GH Research Announces Primary Endpoint Met in Two Phase 2a POC Trials with GH001 and Completion of All FDA Requests to Address IND Hold with No Findings of Respiratory Toxicity in Non-Rodents
GH Research (GHRS) announced successful results from two Phase 2a proof-of-concept trials for GH001. The first trial in postpartum depression (PPD) showed a significant MADRS score reduction of -35.4 points with 100% of patients achieving remission by Day 8. The second trial in bipolar II disorder (BDII) demonstrated a MADRS reduction of -16.8 points with 33.3% of patients in remission by Day 8.
The company completed FDA-requested inhalation toxicology studies in dogs showing no respiratory tract findings, supporting their position that previous findings were rat-specific. GHRS plans to submit a full response to the FDA's IND hold in mid-2025. Their Phase 2b trial in treatment-resistant depression remains on track with top-line data expected in Q1 2025.
The company's financial position remains strong with $182.6 million in cash and equivalents as of December 31, 2024, compared to $222.7 million the previous year.
GH Research (GHRS) ha annunciato risultati positivi da due studi clinici di fase 2a per GH001. Il primo studio sulla depressione postpartum (PPD) ha mostrato una significativa riduzione del punteggio MADRS di -35,4 punti, con il 100% dei pazienti in remissione entro l'ottavo giorno. Il secondo studio sul disturbo bipolare di tipo II (BDII) ha dimostrato una riduzione del MADRS di -16,8 punti, con il 33,3% dei pazienti in remissione entro l'ottavo giorno.
L'azienda ha completato studi di tossicità per inalazione richiesti dalla FDA su cani, non evidenziando problemi alle vie respiratorie, sostenendo così la propria posizione che i risultati precedenti erano specifici per i ratti. GHRS prevede di presentare una risposta completa al blocco IND della FDA a metà 2025. Il loro studio di fase 2b sulla depressione resistente al trattamento è ancora in programma, con i dati principali attesi nel primo trimestre del 2025.
La posizione finanziaria dell'azienda rimane solida, con 182,6 milioni di dollari in contanti e equivalenti al 31 dicembre 2024, rispetto ai 222,7 milioni dell'anno precedente.
GH Research (GHRS) anunció resultados exitosos de dos ensayos clínicos de fase 2a para GH001. El primer ensayo en depresión posparto (PPD) mostró una reducción significativa en la puntuación MADRS de -35.4 puntos, con el 100% de los pacientes alcanzando remisión para el día 8. El segundo ensayo en trastorno bipolar II (BDII) demostró una reducción en el MADRS de -16.8 puntos, con el 33.3% de los pacientes en remisión para el día 8.
La empresa completó estudios de toxicología por inhalación solicitados por la FDA en perros, mostrando que no hubo hallazgos en las vías respiratorias, lo que respalda su posición de que los hallazgos anteriores eran específicos de ratas. GHRS planea presentar una respuesta completa a la suspensión IND de la FDA a mediados de 2025. Su ensayo de fase 2b en depresión resistente al tratamiento sigue en marcha, con datos principales esperados para el primer trimestre de 2025.
La situación financiera de la empresa sigue siendo sólida, con 182.6 millones de dólares en efectivo y equivalentes al 31 de diciembre de 2024, en comparación con 222.7 millones del año anterior.
GH Research (GHRS)는 GH001의 두 가지 2a 단계 개념 증명 시험에서 성공적인 결과를 발표했습니다. 첫 번째 시험에서는 산후 우울증 (PPD)에 대해 MADRS 점수가 -35.4 포인트 감소하였고, 환자의 100%가 8일 차에 완화를 달성했습니다. 두 번째 시험에서는 양극성 II형 장애 (BDII)에 대해 MADRS 점수가 -16.8 포인트 감소하였고, 환자의 33.3%가 8일 차에 완화를 보였습니다.
회사는 FDA가 요구한 개를 대상으로 한 흡입 독성학 연구를 완료하였으며, 호흡계에서의 발견이 없음을 보여주어 이전 발견이 쥐에 특정적이었다는 주장을 뒷받침하고 있습니다. GHRS는 2025년 중반에 FDA의 IND 보류에 대한 전체 대응을 제출할 계획입니다. 치료 저항성 우울증에 대한 2b 단계 시험은 순조롭게 진행 중이며, 주요 데이터는 2025년 1분기에 예상됩니다.
회사의 재정 상태는 여전히 강력하며, 2024년 12월 31일 기준으로 1억 8260만 달러의 현금 및 현금성 자산을 보유하고 있으며, 이는 전년도의 2억 2270만 달러와 비교됩니다.
