The primary endpoint was lumbar puncture opening pressure at 12 weeks; whilst there was no between-arms difference at 12 weeks, there was a significant reduction within the treatment arm at 12 weeks compared to baseline. Importantly, change in opening pressure showed significant correlation with the cortisol:cortisone ratio, a function of 11β-HSD1 activity. This supports a potential role for 11β-HSD1 inhibition in the treatment of IIH, which may also have additional benefits for the broader adverse metabolic phenotype [2], in addition to the core feature of raised intracranial pressure. Image created with Biorender.com.
References
Markey K, Mitchell J, Botfield H, Ottridge RS, Matthews T, Krishnan A, et al. 11β-Hydroxysteroid dehydrogenase type 1 inhibition in idiopathic intracranial hypertension: a double-blind randomized controlled trial. Brain Commun. 2020;2:fcz050. https://doi.org/10.1093/braincomms/fcaa108.
Hardy RS, Botfield H, Markey K, Mitchell JL, Alimajstorovic Z, Westgate CSJ, et al. 11βHSD1 Inhibition with AZD4017 improves lipid profiles and lean muscle mass in idiopathic intracranial hypertension. J Clin Endocrinol Metab. 2021;106:174–87. https://doi.org/10.1210/clinem/dgaa766.
Funding
Alex Sinclair is supported by a Sir Jules Thorn Award for Biomedical Research. This trial was funded by Enterprising Birmingham, University of Birmingham, UK, from 1st August 2016. Further funding was sought, and from 1st August 2019, an investigator-led grant was obtained from Invex Therapeutics.
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Design and draughting: ML, GB. Review: FLA, AS, SPM.
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Susan Mollan reports consultancy fees (Invex Therapeutics); advisory board fees (Ocular Therapeutix); speaker fees (Teva); Travel (AbbVie and European Alliance of Associations for Rheumatology); Receipt of equipment (Heidelberg Engineering). Susan Mollan is a section editor at Eye Journal. Alex Sinclair reports consultancy fees from Novartis, AbbVie, Vertex and Orion Pharma and speaker fees from Teva UK and Novartis. She was previously a Director and Chief Scientific Officer with shares at Invex Therapeutics. Michael Lowe reports speaker fees (Pfizer); travel (AbbVie, Lundbeck, Teva). Authors declare no other financial relationships with any organisations that might have an interest in the submitted work; and no other relationships or activities that could appear to have influenced the submitted work.
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Lowe, M., Berman, G., Labella Álvarez, F. et al. Infographic: landmark trials in Neuro-Ophthalmology—results of the phase II idiopathic intracranial hypertension drug trial. Eye (2025). https://doi.org/10.1038/s41433-025-03973-1
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DOI: https://doi.org/10.1038/s41433-025-03973-1
