First ALS Patient Dosed With NUZ-001 in HEALEY ALS Platform Trial

First ALS Patient Dosed With NUZ-001 in HEALEY ALS Platform Trial

By, Admin 27 February 2026

Neurizon Therapeutics Limited has reached a key clinical milestone. The company announced that the first participant has been enrolled and dosed with NUZ-001 in Regimen I of the HEALEY ALS Platform Trial.

The study is evaluating NUZ-001 for the treatment of amyotrophic lateral sclerosis (ALS) and is being conducted across leading ALS clinical centres in the United States.

About the HEALEY ALS Platform Trial

The HEALEY ALS Platform Trial is a large, adaptive Phase 2/3 study designed to accelerate the development of new ALS therapies.

Key features of the platform include:

  • Multicentre, randomised, double-blind, placebo-controlled design
  • Shared infrastructure across 70+ clinical sites
  • Simultaneous evaluation of multiple investigational drugs
  • A single master protocol to streamline enrolment and execution

The trial is led by the Sean M. Healey & AMG Center for ALS at Mass General Brigham, in partnership with the Network of Excellence for ALS (NEALS).

Regimen I: NUZ-001 Study Design

Regimen I focuses specifically on NUZ-001, Neurizon’s lead drug candidate.

Key study details

  • Approximately 160 participants with ALS
  • 36-week treatment period
  • Randomised 3:1 to NUZ-001 or placebo
  • Daily dosing at 10 mg/kg, the recommended Phase 2 dose

The regimen includes:

  • A randomised, placebo-controlled treatment phase
  • Followed by an active treatment extension phase

The primary objective is to assess the effect of NUZ-001 on ALS disease progression, with secondary endpoints evaluating additional efficacy measures and safety.

Built on Encouraging Phase 1 Data

Entry into the HEALEY ALS Platform Trial follows a completed Phase 1 clinical program.

In a small study population of 12 people living with ALS:

  • NUZ-001 showed encouraging preliminary signals of efficacy
  • The treatment was safe and well tolerated

These results supported NUZ-001’s selection into the highly competitive HEALEY platform, where candidates are reviewed by expert committees based on scientific merit and potential benefit.

A Registrational Pathway for NUZ-001

According to Neurizon, Regimen I represents its registrational study in ALS. The adaptive Phase 2/3 design is intended to:

  • Generate robust clinical evidence
  • Support potential future regulatory submissions
  • Enable ongoing engagement with the U.S. Food and Drug Administration during development

Enrolment in the study is expected to be completed in H2 CY2026.

Leadership and Investigator Perspective

Investigators leading the platform highlighted that beginning enrolment is a significant step made possible by people living with ALS, their families, and dedicated trial sites.

Neurizon’s leadership described first dosing as a defining milestone, emphasizing the urgency of progress in ALS and the company’s commitment to scientific rigour, operational focus, and respect for the ALS community.

About Neurizon Therapeutics

Neurizon Therapeutics is a clinical-stage biotechnology company focused on developing treatments for neurodegenerative diseases.

  • Lead program: NUZ-001 for ALS
  • Strategy: Accelerate access to effective ALS therapies
  • Exploring broader applications in neurodegeneration

NUZ-001 is an investigational product and is not approved for commercial use in any jurisdiction.

Why This Matters?

ALS remains a devastating disease with limited treatment options. Dosing the first patient in Regimen I marks an important step forward—not just for Neurizon, but for the broader effort to deliver faster, more efficient clinical development in ALS through platform trials.

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