GH Research Announces Primary Endpoint Met in Phase 2b Trial with GH001 in TRD Demonstrating -15.5 Point Placebo-adjusted MADRS Reduction
GH Research (NASDAQ: GHRS) announced successful Phase 2b trial results for GH001, their inhalable mebufotenin product candidate for treatment-resistant depression (TRD). The trial's primary endpoint was met, showing a significant -15.5 point placebo-adjusted MADRS reduction in depression scores on Day 8 (p<0.0001).
The trial involved 81 TRD patients, with 40 receiving GH001 and 41 receiving placebo. Key achievements include a 57.5% remission rate in the GH001 group compared to 0% in placebo. The drug demonstrated strong safety profiles with no serious adverse events reported. In the ongoing open-label extension (OLE), 77.8% of completers maintained remission at 6 months, with most patients requiring only 1-4 treatments over this period.
GH Research (NASDAQ: GHRS) ha annunciato risultati positivi della fase 2b per GH001, il loro candidato prodotto mebufotenin inalabile per la depressione resistente al trattamento (TRD). L'obiettivo primario dello studio è stato raggiunto, mostrando una significativa riduzione di -15,5 punti dell'MADRS aggiustata per il placebo nei punteggi di depressione al Giorno 8 (p<0.0001).
Lo studio ha coinvolto 81 pazienti TRD, con 40 che hanno ricevuto GH001 e 41 che hanno ricevuto placebo. Tra i risultati chiave si evidenzia un tasso di remissione del 57,5% nel gruppo GH001 rispetto allo 0% nel gruppo placebo. Il farmaco ha dimostrato un forte profilo di sicurezza, senza eventi avversi gravi segnalati. Nella continuazione in aperto in corso (OLE), il 77,8% dei completatori ha mantenuto la remissione a 6 mesi, con la maggior parte dei pazienti che ha richiesto solo 1-4 trattamenti durante questo periodo.
GH Research (NASDAQ: GHRS) anunció resultados exitosos del ensayo de fase 2b para GH001, su candidato a producto de mebufotenin inhalable para la depresión resistente al tratamiento (TRD). El objetivo primario del ensayo se cumplió, mostrando una significativa reducción de -15.5 puntos ajustada por placebo en la escala MADRS en los puntajes de depresión en el Día 8 (p<0.0001).
El ensayo involucró a 81 pacientes con TRD, de los cuales 40 recibieron GH001 y 41 recibieron placebo. Entre los logros clave se incluye una tasa de remisión del 57.5% en el grupo GH001 en comparación con 0% en el placebo. El fármaco demostró perfiles de seguridad sólidos sin reportes de eventos adversos graves. En la extensión en abierto en curso (OLE), el 77.8% de los completadores mantuvieron la remisión a los 6 meses, siendo la mayoría de los pacientes que requirieron solo 1-4 tratamientos durante este período.
GH Research (NASDAQ: GHRS)는 치료 저항성 우울증(TRD)을 위한 흡입형 메부포테닌 후보 제품 GH001의 2b상 시험 결과를 발표했습니다. 시험의 주요 목표는 달성되었으며, 8일 차에서 우울증 점수가 플라세보 조정으로 -15.5 포인트 감소했음을 보여주었습니다 (p<0.0001).
이 시험에는 81명의 TRD 환자가 참여했으며, 40명이 GH001을, 41명이 플라세보를 받았습니다. 주요 성과로는 GH001 그룹에서 57.5%의 완전 관해율이 나타났고, 플라세보 그룹은 0%였습니다. 이 약물은 심각한 부작용이 보고되지 않아 안전성 프로필이 강한 것으로 나타났습니다. 진행 중인 개방형 연장 시험(OLE)에서 완료자의 77.8%가 6개월 후에도 관해를 유지했습니다, 대부분의 환자는 이 기간 동안 1-4회의 치료만 필요했습니다.
GH Research (NASDAQ: GHRS) a annoncé des résultats positifs de l'essai de phase 2b pour GH001, leur candidat de produit inhalable de mébufoténine pour la dépression résistante au traitement (TRD). L'objectif principal de l'essai a été atteint, montrant une réduction ajustée par placebo de -15,5 points sur l'échelle MADRS des scores de dépression au Jour 8 (p<0.0001).
L'essai a impliqué 81 patients atteints de TRD, dont 40 ont reçu GH001 et 41 ont reçu un placebo. Parmi les résultats clés, on note un taux de rémission de 57,5% dans le groupe GH001 contre 0% dans le groupe placebo. Le médicament a montré un fort profil de sécurité, sans événements indésirables graves rapportés. Dans l'extension en ouvert en cours (OLE), 77,8% des patients ayant terminé l'étude ont maintenu leur rémission à 6 mois, la plupart n'ayant besoin que de 1 à 4 traitements au cours de cette période.
