GH Research PLC (GHRS): Business Model Canvas [Dec-2025 Updated]

You're digging into the operational engine of GH Research PLC, and honestly, what you'll find is a textbook clinical-stage biopharma play, where value is built almost entirely on regulatory milestones, not product sales-since revenue streams are currently zero. This model is burning serious cash, with Research and Development (R&D) expenses hitting $10.6 million in Q3 2025, but they are funding this with a war chest of $293.9 million in cash and marketable securities as of that same quarter. Their entire proposition rests on delivering an ultra-rapid onset treatment for Treatment-Resistant Depression (TRD) via their novel inhalation device, but they are currently navigating an FDA IND hold on their lead candidate, GH001. To see exactly how they are managing this high-stakes R&D spend, who their key partners are in tackling that regulatory hurdle, and what their path to a potential 2026 pivotal program looks like, dive into the full Business Model Canvas below.

GH Research PLC (GHRS) - Canvas Business Model: Key Partnerships

You're planning the next phase of clinical development, so understanding who GH Research PLC relies on for execution is key. The partnerships GH Research PLC has in place are centered around advancing its mebufotenin pipeline, specifically GH001 for treatment-resistant depression (TRD) and GH002.

Clinical Research Organizations (CROs) for global pivotal trials

The planning for the global pivotal program, which GH Research PLC expects to initiate in 2026, involves external expertise for execution. As of July 2025, the CRO and site selection process is ongoing. This is a critical step following the successful Phase 2b trial results for GH001, which showed a 73% remission rate at 6 months in the Open-Label Extension analysis.

The financial backing for these large-scale trials is supported by a strong balance sheet as of late 2025. Here's a quick look at the financial position supporting these operational commitments:

Key Opinion Leaders (KOLs) and academic institutions for trial design

To ensure the pivotal program design is practice-changing, GH Research PLC established a steering committee. This committee includes KOLs (Key Opinion Leaders) who review the Phase 2b results and assist with the design of the pivotal program. This engagement started in Q1 2025. The Phase 2b trial (GH001-TRD-201) met its primary endpoint with a highly significant placebo-adjusted MADRS reduction from baseline of -15.5 points on Day 8 (p<0.0001).

Regulatory consultants to address the remaining FDA IND hold topic

Addressing the regulatory pathway for GH001 remains a key focus. GH Research PLC received a response from the FDA regarding its complete response to the Investigational New Drug Application (IND) clinical hold, with only one hold topic remaining as of November 2025. The company confirms it is actively working with experts to address the remaining topic. The plan is to submit the complete response to the IND hold after addressing this final item, building on the fact that inhalation toxicology studies requested by the FDA related to the hold were completed, supporting the company's assertion that rat-specific histology findings were addressed.

Contract manufacturers for proprietary mebufotenin (GH001/GH002) supply

The supply chain for the active pharmaceutical ingredients (APIs) and final product formulation is a core operational dependency. GH001 is formulated for mebufotenin administration via a proprietary inhalation approach. GH002, the intravenous formulation, has completed a Phase 1 trial. The company expects to submit an IND for GH002 in Q4 2025. While the need for reliable contract manufacturing for both GH001 and GH002 is clear, specific contract manufacturer names aren't detailed in recent public updates, but the focus is on the proprietary delivery methods.

PricewaterhouseCoopers Ireland for statutory audit and financial review

The external validation of financial statements relies on the appointed auditors. PricewaterhouseCoopers Ireland is the firm appointed to audit the statutory financial statements. Shareholders are asked to ratify the appointment of PricewaterhouseCoopers Ireland as GH Research PLC's independent auditors for the year ending December 31, 2025. While the most recent specific fee breakdown relates to prior periods, the structure of their engagement is clear:

• Audit fees billed or billable by PricewaterhouseCoopers, Ireland: $11 thousand.
• Other assurance services billed or billable by PricewaterhouseCoopers, Ireland: $350 thousand.

