PharmAust is rebranding to Neurizon Therapeutics. Pic: Getty Images
Health & Biotech
Special Report: Clinical stage biotech targeting human neurodegenerative diseases PharmAust (ASX:PAA) has rebranded to Neurizon Therapeutics (ASX:NUZ) in what has been a big year for the company after a share price charge of almost 90%.
NUZ is advancing its lead drug monepantel, now known as NUZ-001, for the treatment of neurodegenerative diseases, starting with amyotrophic lateral sclerosis (ALS), which is the most common form of motor neurone disease (MND).
NUZ-001 has been granted orphan drug designation (ODD) by the US Food and Drug Administration (FDA) to treat ALS.
The FDA’s ODD status is designed to support the development of treatments for rare diseases affecting fewer than 200,000 people in the US.
Benefits include tax credits, grants, waived clinical trial fees, and seven years of market exclusivity upon drug approval.
NUZ-001 works by inhibiting a key cellular pathway known as mTOR, which is crucial for regulating cell growth, proliferation, and a process called autophagy, which is how cells recycle and dispose of damaged or abnormal components.
In ALS, misfolded proteins — abnormal proteins that accumulate due to mutant genes — build up in motor neurons.
The misfolded proteins disrupt normal cell function and contribute to the progressive degeneration of nerve cells.
NUZ-001 targets the mTOR pathway, which is thought to help clear these misfolded proteins from the neurons, which in turn could potentially slow or halt ALS disease progression.
The company said NUZ-001 had the potential to treat other neurodegenerative diseases including Alzheimer’s, Parkinson’s, Huntington’s disease and multiple sclerosis.
NUZ-001 has shown promising early efficacy results in patients with ALS by slowing ALS disease progression and increasing life expectancy, which on average is just over two years following diagnosis.
The Phase 1 trial, known as the MEND study, tested NUZ-001 in 12 ALS patients. The study involved a 24-hour single-dose pharmacokinetic analysis, followed by a four-week periods of escalating doses of NUZ-001. Patients were treated with various doses of NUZ-001 continuously for up to 12 months.
The results indicated that NUZ-001 was safe and well-tolerated, with a 39% reduction in the rate of decline in ALS symptoms, compared to historical data from the PRO-ACT database, with no severe side effects noted.
An additional open-label extension (OLE) study was initiated for patients who had completed the Phase 1 MEND study.
Of the 12 patients from the Phase 1 MEND study, 10 rolled over onto the OLE study, and 9 remain on treatment.
Interim results from the OLE study show that NUZ-001 continues to slow disease progression, improves survival, and is well-tolerated at a daily dose of 10mg/kg.
Compared to untreated ALS patients from the historical PRO-ACT database, those on NUZ-001 lived significantly longer, with an 80.3% lower risk of death and a 43.2% slower decline in function. Notably, 56% of patients on NUZ-001 showed no decline in function.
An adaptive Phase 2/3 clinical trial in the US is expected to start in late 2024 and will be carried out as part of the HEALEY ALS Platform.
The HEALEY ALS Platform is a prestigious US program aimed at speeding up the assessment of new ALS treatments by evaluating several experimental drugs at once.
Recognised for its effectiveness in accelerating drug development timelines, it could reduce both the duration and costs associated with bringing new therapies to market.
With a market size exceeding US$2bn annually and high pricing for new therapies, there is a significant opportunity for NUZ-001 if it proves effective.
There is currently no cure for ALS, which affects the cells and nerves in the brain and spinal cord controlling the muscles.
This results in rapid weakness and wasting of the muscles, often progressing to paralysis in swallowing and breathing resulting in respiratory failure.
The exact cause of ALS is currently not fully understood, with only 10% of cases being inherited (familial), while the remaining 90% have no clear genetic or environmental cause (idiopathic).
In the US, the ALS patient population is more than 20,000 with around 5,000 new diagnoses each year. There are currently more than 200,000 patients with ALS worldwide.
Current treatments offer modest benefits, meaning that there’s a clear, critical need for more effective options.
Amylyx’s Relyvrio, a competitor drug for ALS before being withdrawn, was priced at US$158,000. Relyvrio slows disease progression by about 25% after 24 weeks.
Relyvrio was expected to reach peak sales of US$2.6bn with 50% market penetration and in its first year after being conditionally approved earned US$381m.
Managing Director and CEO Dr Michael Thurn said NUZ had a clear mission to advance groundbreaking science to reach a new horizon in neurodegenerative disease treatments.
“The company has a refreshed strategy that strengthens our focus on creating a promising horizon for patients and their families and is driven by three core strategic pillars,” he said.
“First, we’re improving patient access to innovative ALS treatments like NUZ-001.
“In Australia, we’re exploring compassionate use under the Special Access Scheme, and early access programs in the US and Europe.”
He said secondly, through partnering with the world’s leading neurologists and the HEALEY ALS Platform Trial, NUZ was accelerating hope and bringing NUZ-001 closer to ALS patients.
“Finally, we’re unlocking the potential of NUZ-001 to treat a range of neurodegenerative diseases,” he said.
“We are targeting complex and often neglected disease areas where there have been no significant advances in treatments for decades.”
This article was developed in collaboration with Neurizon Therapeutics, a Stockhead advertiser at the time of publishing.
This article does not constitute financial product advice. You should consider obtaining independent advice before making any financial decisions.
Health & Biotech
Health & Biotech
Health & Biotech
