NUZ have been busy over the last few months (unfortunately so have I which is why I am a bit late with this) and have released their interim data from their OLE study as follows:
Positive 8-month Interim Data from Open-Label Extension Study in Patients with ALS
Highlights:
Treatment with NUZ-001 remains well-tolerated, demonstrating encouraging results in slowing disease progression and increasing the life expectancy of patients with ALS
Key findings compared to untreated matched controls from the PRO-ACT Historical Database were:
o NUZ-001 significantly increased survival (χ2=11.67, p=0.00062)
o NUZ-001 significantly reduced the risk of death by 78.1% (HR=0.219, p=0.0044)
The mean rate of reduction in disease progression measured by ALSFRS-R from baseline was -0.77 points/month
Patients are now in their 27th month of continuous treatment with NUZ-001
There have been no serious adverse events related to treatment with NUZ-001
NUZ have also filed their IND application in relation to the Healey phase 2/3 trial as follows
Neurizon Files IND Application to Support HEALEY ALS Platform Trial Highlights:
· IND application submission to the U.S. Food and Drug Administration (FDA) is a pivotal step in initiating a Phase 2/3 clinical study for NUZ-001
· The IND is a comprehensive dossier of information, including animal and human studies, pharmacokinetic analyses, toxicology studies, and manufacturing information for NUZ-001
· The FDA has a period of 30 days to review the IND application · NUZ-001 targets TDP-43 protein aggregation, a hallmark of ALS pathology, supported by its demonstrated safety and preliminary efficacy profile in earlier clinical studies
At this stage NUZ has its Phase 2/3 trial which will be done by Healey almost ready to start with it starting in a month or so and will be finished around the end of the year. If the results from OLE are similar in the Healey trial then there will be significant SP appreciation as it has a market cap of around $84 Million at the moment and should move significantly upwards. I hold this in both my Strawman and actual portfolios and am happy to hold until the end of 2025.