GH Research (NASDAQ: GHRS) gab die erfolgreichen Ergebnisse der Phase 2b-Studie für GH001, ihr inhalierbares Mebufotenin-Produktkandidaten zur Behandlung von behandlungsresistenter Depression (TRD), bekannt. Der primäre Endpunkt der Studie wurde erreicht, wobei eine signifikante Reduktion von -15,5 Punkten im MADRS, placebo-adjustiert, bei den Depressionswerten am Tag 8 zu verzeichnen war (p<0.0001).
Die Studie umfasste 81 TRD-Patienten, wobei 40 GH001 und 41 ein Placebo erhielten. Zu den wichtigsten Ergebnissen gehört eine Remissionsrate von 57,5% in der GH001-Gruppe im Vergleich zu 0% in der Placebo-Gruppe. Das Medikament zeigte starke Sicherheitsprofile, ohne dass ernsthafte unerwünschte Ereignisse gemeldet wurden. In der laufenden offenen Verlängerung (OLE) behielten 77,8% der abschließenden Patienten nach 6 Monaten die Remission bei, wobei die meisten Patienten nur 1-4 Behandlungen in diesem Zeitraum benötigten.
- Significant -15.5 point placebo-adjusted MADRS reduction achieved on Day 8
- High remission rate of 57.5% compared to 0% in placebo group
- Strong 6-month durability with 77.8% of OLE completers maintaining remission
- Minimal treatment frequency needed (1-4 treatments over 6 months)
- Excellent safety profile with no serious adverse events reported
- 18 patients discontinued the trial early
- One discontinuation due to adverse event
Insights
The Phase 2b results for GH001 represent a potential breakthrough in TRD treatment. The -15.5 point placebo-adjusted MADRS reduction is substantially larger than what's typically seen with current TRD treatments, where reductions of 3-5 points are considered clinically meaningful. The 57.5% remission rate is particularly impressive given that typical remission rates in TRD hover around 10-20% with standard treatments.
The rapid onset of action within 8 days and the durability of response are game-changing features. The 77.8% remission rate at 6 months with only 1-4 treatments suggests a paradigm shift from daily medication to episodic intervention. This could dramatically improve treatment compliance and reduce the economic burden of TRD.
The safety profile is robust, with several noteworthy features:
- No serious adverse events
- No treatment-emergent suicidal ideation
- Quick discharge readiness (97.4% within 1 hour)
- No post-discharge restrictions
GH001's clinical profile presents compelling commercial advantages in the TRD market. The combination of rapid onset, high remission rates and infrequent dosing creates a unique value proposition that could command premium pricing and favorable reimbursement terms. The 1-3 hour clinic visits with 1-4 treatments over 6 months represent a dramatic reduction in treatment burden compared to daily oral medications or twice-weekly esketamine sessions.
Key market differentiators include:
- Ultra-rapid onset (8 days vs. weeks/months for conventional treatments)
- Minimal treatment burden (1-4 treatments per 6 months)
- High remission rates maintained long-term
- Simple administration protocol requiring minimal healthcare resources
- Primary endpoint met, GH001 led to an ultra-rapid anti-depressant effect with a significant placebo-adjusted MADRS reduction from baseline of -15.5 on Day 8 (p<0.0001)
- The majority of the patients treated with GH001 achieved remission with a
57.5% remission rate on Day 8 compared with0% in the placebo group (p<0.0001) - All other secondary endpoints were met with clinically and statistically significant improvements on Day 8, compared with placebo
- During the double-blind part, GH001 was well tolerated and no serious adverse events (SAE) were reported. There was no evidence of treatment-emergent suicidal ideation or behavior. As of January 22, 2025, no SAEs have been reported throughout the open label extension (OLE)
- As of January 22, 2025,
77.8% of the OLE completers were in remission at the 6-month visit, with infrequent treatments - Patients who had remission on Day 8 after their first active treatment had a
91.7% remission rate at 6 months
DUBLIN, Feb. 03, 2025 (GLOBE NEWSWIRE) -- GH Research PLC (Nasdaq: GHRS), a clinical-stage biopharmaceutical company, today reported the primary endpoint was met in a randomized, double-blind, placebo-controlled Phase 2b clinical trial with GH001, an inhalable mebufotenin product candidate, in patients with treatment-resistant depression (TRD) (GH001-TRD-201). GH Research will host a conference call and live webcast today at 8.00 a.m. EST. To register for the event, please click here.
The trial recruited 81 patients with TRD. In the double-blind part, 40 patients received GH001 and 41 received placebo. Psychotherapeutic intervention was not a component of either part of this trial.
