NUZ have released the following announcement RE their drug NUZ-001:

US Patent granted for NUZ-001 for the treatment of Neurodegenerative Diseases Highlights:

 Patent granted by the USPTO covering the use of Neurizon’s lead drug candidate, NUZ-001, in neurodegenerative diseases and cancer

 Granted patent provides specific protection for NUZ-001 and structurally related compounds used in treatments for mTOR pathway-related diseases in the US market until 2039

 Considerably strengthen Neurizon’s commercial potential and provides a strong framework for any future licensing negotiations

 Patent grant follows FDA Orphan Drug Designation (ODD) for NUZ-001 in May 2024, further enhancing the Company’s IP estate and market position for NUZ-001 as the clinical trial pipeline advances

At this stage the Phase 2 Healey trial is still held up by the FDA but hopefully in the next few weeks NUZ will have an answer back in regards to the actual reason for the hold so they can move ahead with trial.

Neurizon have released the following announcement about the Phase 2/3 trials for NUZ-001:

Neurizon Therapeutics Limited has received notification from the U.S. Food and Drug Administration (FDA) that it has placed our Investigational New Drug (IND) application for NUZ-001 under clinical hold, pending further clarification and additional information. In their communication, the FDA outlined it has certain concerns about the sufficiency of information to assess the application and any risks to human subjects of the trial and with the proposed dosing regime.

The detailed FDA feedback is expected within 30 days and should provide the specific clarifications needed to progress the IND application. We are committed to thoroughly reviewing this feedback upon receipt, taking all necessary actions, and engaging in constructive dialogue with the FDA to address their feedback and provide the required information so that the FDA provides clearance to the IND. 

The FDA have not informed NUZ of why this has been placed on hold, but it could be something simple such as the FDA asking for a protocol amendment to include some additional patient safety monitoring, if so there will be a hold up of a few months. If it is something more serious then the wait could be longer.

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.

PAA have just released the results of their Open Label Extension study and the results are impressive as follows:

OLE Study update reveals impressive Survival Data Highlights:

 Compared to matched controls from the PRO-ACT Historical Database, treatment with monepantel results in a significantly ( 2=9.39, p=0.0022) longer survival of patients with MND/ALS

 Treatment with monepantel significantly reduces the risk of death by 91% (HR=0.087, p=0.0154) compared to PRO-ACT matched controls  Updated analysis of the rate of decline in ALSFRS-R to include the compassionate use program continued to show monepantel reduces the rate of disease progression

 Enrolment on to the Open Label Extension Study is now complete with 10 of the 12 patients from the Phase 1 MEND Study rolling over

 There were 4 patients that either had no change or had a slight improvement in ALSFRSR score while taking monepantel under compassionate use

What this shows is that Monopatel actually does stop the progression of Motor Nuron disease significantly so there may be something close to a cure,

PAA are moving onto the Phase 2/3 study which will determine actually how effective Monopatel is.

PAA have announced on Friday that Dr Michael Thurn has been appointed as MD & CEO, He initially resigned due to disagreements with the BoD but due to shareholder activism the Bod had been replaced and Dr MT has now been reappointed which is excellent news as he was the driving force in PAA over the last 6 months, anyway the announcement basics are as follows:

PharmAust strengthens executive team with the reappointment of Dr Michael Thurn and new CSO

 PharmAust has reappointed Dr Michael Thurn as Managing Director and Chief Executive Officer

 Dr Nicky Wallis has joined PharmAust as Chief Scientific Officer

 Dr Wallis is a neuroscientist who brings over 12 years of global expertise in preclinical and clinical development, and will lead the evaluation of monepantel as a potential treatment for other neurodegenerative diseases

 These appointments are part of a major reset for the Company as it strives to become a global leader in neurodegenerative diseases 

Definitely a buy