- Neurizon Therapeutics sees positive interim results from the use of NUZ-001 to treat ALS during September quarter
- Regulatory progress with SME status granted by the EMA and submission of OMPD status
- Company successfully raised $7.8m through SPP, far higher than the initial $2m it sought
Special Report: Clinical-stage biotech Neurizon Therapeutics – formerly PharmAust – made significant strides advancing its treatments for neurodegenerative diseases during the September 2024 quarter.
During this period, it advanced clinical-stage development of monepantel, now known as NUZ-001, for neurodegenerative diseases, with a focus on Amyotrophic Lateral Sclerosis (ALS).
Notable milestones include positive interim results showing that NUZ-001, administered at a daily dose of 10mg/kg, continues to be well tolerated and slows disease progression while improving survival rates.
Compared to untreated ALS patients from a historical database, those on NUZ-001 experienced significantly longer survival, an 80.3% reduced risk of death, and a 43.2% slower rate of functional decline.
Notably, 56% of the patients showed no functional decline at all.
Pushing ahead on approvals
Neurizon Therapeutics (ASX:NUZ) also saw regulatory advancements with the company submitting a request for NUZ-001 to receive orphan medicinal product designation (OMPD) for the treatment of ALS to the European Medicines Agency (EMA) during the quarter.
The treatment already has Orphan Drug Designation (ODD) from the US Food and Drug Administration’s (FDA) Office of Orphan Products Development and securing its counterpart in the European Union will provide it with 10 years of market exclusivity.
A decision on the OMPD is expected in December 2024.
Other progress includes being granted Small and Medium-Sized Enterprise (SME) status by the EMA, entitling the company to regulatory fee reductions and enhanced support, significantly streamlining its regulatory engagement.
Separately, the company raised $7.8m through a share purchase plan priced at 19c per share during the quarter, far outstripping the $2m it originally sought.
It follows on a $10m placement to institutional and sophisticated investors that was completed late in the previous quarter and ensures that NUZ is well funded to execute the milestone HEALEY ALS Platform Trial.
The company was selected for the prestigious trial earlier in July, a milestone that ensures that NUZ-001’s efficacy and potential therapeutic benefits can now be independently recognised and validated within the ALS research community.
NUZ had $15m in cash at bank as of September 30, 2024.
Transformational quarter
“This quarter was truly transformational as our company continued to strengthen our position to lead the development of treatments for neurodegenerative diseases,” managing director Dr Michael Thurn said.
“The selection of our lead product candidate, NUZ-001, for inclusion in the prestigious HEALEY ALS Platform Trial and positive regulatory engagement, combined with the strong support from our shareholders through the SPP, has solidified our momentum.
“We are making significant strides towards bringing NUZ-001 to patients with ALS, with encouraging clinical results and ongoing regulatory progress. Our focus remains on advancing these critical programs, and we look forward to the continued development of NUZ-001 in the months ahead.”
Planned activity
During the current quarter, NUZ plans to finalise the Specific Regimen Protocol for the HEALEY ALS Platform Trial, enrolment for which is expected to begin in Q1 2025.
It will also open an Investigational New Drug (IND) application with the US FDA while receiving the results from the OMPD application in the EU.
Additionally, the company expects to see preclinical data readouts in models of neurodegenerative diseases.
NUZ makes big strides in Q1 FY2025
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.
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