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Highlights:

  • GHRS submitted a complete response to the FDA’s clinical hold on GH001 IND in June 2025.
  • The response includes comprehensive data and toxicology studies addressing prior agency feedback.
  • GH Research continues development of GH001 for treatment-resistant depression (TRD) following Phase 2b trial results. 

GH Research PLC (Nasdaq: GHRS), a clinical-stage biopharmaceutical company focused on depression treatments, announced that it has submitted its full response to the U.S. Food and Drug Administration (FDA) concerning the clinical hold placed on its Investigational New Drug (IND) application for GH001. The company confirmed that the response was filed in June 2025, ahead of schedule. 

The IND application for GH001 had previously been placed on hold by the FDA. GH Research’s latest submission is intended to address the agency’s prior concerns, with the company providing additional preclinical data and completed toxicology studies. The outcome of this submission will determine next steps in the regulatory process for the clinical development of GH001 in the United States. 

Dr. Velichka Valcheva, Chief Executive Officer of GH Research, commented on the development: “We submitted our response to the FDA in June, incorporating all requested materials and data. Our team remains engaged with the agency as part of the process to move GH001 forward.” 

The company previously reported results from the Phase 2b GH001-TRD-201 clinical trial, which evaluated GH001’s efficacy and safety in TRD patients. The study achieved its primary endpoint with a statistically significant reduction in depressive symptoms, as measured by the Montgomery–Åsberg Depression Rating Scale (MADRS). On Day 8, the average reduction in MADRS score was 15.5 points from baseline compared with placebo (p<0.0001). 

With these data, GH Research intends to progress GH001 through additional clinical development, pending further regulatory guidance. The company stated that it is working to resume U.S.-based trials once FDA review of the IND response is completed. 

The submission of a complete response to a clinical hold is a key milestone in the drug development process. It does not guarantee that the hold will be lifted but is a prerequisite for continued clinical testing. The FDA will review the materials provided and determine whether the hold can be removed or if additional information is required. 

GH Research has not provided a timeline for when the FDA is expected to complete its review. The company continues to plan for future development activities, both in the U.S. and internationally, based on pending regulatory outcomes.