Neurizon Therapeutics’ disease-modifying drug NUZ-001 has been shown to be safe and effective over 2.5 years of dosing in patients with amyotrophic lateral sclerosis (ALS), further setting it up for its investigation in the HEALEY Platform trial.
In the open-label expansion (OLE) study (NCT06177431), which enrolled patients from the Phase I MEND study (NCT04894240), the Phase II dose of NUZ-001 was evaluated over 12 months.
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In the trial, the estimated mean rate of decline was -0.88 points per month as measured on the ALS Functional Rating Scale-Revised (ALSFRS-R), compared with -0.74 points/month observed in the MEND trial, suggesting stable and durable treatment.
There was no statistical difference in Vital Capacity Percent Predicted (VC PP) from the start of the MEND trial to the end of the OLE study. The mean VC PP reduced from 84.40% at the start of MEND to 62.33% after the OLE trial.
Plasma NfL levels also remained broadly stable over the 12-month OLE, and urinary p75ECD decreased by approximately 17%. Measures of ALS Specific Quality of Life-Revised (ALSSQOL-R) scores and Edinburgh Cognitive and Behavioural ALS Screen (ECAS) both supported a durable, long-term treatment effect.
Neurizon said that daily treatment with NUZ-001 was safe and well-tolerated, with no treatment-related deaths observed. There were 25 adverse events (AEs) reported, of which only three were considered possibly treatment-related.
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By GlobalDataInitial patients from the MEND study have now been receiving daily NUZ-001 for more than 2.5 years.
The long-term data supports the drug’s progression into the HEALEY ALS Platform trial, with NUZ-001 dosing set to commence in Q4 2025. This will only begin, however, if the US Food and Drug Administration (FDA) lifts its clinical hold on the therapy, with a decision expected in October 2025.
The HEALEY Platform trial is “accelerating ALS research” by investigating several treatments at once. The study has investigated drugs, including Zilbrysq (zilucoplan), verdiperstat, CNM-Au8, and pridopidin.
Neurizon CEO Dr Michael Thurn said: “We are very encouraged by these long-term treatment results, which reinforce the potential of NUZ-001 to deliver meaningful clinical benefits for people living with ALS.
“To see sustained functional and respiratory benefits, a clear survival advantage, and supportive biomarker trends after nearly three years of treatment gives us additional confidence as we finalise preparations for entry into the HEALEY ALS Platform trial.”
NUZ-001 is an oral deworming agent used in veterinary medicine that acts in ALS by inhibiting mTOR to prevent the phosphorylation of 4EBP1, which Neurizon hopes will exhibit therapeutic intervention in ALS.
Earlier this month, Mitsubishi Tanabe Pharma America’s (MTPA) oral Radicava (edaravone) showed long-term durability across four ALS trials; however, a non-inferiority trial pitting its on/off version of the drug against a daily version showed no benefit for either therapy.
GlobalData predicts that sales of ALS products will reach $1.28bn by 2029, with growth primarily being led by 12 novel pipeline agents.
GlobalData is the parent company of Clinical Trials Arena.
