GH Research Submits Complete IND Hold Response to FDA Ahead of Schedule

Rhea-AI Summary

GH Research PLC (GHRS) has announced the submission of its complete response to the FDA regarding the clinical hold on its Investigational New Drug Application (IND) for GH001, a treatment in development for depression. The company submitted the response in June 2025, ahead of their planned schedule. According to CEO Dr. Velichka Valcheva, the response includes comprehensive data and completed toxicology studies addressing the FDA's specific requests. The company emphasized its commitment to working with the FDA to advance GH001's development for patient treatment.

Positive

• Early submission of FDA response ahead of schedule demonstrates operational efficiency
• Completion of comprehensive data package and toxicology studies to address FDA concerns

Negative

• IND remains on clinical hold, preventing US clinical trials from proceeding
• Uncertainty regarding FDA's acceptance of the response and timeline for potential hold removal

DUBLIN, June 20, 2025 (GLOBE NEWSWIRE) -- GH Research PLC (Nasdaq: GHRS), a clinical-stage biopharmaceutical company dedicated to transforming the lives of patients by developing a practice-changing treatment in depression, today announced that, in June, it submitted its complete response to the previously announced clinical hold of its Investigational New Drug Application (IND) for GH001 to the U.S. Food and Drug Administration (FDA).

“We are thrilled to have submitted our response to the FDA ahead of schedule, addressing their clear requests with comprehensive data and completed toxicology studies. We remain fully committed to working closely with the agency to bring GH001 to patients,” said Dr. Velichka Valcheva, Chief Executive Officer.

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 Phase 2b GH001-TRD-201 trial, where the primary endpoint was met with a MADRS reduction from baseline of -15.5 points compared with placebo on Day 8 (p<0.0001), we believe that GH001 has potential to change the way TRD is treated today.

Forward-Looking Statements

This press release contains statements that are, or may be 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, medical devices required to deliver these product candidates, research pipeline, ongoing and currently planned preclinical studies and clinical trials, regulatory submissions and approvals and their effects on our business strategy, including our plans and expectations for discussions with the FDA and the outcomes and resolution of such discussions related to the clinical hold on the GH001 IND, research and development costs, cash runway, 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 press release speak only as of the date hereof. 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 is the current status of GH Research's (GHRS) IND application for GH001?

GH Research has submitted its complete response to the FDA regarding the clinical hold on its IND application for GH001, including comprehensive data and toxicology studies

When did GH Research (GHRS) submit its FDA response for the GH001 clinical hold?

GH Research submitted its complete response to the FDA in June 2025, ahead of their planned schedule

What is GH001 being developed for by GH Research (GHRS)?

GH001 is being developed as a potential practice-changing treatment for depression

Who is the CEO of GH Research (GHRS)?

Dr. Velichka Valcheva is the Chief Executive Officer of GH Research

What information did GH Research (GHRS) include in their FDA response?

The response included comprehensive data and completed toxicology studies addressing the FDA's specific requests