Invex Therapeutics’ (ASX: IXC) has filed a pre-investigational new drug application with the United States Food and Drug Administration for the proposed phase III trial evaluating its drug Presendin in idiopathic intracranial hypertension (IIH) patients.
With filing the application, Invex is seeking a pre-IND/Type B meeting with the FDA where it will gain protocol assistance for the proposed trial including guidance on trial endpoints, which include monthly headache days and intracranial pressure.
According to the company, monthly headache days and intracranial pressure harmonise with the protocol advice Invex received from the European Medicines Agency late last year under Presendin’s orphan drug designation.
As part of a previous FDA request, Invex has also filed with the agency a complete proposed study protocol and statistical analysis plan.
Invex chairman Dr Jason Loveridge said the FDA filings represented an “important milestone” for the company.
“The completion of a study protocol and statistical analysis plan within approximately three months of obtaining final European Medicines Agency feedback is a testament to the commitment of the Invex team, our expert medical/scientific and regulatory advisors and study statisticians to our idiopathic intracranial hypertension development program for Presendin.”
Dr Loveridge added the FDA feedback will be an “important consideration” as Invex contemplates filing an IND following the official minutes of the meeting which is anticipated in the September quarter.
“We are excited by the opportunity to share our plans with the FDA upon the granting of our proposed meeting request.”
Dr Loveridge noted the proposed phase III trial was important for idiopathic intracranial hypertension sufferers, who currently cannot rely on any approved therapeutics to effectively manage the disorder and improve their quality of life such as reducing headaches.
Idiopathic intracranial hypertension
According to Invex, idiopathic intracranial hypertension is a disabling disorder which features “severely raised intracranial pressure”. This leads to daily headaches and can compress the optic nerve.
Onset usually occurs between 20 and 30 years of age and the disease is more common in obese women.
Invex noted the disorder is a rapidly growing indication – with incidences increasing more than 350% over the past decade.
Presendin, which is Invex’s brand name for exenatide, previously received approval in the US and EU in 2005 and 2006 for treating type 2 diabetes.
Research indicates the drug can ultimately reduce cerebrospinal fluid in the brain. However, current dosage forms for diabetes have not been optimised for idiopathic intracranial hypertension.