Key Highlights

  • All 250 participants were enrolled in Regimen I across 78 US sites, with topline results now expected in late Q4 FY2027, or late in the second quarter of calendar 2027.
  • The FDA indicated positive HEALEY results could support a traditional approval pathway, subject to substantial evidence of effectiveness.
  • Closing cash was A$11.429 million, with total available funding of A$35.619 million, about 4.5 quarters at the current burn.

Enrolment complete

Neurizon Therapeutics Limited (ASX:NUZ) said in its September quarter report, released on 28 October 2026, that all 250 participants have been enrolled in Regimen I of the HEALEY ALS Platform Trial, across 78 activated sites in the United States. The company said the cohort was expanded from 160 participants and that enrolment took under five months from first dosing. Participants are randomised three to one to NUZ-001 or placebo in a double-blind design, with a 36-week randomised treatment phase followed by a 36-week active treatment extension. The primary endpoint is change in the ALSFRS-R functional scale and survival from baseline to week 36.

Neurizon said the expanded cohort preserves the original statistical power and does not change its funding needs. It now expects topline results in late Q4 of the 2027 financial year, which it equates to late in the second quarter of calendar 2027, earlier than previously guided. NUZ-001 is investigational and is not approved for commercial use in any jurisdiction.

Regulatory feedback

After the quarter ended, the company received Type C feedback from the US Food and Drug Administration. Neurizon said the agency indicated that positive HEALEY results could support a traditional approval pathway, with Regimen I potentially serving as the single registrational study, subject to meeting the standard of substantial evidence of effectiveness. The FDA also agreed with Neurizon's approach to measuring plasma neurofilament light chain and described the evidence needed for it to support accelerated approval. The company may resubmit a request for Fast Track Designation, with timing under evaluation. On Europe, the company said scientific advice indicated an additional Phase 3 study would ordinarily be required, and it will reassess its European strategy after topline results.

Expanded access program and leadership

Neurizon said an expanded access program for NUZ-001 is fully funded by a multi-year grant from the US National Institutes of Health, so it is non-dilutive to shareholders. It will cover up to 200 US people with ALS who are ineligible for trials, for up to 96 weeks, led by the Sean M. Healey and AMG Center for ALS at Mass General Brigham. Enrolment is targeted for the March quarter of 2027, and continuation beyond the topline results depends on a positive outcome.

Dr Chris Bremer started as chief executive on 1 October 2026, and Sergio Duchini moved from interim executive chairman to non-executive chairman on the same date.

Cash and funding

The Appendix 4C shows net operating cash outflow of A$7.899 million for the quarter, including A$6.009 million of research and development and A$1.044 million of staff costs. Financing inflow was A$6.681 million, mainly A$8.31 million drawn from a research and development tax incentive loan facility with Dare Capital, less A$1.614 million of repayments. Closing cash was A$11.429 million, down from A$12.692 million.

The Dare facility is for up to A$17.5 million, secured against the tax incentive refund, with interest of 1.25 per cent a month capitalising, and all amounts due by 31 December 2026 subject to extension terms. Neurizon also has a convertible note facility with Obsidian of up to A$20 million over two years. The company reported total available funding of A$35.619 million, about 4.5 quarters at the current burn, and said it is fully funded through the HEALEY topline readout and study completion.

Milestones and caveats

Neurizon listed the tax incentive refund and preclinical mechanism-of-action updates for the December quarter, expanded access enrolment in the March quarter, HEALEY completion and database lock in the June quarter, and regulatory engagement after topline results. The filing contains some internal inconsistencies, including differing dates for an Obsidian note redemption and an apparent error in the net cash movement line. No efficacy data from the trial are available yet.