The pressure is on for biotech company Invex Therapeutics (ASX: IXC), which is not only struggling to recruit patients but is also dealing with underwhelming results for its flagship drug Presendin, tackling idiopathic intracranial hypertension (IIH).

The Company has initiated a comprehensive assessment of its Phase IIH EVOLVE program, which explores the potential of Presendin as a GLP-1 receptor agonist (GLP-1RA). This evaluation comes in response to the slower-than-anticipated activation of clinical trial sites and the enrolment of patients into the Phase III trial for IIH EVOLVE. Furthermore, the swift adoption of GLP-1RAs for obesity treatment, with or without co-morbidities like type II diabetes, in the evolving market has posed an additional challenge for Invex Therapeutics.

Originally aiming to enrol 240 newly diagnosed IIH patients into the IIH EVOLVE trial, the Company projected a recruitment duration of up to 24 months. The trial commenced its first patient enrolment in November 2022, in Adelaide, Australia. However, the pace of recruitment has fallen short of expectations, primarily due to delayed site activations and sluggish enrolment.

As of June 26, 2023, Invex Therapeutics has managed to enrol only 13 patients into the IIH EVOLVE trial, with a mere 12 out of the targeted 40 global sites activated. If that wasn’t enough, Invex acknowledged that the failure rate among screened IIH patients has been significantly higher than initially anticipated, albeit from a limited number of activated sites.

Compounding the challenges, data collected from enrolling sites reveals that over 50 additional IIH patients were pre-screened but did not progress to formal screening. The main reason for their exclusion was the lapse of the 4-week time threshold for diagnostic lumbar punctures required for screening.

By June 30, 2023, Invex Therapeutics had planned to activate and open the majority of its 40 clinical sites for patient enrolment. While the necessary contracting and site activation processes have been completed for sites in Australia, New Zealand, the United States (US), and the United Kingdom (UK), the overall site activation progress has been slower than anticipated. Obtaining regulatory approvals from Germany, Israel, and France also contributed to the delayed rate of site activations.

In response to the challenges faced, Invex Therapeutics’ Board, in consultation with regulatory and clinical experts, has made the decision to make some changes to the IIH EVOLVE trial’s existing protocol.

The Company plans to seek feedback from relevant authorities and obtain approvals from ethics committees for the revised protocol. The primary endpoint of assessing the change in intracranial pressure (ICP) from baseline at 24 weeks in the Presendin™ arm versus placebo for newly diagnosed IIH patients will remain unchanged. However, the protocol changes will prioritise the inclusion of the more robust Quality of Life Short form 36 (SF-36) Physical component score (PCS) as the key secondary endpoint, replacing PMD. Plus, it will cut down its sample size from the original 240 to 130.

The Company is hoping that its strategic evaluation and protocol amendments will pivot it towards profitability with Presendin. Or at least get it started in that direction. Since the announcement, Invex’s share price has plummeted by 48.4% to $0.23.