Alterity Therapeutics (ATH) investor relations material

Clinical development progress

• Achieved a major milestone with completion of phase II trial in MSA, showing significantly slower symptom decline compared to placebo over 12 months.

• Drug candidate ATH-434 is orally administered, with orphan drug and fast-track designations in the US and Europe.

• Phase II trial demonstrated 30%-48% less decline in disease severity for active groups, with both doses exceeding clinically meaningful thresholds.

• No significant safety signals observed; adverse event rates were similar between active and placebo groups.

• Open-label study in advanced patients showed consistent results with phase II data.

Market opportunity and commercial assessment

• Independent commercial analysis estimated global peak sales potential at $2.4 billion, driven by strong intent to prescribe and substantial unmet need.

• Prevalence estimates for MSA in the US range from 15,000 to 50,000, with commercial models assuming around 20,000 patients.

• Pricing assumptions based on orphan drug status, with an annual price of ~$300,000.

• Market share projections assume competition but anticipate majority share at peak due to superior efficacy.

Regulatory and operational plans

• Preparing for a series of FDA meetings in early next year to address non-clinical, pharmacology, and manufacturing requirements before phase III.

• Targeting end of phase II meeting with FDA by mid-next year, with phase III trial initiation dependent on regulatory feedback.

• Phase III trial expected to enroll up to 200 patients, double the size of phase II.

• Current cash reserves over $50 million are sufficient to reach phase III preparations, with additional capital needed for the trial itself.