After meeting with the FDA, biopharmaceuticals company Alterity Therapeutics (ASX: ATH) has confirmed Phase II clinical trial plans for their ATH434 compound in the treatment of Multiple System Atrophy (MSA), an incurable Parkinsonian disorder with no regulatory precedent.

Due to the unique scenario where there are no accepted efficacy endpoints, guidance has been provided by the U.S Food and Drug Administration (FDA) encouraging Alterity to use data from a natural history study that Alterity has planned with clinical and neuroimaging experts at Vanderbilt University Medical Center.

As there is no approved treatment for MSA, ATH434 has been assigned Orphan Drug designation by the FDA and European Commission. This is reserved for treatment, prevention or diagnosis of rare and/or incurable diseases which enable special funding due to their lack of attention from larger pharmaceutical companies.

“The FDA clearly recognizes the seriousness of MSA and the need for new treatments to address this devastating Orphan disease,” said Alterity Chief Medical Officer, Dr David Stamler.

“Our pre-IND (investigative new drug) meeting was very collegial, and I look forward to again collaborating with the Division of Neurology to determine the best development path for ATH434 in the US.

“With the information obtained from this meeting, we have a clear path forward for conducting our Phase 2 study in MSA.”

The natural history study to be conducted will enroll early stage MSA patients and track changes in clinical parameters and biomarkers for up to one year. Over the course of the year, the study will provide vital information to optimise clinical trial design and inform the selection of biomarkers to evaluate target engagement of drug candidates.

ATH434 is the first in a new generation of small molecules designed to inhibit the aggregation of pathological proteins implicated in neurodegeneration. Tested on animals, it has shown to redistribute labile iron to the brain giving it the potential to treat MSA.

With no known cause or treatment, MSA is a rare and rapidly progressive neurological disorder where onset tends to commence after age 50, evidenced initially by motor impairment symptoms. Individuals with MSA may also experience loss of ability to coordinate voluntary movements and impaired regulation of involuntary body functions such as blood pressure, bowel and bladder control.