Core Insights - GH Research PLC reported financial results for Q3 2025, highlighting advancements in their treatment for depression and ongoing engagement with the FDA regarding their Investigational New Drug Application for GH001 [1][2]. Business Updates - GH001 Update: The company is addressing the last remaining topic from the FDA's clinical hold on their IND for GH001, with ongoing communication with the FDA [2]. - Phase 2b TRD Data: The full dataset from the Phase 2b clinical trial of GH001 in treatment-resistant depression (TRD) was reported, showing a significant reduction in depression scores [3][12]. - Remission Rate: The trial confirmed a 73% remission rate at 6 months with no serious adverse events reported during the trial [4][5]. Financial Highlights - Cash Position: As of September 30, 2025, the company had cash, cash equivalents, and marketable securities totaling $293.9 million, an increase from $182.6 million at the end of 2024 [7][17]. - R&D Expenses: Research and development expenses for Q3 2025 were $10.6 million, up from $8.4 million in Q3 2024, primarily due to increased technical development costs [7][9]. - G&A Expenses: General and administrative expenses rose to $6.0 million in Q3 2025 from $4.2 million in Q3 2024, attributed to higher professional fees and employee expenses [10]. - Net Loss: The net loss for Q3 2025 was $14.0 million, or $0.23 per share, compared to a loss of $12.1 million, or $0.23 per share, in the same quarter of 2024 [10][16].

Core Insights - Needham initiated coverage on GH Research PLC, highlighting the efficacy of its lead drug candidate, GH-001, in Phase 2 trials compared to competitors [1] Company Developments - GH Research submitted a complete response to the FDA regarding the clinical hold on the Investigational New Drug Application (IND) for GH-001, with only one hold topic remaining related to respiratory tract histology findings in rats [2] - There are no additional requests from the FDA concerning dog toxicology or device-related issues [3] Clinical Trial Results - Initial results from the Phase 2b clinical trial of GH-001 in treatment-resistant depression showed a 73% remission rate at six months, with 57.5% of patients achieving remission by day eight and 90% at month six [4] Analyst Insights - Needham assigned a Buy rating with a price forecast of $19 for GH Research, estimating that GH-001 could generate $1.9 billion in U.S. sales by 2035, applying a 35% probability of success to account for risks [5] - The enterprise value of GH Research is currently at $500 million, indicating potential long-term upside [6]

Core Insights - GH Research PLC is focused on developing innovative treatments for treatment-resistant depression (TRD) and has announced a presentation at the 38th Annual European College of Neuropsychopharmacology Congress [1][3] Group 1: Clinical Data and Presentations - A Novel Therapies Symposium Presentation will feature long-term clinical data on the safety and efficacy of GH001 from a Phase 2b clinical trial for TRD [1][4] - Two posters will present additional safety and tolerability data from the open-label extension of the same trial, as well as psychoactive effects of GH001 [2][5] Group 2: Unmet Medical Need and Treatment Efficacy - There is a significant unmet medical need for TRD, as many patients do not achieve adequate relief with existing therapies [3] - GH001 has shown a favorable safety and tolerability profile, with no serious adverse events or treatment-emergent suicidal intent reported during the 6-month open-label extension [3][5] Group 3: Product Information - GH001 is a proprietary formulation of mebufotenin administered via inhalation, which has demonstrated a significant reduction in the Montgomery-Åsberg Depression Rating Scale (MADRS) by 15.5 points compared to placebo on Day 8 in the Phase 2b trial [8]

Core Insights - GH Research PLC is focused on developing innovative treatments for depression, particularly treatment-resistant depression (TRD) [15][16] - The company reported significant progress in its clinical trials, particularly with its lead product candidate GH001, which demonstrated a substantial reduction in depression scores [4][16] Business Updates - The FDA has communicated regarding the clinical hold on the Investigational New Drug Application (IND) for GH001, with only one topic remaining to be addressed [2] - A Phase 1 clinical trial for GH001 using a proprietary aerosol delivery device is ongoing in the UK, aimed at supporting the global program for GH001 [3] - The Phase 2b trial for GH001 in TRD met its primary endpoint with a significant placebo-adjusted reduction of -15.5 points in the Montgomery-Åsberg Depression Rating Scale (MADRS) [4][16] - An open-label extension analysis confirmed a 73% remission rate at 6 months with no serious adverse events reported [5][6][8] Financial Highlights - As of June 30, 2025, the company had cash, cash equivalents, and marketable securities totaling $308.7 million, an increase from $182.6 million at the end of 2024 [11] - Research and development expenses for Q2 2025 were $9.0 million, a decrease from $9.8 million in the same quarter of 2024 [12] - General and administrative expenses rose to $5.7 million in Q2 2025 from $3.5 million in Q2 2024, primarily due to increased professional fees and employee expenses [13] - The net loss for Q2 2025 was $9.3 million, or $0.15 loss per share, compared to a net loss of $10.4 million, or $0.20 loss per share, in Q2 2024 [14] Future Plans - The company is on track to initiate its global pivotal program for GH001 in 2026 [10][8] - Plans for submitting an IND for GH002, another product candidate, are expected in Q4 2025 following a successful Phase 1 trial [9]

