MJFF $495K Grant to Help Move Alterity’s Potential Oral Therapy Into Trials

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by Diana Campelo Delgado |

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Alterity Therapeutics has been awarded a grant from the Michael J. Fox Foundation (MJFF) for animal studies to determine the optimal dose its of lead candidate ATH434, an essential step to opening trials in people with Parkinson’s disease.

“I look forward to seeing the results from the dose optimization studies for PD [Parkinson’s disease] clinical trials and future development in this indication,” Werner Poewe, MD, a professor of neurology at the Medical University Innsbruck, said in a press release.

A hallmark of Parkinson’s is the buildup of alpha-synuclein protein aggregates within brain cells. These clumps are found mainly in dopamine-producing nerve cells (neurons), where they appear to affect neuronal communication, or the brain’s way of sending messages to and from different regions.

Given the key role of alpha-synuclein aggregates in the development and progression of Parkinson’s and other neurodegenerative disorders, these protein clumps are seen as likely therapeutic targets.

ATH434 (formerly PBT434) is a small molecule designed to inhibit the aggregation of alpha-synuclein and tau proteins, both implicated in neurodegeneration — the progressive atrophy and loss of function of nerve cells. In animal models of these diseases, the therapy is reported to have lessened abnormal alpha-synuclein and tau protein buildup by redistributing labile iron in the brain.

Labile iron includes all forms of iron found within cells that are not associated with proteins, and may have toxic effects. These forms of iron are thought to be part of the mechanisms contributing to the development of several neurodegenerative disorders, including Parkinson’s, multiple system atrophy (MSA) and Alzheimer’s disease.

“Aggregates of misfolded alpha-synuclein protein are widely distributed in the brains of individuals affected by these disorders, and by reducing their build-up ATH434 has potential to improve the motor and non-motor symptoms of these devastating conditions,” Poewe said.

The $495,000 MJFF grant was given to a collaborative team of researchers led by Margaret Bradbury, PhD, vice president of nonclinical development at Alterity.

With this award, the team will evaluate the pharmacologic profile of ATH434 in a primate model of Parkinson’s. The goal is to determine the therapy’s optimal dose for future clinical trials. (Pharmalogical profiles look at how a drug reaches, interacts with, and accumulates in cells and organs; how it interacts with proteins; and the like.)

“This funding allows us to take another step towards realizing a program in Parkinson’s,” said David Stamler, MD, CEO of Alterity Therapeutics. It is the second MJFF grant given the company for its development work with this potential therapy.

ATH434 has been designated an orphan drug by the U.S. Food and Drug Administration and the European Commission as a possible treatment of MSA.

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