Company: ATHE
Filing Date: 2025-08-29
Form Type: 20-F
Source: 0001213900-25-082027
Chunk: 49

Company: ALTERITY THERAPEUTICS LTD
Filing Date: 2025-08-29
Form: 20-F
Item: Item 4
Chunk 49
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 benefits including assistance in developing clinical protocols, reduced fees and access to EU-funded research grants.

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ATH434-201 Phase 2 Clinical Trial

In July 2022, we commenced our first Phase 2 clinical trial of ATH434 in patients with MSA. The trial, known as ATH434-201, is a randomized, double-blind, placebo-controlled investigation that explored the effect of ATH434 treatment on clinical and biomarker endpoints. Activity in the outpatient setting was assessed with wearable movement sensors. The study was expected to enrol 60 adult patients with MSA to receive 12 months treatment with one of two dose levels (50 mg and 75 mg twice daily) of ATH434 or matching placebo.

In November 2023, we announced completion of enrollment in the ATH434-201 clinical trial. The study enrolled 77 adults with MSA.

In May 2024, we announced that an independent Data Monitoring Committee (DMC) completed its third prespecified review of unblinded clinical trial data from the ATH434-201 Phase 2 study. Consistent with the first two reviews, the DMC expressed no concerns about safety and recommended that the study continue as planned without modification. This recommendation is an important milestone as participants were able to safely tolerate ATH434 as their time on study increased.

In December 2024, we reported the completion of the ATH434-201 study as the last patient finished all clinical evaluations leading to the announcement of the topline results.

In January 2025, we announced topline results for the trial and have since published additional data supporting the positive results for ATH434 in the treatment of MSA. ATH434 demonstrated significant slowing of clinical progression and a favorable safety profile in MSA. The results show that ATH434’s targeting of labile iron may have a disease modifying effect. The fact that we achieved statistical significance on the key clinical endpoint, Modified Unified MSA Rating Scale Part 1 (UMSARS Part 1), is extremely meaningful because it assesses the functional areas affected in MSA and it is the endpoint needed to support drug approval by the FDA.

In May 2025, additional analyses evaluated the clinical analysis population (n=71) who had at least one post-baseline assessment of the key clinical endpoint, the modified UMSARS part I activities of daily living scale. On this endpoint, ATH434 demonstrated a clinically significant reduction in disease severity versus placebo, with a 48% relative treatment effect at the 50 mg dose (p=0.02) and