Core Insights - GH Research PLC is focused on developing innovative treatments for depression, particularly targeting treatment-resistant depression (TRD) [12] GH001 Update - The company submitted a complete response to the FDA regarding the clinical hold on its Investigational New Drug Application (IND) for GH001, with only one hold topic remaining related to respiratory tract histology findings in rats [2][3] - The primary endpoint of the Phase 2b clinical trial for GH001 was met, showing a significant placebo-adjusted reduction of -15.5 points in the Montgomery-Åsberg Depression Rating Scale (MADRS) total score on Day 8 (p<0.0001) [4][12] - The full analysis of the open-label extension (OLE) phase confirmed a 73% remission rate at 6 months, with 57.5% of patients achieving remission by Day 8 and 90% of those remaining in remission at month 6 [5][10] - Safety analysis indicated that 100% of patients from the double-blind part continued in the OLE, with no treatment-related serious adverse events reported [6][10] GH002 Update - GH Research PLC completed a Phase 1, dose-ranging clinical pharmacology trial of GH002, which was well-tolerated with no severe adverse events and demonstrated ultra-rapid psychoactive effects [8] - The pharmacokinetic profile of GH002 was found to be equivalent to that of GH001, with plans to submit an IND for GH002 in Q4 2025 [8] Global Pivotal Program Plans - The company is on track to initiate its global pivotal program in 2026, with planning ongoing since Q1 2025 [11][10] - A steering committee with key opinion leaders (KOLs) has been established to review Phase 2b results and assist in the design of the pivotal program [14]

Core Insights - GH Research PLC has submitted a complete response to the FDA regarding the clinical hold of its Investigational New Drug Application (IND) for GH001, ahead of schedule [1][2] - The company is focused on developing GH001, a treatment for treatment-resistant depression (TRD), utilizing a proprietary inhalation method [3] Company Developments - The response to the FDA includes comprehensive data and completed toxicology studies, demonstrating the company's commitment to regulatory compliance and patient care [2] - GH001 has shown promising results in the Phase 2b GH001-TRD-201 trial, achieving a significant reduction in the Montgomery-Åsberg Depression Rating Scale (MADRS) score by -15.5 points compared to placebo on Day 8 (p<0.0001) [3]

Core Insights - GH Research PLC announced the acceptance of a Pharmaceutical Pipeline Presentation at the ASCP Annual Meeting, where clinical data from a Phase 2b trial of GH001 for treatment-resistant depression will be presented [1][7] - The Phase 2b trial results indicate robust evidence of GH001's efficacy and safety, potentially transforming treatment for patients with treatment-resistant depression [2][10] - Additional late-breaking posters will present safety and tolerability data from the Phase 2b trial and a proof-of-concept trial for postpartum depression [3][4] Company Overview - GH Research PLC is a clinical-stage biopharmaceutical company focused on developing innovative treatments for depression, particularly targeting treatment-resistant depression with its lead product candidate, GH001 [9][10] - GH001 is administered via a proprietary inhalation method and has shown promising clinical activity in trials, meeting primary endpoints with significant reductions in depressive symptoms [10] Clinical Trial Highlights - The Phase 2b trial (GH001-TRD-201) demonstrated a mean reduction of 15.5 points in the Montgomery-Åsberg Depression Rating Scale (MADRS) compared to placebo on Day 8, with a p-value of less than 0.0001, indicating strong efficacy [10] - In a proof-of-concept trial for postpartum depression, all ten participants achieved remission by Day 8, highlighting GH001's potential as an effective treatment option [4][8] - A separate Phase 2a trial for bipolar II disorder showed a 52% mean reduction in depressive symptoms by Day 8, suggesting rapid onset and substantial efficacy [6][9]

