Alternative drug developer Alterity Therapeutics (ASX: ATH) has received guidance from the US Food and Drug Administration (FDA) on the development pathway for its experimental treatment for multiple system atrophy (MSA), a Parkinsonian disorder that currently has no approved treatment.
The company, which is dual listed on the ASX and NASDAQ (under ticker code ATHE), met with the FDA following last year’s successful completion of a phase one clinical trial for its lead drug candidate ATH434.
The meeting was intended to gain input on the compound’s clinical development plan and for Alterity to receive feedback on its phase two study design.
Conclusions from the meeting included consensus of the non-clinical investigations needed to support the phase two study, and FDA’s support of key aspects of the study design including the proposed patient population, safety monitoring plan and the strategy for evaluating drug exposure during the trial.
Alterity chief medical officer Dr David Stamler said the meeting has provided the company with a “clear path forward” for conducting its phase two study.
“The FDA clearly recognises the seriousness of MSA and the need for new treatments to address this devastating orphan disease,” he said.
No approved treatment for MSA
MSA is a rare and rapidly progressive neurological disorder that affects adults and has no known cause.
The condition presents with similar motor symptoms as found in patients with Parkinson’s disease such as loss of muscle control.
Individuals with MSA may also lose the ability to coordinate voluntary movements and experience an impaired regulation of involuntary body functions including blood pressure, bowel and bladder control.
Unfortunately, most of these symptoms are not addressed by the drugs used to treat Parkinson’s disease.
Because there are no approved treatments for MSA, there is also no regulatory precedent regarding accepted efficacy endpoints.
Alterity said it will work with the FDA to develop an endpoint that is best suited for the MSA patients to be studied.
Natural history study
To develop an endpoint for its MSA clinical study, the FDA has encouraged Alterity to use data from its natural history study planned with clinical and neuroimaging experts at Vanderbilt University Medical Centre in Nashville, Tennessee.
The study, referred to as bioMUSE, will enrol early-stage MSA patients and track change in clinical parameters and biomarkers for up to one year.
According to Alterity, the study is important for characterising disease progression over time in selected patient populations and in optimising clinical trial design.
International regulatory strategy
Alterity is also pursuing a regulatory pathway in Europe and Australia with planning already underway to meet with European authorities.
“Given the uncertainty of study conduct and recruitment in the COVID-19 era, and with the need to target sites that are minimally impacted, it is prudent for the company to be flexible in identifying and recruiting sites around the world and maintaining optionality,” it said.