Company: ATHE
Filing Date: 2025-08-29
Form Type: 20-F
Source: 0001213900-25-082027
Chunk: 46

Company: ALTERITY THERAPEUTICS LTD
Filing Date: 2025-08-29
Form: 20-F
Item: Item 4
Chunk 46
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 motor impairment in older animals, which was associated with a reduction in iron levels and preservation of neurons in the substantia nigra, the brain region affected in Parkinson’s. These data support other studies indicating that ATH434 has a beneficial effect on the motor and non-motor symptoms in animal models of PD.

In October 2021, The Journal of Parkinson’s Disease published the results from a preclinical study investigating the effect of ATH434 on gastrointestinal complications titled “ATH434 Reverses Colorectal Dysfunction in the A53T Mouse Model of Parkinson’s Disease”. Non-motor symptoms are common in patients with Parkinsonian disorders, such as Parkinson’s disease and MSA. Parkinson’s disease patients experience gastrointestinal complications, cognitive deficits, autonomic dysfunction, and mood disturbance and these non-motor manifestations are an important source of morbidity and reduced quality of life.

Friedreich’s Ataxia 

In April 2024, a poster was presented at the World Orphan Drug Congress, entitled “Biophysical Characteristics of ATH434, a Unique Iron-Targeting Drug for Treating Friedreich’s Ataxia”. The study evaluated the ability of ATH434 to target the toxic form of iron that drives the pathology of Friedreich’s Ataxia, a rare neurodegenerative disease that affects young children to young adults. The study also evaluated traditional iron chelators that are designed to bind iron and remove iron from the body. Conversely, an iron chaperone is designed to bind and redistribute iron within the body. The results confirmed that ATH434 has properties consistent with a chaperone. These studies were published in the peer-reviewed journal Metallomics entitled “ATH434, a promising iron-targeting compound for treating iron regulation disorders”.

Alzheimer’s disease

PBT2 was our product candidate for Alzheimer’s disease. The drug candidate is orally bioavailable and has been shown to cross the blood-brain barrier. While PBT2 was found to be well tolerated in Phase 1 and Phase 2a trials, a Phase 2 trial did not meet its primary endpoint and the program was ended.

In March 2023, we announced a sub-licensing agreement for PBT2 to Professor Colin Masters, M.D., A.O., to advance compounds for the treatment of Alzheimer’s and related diseases. Under the license agreement, Alterity granted the entire rights to the acyl hydrazone patent protecting novel zinc modulators mentioned above, as well as an exclusive worldwide license to develop and commercialize both the novel zinc modulators