Core Insights - GH Research PLC reported financial results for Q1 2025, highlighting significant advancements in its clinical trials for GH001, a treatment for treatment-resistant depression (TRD) [1][9]. Business Updates - The Phase 2b trial of GH001 in 81 patients with TRD has completed, achieving its primary endpoint with a significant placebo-adjusted reduction of -15.5 on the Montgomery-Åsberg Depression Rating Scale (MADRS) by Day 8 [2][3]. - A 57.5% remission rate was observed in patients treated with GH001 on Day 8, compared to 0% in the placebo group [3]. - Safety analysis for the open-label extension (OLE) has not been completed, but as of January 22, 2025, no serious adverse events (SAEs) were reported [4]. - In the OLE, 77.8% of completers were in remission at the 6-month visit, with a 91.7% remission rate for those who achieved remission on Day 8 [4]. - The company is preparing to submit a full response to the FDA regarding the clinical hold on the Investigational New Drug Application (IND) for GH001, with submission expected in mid-2025 [6][7]. Financial Highlights - As of March 31, 2025, the company had cash, cash equivalents, and marketable securities totaling $315.3 million, up from $182.6 million as of December 31, 2024 [10]. - Gross proceeds from a public offering in Q1 2025 amounted to $150 million [10]. - Research and development expenses for Q1 2025 were $7.9 million, a decrease from $8.7 million in the same quarter of 2024 [11]. - General and administrative expenses increased to $4.9 million in Q1 2025 from $2.9 million in Q1 2024 [12]. - The net loss for Q1 2025 was $10.8 million, or $0.19 loss per share, compared to a net loss of $7.7 million, or $0.15 loss per share, in Q1 2024 [12]. Clinical Development - GH001 is being developed for various indications, including bipolar II disorder and postpartum depression, with ongoing proof-of-concept trials [5]. - The Phase 1 clinical pharmacology trial for GH001 using a proprietary aerosol delivery device is ongoing in the UK [8].

Core Insights - GH Research PLC reported financial results for the year ended December 31, 2024, and provided updates on its clinical programs and financial position [1] Business Updates - GH001, an inhalable mebufotenin product candidate, is in a Phase 2b trial for treatment-resistant depression (TRD) involving approximately 80 patients [2] - The trial met its primary endpoint with a significant placebo-adjusted reduction of -15.5 in the Montgomery-Åsberg Depression Rating Scale (MADRS) on Day 8 (p<0.0001) [3] - A 57.5% remission rate was observed in patients treated with GH001 on Day 8 compared to 0% in the placebo group (p<0.0001) [3] - Safety analysis for the ongoing open-label extension (OLE) has not been completed, but as of January 22, 2025, 77.8% of OLE completers were in remission at the 6-month visit [4] - The Phase 1 trial for the proprietary aerosol delivery device is ongoing in the UK [5] Regulatory Updates - The Investigational New Drug Application (IND) for GH001 was placed on clinical hold by the FDA, which requested additional inhalation toxicology studies and device design verification [6] - The company has completed the requested inhalation toxicology studies, showing no adverse findings in the respiratory tract of dogs [7][9] - A full response to the IND hold is on track for submission in mid-2025 [10] Clinical Trials - Two Phase 2a proof-of-concept trials for GH001 in postpartum depression (PPD) and bipolar II disorder (BDII) have been completed [11] - The PPD trial met its primary endpoint with a MADRS reduction of -35.4 points (96.3%) on Day 8, with 100% of patients in remission [12] - The BDII trial also met its primary endpoint with a MADRS reduction of -16.8 points (51.9%) on Day 8, with 33.3% of patients in remission [13] Financial Highlights - As of December 31, 2024, the company had cash, cash equivalents, and marketable securities totaling $182.6 million, down from $222.7 million in 2023 [14] - Research and development expenses increased to $35.0 million in 2024 from $29.8 million in 2023, primarily due to clinical development activities [15] - General and administrative expenses rose to $15.3 million in 2024 from $11.4 million in 2023, driven by higher professional fees and increased headcount [16] - The net loss for 2024 was $39.0 million, or $0.75 loss per share, compared to a net loss of $35.6 million, or $0.68 loss per share, in 2023 [17]