The first participant in Alterity Therapeutics’ phase 2 clinical trial of ATH434 in multiple system atrophy (MSA), a rare and severely handicapping Parkinson’s disease, has been dosed in the US, according to the biotechnology company’s announcement. Alterity Therapeutics is dedicated to developing disease-modifying therapies for neurodegenerative diseases.
According to David Stamler, M.D., chief executive officer of Alterity, “the US is a crucial region for our ATH434 phase 2 clinical trial, and we are happy to have dosed our first patient at Vanderbilt University Medical Center. “We are appreciative of the ongoing work that our Vanderbilt colleagues have been doing to support the ATH434 development programme for a very long period. As we work to provide MSA patients with a potential new therapy option, the phase 2 trial is gathering speed with active enrollment in five countries.
In the phase 2 clinical trial, ATH434 was examined in patients with early-stage MSA in a randomised, double-blind, placebo-controlled fashion. In addition to studies of safety and pharmacokinetics, the trial will look at how the therapy with ATH434 affects neuroimaging and protein biomarkers to show target engagement and clinical endpoints to show efficacy. The chosen indicators, such as brain iron and aggregating a-synuclein, play a significant role in MSA pathophysiology and are thus suitable targets for therapeutic activity demonstration. Moreover, the use of wearable sensors will be used to assess key motor functions for MSA patients. About 60 adults are anticipated to participate in the study and will either receive a placebo or one of two dosing levels of ATH434. Twelve months of treatment will be given to participants, giving researchers the chance to monitor changes in efficacy outcomes and improve the design of a conclusive phase 3 study.
The leading candidate from Alterity, ATH434, is an oral medication that is intended to prevent the aggregation of pathogenic proteins linked to neurodegeneration. By restoring the proper iron balance in the brain, ATH434 has been demonstrated in preclinical studies to minimise a-synuclein disease and protect nerve cells. It has good potential to treat Parkinson’s disease as well as other Parkinsonian illnesses including multiple system atrophy since it functions as an iron chaperone. In phase 1 investigations, ATH434 was shown to be well tolerated and to have reached brain levels that were close to those seen in animal models of MSA.
A randomised, double-blind, placebo-controlled phase 2 clinical trial of ATH434 is now being conducted in patients with early-stage MSA. The US FDA and the European Commission have designated ATH434 as an orphan drug for the treatment of MSA. A uncommon form of neurodegenerative disease called multiple system atrophy causes the autonomic nervous system to malfunction and impairs movement. The symptoms are a result of many types of nerve cells in the brain and spinal cord gradually losing their ability to function and dying. It is a disease that progresses quickly and leaves patients severely disabled. The symptoms of MSA, a Parkinsonian condition, can range from slower movement and/or rigidity to autonomic instability that impairs involuntary processes including blood pressure regulation and bladder control, as well as decreased balance and/or coordination that increases the risk of falling.
The buildup of the protein a-synuclein within glia, the central nervous system’s support cells, as well as the death of neurons in various brain regions, are pathogenic symptoms of MSA. Around 15,000 people in the US suffer from MSA, and while some of its symptoms can be managed with medication, there is currently no treatment that can stop the disease from progressing and no cure. A biotechnology business in the trial stages called Alterity Therapeutics is committed to forging a different future for patients with neurological disorders.