This ratification process is a standard part of the Annual General Meeting agenda, which was scheduled for July 31, 2025. Finance: draft 13-week cash view by Friday.

GH Research PLC (GHRS) - Canvas Business Model: Key Activities

You're looking at the core engine of GH Research PLC right now, which is all about pushing their mebufotenin candidates, GH001 and GH002, through the final stages before a potential pivotal program launch. The key activities are heavily weighted toward regulatory navigation and clinical execution as of late 2025.

The engagement with the U.S. Food and Drug Administration (FDA) to lift the clinical hold on the Investigational New Drug Application (IND) for GH001 is a critical, ongoing activity. GH Research PLC submitted its complete response to the FDA in June 2025, ahead of schedule. As of the latest updates, the company is still addressing only one remaining hold topic, which relates to respiratory tract histology findings observed in rats. There are no outstanding issues remaining regarding dog toxicology or the proprietary aerosol delivery device itself.

The planning for the next major step, the global pivotal program for GH001 in treatment-resistant depression (TRD), is firmly set. Initiation is on track for 2026, with planning activities, including Clinical Research Organization (CRO) selection, having been underway since Q1 2025. This planning follows highly compelling data from the Phase 2b trial, which informs the design of these upcoming pivotal studies.

Technical development of the proprietary mebufotenin product candidates is supported by the financial outlay reported in the third quarter of 2025. Research and Development (R&D) expenses for the quarter ended September 30, 2025, totaled $10.6 million. This spend reflects the ongoing work on the mebufotenin platform, including the successful completion of the Phase 1, dose-ranging clinical pharmacology trial for GH002 in healthy volunteers.

The IND submission for the intravenous candidate, GH002, is a near-term milestone activity scheduled for Q4 2025. This follows the positive Phase 1 trial results which showed the candidate was well-tolerated with ultra-rapid psychoactive effects. The execution of the Phase 1 trial for the proprietary aerosol delivery device, while not having a specific late-2025 completion date in the latest reports, is implicitly advanced, as no device-related issues remain outstanding with the FDA for GH001.

Here's a quick look at the key performance indicators driving the urgency of these activities:

• GH001 Phase 2b OLE Remission Rate at 6 months: 73%.
• GH001 Primary Endpoint Achievement: -15.5 points placebo-adjusted reduction in MADRS score (p<0.0001).
• GH001 Remission Maintenance: 90% of Day 8 remitters maintained remission at 6 months.
• GH001 Safety: 0 treatment-related Serious Adverse Events (SAEs) over the 6-month trial duration.
• GH001 Dosing Profile: Majority of patients needed 1-2 doses, suggesting a 2-hour visit or less commercially.

The financial health underpinning these key activities, as of the end of Q3 2025, shows the company is actively investing:

To be fair, the cash position of $293.9 million as of September 30, 2025, provides a runway, but the quarterly net loss of $14.0 million means you need to watch the burn rate closely as pivotal trial costs ramp up next year. Finance: draft 13-week cash view by Friday.

GH Research PLC (GHRS) - Canvas Business Model: Key Resources

When we look at GH Research PLC (GHRS) as of late 2025, their key resources are heavily weighted toward their clinical pipeline and the data supporting it. Honestly, for a clinical-stage company, the data is the asset.

Financially, you want to see a solid runway. GH Research PLC reported $293.9 million in cash, cash equivalents, and marketable securities as of September 30, 2025. That's a healthy increase from the $182.6 million they held at the end of 2024. This capital fuels the next big steps, like the planned global pivotal program initiation in 2026.

The core of their value rests on their proprietary mebufotenin product candidates. You've got two main assets here:

• The lead candidate, GH001, an inhalable mebufotenin product for Treatment-Resistant Depression (TRD).
• GH002, another mebufotenin product candidate, which completed Phase 1 and showed ultra-rapid psychoactive effects; the company expects to submit an Investigational New Drug (IND) application to the FDA for GH002 in Q4 2025.

