• Neurizon submits formal request to US FDA for clarification on pharmacokinetic studies needed to lift hold on its IND application for NUZ-001

• FDA expected to issue formal response to request within 60 days

• Neurizon to proactively start pharmacokinetic studies required to generate additional animal exposure data 

Special Report: Neurizon Therapeutics has submitted a formal request to the US Food and Drug Administration (FDA) for clarification on two short-term, low-cost pharmacokinetic (PK) studies necessary to lift a clinical hold on the Investigational New Drug (IND) application for its lead drug candidate NUZ-001.

Neurizon Therapeutics (ASX: NUZ) announced last month that the regulator had requested additional animal exposure data be provided to assess adequacy of systemic exposure to NUZ-001 during the relevant studies that have been undertaken, after placing its IND application for the drug on a hold in January. 

The clinical-stage biotech company, which is focused on advancing treatments for neurodegenerative diseases, said it had been in communication with the FDA, including submitting a formal request for advice detailing the two short-term PK studies required to lift the hold on IND for NUZ-001.

The company said the PK studies were anticipated to provide additional animal exposure data requested by the FDA to assess the adequacy of systemic exposure to NUZ-001. The FDA has indicated that it will provide a formal response within the next 60 days.

PK studies to begin in coming weeks

Wasting no time, Neurizon plans to proactively start the PK studies in the coming weeks. 

The company said the studies were expected to take approximately four months from start to completion, including study start-up, the 28-day study treatment period, data analysis and reporting. 

Neurizon said the PK studies were expected to cost between $400,000 to $600,000 and be eligible for rebate under the Australian government’s R&D Tax Incentive Scheme.

The extra PK data generated from the studies will support ongoing clinical development of NUZ-001 and future regulatory approval applications.

Managing director and CEO Dr Michael Thurn said the decision to proactively undertake the two PK studies follows ongoing review of its FDA correspondence to date, as well as close consultation between the board, management and the company’s scientific and regulatory advisors. 

“The two studies, which are anticipated to be completed in around four months, are expected to generate additional animal exposure data in line with the FDA’s straightforward request to date to lift the clinical hold,” Thurn said. 

Neurizon’s MD emphasised the company remained committed to advancing NUZ-001 as an effective potential treatment for amyotrophic lateral sclerosis/motor neurone disease (ALS/MND) and other neurodegenerative diseases.

“Undertaking these studies prior to the receipt of a formal response from the FDA highlights the company’s proactive approach to satisfying the regulator’s request and our ongoing commitment to advancing NUZ-001 as an effective potential treatment for ALS and other neurodegenerative diseases,” he said. 

“The timely execution of these studies will allow NUZ-001 entry into the Healey ALS Platform Trial during the second half of CY2025.” 

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.