Vistagen Announces Positive Results from Exploratory Phase 2A Study of PH284 in Cancer Cachexia
Vistagen (VTGN) announced positive results from an exploratory Phase 2A study of PH284, a pherine nasal spray designed to treat cancer cachexia. The double-blind, placebo-controlled study involved 40 female patients with terminal cancer-induced cachexia. PH284 demonstrated significant improvements in subjective feelings of hunger (SFH), with patients reporting a 71% improvement versus baseline prior to dinner on Day 7, compared to less than 1% in the placebo group.
The treatment protocol involved administering PH284 nasal spray (0.4 µg/50 µL) four times daily before meals. The study showed cumulative positive effects on hunger scores throughout the treatment period. Safety results were favorable, with no serious adverse events reported and an adverse event profile similar to placebo. PH284 becomes the fifth pherine product candidate in Vistagen's neuroscience pipeline to show positive efficacy signals.
Vistagen (VTGN) ha annunciato risultati positivi da uno studio esplorativo di Fase 2A su PH284, uno spray nasale a feromoni progettato per trattare la cachessia cancerosa. Lo studio, in doppio cieco e controllato con placebo, ha coinvolto 40 pazienti femminili con cachessia terminale indotta da cancro. PH284 ha dimostrato significativi miglioramenti nei sentimenti soggettivi di appetito (SFH), con i pazienti che hanno riportato un miglioramento del 71% rispetto al valore di base prima della cena nel Giorno 7, rispetto a meno dell'1% nel gruppo placebo.
Il protocollo di trattamento prevedeva l'amministrazione dello spray nasale PH284 (0,4 µg/50 µL) quattro volte al giorno prima dei pasti. Lo studio ha mostrato effetti positivi cumulativi sui punteggi di appetito per tutto il periodo di trattamento. I risultati di sicurezza sono stati favorevoli, senza eventi avversi gravi segnalati e un profilo di eventi avversi simile a quello del placebo. PH284 diventa il quinto candidato prodotto a feromoni nel pipeline neuroscientifico di Vistagen a mostrare segnali di efficacia positivi.
Vistagen (VTGN) anunció resultados positivos de un estudio exploratorio de Fase 2A de PH284, un spray nasal de feromona diseñado para tratar la caquexia cancerosa. El estudio, doble ciego y controlado con placebo, involucró a 40 pacientes femeninas con caquexia terminal inducida por cáncer. PH284 mostró mejoras significativas en las sensaciones subjetivas de hambre (SFH), con pacientes reportando una mejora del 71% respecto a la línea base antes de la cena en el Día 7, en comparación con menos del 1% en el grupo placebo.
El protocolo de tratamiento consistió en administrar spray nasal PH284 (0.4 µg/50 µL) cuatro veces al día antes de las comidas. El estudio mostró efectos positivos acumulativos en los puntajes de hambre durante todo el período de tratamiento. Los resultados de seguridad fueron favorables, sin eventos adversos graves reportados y un perfil de eventos adversos similar al del placebo. PH284 se convierte en el quinto candidato a producto de feromona en la cartera de neurociencia de Vistagen que muestra señales de eficacia positiva.
Vistagen (VTGN)는 암 카헉시아 치료를 목적으로 하는 페로몬 비강 스프레이 PH284의 탐색적 2A상 연구에서 긍정적인 결과를 발표했습니다. 이 이중 맹검, 위약 대조 연구에는 말기 암으로 인한 카헉시아를 앓고 있는 여성 환자 40명이 참여했습니다. PH284는 주관적인 배고픔 감각(SFH)에서 유의미한 개선을 보여주었습니다, 환자들이 7일차 저녁 식사 전 기준선 대비 71% 개선을 보고한 반면, 위약 그룹에서는 1% 미만이었습니다.
치료 프로토콜에는 PH284 비강 스프레이(0.4 µg/50 µL)를 식사 전 하루 네 번 투여하는 것이 포함되었습니다. 연구는 치료 기간 동안 배고픔 점수에 대한 누적 긍정적 효과를 보여주었습니다. 안전성 결과는 긍정적이었습니다, 심각한 부작용 보고는 없었고, 부작용 프로필은 위약과 유사했습니다. PH284는 Vistagen의 신경과학 파이프라인에서 긍정적인 효능 신호를 보인 다섯 번째 페로몬 후보 제품이 됩니다.
