GH Research reports progress on FDA engagement for GH001 IND

DUBLIN - GH Research PLC (NASDAQ:GHRS), a $1.04 billion market cap biotech company that has seen its stock surge 90% over the past six months, reported Wednesday that it continues to engage with the U.S. Food and Drug Administration regarding the clinical hold on its Investigational New Drug Application for GH001, with only one remaining issue to resolve. InvestingPro analysis reveals 8 key investment tips for GHRS, including strong financial health indicators and recent market performance metrics.

The FDA has requested additional data or further justification related to respiratory tract histology findings in rats. The company, which maintains a healthy current ratio of 29.7 and holds more cash than debt on its balance sheet, stated it believes these findings are rat-specific, based on scientific evidence. No additional requests related to dog toxicology or device issues remain outstanding.

GH Research also announced final data from its completed Phase 2b trial of GH001 in treatment-resistant depression. The company reported that the trial met its primary endpoint with a placebo-adjusted reduction of 15.5 points in the Montgomery-Åsberg Depression Rating Scale total score on Day 8.

The full open-label extension analysis showed a 73% remission rate at six months with infrequent treatment visits and no mandated psychotherapy. According to the company, 57.5% of patients achieved remission at day 8, with 90% of those maintaining remission at month 6.

Safety analysis indicated no treatment-related serious adverse events during the six-month trial period. The company reported no treatment-emergent events of suicidal intent or behavior throughout the study.

GH Research also provided an update on GH002, its intravenous mebufotenin HBr product candidate. The company plans to submit an IND application for GH002 to the FDA in Q4 2025.

The company stated it remains on track to initiate its global pivotal program for GH001 in 2026, with ongoing preparations including establishment of a steering committee and selection processes for clinical research organizations and sites.

This information is based on a press release statement from GH Research PLC.

In other recent news, GH Research PLC has been actively addressing a clinical hold from the U.S. Food and Drug Administration on its investigational new drug application for GH001, an inhalable treatment for depression. The company submitted its complete response to the FDA in June, including data and toxicology studies, although specific details about the FDA’s initial concerns were not disclosed. Analyst Sumant Kilkami from Canaccord Genuity reiterated a Buy rating with a $35.00 price target for GH Research, indicating confidence in the company’s prospects. Meanwhile, Cantor Fitzgerald analysts have assumed coverage of GH Research with an Overweight rating and a $25.00 price target, highlighting the transformative potential of psychedelics in treating mental health disorders. These analysts emphasize the significant unmet need for effective treatments in areas such as depression, PTSD, and anxiety. They also note the growing infrastructure in the U.S. for delivering psychedelic therapies. These developments reflect the ongoing interest and optimism in GH Research’s efforts within the mental health sector.

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