Nyrada NYR-BI03 trial success heralds breakthrough in stroke and brain injury treatment

Drug discovery and development company Nyrada (ASX: NYR) has announced positive results from a pre-clinical study evaluating lead candidate NYR-BI03 in the prevention of secondary brain injury.

The drug is part of the company’s program seeking to develop therapies to reduce the long-term disabilities associated with stroke or traumatic brain injury (TBI) by limiting progressive cell death.

Conducted in collaboration with the University of New South Wales, The study involved inducing a focal ischemic stroke using a photothrombotic model—considered to be a minimally-invasive and reproducible way of inducing focal brain injury in test animals.

A total of 16 test animals were blind-treated with either NYR-BI03 or a vehicle control 30 minutes following the induced brain injury, with treatment conducted for 72 hours via continuous intravenous infusion.

Magnetic resonance imaging (MRI) was then performed to quantify the brain injury in drug-treated and vehicle control animals and determine the amount of tissue damage in the penumbra region, the area of secondary brain injury that NYR-BI03 is designed to target.

Blood samples were also analysed for changes in the biomarker neurofilament light (NfL), a protein associated with neuron fibre tracts released into the bloodstream following a brain injury.

Neuroprotection achievement

MRI scans showed that a “statistically significant” neuroprotection was achieved when the test animals received treatment with NYR-BI03.

On average, NYR-BI03 therapy rescued 42% of brain injury in the penumbra region compared to vehicle animals and the mean NfL level for the animals treated with NYR-BI03 was 41% lower than that of the vehicle control.

All animals survived the brain injury and drug treatment with no drug-related adverse effects reported.

The results are believed to build upon NYR-BI03’s good safety profile for continuous intravenous delivery in the sub-acute brain injury treatment interval.

This confirmation of brain injury neuroprotection efficacy enables Nyrada to commence good laboratory practice (GLP) studies this quarter to assess the safety of the NYR-BI03 molecule in two animal species.

‘Compelling outcome’

Chief executive officer James Bonnar said the results marks a key milestone in the company’s brain injury program and provides strong evidence that NYR-BI03 has the potential to protect the brain from secondary injury.

“The magnitude of rescue achieved in this study is a compelling outcome and signals a significant therapeutic and market opportunity.”

“This work is critical for our development pathway for NYR-BI03, giving us confidence as we advance it through to GLP safety and toxicology studies ahead of a first-in-human clinical trial currently planned in the second half of this calendar year,” he added.

Leading cause of death

Research shows that stroke and TBI are among the leading causes of death and disability worldwide.

NYR-BI03 is a first-in-class therapy with a novel mechanism of action that can limit the secondary brain injury that occurs following a stroke or TBI.

The drug has been developed to selectively block the “canonical” transient receptor potential (TRPC) ion channels that are over-activated during brain trauma, causing calcium overload which leads to brain cell death.

The US Food and Drug Administration is yet to approve any drugs for the treatment of secondary brain injury.