Neurizon’s $16.6M Loss Raises Questions as FDA Review Delays ALS Trial Start

Financial Overview

Neurizon Therapeutics Limited, a clinical-stage biopharmaceutical company focused on neurodegenerative diseases, reported a net loss of $16.6 million for the year ended 30 June 2025. This represents a significant increase from the $7.7 million loss recorded in the previous year. The company’s net assets declined to $2.8 million from $10.2 million, reflecting ongoing investment in research and development activities.

Clinical and Regulatory Progress

Despite the widening loss, Neurizon made notable strides with its lead drug candidate, NUZ-001, an orally available small molecule targeting key pathological drivers of neurodegenerative diseases such as Amyotrophic Lateral Sclerosis (ALS) and Motor Neurone Disease (MND). The company successfully completed Phase 1 MEND and Open-Label Extension studies, which demonstrated encouraging safety, tolerability, and signals of survival benefit.

NUZ-001 received Orphan Drug Designation from both the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA), providing regulatory incentives and market exclusivity. The company is preparing to commence participation in the HEALEY ALS Platform Trial, a large-scale, multi-site clinical study in the US, pending FDA clearance to lift a clinical hold placed in early 2025. Neurizon submitted a comprehensive Clinical Hold Complete Response in July 2025, with the FDA expected to complete its review by October 2025.

Strategic Partnerships and Intellectual Property

In a key commercial development, Neurizon signed an exclusive global licensing agreement with Elanco Animal Health for Monepantel, the active pharmaceutical ingredient in NUZ-001. This deal grants Neurizon access to Elanco’s extensive non-clinical and manufacturing data, supporting regulatory submissions and reducing development costs. The agreement also secures a scalable supply of the drug to support clinical and commercial activities.

The company strengthened its intellectual property portfolio with the grant of a US 'method of use' patent for NUZ-001, extending protection until 2041. This patent covers the treatment of ALS and other neurodegenerative diseases, reinforcing Neurizon’s competitive position.

Operational and Financial Management

Neurizon continued to enhance its operational capabilities with strategic appointments, including a new Chief Financial Officer, and expanded its team to support global clinical execution and regulatory engagement. The company raised $7.8 million through a Share Purchase Plan in July 2024 and secured a $1.5 million non-dilutive loan from Radium Capital, backed by its expected R&D tax rebate, to maintain financial momentum without diluting shareholders.

Additionally, Neurizon developed a patient-friendly oral liquid formulation of NUZ-001 to improve treatment accessibility for ALS patients with swallowing difficulties, aligning with its patient-centric approach.

Looking Ahead

With a clear clinical pathway and strengthened regulatory foundation, Neurizon is focused on lifting the FDA clinical hold and initiating the HEALEY ALS Platform Trial in the final quarter of 2025. The company also aims to explore NUZ-001’s potential as a platform therapy for a broader range of neurodegenerative diseases, leveraging emerging preclinical data.

While the extended FDA review timeline introduces some uncertainty, Neurizon’s proactive engagement with regulators and stakeholders underscores its commitment to advancing a much-needed disease-modifying therapy for ALS and related conditions.