Vistagen Reports Positive Phase 2A Results for PH284 in Cancer Cachexia

Vistagen, a South San Francisco-based company focused on advanced neuroscience, has announced encouraging findings from a Phase 2A clinical trial of PH284, an innovative nasal spray designed to combat cancer cachexia. The condition, characterized by severe weight loss and muscle wasting, is a frequent complication associated with cancer. PH284 is unique compared to existing treatments, as it specifically targets the loss of appetite that accompanies chronic illnesses like cancer.

The clinical study aimed to assess the effectiveness, safety, and tolerability of PH284 in women suffering from cachexia due to terminal cancer. Conducted in a double-blind, placebo-controlled manner, the study involved 40 participants. The treatment regimen included administering PH284 intranasally four times daily before meals. From Days 1 to 4, participants received a placebo. Starting Day 5 through Day 11, they were randomly assigned to receive either PH284 or a placebo.

The study's efficacy was measured using the Subjective Feeling of Hunger (SFH) scores, recorded ten minutes before meals. PH284 demonstrated a significant impact, showing a cumulative increase in SFH scores. By Day 7, participants receiving PH284 reported a 71% improvement in hunger compared to baseline measurements, whereas the placebo group showed less than a 1% improvement. These results underscore the potential of PH284 to significantly enhance appetite in patients suffering from cancer cachexia.

In terms of safety, PH284 was well-tolerated among the participants. No notable changes in body weight were recorded between the PH284 and placebo groups, although there was a slight increase in body weight in the PH284 group compared to a minor decrease in the placebo group. Importantly, no serious adverse events were associated with PH284. The adverse events observed were consistent with those expected in the context of the underlying cancer condition and were not linked to the treatment itself.

Cachexia, often referred to as wasting syndrome, involves a complex metabolic process leading to a gradual loss of muscle and body weight. It is a common occurrence in chronic diseases like cancer, affecting up to 80% of individuals with advanced cancer. The syndrome significantly contributes to cancer-related mortality, often due to complications arising from muscle loss, impacting heart and respiratory functions. Currently, there are no approved medical treatments specifically targeting cachexia, highlighting the potential impact of PH284.

The data from this exploratory Phase 2A study, originally conducted by Pherin Pharmaceuticals in Mexico City in 2005, became accessible to Vistagen following their acquisition of Pherin in February 2023. Key investigators in the study included notable figures such as Hector Burges, MD, and Marcos Cano Guardiana, MD, from the National Institute of Oncology, as well as Ricardo Plancarte Sanchez, MD, from the Institute of Oncology's Pain Clinic.

PH284 is characterized by a novel mechanism of action that differentiates it from other appetite loss treatments. It is believed to influence neural circuits connected to appetite control via the olfactory system, demonstrating a promising safety profile in various clinical trials. Vistagen is currently evaluating further developmental pathways for PH284, intending to seek regulatory approval in the United States for additional Phase 2 clinical trials targeting cachexia.

Vistagen is committed to leveraging its innovative neuroscience platform to develop a diverse range of intranasal products, known as pherines, aimed at rapidly activating brain neurocircuitry to achieve therapeutic benefits. The company's broad pipeline also includes AV-101, an oral prodrug targeting neurological conditions related to the NMDA receptor. Vistagen continues to pursue treatments that address significant unmet medical needs, particularly for disorders like social anxiety, depression, and menopause-related symptoms.