A Western Australian developed potential therapy ARG-007, designed to reduce brain tissue death after brain injury and other neurological conditions, has been granted Orphan Drug Designation status by the U.S. Food and Drug Administration for treatment of Hypoxic Ischaemic Encephalopathy (HIE).

HIE, a form of brain injury caused by babies being deprived of oxygen before, during or immediately after birth, contributes to one of the leading causes of death and disability in children under two.

In an ASX announcement, Argenica Therapeutics, the WA biotech company working towards commercialisation of ARG-007, said FDA Orphan Drug Designation status qualified companies for incentives, including tax credits for qualified clinical trials, user fees exemption, and potential seven years of market exclusivity after approval.

The ARG-007 therapeutic has been developed through research led by Argenica’s Chief Scientific Officer Professor Bruno Meloni (Perron Institute and The University of Western Australia) and Clinical Professor Neville Knuckey (Perron Institute, UWA and Sir Charles Gairdner Hospital).

The application of ARG-007 for HIE is being led by Argenica’s Neonatal Scientific and Regulatory Advisor Dr Adam Edwards (Perron Institute, UWA and SCGH).

Argenica recently announced further encouraging preclinical data on the potential for reducing brain injury following HIE, showing ARG-007 significantly reduces brain cell death in preclinical models.

Dr Liz Dallimore, Argenica’s Managing Director, said achieving Orphan Drug Designation status for ARG-007 in HIE was a key pillar of Argenica’s commercialisation strategy for the company’s lead neuroprotective peptide candidate.

“The potential for extensive market exclusivity following approval of ARG-007 is an extremely compelling commercial driver for the company,” she said.

“We look forward to continuing to progress the development of ARG-007 as a treatment for HIE in newborn infants.”

Following a successfully completed Phase 1 clinical trial in healthy human volunteers to assess the safety and tolerability of a single dose of ARG-007, Argenica is progressing towards a Phase 2 clinical trial in ischaemic stroke patients.