FDA Lifts Clinical Hold on Neurizon's ALS Drug, Clearing Path for Human Trials

The U.S. Food and Drug Administration (FDA) has lifted a clinical hold on Neurizon's investigational drug for amyotrophic lateral sclerosis (ALS), paving the way for human trials to begin. The decision, announced by the Melbourne-based biotech company on Monday, marks a significant milestone in the development of NUZ-001, a potential treatment for ALS and other neurodegenerative diseases.

Clinical Hold Lifted After Preclinical Data Review

The FDA initially placed a hold on Neurizon's Investigational New Drug (IND) application for NUZ-001 in February, citing the need for additional preclinical animal exposure studies. The company stressed that no safety issues had been identified with the drug at that time.

In response to the FDA's request, Neurizon conducted new preclinical studies in rats and dogs, submitting a clinical hold complete response letter in July. Despite the company's expectation of a response within 30 days, the FDA informed Neurizon in August that a decision would be delayed until October 3rd.

Neurizon attributed the extended timeline to broader issues affecting the FDA, including agency-wide restructuring and staffing reductions resulting from recent administrative reforms. These changes have reportedly impacted the FDA's ability to maintain timely review cycles.

HEALEY ALS Platform Trial Integration

With the clinical hold now lifted, NUZ-001 is set to enter clinical trials as part of the HEALEY ALS Platform Trial. This privately funded, adaptive trial hosted by Massachusetts General Hospital aims to study potential therapies for ALS.

Neurizon expects Mass General Hospital to file a protocol amendment to its IND as part of the HEALEY trial, with patient enrollment targeted to begin in the fourth quarter of 2025. The integration into the HEALEY trial represents a significant step forward for NUZ-001, potentially accelerating its development process.

Challenges in ALS Drug Development

The inclusion of NUZ-001 in the HEALEY ALS Platform Trial comes against a backdrop of challenging developments in ALS drug research. Since its inception in 2020, the HEALEY trial has seen several setbacks, mirroring the broader difficulties faced in developing effective treatments for ALS.

Notable failures within the HEALEY trial include investigational drugs from Denali, AbbVie, and Calico. To date, only two companies participating in HEALEY - Prilenia Therapeutics and Clene - have managed to produce sufficient positive biomarker data to advance their molecules to further stages of development.

These challenges underscore the complexity of ALS as a disease target and highlight the potential significance of Neurizon's progress with NUZ-001. As the drug moves into human trials, it joins a select group of candidates that have overcome initial regulatory hurdles in the pursuit of an effective ALS treatment.