Clinical-stage biopharmaceutical company Invex Therapeutics (ASX: IXC) has been granted a meeting with the US Food and Drug Administration to discuss the planned development pathway for its lead drug Presendin in the treatment of disorders relating to raised intracranial pressure.
The company will meet with the FDA’s Division of Neurology to seek guidance on the design of a planned phase III trial investigating the use of Presendin for neurological conditions such as idiopathic intracranial hypertension (IIH), which have no known cause and can mimic brain tumours in their symptoms.
The planning will include proposed clinical endpoints and statistical analysis methods for the trial, which is expected to be conducted in the US, Europe and Australia.
Invex’s meeting has been labelled a “Type C” requiring written responses only to questions on study protocol and planning previously submitted as part of the company’s meeting request application.
Chairman Dr Jason Loveridge said the US FDA meeting would help advance the development pathway of Presendin.
“Currently, IIH patients are unable to rely on any regulatory-cleared therapeutic agents to manage high intracranial pressure and improve their quality of life by positively impacting important clinical outcomes such as a reduction in headaches,” he said.
“The Division of Neurology’s input will provide valuable feedback as our development progresses in this important market where treatment options for IIH patients are limited.”
About the condition
IIH occurs when the cerebrospinal fluid which exists around the brain and spinal cord builds up in the skull, increasing pressure and causing symptoms such as vision changes (due to compression of the optic nerve) and headaches.
The condition is most common in obese women of childbearing age and is a rapidly-growing orphan indication, with patient numbers having increased by more than 350% over the last 10 years.
Invex is focused on re-purposing the approved drug Exenatide (trademarked as Presendin) for the treatment of neurological conditions derived from or involving raised intracranial pressure such as IIH, acute stroke and traumatic brain injury.
Exenatide received approval in the US and European Union in the mid-2000’s for the treatment of Type 2 diabetes.
Last May, a phase II, double-blind, placebo-controlled clinical trial involving patients with IIH demonstrated a statistically-significant reduction in key endpoints in the Exenatide arm, including all those related to intracranial pressure.
Dr Loveridge said the data supported the progression of Presendin into the phase III trial according to its orphan drug designation.
Invex closed the March quarter in a strong financial position with cash and cash equivalents of $33.2 million.
The company’s quarterly report highlighted cash outflows of $0.42 million included $0.29 million in research and development expenditure, of which $0.17 million was dedicated to regulatory submissions for the phase III trial.
Invex also strengthened its board during the March quarter with the appointment of Dr Megan Baldwin as a non-executive director.
Dr Baldwin is the managing director of late-stage biopharmaceutical company Opthea (ASX: OPT) which is developing a novel therapy to address unmet needs in the treatment of retinal eye diseases, including wet age-related macular degeneration (wet AMD).
Under Dr Baldwin’s leadership, Opthea has advanced its ophthalmology program through phase I and phase II clinical development, and recently completed a $180 million initial public offering and listing on the US NASDAQ exchange to progress two pivotal phase III studies in wet AMD.
“Dr Baldwin’s experience in drug development, regulatory engagement and clinical trial design and execution will be invaluable for Invex as we progress Presendin into our phase III studies this year,” Dr Loveridge said.