Investigational Nasal Spray May Improve Symptoms in Cancer Cachexia

Study shows potential benefits of PH284 for patients with cancer cachexia.

PH284, an investigational pherine nasal spray, showed improvements in the feelings of hunger vs placebo in patients with cancer experiencing cachexia.

Findings from the exploratory phase 2a study, which were announced in a press release from Vistagen, the manufacturer of PH284, demonstrated a higher mean subjective feeling of hunger (SFH) with PH284 compared with placebo. In addition, the nasal spray was well-tolerated and safe, with a similar adverse event profile as placebo in patients with terminal cancer.

“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,” Shawn Singh, president and chief executive officer of Vistagen, said in the release. “Loss of appetite from cancer and other illnesses not only negatively impacts overall health and quality of life, but can also reduce the effectiveness of critical therapies, such as chemotherapy in cancer patients.”

In the double-blind, placebo-controlled phase 2a study, researchers aimed to evaluate the safety, efficacy, and tolerability of PH284 in 40 women diagnosed with cachexia induced by chronic appetite loss from terminal cancer, according to the release.

PH284 was administered intranasally, with 1 spray in each nostril, for a total daily dose of 3.2 µg. Patients received the nose spray 4 times a day before meals, particularly breakfast, mid-morning snack, lunch, and dinner, as announced in the release. From day 1 to day 4, all patients were given placebo 30 minutes before each meal. Starting on day 5 through 11, patients were randomly assigned in a 1:1 fashion to receive either PH284 or placebo.

Ten minutes before each meal, patients measured SFH, according to the release. Compared with placebo, PH284 resulted in a cumulative effect on mean SFH scores. Scores increased from breakfast to lunch, and from lunch to dinner throughout the study period. Before dinner on day 7 of treatment, patients assigned PH284 experienced a 71% improvement in SFH compared with baseline, whereas patients assigned placebo had a less than 1% improvement.

The release also noted no unusual body weight changes in patients regardless of treatment assignment, although there was a small gain in body weight for the PH284 group compared with a small loss in the placebo group. Patients assigned PH284 reported no serious adverse events. Any adverse events reported by the PH284 group were similar to those in the placebo group, per the release. The reported adverse events were related to cancer and not from treatment with PH284 or placebo.

PH284 is a neuroactive pherine nasal spray that aims to regulate the olfactory to mediobasal-hypothalamus neural circuits typically involved in appetite control, as noted in the release. With the findings from this 2a study in addition to previously conducted studies, Vistagen noted it may have sufficient evidence to support an Investigational New Drug application to the FDA for potential phase 2 clinical development of PH284 to treat patients with cachexia.

Reference

Vistagen Announces Positive Results from Exploratory Phase 2A Study of PH284 in Cancer Cachexia. News release. Adaptimmune. January 14, 2025. Accessed January 16, 2025. https://www.vistagen.com/news-releases/news-release-details/vistagen-announces-positive-results-exploratory-phase-2a-study-1