Invex Therapeutics Advances Presendin Phase III Trial for Idiopathic Intracranial Hypertension with FDA Guidance
Key Insights
Invex Therapeutics (search) has been granted an FDA meeting to discuss the development pathway for Presendin (search), a repurposed diabetes drug being tested for idiopathic intracranial hypertension (search) (IIH (search)).
The company's IIH (search) EVOLVE phase III trial will enroll 240 newly diagnosed patients across 37 centers in Europe, UK, Australia, and the US to assess intracranial pressure (search) reduction.
Previous phase II results demonstrated statistically significant reduction in key endpoints related to intracranial pressure (search), supporting progression to phase III development.
Clinical-stage biopharmaceutical company Invex Therapeutics (search) has secured a crucial FDA meeting to advance its lead drug candidate Presendin (search) for treating idiopathic intracranial hypertension (search) (IIH (search)), a debilitating neurological condition with limited treatment options. The company will meet with the FDA's Division of Neurology to seek guidance on the design of its planned phase III trial investigating Presendin for neurological conditions characterized by raised intracranial pressure (search).
FDA Meeting and Regulatory Strategy
The FDA meeting, classified as "Type C" requiring written responses only, will focus on proposed clinical endpoints and statistical analysis methods for the upcoming trial. The study is expected to be conducted across the US, Europe, and Australia as part of a comprehensive global development strategy.
"Currently, IIH (search) patients are unable to rely on any regulatory-cleared therapeutic agents to manage high intracranial pressure (search) and improve their quality of life by positively impacting important clinical outcomes such as a reduction in headaches," said Chairman Dr. Jason Loveridge. "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."
IIH EVOLVE Phase III Trial Design
Invex has made significant progress in preparing for its IIH (search) EVOLVE phase III clinical trial, designed to achieve global registration of Presendin (search). The company filed its first clinical trial application in Australia in December and anticipates completing additional applications for the UK, Europe, and US during the first half of 2022.
The single phase III trial will enroll 240 newly diagnosed IIH (search) patients randomized to receive either once-weekly subcutaneous injections of Presendin (search) or placebo across 37 trial centers. The primary endpoint will assess the mean difference in intracranial pressure (search) from baseline at 24 weeks between patients receiving Presendin and placebo. Secondary endpoints will evaluate relative differences in vision and monthly headache days between the two groups over 24 weeks.
Understanding Idiopathic Intracranial Hypertension
IIH (search) occurs when cerebrospinal fluid around the brain and spinal cord builds up in the skull, increasing pressure and causing symptoms such as vision changes due to optic nerve (search) compression and severe headaches. The condition most commonly affects obese women of childbearing age and represents a rapidly growing orphan indication, with patient numbers having increased by more than 350% over the last 10 years.
Repurposing Exenatide for Neurological Applications
Invex is focused on repurposing Exenatide, originally approved in the US and European Union in the mid-2000s for Type 2 diabetes (search) treatment, for neurological conditions involving raised intracranial pressure (search). The company has trademarked the repurposed formulation as Presendin (search) for treating conditions such as IIH (search), acute stroke (search), and traumatic brain injury (search).
The development strategy received significant validation from a phase II, double-blind, placebo-controlled clinical trial conducted in May, which demonstrated statistically significant reduction in key endpoints in the Exenatide arm, including all measures related to intracranial pressure (search). Dr. Loveridge noted that this data supported progression of Presendin (search) into phase III development according to its orphan drug designation.
Manufacturing and Financial Position
Following a major collaboration and manufacturing agreement with Peptron (search), Invex completed a satisfactory independent audit of Peptron's Korean manufacturing facility, which can produce more than 48,000 vials of Presendin (search) per month. The company has placed an initial purchase order with Peptron to supply clinical batches of Presendin and placebo for the IIH (search) EVOLVE trial.
Invex maintains a strong financial position with $31.4 million in cash and cash equivalents at the end of the December quarter. The company stated it is fully funded to complete its IIH (search) EVOLVE phase III trial from current cash reserves, with quarterly cash outflows of $600,000 including product manufacturing and research and development expenditure.
Leadership Strengthening
The company strengthened its board with the appointment of Dr. Megan Baldwin as non-executive director. Dr. Baldwin serves as managing director of Opthea, a late-stage biopharmaceutical company that recently completed a $180 million NASDAQ IPO to advance phase III studies in wet age-related macular degeneration (search). Professor Michael Wall, a distinguished expert in IIH (search) with extensive clinical trial experience in neurology and ophthalmology, was appointed as trial steering group chairperson.