Vistagen (VTGN) a annoncé des résultats positifs d'une étude exploratoire de Phase 2A sur PH284, un spray nasal à phéromones conçu pour traiter la cachexie cancéreuse. L'étude, en double aveugle et contrôlée par placebo, a impliqué 40 patientes atteintes de cachexie terminale induite par le cancer. PH284 a montré des améliorations significatives des sensations subjectives de faim (SFH), les patientes rapportant une amélioration de 71 % par rapport à la ligne de base avant le dîner le jour 7, contre moins de 1 % dans le groupe placebo.
Le protocole de traitement prévoyait l'administration de spray nasal PH284 (0,4 µg/50 µL) quatre fois par jour avant les repas. L'étude a montré des effets positifs cumulatifs sur les scores de faim tout au long du traitement. Les résultats de sécurité étaient favorables, sans événements indésirables graves signalés et un profil d'événements indésirables similaire à celui du placebo. PH284 devient le cinquième candidat produit de phéromone dans le pipeline des neurosciences de Vistagen à montrer des signaux d'efficacité positifs.
Vistagen (VTGN) gab positive Ergebnisse aus einer explorativen Phase-2A-Studie zu PH284 bekannt, einem Pheromon-Nasenspray, das zur Behandlung der Krebs-cahexie entwickelt wurde. Die doppelblinde, placebo-kontrollierte Studie umfasste 40 weibliche Patientinnen mit terminaler krebsbedingter Cahexie. PH284 zeigte signifikante Verbesserungen in den subjektiven Hungerempfindungen (SFH), wobei die Patientinnen eine Verbesserung von 71 % im Vergleich zur Basislinie vor dem Abendessen am Tag 7 berichteten, im Vergleich zu weniger als 1 % in der Placebo-Gruppe.
Das Behandlungsprotokoll umfasste die Verabreichung des PH284-Nasensprays (0,4 µg/50 µL) viermal täglich vor den Mahlzeiten. Die Studie zeigte kumulative positive Effekte auf die Hungerwerte während des gesamten Behandlungszeitraums. Die Sicherheitsresultate waren günstig, ohne schwerwiegende unerwünschte Ereignisse und ein ähnliches unerwünschtes Ereignisprofil wie beim Placebo. PH284 wird das fünfte Pheromon-Produktkandidat im Neuroscience-Pipeline von Vistagen, das positive Wirksamkeitssignale zeigt.
- 71% improvement in subjective feelings of hunger versus baseline compared to <1% for placebo
- No serious adverse events reported with safety profile similar to placebo
- Small weight gain observed in treatment group versus weight loss in placebo group
- Fifth successful pherine candidate showing positive Phase 2 or later clinical data
- Study to female patients only
- Small sample size of 40 patients
- Early-stage (Phase 2A) exploratory study requiring further validation
Insights
The Phase 2A study of PH284 represents a significant advancement in cancer cachexia treatment. The
The trial design was robust - double-blind, placebo-controlled with a reasonable sample size (n=40) for an exploratory study. The dosing regimen (4x daily, 3.2µg total) and the 11-day duration provided sufficient data to evaluate both efficacy and safety. The placebo lead-in period (Days 1-4) helps establish a reliable baseline and reduces placebo effect influence.
Most compelling is the safety profile - no serious adverse events and a side effect profile similar to placebo, particularly noteworthy in a terminally ill population. The slight weight gain trend in the treatment group, while not statistically detailed, aligns with improved appetite signals.
This data positions VTGN strategically in the cancer supportive care market. Cancer cachexia affects approximately 50-80% of cancer patients and contributes significantly to mortality, making it a substantial market opportunity. PH284's novel nose-to-brain delivery mechanism differentiates it from current treatments, potentially offering faster onset and better tolerability.
The positive Phase 2A results mark PH284 as the fifth successful pherine candidate in Vistagen's pipeline, demonstrating consistent R&D execution and platform validation. This strengthens the company's position for potential partnerships or licensing deals, particularly valuable given their
While early-stage, these results could catalyze near-term value creation through partnership discussions or strategic interest, especially given the unmet need in cancer supportive care. The data quality and safety profile reduce development risk for potential future trials.
PH284 nasal spray demonstrated improvements to subjective feelings of hunger in cancer patients
PH284 is the fifth pherine product candidate in Vistagen’s neuroscience pipeline with a positive efficacy signal
“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,” stated Shawn Singh, President and Chief Executive Officer of Vistagen. “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. PH284 is our fifth novel investigational pherine, each supported by positive Phase 2 or later clinical data and placebo-like tolerability, underscoring the breadth, diversity and potential of our neuroscience pipeline to address multiple significant unmet needs.”