The positive Phase 2b clinical data for GH001 in TRD is definitely the most compelling resource right now. The results, fully analyzed from the Open-Label Extension (OLE), show significant efficacy and a strong safety profile. Here's a quick look at the numbers that matter most from that trial:

Beyond the molecules themselves, GH Research PLC holds the intellectual property protecting the drug formulation and the proprietary aerosol delivery device used for GH001. This technology is key because it supports the infrequent treatment visits and the fact that no mandated psychotherapeutic intervention was required in the OLE.

Finally, the human capital-the scientific and regulatory expert teams-is a critical resource. You see this in action with the ongoing engagement with the FDA to address the single remaining clinical hold topic related to rat histology findings for GH001. Plus, they've established a steering committee with Key Opinion Leaders (KOLs) to help design the upcoming global pivotal program, showing they are actively building the expertise needed for the next phase.

GH Research PLC (GHRS) - Canvas Business Model: Value Propositions

You're looking at the core reasons why GH Research PLC's lead asset, GH001, is positioned to disrupt the Treatment-Resistant Depression (TRD) space. The value here isn't just incremental improvement; it's about a fundamental shift in how severe depression is managed.

Ultra-rapid onset of action for severe depression symptoms.

The speed of effect is a major differentiator from conventional antidepressants that often take weeks to show clinical benefit. In the Phase 2b trial (GH001-TRD-201), patients treated with GH001 showed a highly significant reduction in depressive symptoms very quickly. The primary endpoint was met with a placebo-adjusted reduction from baseline of -15.5 points on the Montgomery-Åsberg Depression Rating Scale (MADRS) total score on Day 8. This rapid effect was also seen in a prior Phase 2 part of a trial where 87.5% of patients with TRD achieved ultra-rapid remission.

High and durable remission rate, with 73% remission at 6 months for TRD.

Efficacy isn't just fast; it's sustained. The Open-Label Extension (OLE) analysis of the Phase 2b trial confirmed that the treatment effect endures. Specifically, 73.0% of patients who completed the 6-month OLE achieved remission (MADRS $\le$10). To be fair, the initial Day 8 remission rate in the double-blind portion was 57.7% versus 0% for placebo. What's more, 90% of those who achieved remission at Day 8 were still in remission at month 6.

Potential for a single-day, infrequent treatment regimen.

This value proposition addresses the burden of chronic medication adherence and frequent clinic visits. The treatment is designed around an Individualized Dosing Regimen (IDR) administered on a single day, consisting of up to 3 increasing doses (6 mg, 12 mg, and 18 mg). The long-term data supports infrequent re-treatment; most patients (60.3%) in the 6-month period received only 1-4 GH001 treatments. The treatment was associated with short clinic visits, potentially only 1-3 hours long. The safety profile supported this, with almost all patients (97.4%) being discharge ready within an hour of the last dose.

The key efficacy and durability metrics from the Phase 2b trial are summarized below:

Novel, proprietary inhalation delivery method (GH001).

GH001 utilizes a novel delivery system for mebufotenin (synthetic 5-methoxy-N,N-dimethyltryptamine or 5-MeO-DMT). The drug is formulated for administration via a proprietary inhalation approach. This method is critical because mebufotenin is not orally active due to significant first-pass metabolism. The inhalation delivery is fast-acting, with a minimal median psychoactive effect duration of only 11 minutes per dose reported. The company is advancing a Phase 1 trial to bridge clinical data to a commercially available device.

Practice-changing treatment for Treatment-Resistant Depression (TRD).

The combination of rapid, profound effect, high durability, and infrequent dosing positions GH001 as a potential paradigm shift. The data suggests it could change the way TRD is treated today. The company's financial position as of September 30, 2025, stood at $293.9 million in cash, cash equivalents, and marketable securities, supporting the advancement toward initiating the global pivotal program in 2026.

