Vistagen Therapeutics announced positive results from an exploratory Phase 2A study of PH284, an investigational neuroactive pherine nasal spray, for the treatment of cancer cachexia. The study demonstrated that PH284 significantly improved the subjective feeling of hunger (SFH) in patients with cancer cachexia, a debilitating wasting syndrome, while exhibiting a safety profile comparable to placebo.
The double-blind, placebo-controlled Phase 2A trial, sponsored by Vistagen's subsidiary Pherin Pharmaceuticals, involved 40 female patients with cachexia due to terminal cancer. Participants received either PH284 (0.4 μg/50 μL per nostril, four times daily before meals) or placebo. The primary endpoint was the change in SFH scores, measured before each meal.
Improved Hunger Feelings
Results showed that PH284 led to a cumulative increase in mean SFH scores throughout the treatment period. On day 7, before dinner, patients treated with PH284 reported a 71% improvement in SFH compared to baseline, while the placebo group showed less than a 1% improvement. Shawn Singh, President and CEO of Vistagen, stated, "We are highly encouraged by the potential of PH284 to improve the quality of life for those challenged by the debilitating impacts of cancer cachexia."
Safety and Tolerability
In terms of safety, the study found no serious adverse events related to PH284. Adverse events reported in the PH284 group were similar to those in the placebo group and were attributed to the underlying cancer. There were no unusual changes in body weight, although a slight weight gain was observed in the PH284 group compared to a slight weight loss in the placebo group.
Mechanism of Action
PH284 is believed to work by regulating olfactory to mediobasal-hypothalamus neural circuits involved in appetite control, offering a novel mechanism of action compared to existing treatments for appetite loss associated with chronic disorders. Vistagen is currently evaluating the potential path forward for PH284, including further Phase 2 clinical development.
About Cancer Cachexia
Cancer cachexia is a complex metabolic syndrome characterized by a loss of muscle and body weight, affecting up to 80% of people with advanced cancer. It is associated with reduced quality of life and can directly cause up to 30% of cancer deaths. There are currently no effective medical interventions or approved drugs to alleviate cachexia, highlighting the unmet medical need this study addresses.
