Currently, treatment for multiple system atrophy (MSA) is symptomatic; there are no available therapies designed to slow disease progression. This represents a huge unmet need for this patient community. Biotechnology company Alterity Therapeutics (“Alterity”) is working towards addressing this through the development of ATH434, a potential therapeutic option. According to a July 6 news release, Alterity shared that the first patient had been dosed within a Phase 2 clinical trial evaluating the therapy’s safety, efficacy, and pharmacokinetics.
ATH434 is a next-gen small-molecule therapy designed to inhibit protein aggregation, such as that of α-synuclein, related to neurodegeneration. Through this, the drug works to maintain brain and nerve cell health, and stop the progression of MSA and other forms of Parkinsonism. In a Phase 1 study, researchers found that the treatment was able to cross the blood-brain barrier, and was safe and well-tolerated. The treatment has received Orphan Drug designation in both Europe and the United States. Learn more about ATH434.
During the trial, patients will receive either ATH434 – one of two doses available – or a placebo. Altogether, 60 patients will enroll. Over a 12-month period, researchers will evaluate safety, efficacy, and how the treatment affects various biomarkers.
Multiple System Atrophy (MSA): An Overview
Multiple system atrophy (MSA) is a rare and progressive neurodegenerative disorder which affects the autonomic nervous system. Though researchers have hypothesized genetic or environmental factors to play a role, MSA currently has no known cause. MSA may be considered cerebellar or Parkinsonian, depending on symptoms. On average, patients typically survive for around 10 years following symptom onset, which usually occurs in the 50s. Symptoms can include:
- Sleep disruptions
- Sexual dysfunction
- Postural orthostatic hypotension
- Irregular heartbeat
- Fatigue
- Decreased ability to sweat
- Leg weakness
- Tremors
- Problems with balance and coordination
- Slurred or slowed speech
- Difficulty swallowing
- Problems with bladder control
