Initial data for Alterity Therapeutics Phase 1 clinical trial released at American Academy of Neurology Annual Meeting

Alterity Therapeutics Limited (ASX: ATH, NASDAQ: ATHE) (“Alterity” or “the Company”) is today releasing interim clinical data of its Phase 1 clinical trial program for its investigative drug PBT434 at the American Academy of Neurology Annual Meeting in Philadelphia, USA.

The Platform Presentation titled: A First in Human Study of PBT434, a Novel Small Molecule Inhibitor of α-Synuclein Aggregation is being delivered by David Stamler, MD, Chief Medical Officer & Senior VP Clinical Development.  The American Academy of Neurology Annual Meeting is one of the largest gatherings of clinicians and researchers focusing on neurology in the world.

The study is expected to complete mid-2019, however Alterity has been invited to present interim data from the initial four single dose cohorts and the initial three multiple dose cohorts. The data being released indicate that PBT434 was well tolerated with adverse event rates comparable to placebo and dose dependent systemic exposure following oral administration. 

Importantly, the results indicate that PBT434 not only crosses the blood brain barrier in humans, confirming previous observations in animal studies, but that clinically tested doses achieve concentrations in the brain that exceed those associated with efficacy in animal models of disease.

No serious adverse events were reported and no subject discontinued dosing with PBT434 due to adverse events.

Dr David Stamler said: “The notable finding from this study is that PBT434 penetrates into the human brain and achieves concentrations that are potentially clinically relevant at doses that were well tolerated in healthy volunteers.  We are very encouraged by these results which represent an important step in the advancement of PBT434.  We look forward to providing final data at a medical conference later this year.”

PBT434 is an oral small molecule drug candidate with potential for treating synucleinopathies such as Parkinson’s Disease and Multiple System Atrophy, a rare and rapidly progressive neurological disorder that affects adults.

The Phase 1 Clinical Trial for PBT434 commenced in 2018 in Australia, recruiting healthy adult and older adult (≥ 65) volunteers with the primary goals of assessing the safety and tolerability of PBT434 after single and multiple oral dose administration. Secondary goals include evaluating pharmacokinetic measures that will allow Alterity understand how PBT434 is absorbed and metabolised by the body.

PBT434 is the first of a new generation of small molecules designed to block the accumulation and aggregation of α-synuclein.  α-synuclein is of great interest because aggregated forms of the protein are considered a pathological hallmark of Parkinsonian conditions and are a recognised therapeutic target by neuroscientists and clinicians.

End Note

The Company changed its name on 8 April 2019 from Prana Biotechnology Limited to Alterity Therapeutics Limited, (ASX: ATH, NASDAQ:ATHE).

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