Neurizon has filed an IND with the FDA for NUZ-001. Pic via Getty Images
Health & Biotech
Special Report: Clinical stage biotech Neurizon Therapeutics (ASX:NUZ) has filed an Investigational New Drug (IND) application to the US Food and Drug Administration (FDA) for its lead drug candidate NUZ-001.
An IND application provides the FDA with essential information to authorise the safe testing of an investigational drug in humans or an approved drug for a new use or patient population.
The FDA evaluates data from animal and human studies, pharmacokinetics, toxicology and manufacturing processes to ensure the proposed research meets safety standards.
The FDA has a period of 30 days to review the IND application.
Neurizon said that filing of the IND application was a pivotal step in enabling the start of a phase 2/3 clinical trial of NUZ-001 within the Healey ALS Platform Trial framework.
The trial will take place for patients with amyotrophic lateral sclerosis (ALS), also known as motor neurone disease.
The Healey ALS Platform is a prestigious US program aimed at speeding up the assessment of new ALS treatments by evaluating several experimental drugs at once.
Neurizon said there was no cure for the ultimately fatal ALS disease, which is marked by the gradual breakdown of nerve cells in the spinal cord and brain, impairing voluntary muscle control of the limbs, which can ultimately result in respiratory difficulties and death.
Pending FDA clearance of the IND application, Neurizon said it anticipated Massachusetts General Hospital filing a protocol amendment to their IND for the Healey ALS Platform Trial to incorporate its regimen specific appendix in Q1 CY25.
Neurizon said it expected to start enrolling patients in the trial in H1 CY25.
This week Neurizon also reported a positive interim eight-month update on its ongoing 12-month open-label extension (OLE) study of NUZ-001.
The latest results confirm NUZ-001’s long-term safety and tolerability, as well as its significant potential to extend life expectancy for patients with ALS.
Neurizon said treatment continued to be well-tolerated at the recommended 10 milligrams per kilogram daily dose.
This is the same dose planned for the company’s phase 2/3 Healey ALS Platform Trial.
Managing director and CEO Dr Michael Thurn said the submission of its IND application to the FDA represented a critical milestone in Neurizon’s mission to address the devastating impacts of ALS.
“The application represents the culmination of several years of research and development and contains many elements that are ultimately required for our New Drug Application (NDA) submission,” Thurn said.
“Our team has worked tirelessly around the clock to produce this foundational regulatory document to realise our goal of commencing the Healey ALS Platform Trial.
“This trial provides an unparalleled opportunity to evaluate the therapeutic potential of NUZ-001 in a collaborative, streamlined manner with the world’s leading ALS neurologists.
“Our team remains committed to advancing this program and delivering hope to patients worldwide who are in urgent need of effective ALS treatments.”
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.
Health & Biotech
Health & Biotech
Health & Biotech
