Seven to ten years. That’s how long someone diagnosed with multiple system atrophy (MSA) has to live from when symptoms first appear. The progressive disease results in the gradual functional decline of the body’s involuntary functions such as blood pressure, breathing, bladder function and motor control.
Often confused with Parkinson’s which is characterised by the progressive degradation of nerves that control movement, MSA affects the autonomic nervous system.
There is currently no evidence that MSA is genetic or caused by environmental factors, meaning this ‘sporadic’ rare disease can affect anyone.
With no specific treatment for MSA, doctors are resigned to managing symptoms to varying degrees of success. Alterity Therapeutics (ASX: ATH, NASDAQ: ATHE) is developing a novel medication named ATH434 to reduce toxic levels of alpha-synuclein, a neuronal protein responsible for certain nerve cell functions. Alpha-synuclein accumulation is a pathological characteristic of MSA and plays a role in destruction of nerve cells. In theory, reducing accumulation of the protein in the brain will preserve function in patients.
The Drug has been granted Orphan Drug designation by the US Food and Drug Administration (FDA) and the European Commission (EC), and recently passed Phase 1 safety trials.
The Company is now furthering their work on ATH434, initiating a Phase 2 clinical trial for treatment of early stage MSA patients with enrolment now open in New Zealand. The randomised, double-blind and placebo controlled trial will evaluate the effect of the medication on protein levels in the brain and motor control in 60 patients. Patients will wear sensors that allow researchers to gather data on motor parameters. The trial will also further inform on the safety and pharmacokinetics of the drug.
ATH434 has potential applications in Parkinson’s Disease and other types of atypical parkinsonism as it inhibits the build up of proteins that cause neurodegeneration. With over 1 million people suffering from Parkinson’s in the US alone the treatment market is expected to exceed USD $11.5 billion by 2029- somewhat taking the sting out of having just one pipeline asset.
For the March quarter, Alterity reported a cash balance of $32 million