GH Research (GHRS) a annoncé des résultats positifs issus de deux essais cliniques de phase 2a pour GH001. Le premier essai sur la dépression postpartum (PPD) a montré une réduction significative du score MADRS de -35,4 points, avec 100 % des patients atteignant la rémission au huitième jour. Le deuxième essai sur le trouble bipolaire de type II (BDII) a démontré une réduction du MADRS de -16,8 points, avec 33,3 % des patients en rémission au huitième jour.
L'entreprise a complété les études de toxicologie par inhalation demandées par la FDA sur des chiens, montrant qu'il n'y avait pas de résultats dans les voies respiratoires, soutenant ainsi leur position que les résultats précédents étaient spécifiques aux rats. GHRS prévoit de soumettre une réponse complète à la suspension de l'IND de la FDA à la mi-2025. Leur essai de phase 2b sur la dépression résistante au traitement reste sur la bonne voie, avec des données clés attendues au premier trimestre 2025.
La position financière de l'entreprise reste solide avec 182,6 millions de dollars en espèces et équivalents au 31 décembre 2024, contre 222,7 millions de dollars l'année précédente.
GH Research (GHRS) hat erfolgreiche Ergebnisse aus zwei Phase-2a-Pilotstudien für GH001 angekündigt. Die erste Studie zur postpartalen Depression (PPD) zeigte eine signifikante Verringerung der MADRS-Punkte um -35,4 Punkte, wobei 100 % der Patienten bis Tag 8 in Remission waren. Die zweite Studie zu bipolaren Störung Typ II (BDII) wies eine Verringerung der MADRS um -16,8 Punkte auf, wobei 33,3 % der Patienten bis Tag 8 in Remission waren.
Das Unternehmen hat die von der FDA geforderten inhalativen Toxizitätsstudien an Hunden abgeschlossen, die keine Atemwegsergebnisse zeigten, was ihre Position unterstützt, dass frühere Ergebnisse spezifisch für Ratten waren. GHRS plant, Mitte 2025 eine vollständige Antwort auf die IND-Sperre der FDA einzureichen. Ihre Phase-2b-Studie zur behandlungsresistenten Depression bleibt auf Kurs, und die wichtigsten Daten werden im ersten Quartal 2025 erwartet.
Die finanzielle Lage des Unternehmens bleibt stark mit 182,6 Millionen Dollar an Bargeld und liquiden Mitteln zum 31. Dezember 2024, im Vergleich zu 222,7 Millionen Dollar im Vorjahr.
- Phase 2a PPD trial met primary endpoint with 100% remission rate
- Phase 2a BDII trial met primary endpoint with 33.3% remission rate
- No respiratory toxicity found in dog studies, supporting IND progression
- Strong cash position of $182.6M as of December 2024
- Phase 2b TRD trial on track for Q1 2025 data readout
- FDA IND hold remains in effect until mid-2025
- Cash position decreased by $40.1M year-over-year
- Lower remission rate in BDII trial (33.3%) compared to PPD trial (100%)
Insights
The clinical trial results for GH001 represent a remarkable breakthrough in psychiatric treatment. The 100% remission rate in postpartum depression patients with a MADRS reduction of -35.4 points is exceptionally strong, particularly given the rapid onset of action within 2 hours. For context, traditional antidepressants typically take 4-6 weeks to show meaningful effects and rarely achieve such high remission rates.
The bipolar II disorder trial, while showing more modest results with a 33.3% remission rate, still demonstrated statistically significant improvements. The absence of mania or hypomania events is particularly noteworthy, as this is a common concern with antidepressant treatment in bipolar patients.
The resolution of FDA's safety concerns through the non-rodent toxicology studies, showing no respiratory tract issues, substantially de-risks the regulatory pathway. This positions GH001 favorably for potential FDA approval, pending the upcoming Phase 2b TRD results.
The positive clinical data and regulatory progress significantly strengthen GHRS's market position. With
The upcoming Q1 2025 readout of the Phase 2b TRD trial represents a major catalyst. The strong efficacy signals in PPD and BDII trials suggest increased probability of success for the TRD study. A positive outcome could drive significant value appreciation, given the multi-billion dollar market opportunity in treatment-resistant depression.
The therapeutic profile of GH001 - rapid onset, high remission rates and good tolerability - addresses major unmet needs in psychiatric treatment. The postpartum depression market, while smaller than TRD, represents a compelling commercial opportunity with competition. The 96.3% reduction in MADRS scores surpasses current treatment options.
The broader implications for psychiatric treatment are substantial. The ultra-rapid onset (2-hour response) could revolutionize emergency psychiatric care, particularly for severe depression. The upcoming TRD trial results will be important - success in this larger market could position GH001 as a paradigm-shifting therapy in mental health treatment.
