- The disease
- Idiopathic intracranial hypertension (IIH) is a condition in which pressure in the fluid surrounding the brain is abnormally elevated without a detectable underlying cause such as a tumour or infection. It primarily affects women of reproductive age who are overweight, though the exact prevalence is not established here. Symptoms typically include persistent headache and vision changes, with the risk of permanent vision loss if untreated. Currently approved pharmacological options are limited, and weight loss is a cornerstone of management.
- What the asset is trying to do
- Presendin is a repurposed small molecule being developed by Invex Therapeutics to reduce intracranial pressure in IIH patients (per ClinicalTrials.gov NCT05347147). The specific molecular target of Presendin is not publicly disclosed in the available data. As a repurposed drug, it has an existing safety and pharmacology profile from prior use, which informed its development pathway in this new indication.
- What the trial is measuring, and why it matters
- The Phase 3 trial (NCT05347147) was designed to evaluate the efficacy and safety of Presendin compared with placebo in patients with IIH (per ClinicalTrials.gov NCT05347147). The specific primary endpoint is not detailed in the available trial registry data beyond the trial title. Efficacy and safety assessments in IIH trials typically include measures of intracranial pressure and visual function, though the precise endpoints used in this trial are .
- Efficacy benchmarks in this setting
- In IIH, acetazolamide — the most commonly used pharmacological treatment — was evaluated in the IIHTT randomised controlled trial, where it was associated with improvement in visual field mean deviation compared with placebo when combined with a low-sodium weight-reduction diet (Wall et al, New England Journal of Medicine 2014). Topiramate has also been studied as a comparator in this setting (per published literature). Because this asset's Phase 3 trial was terminated after enrolling 14 of a planned number of patients (per ClinicalTrials.gov NCT05347147), any figures from that trial are not statistically comparable to data from completed trials of approved therapies.
- Commercial context
- IIH does not have a specifically approved pharmacological treatment in most major markets; acetazolamide is used off-label as standard of care in many jurisdictions (per published clinical guidelines). The Phase 3 trial for Presendin (NCT05347147) was terminated, and the company has disclosed it is reassessing its clinical strategy. An April 2026 ASX announcement indicates Invex intends to expand its preclinical research program (per ASX release, 23 April 2026). No licensing deals, partnership agreements, or manufacturing arrangements for Presendin have been disclosed in the available data.