Interim Phase 2 Data for Cancer Anorexia-Cachexia Treatment Show Promise

Encouraging interim results from the phase 2 Cancer Appetite Recovery Study (CAReS), a pivotal trial evaluating the investigational cannabinoid receptor agonist ART27.13 for the treatment of cancer anorexia-cachexia syndrome (CACS), suggest the drug may offer a new therapeutic modality for a condition that affects up to 80% of patients with cancer and currently has no FDA-approved treatment.¹

The CAReS trial (EudraCT-2020-000464-27)² is a phase 1/2 randomized, placebo-controlled study designed to assess the efficacy, safety, and tolerability of ART27.13. The interim analysis focuses on a small cohort of patients and provides a compelling signal of activity. Patients who were escalated to the highest dose of 1300 mcg per day demonstrated a mean weight gain of +6.38% over 12 weeks, in stark contrast with the placebo group, which experienced an average weight loss of –5.42%. This notable difference of approximately 12% is considered clinically significant and represents a potential reversal of the debilitating weight loss associated with cachexia.

Beyond body weight, the study also revealed a significant increase in lean body mass in the treatment group. At one month, patients receiving ART27.13 showed a mean gain of +4.23% in lean body mass, while the placebo group lost -3.15%. This finding is particularly important for a clinical audience, as the loss of skeletal muscle is a primary and often irreversible characteristic of cachexia. The preservation and increase of lean mass suggest that ART27.13's mechanism of action, which involves modulating peripheral cannabinoid CB1 and CB2 receptors, may directly address the muscle-wasting component of the syndrome. Preclinical research cited in the company's materials supports this, indicating that the drug protects human muscle cells from cancer-induced degeneration, primarily via a CB2-mediated pathway.

“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,” said Barry Laird, professor of palliative medicine at Oslo University Hospital and chief investigator of CAReS, in a press release.

A favorable safety profile was also observed, consistent with prior phase 1 findings. The most common adverse events were reported as mild to moderate, and no new safety signals or drug-related serious adverse events were noted. The well-tolerated nature of the drug is a critical factor for its potential use in a cancer population often debilitated by disease and therapy.

The Unmet Need in CACS

Cancer anorexia-cachexia syndrome is a complex, multifactorial condition that is a leading cause of mortality in cancer patients. It is characterized by progressive weight loss, anorexia, asthenia, and a significant decline in physical function. This syndrome can negatively impact a patient's quality of life, reduce their tolerance for anticancer therapies, and ultimately shorten survival. While off-label treatments like megestrol acetate have been used, they lack specific regulatory approval for CACS, and their efficacy remains a subject of debate within the clinical community. The absence of a dedicated, approved therapy highlights the substantial unmet medical need that ART27.13 aims to address.

The interim results of the CAReS trial are a promising step toward filling this therapeutic gap. By demonstrating a tangible reversal of weight and lean body mass loss, ART27.13 presents a potential paradigm shift in the supportive care of cancer patients. The positive findings have reportedly accelerated discussions with Artelo Biosciences, the sponsor, and potential pharmaceutical partners for the future development and commercialization of the drug, signaling a strategic focus on external collaboration to advance the program through late-stage trials.

The clinical significance of these interim findings, particularly the improvements in weight and lean body mass, provides a foundation for further investigation. The study's ability to demonstrate a clear drug effect at the highest dose level supports the next steps of the development program, which may involve starting future cohorts at the most effective dose to maximize the therapeutic benefit for patients. Continued research and the eventual publication of full trial data will be essential to fully understand the role of ART27.13 in managing this devastating syndrome.

REFERENCES:

1. Artelo Biosciences Announces Positive Interim Phase 2 CAReS Results for the Treatment of Cancer Anorexia-Cachexia Syndrome (CACS). News release. Artelo Biosciences. September 3, 2025. Accessed September 8, 2025. https://tinyurl.com/ymzrkfmb

2. A Phase 1/2 Trial of the Synthetic Cannabinoid ART27.13 in Patients with Cancer Anorexia and Weight Loss. European Union Clinical Trials Register. Accessed September 8, 2025. https://tinyurl.com/mrxffum6