European Union Regulatory Guidance for ATH434 Phase 2 Clinical Trial

Highlights:

• Alterity receives positive guidance from the European Medicines Agency’s Committee for Medicinal Products for Human Use on its Phase 2 clinical trial for Multiple System Atrophy.
• Concurrence with Alterity’s plan to target early stage MSA patients.
• Endorsement on selection of biomarker endpoints to assess pathological hallmarks of MSA.
• Agreement that ATH434 has potential as a disease modifying treatment.

Alterity Therapeutics Limited (ASX: ATH, NASDAQ: ATHE) (“Alterity” or “the Company”) has received guidance from the European Medicines Agency (EMA)regarding key aspects of the Company’s Phase 2 clinical trial for investigational drug ATH434 in the treatment of Multiple System Atrophy (MSA).

Alterity is actively preparing for the launch of its Phase 2 trial expected to commence in 2H of this calendar year.  The EMA is the agency of the European Union responsible for the evaluation and supervision of medicinal products.  More specifically, EMA provides an important role in supporting the timely and sound development of high-quality, effective, and safe medicines, for the benefit of patients. Whilst their advice is non-binding, it has the benefit of influencing improved trial designs that are more likely to generate robust and complete data to show whether a treatment works and is safe.

Given there is no approved treatment for MSA, there is currently no regulatory precedence for defining the most suitable patient population or clinical endpoints in efficacy studies, thus requiring greater consideration in developing an optimal trial design.  Accordingly, Alterity has sought diverse input from clinical experts and global regulatory authorities, and is conducting a natural history study, called BioMUSE, to identify biomarkers and clinical endpoints best suited to capture efficacy signals in the Phase 2 study.

The EMA has given its support to Alterity’s intention to enroll early-stage MSA patients and to utilize biomarkers to accurately diagnose these patients prior to enrolment.  Improving diagnostic accuracy and targeting early-stage patients will enable Alterity to maximize the opportunity to demonstrate the efficacy of ATH434, its potentially disease modifying therapy.

ATH434 is the first of a new generation of small molecule drug candidates designed to block the accumulation and aggregation of α-synuclein.  Alpha-synuclein, when aggregated in the brain, is a pathological hallmark of Parkinsonian disorders such as MSA and is considered an important biologic target for treating these neurodegenerative diseases.  ATH434 is thought to achieve its biological effect on α-synuclein by binding and redistributing excess iron in areas of pathology. The EMA recognized the potential role of iron in the pathogenesis of MSA and accordingly supported the use of biomarker endpoints to assess iron content and α-synuclein pathogenesis.

As a result of interactions with the EMA, and previously with the US Food and Drug Administration (FDA), Alterity is finalizing its Phase 2 trial design including patient selection, sample size, treatment duration, as well as primary and secondary endpoints.

In addition, the bioMUSE Natural History Study being conducted at Vanderbilt University Medical Center in the US, has enrolled more than 50% of targeted patients and continues to collect vital clinical and biomarker data to inform the Phase 2 study design.

Alterity CEO Dr David Stamler said: “MSA is a devastating disease with no cure and few effective treatment options. With the valuable advice received from the EMA, we now have a clear path forward to finalize the study design and generate data that global regulatory authorities are seeking. This is another important step toward bringing much needed disease modifying treatments to individuals with MSA.”

END

Authorisation & Additional information

This announcement was authorized by David Stamler, CEO of Alterity Therapeutics Limited.