The previously unreported double-blind, placebo-controlled exploratory Phase 2A study was designed to evaluate the efficacy, safety, and tolerability of intranasal administration of PH284 in female patients diagnosed with cachexia (induced by chronic loss of appetite) due to terminal cancer (n=40). PH284 nasal spray (0.4 µg/50 µL) was administered intranasally, one spray in each nostril (total daily dose = 3.2µg), four times daily before meals (breakfast, mid-morning snack, lunch, and dinner). From Day 1 through Day 4, all subjects were administered placebo 30 minutes prior to each meal. Beginning on Day 5 through Day 11 subjects were randomized in a 1:1 fashion to receive either PH284 or placebo.
Efficacy
Patients measured Subjective Feeling of Hunger (SFH) ten minutes before each meal. PH284, as compared to placebo, induced a cumulative effect on mean SFH scores, with scores increasing from breakfast to lunch and lunch to dinner throughout the treatment period. Specifically, prior to dinner on Day 7 of treatment, PH284 subjects reported a
Safety and Tolerability
No unusual changes in body weight were observed in either the PH284 or placebo groups, though on average, there was a small gain in body weight for PH284 versus a small loss in placebo. PH284 demonstrated no serious adverse events, and adverse events reported for the PH284 group were similar to those reported in the placebo-treated group. All the adverse events reported were attributed to the underlying medical condition (cancer) and were not deemed to be related to the administration of PH284 or placebo.
About Cachexia
Cachexia, also known as wasting syndrome, is a complex metabolic syndrome that causes a gradual loss of muscle and body weight. Cachexia is associated with chronic diseases like cancer, AIDS, heart failure, chronic obstructive pulmonary disease (COPD), anorexia nervosa, multiple sclerosis, tuberculosis, and anemias. The current definition of cancer cachexia is a loss of
This previously unreported Phase 2A exploratory study of PH284 was sponsored by Pherin Pharmaceuticals (Pherin), now a wholly owned subsidiary of Vistagen, and conducted at the National Institute of Oncology (INCAN) and National Institute of Nutrition (INNSZ) in
About PH284
PH284 is an investigational neuroactive pherine nasal spray with a novel, rapid-onset potential mechanism of action (MOA) that is fundamentally differentiated from the MOA of all current treatments for the loss of appetite associated with chronic disorders, such as cancer or heart disease. PH284 is thought to act by regulating olfactory to mediobasal-hypothalamus neural circuits involved in appetite control. PH284 has demonstrated an excellent safety profile in all clinical trials completed to date. Vistagen is currently evaluating the potential path forward for PH284, including an assessment of completed studies and studies we believe are necessary to support a
About Vistagen
Headquartered in
Forward-looking Statements
This press release contains certain forward-looking statements within the meaning of the federal securities laws. These forward-looking statements involve known and unknown risks that are difficult to predict and include all matters that are not historical facts. In some cases, you can identify forward-looking statements by the use of words such as “may,” “could,” “expect,” “project,” “outlook,” “strategy,” “intend,” “plan,” “seek,” “anticipate,” “believe,” “estimate,” “predict,” “potential,” “strive,” “goal,” “continue,” “likely,” “will,” “would” and variations of these terms and similar expressions, or the negative of these terms or similar expressions. Such forward-looking statements are necessarily based upon estimates and assumptions that, while considered reasonable by Vistagen and its management, are inherently uncertain. As with all pharmaceutical products, there are substantial risks and uncertainties in the process of development and commercialization and actual timelines, results or development may differ materially from those projected or implied in these forward-looking statements. There can be no guarantee that PH284, or any of Vistagen’s product candidates, will successfully replicate past clinical trials, complete ongoing or future clinical trials within the timeframe estimated by Vistagen or at all, receive regulatory approval or be commercially successful. Other factors that may cause such a difference include, without limitation, risks and uncertainties relating to delays in launching, conducting and/or completing ongoing and planned clinical trials; the availability and scope of applicable patents; fluctuating costs of materials and other resources and services required to conduct Vistagen’s ongoing and/or planned clinical and nonclinical trials; market conditions; the impact of general economic, industry or political conditions in
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Investors:
Mark A. McPartland
Vistagen Therapeutics
markmcp@vistagen.com
Media:
Michelle Wellington
Vistagen Therapeutics
mwellington@vistagen.com
Source: Vistagen
FAQ
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