GH001 led to a significant reduction from baseline of -15.2 points in Montgomery-Åsberg Depression Rating Scale (MADRS) total score on Day 8, compared with +0.3 points in the placebo group (difference of -15.5 points, p<0.0001).
All secondary endpoints in the trial were met, with results consistent with the primary endpoint. Treatment with GH001 led to clinically and statistically significant improvements on the CGI-S and HAM-A scales and the Q-LES-Q-SF Questionnaire on Day 8, compared with placebo.
"Patients treated with GH001 experienced a difference of -15.5 points in MADRS score at Day 8 compared to placebo, which is truly remarkable,” said Professor Michael E. Thase, MD, Professor of Psychiatry, Perelman School of Medicine, University of Pennsylvania. “Most TRD patients have not benefited from a number of established treatment options and this illness frequently imposes years of insurmountable mental suffering and disabling effects on social and vocational functioning. A novel treatment with such a large and rapid effect, particularly one that may require only infrequent, short 1-3 hours clinic visits, has the potential to be a practice changing treatment.”
GH001 was well tolerated and no serious adverse events were reported in the double-blind part of the trial. All treatment emergent adverse events (TEAEs) were mild or moderate with no severe adverse events observed. There were no TEAEs of flashbacks reported. No clinically significant changes were observed in any of the vital parameters, including heart rate, blood pressure and ECG. No dissociative state symptoms or sedation were observed at discharge after treatment with GH001 and
As of January 22, 2025, 9 patients are ongoing, 54 patients have completed and 18 patients have discontinued early (with one discontinuation due to an adverse event). Of the OLE completers,
"Today, as we share our unprecedented positive Phase 2b data, we celebrate a significant milestone in our journey to interventional psychiatry and pave the way for our future commercial success with GH001 in treatment-resistant depression," said Dr. Villy Valcheva, Chief Executive Officer of GH Research. "The ultra-rapid and profound reduction in depressive symptoms, coupled with sustained remission through infrequent, short treatment visits, positions us uniquely."
Webcast Information
GH Research will host a conference call and live webcast today at 8:00 a.m. EST. A live question and answer session will follow the formal presentation. To register for the event, please click here.
A live webcast of the call will be available under “Events & Presentations” in the Investors section of GH Research’s website at ghres.com.
About GH Research PLC
GH Research PLC is a clinical-stage biopharmaceutical company dedicated to transforming the lives of patients by developing a practice-changing treatment in depression. GH Research PLC's initial focus is on developing its novel and proprietary mebufotenin therapies for the treatment of patients with treatment-resistant depression (TRD).
About GH001
Our lead product candidate, GH001, is formulated for 5-MeO-DMT administration via a proprietary inhalation approach.
About Notation for Trial Timepoints
In relation to our clinical trials, we have previously referred to the day of dosing as Day 0 (D0), the day after dosing as Day 1 (D1), and the seventh day after dosing as Day 7 (D7). In this press release, and going forward, we shall refer to the day of dosing as Day 1 (D1), the day after dosing as Day 2 (D2) and the seventh day after dosing as Day 8 (D8).
Forward-Looking Statements
This press release contains statements that are, or may deemed to be, forward-looking statements. All statements other than statements of historical fact included in this press release, including statements regarding the ongoing OLE part of our Phase 2b trial with GH001 in TRD, our future results of operations and financial position, business strategy, product candidates, research pipeline, ongoing and currently planned preclinical studies and clinical trials, regulatory submissions and approvals, research and development costs, timing and likelihood of success, as well as plans and objectives of management for future operations are forward-looking statements. Forward-looking statements appear in a number of places in this press release and include, but are not limited to, statements regarding our intent, belief or current expectations. Forward-looking statements are based on our management’s beliefs and assumptions and on information currently available to our management. Such statements are subject to risks and uncertainties, and actual results may differ materially from those expressed or implied in the forward-looking statements due to various factors, including, but not limited to, those described in our filings with the U.S. Securities and Exchange Commission. No assurance can be given that such future results will be achieved. Such forward-looking statements contained in this document speak only as of the date of this press release. We expressly disclaim any obligation or undertaking to update these forward-looking statements contained in this press release to reflect any change in our expectations or any change in events, conditions, or circumstances on which such statements are based unless required to do so by applicable law. No representations or warranties (expressed or implied) are made about the accuracy of any such forward-looking statements.
Investor Relations
Julie Ryan
GH Research PLC
investors@ghres.com
FAQ
What were the primary results of GHRS's Phase 2b trial for GH001?
How long did the antidepressant effects of GH001 (GHRS) last in the trial?
What safety concerns were reported in GHRS's Phase 2b trial of GH001?
How many patients participated in GHRS's Phase 2b trial for GH001?
What is the discharge time after GH001 treatment for GHRS patients?