The key attributes supporting this claim include:

• Rapid MADRS improvement of -15.5 points placebo-adjusted by Day 8.
• Sustained remission of 73% at 6 months.
• No mandated psychotherapeutic intervention required.
• Well-tolerated with no treatment related serious adverse events across the 6-month trial duration.

Finance: draft 13-week cash view by Friday.

GH Research PLC (GHRS) - Canvas Business Model: Customer Relationships

You're managing the relationships that keep GH Research PLC moving from clinical data to potential market readiness. This isn't just about selling; it's about deep scientific collaboration and rigorous regulatory navigation.

High-touch, collaborative relationships with clinical investigators and sites

The relationship with clinical investigators centers on the data generated from the GH001 program. The full dataset from the Phase 2b trial in treatment-resistant depression (TRD), GH001-TRD-201, was reported in July 2025, providing the core evidence for these collaborations.

The performance metrics from the key trials define the collaborative success:

The Open-Label Extension (OLE) analysis confirms this sustained efficacy, showing a 73% remission rate at 6 months with infrequent treatment visits and no mandated psychotherapy. Also, the Phase 1 clinical pharmacology trial (GH001-HV-106) evaluating the proprietary aerosol delivery device is ongoing in the United Kingdom, requiring close coordination with that site.

Direct, formal engagement with regulatory bodies like the FDA

Formal engagement with the U.S. Food and Drug Administration (FDA) is critical, specifically regarding the Investigational New Drug Application (IND) clinical hold for GH001. Engagement on the complete response submission is ongoing as of November 2025.

Key points of the ongoing regulatory dialogue include:

• The company submitted its complete response to the IND clinical hold in June 2025.
• Only one hold topic remains outstanding as of July 2025.
• The remaining issue relates to respiratory tract histology findings in rats, which GH Research PLC believes are rat-specific based on scientific evidence.
• There are no additional requests outstanding related to dog toxicology or device issues.
• GH Research PLC plans to submit an IND application for its second product candidate, GH002, in Q4 2025.

The initiation of the global pivotal program for GH001 remains on track for 2026, contingent on resolving this final FDA topic.

Investor relations and communication with the financial community

GH Research PLC maintains active communication with stockholders and potential investors, highlighted by public reporting and conference participation. The company reported its Third Quarter 2025 Financial Results in November 2025 and its Second Quarter 2025 Financial Results in August 2025.

Financial standing directly impacts investor confidence. As of June 30, 2025, cash, cash equivalents and marketable securities stood at $308.7 million. The company also participated in the Stifel 2025 Healthcare Conference in New York from November 11-13, 2025, where they conducted one-on-one investor meetings.

Scientific communication via conferences (ECNP, ASCP) and publications

Disseminating clinical findings to the scientific community is a core relationship activity. GH Research PLC announced the acceptance of presentations at major 2025 meetings.

Scientific engagement highlights for 2025 include:

• Presentation acceptance at the 2025 ECNP Congress in Amsterdam (October 11 - 14), featuring a Novel Therapies Symposium Presentation and posters.
• Presentation acceptance at the ASCP 2025 Annual Meeting in Arizona (May 27 - 30), including a Pharmaceutical Pipeline Presentation.

The ECNP presentation included long-term data for GH001 in TRD patients, reinforcing the data shared with the FDA.

GH Research PLC (GHRS) - Canvas Business Model: Channels

You're hiring before product-market fit, so every interaction point, from the lab bench to the investor call, has to be precise. Here's how GH Research PLC moves its science and capital.

Global clinical trial sites for product development and testing

The channel for product development centers on clinical trials across various jurisdictions. The global pivotal program for GH001 is on track for initiation in 2026. Currently, a Phase 1 clinical pharmacology trial (GH001-HV-106) evaluating the proprietary aerosol delivery device is ongoing in the United Kingdom.

Key data points from completed trials, which inform site selection and protocol design for future phases, include:

Scientific and medical conferences for data dissemination

Dissemination of clinical data occurs through presentations and posters at major scientific meetings. GH Research PLC actively presented data throughout 2025, focusing on its lead candidate, GH001.

