An injectable peptide used for type2 diabetes may be just what’s needed to treat patients with Idiopathic Intercranial Hypertension (IIH), according to a Phase 2 trial published in the journal Brain. IIH causes disabling daily headaches and can even lead to vision loss.
The Phase 2 trial, led by the University of Birmingham and University Hospitals Birmingham, found that regular injections of a drug called exenatide – a GLP-1 receptor agonist, led to a drop in pressure in the brain during both short (2.5hrs and 24hrs) and long term (12 weeks) measurements.
The trial also saw significant reductions in the numbers of headaches across the 12 weeks during which the seven participants took part, with an average of 7.7 fewer days per month of headaches compared to the baseline, compared to only 1.5 fewer days in the placebo arm.
A key finding from the Phase 2 study was the rapid action of the drug, with results indicating that brain pressure was significantly reduced within two-and-a-half hours of taking the medication
Following the success of this Phase 2 trial, patients with Idiopathic Intercranial Hypertension (IIH) are now required for a Phase 3 trial – IIH EVOLVE – which will run in 40 sites across Australia, New Zealand, United Kingdom, Europe, Israel, and the United States.
IIH EVOLVE will be run by Invex Therapeutics, an Australian biotechnology company, led by Professor Alexandra Sinclair from Birmingham UK.
The placebo-controlled, double-blind Phase 3 clinical trial will randomise 240 adult patients with newly diagnosed IIH with papilloedema to determine the efficacy and safety of Presendin versus placebo, administered once weekly over 24 weeks.
Exciting Opportunity for Australia
The lead Australian principal investigator, Associate Professor Clare Fraser, from The University of Sydney, told mivision that this study is particularly exciting because it will evaluate “the first new treatment we potentially have available for this condition, which is specifically designed for IIH”.
“The rates of IIH are rising across the world, and it can cause headaches and, in some cases, severe vision loss. So, it is vital that we get a better understanding of why this condition happens, and how we can best treat it…Australian sites are effort,” Assoc Prof Fraser said.
The trial is for patients who have mild to moderate disease, and all patients will be closely monitored through the trial.
Ultimately, the aim is to gain enough evidence to allow the drug to be licensed for use in IIH patients in the future, she said.
Shot in the Arm for IIH Treatment
IIH is a debilitating condition that raises pressure in the brain and can lead to chronic headaches and even permanent sight loss. The illness, which often leaves patients with a reduced quality of life, predominately affects women aged 25–36. Weight gain is a major risk factor of developing IIH and relapses of the disease.
Once regarded as rare, the incidence of IIH is now rising dramatically in line with the global rise in obesity and there has been a 350% rise in incidence in the past 10 years.
Currently there are no licenced drugs options and existing medications used off label are complicated by troublesome side effects.
A key finding from the Phase 2 study was the rapid action of the drug, with results indicating that brain pressure was significantly reduced within two-and-a-half hours of taking the medication.
This rapid onset of action is vital in a condition that can cause rapid blindness if left untreated.
Patients Need ted for EVOLVE
Information on the trial is available at clinicaltrials.gov under Identifier NCT05347147.
Eye health professionals in Western Australia or South Australia should contact Associate Professor Celia Chen from Vision SA: celiachen@visionsaeye.com.au.
Eye health professionals in the eastern states of Australia should contact Assoc Prof Fraser: clare.fraser@sydney.edu.au.
