Investors have backed Alterity Therapeutics (ASX: ATH) with the company securing $35 million to pursue its lead drug for treating Parkinsonian diseases, particularly multiple system atrophy (MSA).
The company’s lead compound ATH434 has attracted mounting scientific and clinical interest, with the latest raising receiving funds from institutional investors across Australia, the US and UK.
Speaking with Small Caps Alterity chairman and chief executive officer Geoffrey Kempler said he was “very excited” about where the company is heading, with shareholders recognising their money was in “smart, responsible and proven hands”.
He said ATH434 had “massive potential to help a lot of people”, especially when there is a “desperate unmet medical need for Parkinsonian treatments”.
As part of the company’s strategy to ensure ATH434’s success, Mr Kempler said he had put together an “A Team” headed by chief medical officer Dr David Stamler.
Mr Kempler said Dr Stamler has previously successfully navigated similar drugs through trials and locked-in regulatory approvals with the FDA and in Europe.
He added Dr Stamler had chosen to be part of ATH434’s development and that investors’ had taken “great comfort” with him on board.
Alterity has already collared orphan drug status for ATH434 in the EU and US, which places it in good stead with shareholders.
Other pharmaceutical companies and stakeholders are also following Alterity closely as it advances ATH434, Mr Kempler said.
As a neurodegenerative disease, MSA has similar motor symptoms to Parkinson’s disease, causing slowed movement, stiffness and tremor. MSA patients also have reduced coordination of voluntary movements and unstable gait early in disease.
It also impairs capacity to maintain normal blood pressure, bowel function and bladder control.
The disease tends to become symptomatic after the age of 50 and advances rapidly. As it progresses, patients also experience increased difficulty swallowing, vocal cord paralysis and greater immobility.
It ultimately leads to profound disability and death.
Mr Kempler pointed out current treatments include medication and lifestyle changes to manage systems. However, there is no cure or treatments that address the underlying causes behind the “devastating” condition.
Phase 1 trial results
Alterity’s phase 1 study evaluated the safety, tolerability, and pharmacokinetics in healthy adult and older adult volunteers.
Results showed ATH434 crossed the blood brain barrier, while also being well tolerated and deemed safe.
With ATH434, Alterity is attempting to treat the underlying causes of MSA by targeting the alpha synuclein protein, which is critical for normal neuron function. Given orally, ATH434 works by redistributing labile iron, thus inhibiting aggregation of the protein and eliminating the root cause of oxidative stress. In animal models of MSA, ATH434 preserved neurons and improved motor function.
In conditions such as Parkinson’s disease and MSA, alpha synuclein aggregates, resulting in cellular dysfunction and death. It is this neuronal impairment that underpins the symptoms of MSA.
Dr Stamler said the drug has the potential to “affect all aspects of the disease – the motor symptoms, the blood pressure problems, along with gait and balance and even bowel and bladder dysfunction”.
BioMUSE trial to underpin phase 2 success
Late last month, Alterity announced it has begun enrolling MSA patients in a bioMUSE study in the US, which is being carried out in collaboration with Vanderbilt University Medical Centre under the direction of Associate Professor of Neurology and Principal Investigator Daniel Claassen.
According to Alterity, bioMUSE is a natural history trial that will track the progression of MSA patients, without investigational drug treatment.
Mr Kempler said the study will provide “vital information” on early stage MSA patients for the planned phase 2 study mid-next year.
He added the bioMUSE trial will de-risk the phase 2 study, enhancing its chances of success, by identifying suitable efficacy endpoints and following the change in MSA symptoms over time.
Given the lack of available treatments for MSA, Mr Kempler estimates that ATH434 could potentially generate between US$550-725 million in US sales alone.
Ongoing research and discovery
In addition to ATH434, Mr Kempler said Alterity has other novel drugs in the pipeline with potential to treat a number of conditions.
Currently, the company is evaluating its drug PBT2 for use as an antimicrobial agent.
As a result, Mr Kempler anticipates “many new successes” for Alterity as it continues its research and development journey.