• American Society of Clinical Psychopharmacology (ASCP) Annual Meeting in Arizona, May 27 - 30, 2025: Included a Pharmaceutical Pipeline Presentation.
• International Society for Bipolar Disorders (ISBD) Annual Conference in Chiba, Japan, September 17 - 19, 2025: Accepted poster presentation.
• 38th Annual European College of Neuropsychopharmacology Congress (ECNP) in Amsterdam, October 11-14, 2025: Included a Novel Therapies Symposium Presentation on long-term data.

Regulatory submissions (INDs, NDAs) to the FDA and other agencies

The primary channel for regulatory interaction involves submissions to the U.S. Food and Drug Administration (FDA) concerning the Investigational New Drug (IND) applications for its product candidates.

For GH001, the company submitted its complete response to the clinical hold on its IND in June 2025. This submission was ahead of schedule. The FDA response indicated only one remaining topic, concerning respiratory tract histology findings in rats, which GH Research PLC believes are rat-specific. There are no remaining device-related issues.

For the second candidate, GH002, GH Research PLC plans to submit an IND application to the FDA in Q4 2025.

Investor presentations and press releases for capital markets

Capital markets communication is managed through financial reporting, corporate presentations, and conference participation. As of September 30, 2025, GH Research PLC reported cash, cash equivalents, and marketable securities of $293.9 million.

Key financial metrics from the Third Quarter 2025 results include:

• Net loss for the quarter: $14.0 million (or $0.23 loss per share).
• R&D expenses for the quarter: $10.6 million.
• General & Administrative (G&A) expenses for the quarter: $6.0 million.

The company held investor meetings and participated in the Stifel 2025 Healthcare Conference in New York from November 11-13, 2025. A Corporate Presentation was released on November 06, 2025. To be fair, the stock surged 90% over the six months leading up to July 2025, when its market cap was reported at $1.04 billion. Finance: draft 13-week cash view by Friday.

GH Research PLC (GHRS) - Canvas Business Model: Customer Segments

Patients with Treatment-Resistant Depression (TRD), the primary focus.

GH Research PLC's lead candidate, GH001, is specifically targeting patients with Treatment-Resistant Depression (TRD), defined as depression not responding to at least two adequate antidepressant therapies. The potential patient pool in the US alone was estimated to be 2.8 million adults in 2021. The US TRD treatment market was valued at USD 873.2 million in 2022, while the global market across seven major regions reached USD 3,494.7 Million in 2024.

The clinical data from the Phase 2b trial (GH001-TRD-201) provides concrete numbers on the segment's response profile:

Patients with Postpartum Depression (PPD) and Bipolar II Depression (secondary).

GH Research PLC has also advanced GH001 into earlier-stage trials for these secondary indications, suggesting these patient populations are key to future expansion. The company has completed Phase 2 open-label trials in both indications.

• Phase 2 open-label trials completed for Postpartum Depression (PPD).
• Phase 2 open-label trials completed for Bipolar II Disorder (BD).
• Positive results from Phase 2a trials in PPD and Bipolar II indicate potential broad applications.

Psychiatrists and mental health specialists who treat severe depression.

This group represents the prescribers and gatekeepers for novel therapies like GH001. The overall Psychiatrists market size in 2024 was $195.22 billion, projected to grow to $215.02 billion in 2025 at a compound annual growth rate (CAGR) of 10.1%. North America was the largest region in this market in 2024. These specialists are the direct target for education on the ultra-rapid onset and durable effects demonstrated by GH001.

Healthcare payers and government agencies (future commercial segment).

This segment becomes critical upon potential commercialization, as payers determine formulary access and reimbursement rates. The company is preparing for its global pivotal program initiation in 2026, which directly precedes commercial launch discussions with payers. As of September 30, 2025, GH Research PLC held $293.9 million in cash, cash equivalents and marketable securities, supporting the R&D required to reach this commercial stage, while reporting a net loss of $14.0 million for the quarter ended September 30, 2025.

