‘One straightforward request’: Neurizon buoyant on FDA clarity

The US FDA has provided further clarification on Neurizon’s IND application for NUZ-001.

Neurizon has received clarification from the FDA on its IND application for NUZ-001. Pic via Getty Images

Special Report: Neurizon Therapeutics is buoyant after receiving a 'straightforward request' from the FDA following a clinical hold placed on the company’s Investigational New Drug application for its lead drug candidate, NUZ-001.

• The FDA has provided further clarification on Neurizon’s IND application for NUZ-001 
• The US regulatory authority has responded with only one straightforward request for additional information 
• Company to provide additional animal exposure data to assess the adequacy of systemic exposure to NUZ-001 in previous studies

Neurizon Therapeutics (ASX: NUZ), a clinical-stage biotech company focused on advancing treatments for neurodegenerative diseases, has revealed it received some important clarification from the US Food and Drug Administration (FDA) on Saturday. 

The regulator has requested additional animal exposure data be provided to assess the adequacy of systemic exposure to NUZ-001 during the relevant studies that have been undertaken. 

Neurizon said it had started planning to generate the additional exposure data and remained confident that further information would assist in reinforcing and better defining safety margins for NUZ-001’s clinical development program. 

The company noted its board, management and scientific and regulatory teams were working to expeditiously advance the process, leveraging its established expertise and strong industry partnerships. 

Neurizon said the FDA did not specify any safety concerns arising from previously undertaken clinical studies using NUZ-001.

The company announced on January 17 that the FDA had placed its Investigational New Drug (IND) application for NUZ-001 under clinical hold, pending further clarification and additional information. 

The company said the regulator expressed 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 regimen.

At the time of writing, detailed FDA feedback was expected within 30 days and the company expected that this should provide specific clarifications needed to progress the IND application.

‘One straightforward request’

Managing director and CEO Dr Michael Thurn said Neurizon considers that it was well-positioned to meet the FDA’s additional request following the clarification. 

“We appreciate the FDA’s guidance and are pleased that the FDA has responded with only one straightforward request for additional information,” he said. 

“This request focuses on enhancing and ensuring confidence in the previous animal safety data generated for NUZ-001 used in veterinary applications.

“With established regulatory and scientific expertise, strong industry partnerships, and an open and constructive relationship with the FDA, we will work swiftly to generate the requested information to further strengthen the regulatory package for NUZ-001’s clinical development. 

He said the company remains committed to advancing NUZ-001 as an effective potential treatment for amyotrophic lateral sclerosis/motor neurone disease (ALS/MND) and other neurodegenerative diseases.

Thurn said the impact of the FDA’s request on the timeline of the company's planned ALS clinical trial was currently being assessed.

Neurizon announced earlier this month it had been granted a patent in the US for NUZ-001 to treat (ALS/MND), other neurodegenerative diseases and cancer. 

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.

Originally published as ‘One straightforward request’: Neurizon buoyant on FDA clarity