A year ago, in Medical Forum, we outlined the progress at the Perron Institute in developing the neuroprotective peptide poly-arginine-18 (R18; renamed ARG-007) for acute ischaemic stroke. Since then, there have been significant developments that suggest a major breakthrough is imminent.

In February, our Canadian colleagues published the results of their phase III trial of the neuroprotective peptide, NA-1, also called nerinetide. The NA-1 trial randomised 1105 patients with acute ischaemic stroke to receive nerinetide or placebo, in addition to standard stroke care comprising endovascular thrombectomy, with or without thrombolysis, with tissue plasminogen activator (tPA).

Dr David Blacker, Neurologist, Hollywood.

Overall, the results were neutral. However, in those subjects not receiving tPA there was a statistically significant reduction in infarct volume, increased chance of a good outcome and lower risk of death. A pharmacokinetic sub-analysis suggested plasmin, the proteolytic enzyme activated by tPA, was degrading nerinetide, rendering it ineffective.

Experiments by the team at Perron Institute have consistently found ARG-007 to be superior to NA-1, and critical new data has demonstrated that the D-enantiomer of ARG-007 (i.e. peptide synthesised with D-arginine as opposed to L-arginine) to be resistant to degradation by plasmin. This has important implications for ARG-007 to be potentially a much more useful drug when used in combination with standard stroke care.

This, together with recent encouraging immunotoxicity safety data, has further accelerated the work leading up to a first in-human trial of ARG-007, which will probably occur in the first half of 2021.

A pre-clinical drug development bio-tech company, Argenica Therapeutics (argenica.com.au), has been formed to fund the next stages of development. The goal is to list the company on the ASX in March 2021 which should generate funds to support clinical trials of ARG-007.

Key messages

  • ARG-007 may be useful in combination with standard stroke care
  • Recent trials have been encouraging
  • Funding for clinical trials planned for 2021.

Questions? Contact the editor.

Author competing interests: The author is an advisor to and shareholder in Argenica Therapeutics.

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