Poster The Joint Annual Meeting of the Stroke Society of Australasia (SSA) and Smartstrokes 2023

Phase II, double-blinded, randomised, placebo-controlled study to determine the safety, preliminary efficacy and pharmacokinetics of ARG-007 in acute ischaemic stroke patients (SEANCON) (#121)

David J Blacker 1 2 3 4 , Graeme J Hankey 1 3 , Meghan Thomas 5 , Tim Phillips 6 7 , Paul Bailey 5 8 , Geoffrey Donnan 6 9 , Jeffrey L Saver 6 10 , Samantha South 11 , Neville Knuckey 1 3 11 12 , Bruno Meloni 1 3 11
  1. Perron Institute, NEDLANDS, WA, AUSTRALIA
  2. Neurology, Sir Charles Gairdner Hospital, NEDLANDS, WA, AUSTRALIA
  3. Medicine and Pharmacology, University of Western Australia, CRAWLEY, WA, AUSTRALIA
  4. Chair, Clinical advisory committee, Argenica Therapeutics, NEDLANDS, WA, AUSTRALIA
  5. Clinical advisory committee, Argenica Therapeutics, NEDLANDS, WA, AUSTRALIA
  6. Clinical advisory committee, Argenica Therapeutics, NEDLANDS, WA, AUSTRALIA
  7. NIISWA, Neurological Intervention and Imaging Service of Western Australia, NEDLANDS, WA, AUSTRALIA
  8. Emergency Physician , WA Country Health Service , PERTH, WA, AUSTRALIA
  9. Melbourne Brain Centre, University of Melbourne, Melbourne, VIC, AUSTRALIA
  10. Neurology, School of Medicine, UCLA, LOS ANGELES, CALIFORNIA, USA
  11. Argenica Therapeutics, NEDLANDS, WA, AUSTRALIA
  12. Neurosurgery, Sir Charles Gairdner Hospital, NEDLANDS, WA, AUSTRALIA

Background: ARG-007 is a cationic poly-arginine peptide consisting of 18 D-arginine residues developed by Argenica Therapeutics, with the goal of neuroprotection in ischaemic stroke and other neurological conditions. Pre-clinical and a phase 1 study have now provided support for a phase II study in subjects with large vessel ischaemic stroke receiving guideline based standard of care.

Aims: To publicise this study amongst Australian clinical stroke researchers, and to invite up to 10 leading stroke centres to participate. The safety, preliminary efficacy and pharmacokinetics will be assessed.

Methods: The protocol for a phase II, randomised, double-blinded, placebo controlled, multi-centre study will be presented. Subjects will have ischaemic stroke due to large vessel occlusion undergoing mechanical thrombectomy (with or without thrombolysis), in accordance with local institutional practices. Subjects will be randomised 1:1 (placebo: ARG-007 0.3 mg/kg), to be administered by intravenous infusion over 10 minutes, as soon as enrolment criteria are confirmed, before completion of revascularization. Up to 100 subjects will be enrolled. In a subset, blood samples will be taken for pharmacokinetics. Follow up will be on days 2, 5 (or discharge), 30 and 90 and include standard stroke scales such as NIHSS, modified Rankin, Barthel Index, EQ-5D and tele-MOCA.

Results: Primary endpoints include mortality rate, incidence of SAEs and AEs, symptomatic intra-cranial haemorrhage, worsening of stroke severity and incidence of clinically relevant changes in routine blood tests and clinical vital signs. A secondary endpoint is change in infarct volume at 48 hours. An exploratory endpoint will be pharmacokinetic analysis of subjects treated with and without thrombolysis.

Conclusion: Results of this phase II study of ARG-007 will guide further studies.