Alterity Therapeutics has announced the granting of a new composition of matter patent by the United States Patent and Trademark Office (USPTO).  The patent secures a broad monopoly over a new class of iron chaperones, a technology capable of redistributing excess iron in the central nervous system. The structural backbone depicted in the patent provides the foundation for small molecule drug candidates with potential to cross the blood brain barrier and directly attack a source of neuropathology.

Excess iron in the brain is implicated in the pathology of many important neurodegenerative diseases, such as Alzheimer's and Parkinson's diseases[1].  

The patent, entitled "Compounds for and Methods of Treating Diseases" (Application No. 16/818,641), was granted following expedited review by the USPTO.  It covers more than 150 novel pharmaceutical compositions that are designed to redistribute the labile iron implicated in many neurodegenerative conditions.

Alterity is on track to launch the Phase 2 trial of its lead clinical candidate ATH434 by the end of the calendar year.  ATH434 is a small molecule drug being developed for Multiple System Atrophy (MSA), a form of atypical parkinsonism where iron plays a key role in pathogenesis by promoting α-synuclein aggregation.  The scientific investigation of ATH434, along with the results from the Phase 2 study, will augment the development and optimization of novel compounds expected to emerge from the new patent.

The patent confers on Alterity 20 years of exclusivity, providing a strong basis for drug development and commercialization in major neurodegenerative diseases. 

Alterity's CEO, Dr David Stamler said: "This new patent is an important part of our corporate strategy to expand our portfolio of potential disease modifying treatments for neurodegenerative diseases affecting many individuals. The newly covered compounds target excess brain iron that is increased in these conditions, and we hope to identify a new clinical candidate by the time we get results from our lead clinical program."

In addition to the US, the company is pursuing patent protection in other jurisdictions.

Authorisation & Additional information
This announcement was authorized by David Stamler, CEO of Alterity Therapeutics Limited.

About Alterity Therapeutics Limited and ATH434

Alterity's lead candidate, ATH434, is the first of a new generation of small molecules designed to inhibit the aggregation of pathological proteins implicated in neurodegeneration. ATH434 has been shown to reduce abnormal accumulation of α-synuclein in animal models of disease by restoring normal iron balance in the brain. In this way, it has excellent potential to treat Parkinson's disease as well as various forms of atypical Parkinsonism such as Multiple System Atrophy (MSA).

ATH434 has been granted Orphan designation for the treatment of MSA by the US FDA and the European   Commission.

[1] Hagemeier J, Geurts J, Zivadinov R. Brain iron accumulation in aging and neurodegenerative disorders. Expert Review of Neurotherapeutics, 2012,12:12, 1467-1480, DOI: 10.1586/ern.12.128

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Zucca F, Segura-Aguilar J, Ferrari E, Mu?oz P, Paris I, Sulzer D, Sarna T, Casella L, Zecca L. Interactions of iron, dopamine and neuromelanin pathways in brain aging and Parkinson's disease. Progress in Neurobiology, Volume 155, 2017, Pages 96-119, ISSN 0301-0082, https://doi.org/10.1016/j.pneurobio.2015.09.012