The Phase 2 CAReS study evaluated ART27.13 as a once-daily oral treatment in cancer patients who had lost a minimum of 5% of their body weight in the prior six months. The interim analysis included patients primarily with lung and gastrointestinal cancers not receiving cyclic chemotherapy. Effectiveness was measured by changes in lean body mass, weight, appetite, and activity over 12 weeks and at a 30-day follow-up, with activity and quality of life assessed using wearable monitors and standardized questionnaires.
ART27.13 is a novel benzimidazole derivative that selectively targets peripheral CB1 and CB2 receptors, enabling systemic metabolic effects while minimizing central nervous system-mediated toxicity. Initially developed by AstraZeneca, the compound has been evaluated in seven clinical studies with over 280 participants, previously demonstrating statistically significant and dose-dependent increases in body weight in healthy individuals with back pain.