- Primary endpoint met in phase 2a POC trial in postpartum depression with a MADRS reduction from baseline of –35.4 points (p<0.0001, n=10) and
100% of patients in remission at Day 8 - Primary endpoint met in phase 2a POC trial in bipolar II disorder with a current major depressive episode with a MADRS reduction from baseline of –16.8 points (p=0.0099, n=6) and
33% of patients in remission at Day 8 - In both trials, GH001 was well tolerated and no treatment-related serious adverse events were reported
- Inhalation toxicology study in a non-rodent species was completed with no histology findings in the respiratory tract of any dogs in the study at any dose level
- Additional inhalation toxicology study in rats was completed supporting our position that respiratory tract histology findings are rat specific
- Our response to FDA’s request for additional device design verification information is being prepared
- Full response to the IND hold planned to be submitted in mid-2025
- Top-line data from our randomized, double-blind, placebo-controlled Phase 2b trial in TRD on track to be announced in Q1 2025
DUBLIN, Jan. 10, 2025 (GLOBE NEWSWIRE) -- GH Research PLC (Nasdaq: GHRS), a clinical-stage biopharmaceutical company dedicated to transforming the treatment of psychiatric and neurological disorders, today provided updates on its business and highlighted key upcoming milestones.
Primary Endpoint Met in Phase 2a Proof-of-Concept Trial in PPD
The primary endpoint of the Phase 2a proof-of-concept (POC) trial for GH001 in postpartum depression (PPD) was met with a significant reduction from baseline of –35.4 points (
GH001 led to an ultra-rapid antidepressant effect with a significant reduction in MADRS score at 2 hours after administration of –31.4 points (p<0.0001) and on Day 2 of –36.0 points (p<0.0001).
The trial recruited 10 patients with PPD. All patients were administered an individualized dosing regimen (IDR) of up to three escalating doses of GH001. There was no psychotherapeutic intervention in this trial. The mean total MADRS score on Day 8 was 1.3 and all 10 patients were in remission.
GH001 was well tolerated and no treatment-related serious adverse events were reported. All treatment-emergent adverse events (TEAEs) were mild or moderate.
Primary Endpoint Met in Phase 2a Proof-of-Concept Trial in BDII
The primary endpoint of the Phase 2a POC trial for GH001 in bipolar II disorder with a current major depressive episode (BDII) was met with a significant reduction from baseline of –16.8 points (
GH001 led to an ultra-rapid antidepressant effect with a reduction in MADRS score at 2 hours after administration of –16.3 points (p=0.0006) and on Day 2 of -13.3 points (p=0.0299).
The trial recruited 6 patients with BDII. All patients were administered an IDR of up to three escalating doses of GH001. There was no psychotherapeutic intervention in this trial.
GH001 was well tolerated and no treatment-related serious adverse events were reported. The majority of TEAEs were mild or moderate and there were no reported TEAEs of hypomania or mania.
Update on IND for GH001
As previously announced, our Investigational New Drug Application (IND) for GH001 administered using our proprietary aerosol delivery device has been placed on clinical hold by the U.S. Food and Drug Administration (FDA), with the FDA requesting that we provide (i) an inhalation toxicology study in a non-rodent species and an additional inhalation toxicology study in rats, (ii) additional device design verification information and (iii) updates to our investigator brochure, to resolve the hold.
The requested additional inhalation toxicology study in a non-rodent species has now been completed. The pathology report concludes that there are no histology findings in the respiratory tract of any dogs at any dose level evaluated in the study.
The requested additional inhalation toxicology study in rats has now been completed which showed histology findings consistent with our previously completed study in rats. This supports our position that these findings are rat specific.
Based on previously announced FDA interactions, the response to their request for additional device design verification information is being prepared and, together with the completion of the inhalation toxicology studies, provides the final piece of information requested by the agency.
We are preparing to engage with the FDA in advance of providing a full response to the IND hold which we plan to submit in mid-2025.
Business Updates
GH001 in Patients with TRD
As previously announced, we completed enrolment of the double-blind phase of our randomized, double-blind, placebo-controlled Phase 2b trial in 80 treatment-resistant depression (TRD) patients in the third quarter of 2024, with top-line data on track to be announced in the first quarter of 2025. This trial also includes a 6-month open-label extension which is on track for completion of last patient visit in the first quarter of 2025.
Cash Position
Cash, cash equivalents, other financial assets and marketable securities were
About GH Research PLC
GH Research PLC is a clinical-stage biopharmaceutical company dedicated to transforming the treatment of psychiatric and neurological disorders. 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 mebufotenin administration via a proprietary inhalation approach. Based on the observed clinical activity in our completed phase 1/2 GH001-TRD-102 trial, where
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 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
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