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news.The US Food and Drug Administration (FDA) has granted fast track designation to Alterity Therapeutics' oral agent ATH434 for treating multiple system atrophy (MSA).
The Phase II clinical trial results for the agent have shown strong clinical efficacy and a favourable safety profile. Credit: Louis Reed on Unsplash.
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The fast-track application included top-line data from the double-blind randomised Phase II clinical trial, which showed efficacy on the modified Unified Multiple System Atrophy Rating Scale (UMSARS)I alongside preclinical data confirming that the agent is a low to moderate affinity iron chaperone.
ATH434, which acts as an iron chaperone, is aimed at inhibiting the aggregation of pathological proteins associated with neurodegeneration.
It claims to have shown potential in treating not only MSA but also Parkinson’s disease and related disorders.
According to Alterity, Phase I trials indicated that the agent is tolerated well and reaches brain levels comparable to efficacious levels in MSA animal models.
The Phase II clinical trial results for ATH434 have shown strong clinical efficacy and a favourable safety profile.
Additionally, ATH434 holds orphan drug designation from both the US regulator and the European Commission for MSA treatment.
ATH434’s development continues with a second Phase II open-label biomarker trial, focusing on individuals with more advanced stages of MSA.
Alterity Therapeutics CEO David Stamler said: “Receiving the FDA’s fast track designation for ATH434, alongside the orphan drug designation we have already received, underscores the promise of this novel agent to address the urgent need for a disease modifying therapy for individuals with MSA.
“This designation reinforces the potential of ATH434 as demonstrated by recent scientific findings related to its mechanism of action and the robust and clinically meaningful efficacy from our double-blind Phase II clinical trial. Importantly, the fast track designation provides us the opportunity to interact with the FDA more frequently on the advancement of ATH434, potentially accelerating its development path and approval.”
