The US Food and Drug Administration (FDA) has released its clinical hold on Neurizon Therapeutics’ NUZ‑001, paving the way for its advancement to the HEALEY-ALS trial.
The company’s lead drug in amyotrophic lateral sclerosis (ALS) was put on developmental pause in February 2025.
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The lift opens the door for Neurizon to investigate the drug in the Phase II/III HEALEY-ALS trial; however, the company is preparing to file a protocol amendment to the investigational new drug (IND) application for the study, which is due to initiate in Q4 2024. 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.
CEO of Neurizon, Dr Michael Thurn, said: “The FDA’s clearance of our IND application marks a significant milestone in our mission to bring NUZ-001 closer to patients living with ALS. This achievement reflects both our unwavering commitment to rigorous scientific and clinical development and the tireless dedication of our team.
“We are now partnering with the HEALEY ALS Platform Trial team to take the next critical steps in advancing NUZ-001. Supported by our compelling TDP-43 preclinical data and encouraging survival outcomes from the Open-Label Extension (OLE) study in ALS patients, we believe NUZ-001 has the potential to meaningfully slow the progression of this devastating disease,” Thurn added.
In the OLE trial (NCT06177431), which enrolled patients from the Phase I MEND study (NCT04894240), 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.
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By GlobalDataNUZ-001 is an oral deworming agent used in veterinary medicine that inhibits mTOR to prevent the phosphorylation of 4EBP1, which Neurizon hopes will exhibit therapeutic intervention in ALS in humans.
ALS market set to expand
In September 2025, AL-S Pharma’s monoclonal antibody (mAb) met its co-primary endpoints in a Phase II trial. In the randomised, double-blind, placebo-controlled trial (NCT05039099), AP-101 combined with standard of care (SoC) met the safety and tolerability endpoints in patients with sporadic ALS and mutant SOD1-ALS; however, AL-S Pharma is yet to share specific data.
GlobalData predicts that sales of ALS products will reach $1.28bn by 2029, from $317m in 2019, with growth primarily being led by 12 novel pipeline agents.
GlobalData is the parent company of Clinical Trials Arena.
