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Perth-headquartered PharmAust Limited (ASX: PAA) has received Orphan Drug Designation (ODD) from the US Food and Drug Administration (FDA) for monepantel and its treatment of Motor Neurone Disease (MND) / Amyotrophic Lateral Sclerosis (ALS).

The FDA grants ODD status to assist and encourage companies to develop safe and effective treatments for rare diseases and disorders (impacting less than 200,000 persons in the US). Under the US Orphan Drug Act, Orphan Drug status provides incentives, including tax credits, grants, waiver of some administrative fees for clinical trials, and seven years of market exclusivity following drug approval.

PharmAust’s application was based on preclinical mechanistic data that demonstrates MPL can induce autophagy in diseased cells, and consideration of the pathology of the disease. PharmAust initially submitted the request for ODD to the FDA’s Office of Orphan Products Development early in November 2023. In January 2024, the FDA responded, requesting further supporting data to establish the drug's potential for effectiveness in MND/ALS. In March, PharmAust submitted the positive clinical data from its Phase 1 MEND study involving 12 patients with MND/ALS.

The positive top-line data from the Phase 1 MEND Study was based on daily administration of MPL over a 7 to 12-month period was well-tolerated and did not result in any dose-limiting toxicities or serious adverse effects.

Daily administration of MPL resulted in a clinically meaningful therapeutic effect as evidenced by a small and non-significant numerical reduction in ALS Functional Rating Scale-Revised (ALSFRS-R) scores from baseline to end of treatment.

Quality of life and cognitive and behavioural function were also not significantly impacted, and there was no change in respiratory function (Slow Vital Capacity). The effects of treatment with MPL on biomarkers demonstrated a large reduction in cerebrospinal fluid (CSF), which supported a meaningful clinical effect.

"This is an outstanding milestone for PharmAust and monepantel, providing an even stronger pathway forward for the drug, particularly in light of recent failures of other MND/ALS treatments,” Managing Director, John Clark, said.

“We are now increasingly optimistic as we progress to our pivotal registration adaptive Phase 2/3 study which will commence in H2 CY2024.”

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