Core Insights - Alterity Therapeutics announced positive results from the ATH434-201 Phase 2 clinical trial for Multiple System Atrophy (MSA), demonstrating that ATH434 slows disease progression and stabilizes orthostatic hypotension [1][2] Group 1: Clinical Trial Results - The ATH434-201 trial involved 77 participants, comparing two doses of ATH434 (50 mg and 75 mg) against a placebo, with results indicating a 48% relative treatment effect at the 50 mg dose and a 30% effect at the 75 mg dose on the UMSARS I scale at 52 weeks [3][13] - A new analysis accounting for baseline differences in orthostatic hypotension (OH) showed a strengthened efficacy signal in the 75 mg group, with a relative treatment effect of 35% [5][13] - ATH434 demonstrated a beneficial effect on OH symptoms, with placebo patients worsening significantly while both treatment groups remained stable [5][7] Group 2: Neuroimaging and Biomarkers - The trial utilized advanced neuroimaging and biomarkers to refine MSA diagnosis and track disease evolution, showing that ATH434 reduced iron accumulation in affected brain regions [7][8] - Imaging biomarkers supported MSA diagnosis in 96.1% of participants, indicating a multimodal approach may enhance diagnostic accuracy [8][9] Group 3: Safety and Tolerability - ATH434 was well tolerated, with adverse event rates similar to placebo and no serious adverse events attributed to the drug [7][13] - The study also indicated increased outpatient activity levels in participants treated with ATH434, as measured by wearable sensors [7][13] Group 4: Company Overview - Alterity Therapeutics is focused on developing disease-modifying therapies for neurodegenerative diseases, with ATH434 being a lead candidate showing robust clinical efficacy in MSA [11][15] - The company has received Fast Track Designation and Orphan Drug Designation from the FDA for ATH434, highlighting its potential in treating MSA [11][15]

Core Insights - Alterity Therapeutics announced positive results from the ATH434-201 Phase 2 clinical trial for Multiple System Atrophy (MSA) at the MSA Research Symposium [1][2] - The trial demonstrated significant clinical efficacy of ATH434, with a 48% relative treatment effect at the 50 mg dose and a 30% effect at the 75 mg dose [3][5] - ATH434 is designed to inhibit the aggregation of pathological proteins and has shown potential in treating neurodegenerative diseases [4][8] Company Overview - Alterity Therapeutics is focused on developing disease-modifying therapies for neurodegenerative diseases, particularly Parkinson's disease and related disorders [8] - The company is based in Melbourne, Australia, and San Francisco, California, and is advancing its lead candidate, ATH434, through clinical trials [8] Clinical Trial Details - The ATH434-201 Phase 2 trial involved 77 patients and assessed the efficacy, safety, and pharmacokinetics of ATH434 over 12 months [5][6] - Key findings included a clinically significant reduction in disease severity on the modified UMSARS I scale and improvements in motor activities measured by wearable sensors [3][5] - ATH434 was well tolerated, with no serious adverse events reported, and showed trends in reducing iron accumulation in MSA-affected brain regions [3][4][6] Disease Context - Multiple System Atrophy (MSA) is a rare, rapidly progressive neurodegenerative disease affecting the autonomic nervous system and movement [7] - MSA currently has no approved treatments that can slow disease progression, highlighting the potential impact of successful therapies like ATH434 [7]