GH Research PLC (GHRS) - Canvas Business Model: Cost Structure

You're looking at the operational burn rate for GH Research PLC as of late 2025, which is heavily weighted toward advancing its clinical pipeline.

The most recent reported figures, covering the quarter ended September 30, 2025, give us a clear picture of where the cash is going right now. Honestly, for a company at this stage, the cost structure is almost entirely driven by R&D.

The R&D spend increase in Q3 2025 was primarily fueled by technical development activities and employee costs, though this was partly offset by a decrease in clinical development expenses for that specific quarter. The G&A rise reflects higher professional fees and employee expenses too.

Here's the quick math: R&D made up about 63.5% of the total operating expenses ($10.6M / ($10.6M + $6.0M)) for the quarter. That's where the focus defintely is.

The specific components driving these costs include:

• Clinical trial costs, including CRO fees and site payments.
• Employee expenses for scientific, technical, and professional staff.
• Regulatory and professional fees for IND submissions and legal work.

What this estimate hides is the exact split between CRO fees versus site payments within that clinical development bucket, as the search results only give the aggregate R&D number.

The cost drivers within the R&D spend for the quarter ended September 30, 2025, were:

• Increased expenses relating to technical development activities.
• Increased employee expenses.
• A decrease in clinical development expenses.

The G&A increase for the quarter ended September 30, 2025, was driven by:

• An increase in professional fees.
• An increase in employee expenses.

Finance: draft 13-week cash view by Friday.

GH Research PLC (GHRS) - Canvas Business Model: Revenue Streams

You're looking at the revenue side for GH Research PLC (GHRS) as of late 2025, and honestly, it's what you'd expect for a clinical-stage company deep in development. There's no product revenue right now; GH Research PLC is pre-commercial, focused entirely on getting its lead candidate, GH001, through the final stages of regulatory review and into pivotal trials. That means the current financial lifeblood comes from outside the lab-to-market pipeline.

The primary, most significant cash inflow you see is from equity financing. This is how GH Research PLC funds its operations while waiting for a marketable product. A major example of this was the underwritten public offering that closed in early 2025. This inflow was crucial for maintaining the large cash buffer needed for ongoing research and development expenses, which hit $10.6 million in the third quarter of 2025 alone. You need to track these capital raises closely.

Here's a quick look at that key financing event:

Next up, you have interest income. Since GH Research PLC is sitting on a substantial balance of unspent capital, the interest earned on those holdings is a reliable, albeit secondary, revenue component. This income is generated from their large holdings of cash, cash equivalents, and marketable securities, which are primarily investment-grade bonds and money market funds. It's not product revenue, but it helps offset the net losses, which were $14.0 million for the quarter ended September 30, 2025.

The cash position itself is the key metric here, showing the runway provided by past financing. As of September 30, 2025, the balance was $293.9 million. That's a significant increase from the $182.6 million reported at the end of 2024. You should definitely keep an eye on this burn rate versus the cash on hand.

• Cash, cash equivalents and marketable securities as of September 30, 2025: $293.9 million.
• Cash, cash equivalents and marketable securities as of June 30, 2025: $308.7 million.
• Cash, cash equivalents and marketable securities as of March 31, 2025: $315.3 million.

Looking forward, the potential revenue streams are entirely contingent on clinical and regulatory success. You should be watching for two main future possibilities. First, there are potential milestone payments that could come from any existing or future licensing or collaboration deals that GH Research PLC might strike for its pipeline assets, though no specific amounts are public right now. Second, and most important, is the eventual revenue from product sales, which is contingent on successful regulatory approval. GH Research PLC continues to work on its Investigational New Drug (IND) application response with the FDA, and the company expects to initiate its global pivotal program in 2026. That 2026 timeline sets the stage for any potential commercial revenue post-approval, which is definitely not expected before 2027.