Alterity Therapeutics (ATH) Study Result summary

Study design and patient population

• Phase II randomized, double-blind, placebo-controlled trial in early-stage MSA enrolled 77 participants treated for 12 months.

• Evaluated efficacy, safety, and pharmacokinetics of ATH434 at 50 mg and 75 mg BID versus placebo.

• Key endpoint was change in UMSARS Part I (activities of daily living).

• Additional endpoints included global severity, motor function, autonomic symptoms, brain iron and volume, and wearable sensor data.

• Inclusion required parkinsonism, autonomic impairment, ambulatory status, and elevated brain iron on MRI.

Patient population and baseline characteristics

• Participants had a mean age of 62.8 years and 59% were male.

• Modified UMSARS I baseline scores averaged 15.5.

• Mean duration of motor symptoms was 2.5 years.

• Baseline characteristics were consistent with early-stage MSA.

Efficacy and clinical outcomes

• 50 mg dose slowed clinical progression by 48% (p=0.03); 75 mg dose by 29–30% (p=0.2) on UMSARS I at 52 weeks.

• Clinically meaningful slowing of disease progression observed at both dose levels.

• Clinical benefit observed on secondary endpoints: global impression, motor scores, wearable sensor activity, and orthostatic hypotension.

• Patients on active treatment had more steps, walking bouts, and time standing compared to placebo.

• Improvement in multiple domains relevant to patient quality of life.