Neurizon Seeks FDA Guidance required to lift clinical trial hold on NUZ-001

Neurizon Therapeutics (ASX: NUZ) has formally requested guidance from the FDA, seeking to understand what specific new pharmacokinetic (PK) studies are required to lift the clinical hold on its Investigational New Drug (IND) application for NUZ-001.

The drug candidate is being developed as a potential treatment for amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases. The FDA is expected to provide a formal response to Neurizon's request within 60 days.

Additional PK studies are required to release FDA hold on pivotal IND application.

Regulatory Background and FDA’s Request

Earlier this year, the FDA placed Neurizon’s IND application for NUZ-001 on hold, citing the need for additional animal exposure data to ensure the adequacy of systemic exposure during previously conducted studies. In response, Neurizon has been in active discussions with the FDA and has now taken the step of formally requesting clarification on the design of the necessary PK studies.

An IND application is submitted to the FDA to obtain authorization to begin clinical trials of a new drug in humans in the United States. It includes preclinical data, manufacturing details, and study protocols to ensure the drug is safe for initial testing.

Rather than waiting for the FDA’s official response, Neurizon plans to initiate the required PK studies in the coming weeks. These studies, expected to span approximately four months, will encompass study start-up procedures, a 28-day treatment period, data collection and analysis.

By launching these studies ahead of the FDA’s response, Neurizon aims to expedite the regulatory process and ensure that NUZ-001 remains on track for further clinical development.

The estimated cost of the PK studies is anticipated to be up to $600,000, however, may well qualify for reimbursement under the Australian government’s R&D Tax Incentive Scheme to soften the blow.

The additional PK data is anticipated to address the FDA’s concerns but will also support Neurizon’s broader clinical development and regulatory strategy for NUZ-001. The drug candidate is being developed as a potential treatment for amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases.

Neurizon’s Commitment to Advancing NUZ-001

Managing Director and CEO Dr. Michael Thurn emphasized the company’s dedication to advancing NUZ-001 through a proactive and transparent regulatory strategy.

"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. 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."

Neurizon has a market cap of approximately A$47M at 10c per share and has a 52-week high of 24c.