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Neurizon Therapeutics Develops Oral Liquid Formulation of NUZ-001 for ALS Patients with Swallowing Difficulties

2 months ago2 min read

Key Insights

  • Neurizon Therapeutics has developed a new oral liquid formulation of NUZ-001, its lead investigational therapy for amyotrophic lateral sclerosis treatment.

  • The liquid formulation was created in direct response to patient and caregiver feedback to address swallowing difficulties, particularly for those with bulbar onset ALS.

  • The new formulation will be evaluated for bioequivalence and patient acceptability alongside the standard tablet form, with human bioequivalence studies scheduled to begin in the first half of 2026.

Neurizon Therapeutics (ASX: NUZ), a clinical-stage biotech company based in Australia, has announced the development of a new oral liquid formulation of NUZ-001, its lead investigational therapy for the treatment of amyotrophic lateral sclerosis (ALS). The development addresses a critical unmet need for ALS patients who experience progressive swallowing difficulties as their disease advances.

Patient-Centered Development Approach

The oral liquid formulation was developed in direct response to feedback from patients and carers, reflecting Neurizon's commitment to ensuring that the patient voice guides both trial design and treatment delivery. For many patients, especially those experiencing bulbar onset or progressive difficulty swallowing, tablet-based medication can become increasingly hard to manage as the disease advances.
The liquid formulation is designed to support patients with all stages of the disease, particularly those with swallowing difficulties such as bulbar onset. This patient-centered approach ensures more patients can continue to benefit from NUZ-001 throughout their disease journey.

Key Clinical Advantages

The new formulation offers several important clinical benefits for ALS patients and their caregivers:
  • Improved ease of swallowing for patients with dysphagia or speech impairment
  • Flexible dosing across a range of patient weights and tolerances
  • Enteral administration through feeding tubes, ensuring continuity of treatment
  • Simplified administration for caregivers and clinical teams

Life Cycle Management Strategy

The development of the liquid formulation marks the launch of a life cycle management (LCM) strategy for NUZ-001. This approach transforms the drug from a single-asset product into a long-term, scalable treatment platform, enhancing both patient value and commercial success. By strategically evolving the product, Neurizon aims to maximize patient impact, support long-term commercial growth, and drive innovation well beyond the original patent expiry.

Clinical Development Timeline

The liquid formulation is currently being integrated into Neurizon's ongoing clinical development program for NUZ-001. The new formulation will be evaluated for bioequivalence and patient acceptability alongside the standard tablet form. The human bioequivalence study is scheduled to commence in the first half of 2026.
Following the announcement, Neurizon's shares fell 3% on market close, according to the Australian bourse filing made on Friday.
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