NUZ-001 arm in HEALEY ALS trial expands to 240 participants
Neurizon says rapid enrollment may support earlier top-line results
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- The NUZ-001 arm of the HEALEY ALS trial is expanding enrollment to 240 participants.
- NUZ-001 is an experimental ALS therapy designed to target the mTOR pathway and reduce toxic protein buildup.
- Neurizon says the expansion may strengthen data analyses and support earlier top-line results.
An arm of the HEALEY ALS platform trial testing Neurizon Therapeutics’ experimental therapy NUZ-001 as a treatment for amyotrophic lateral sclerosis (ALS) is expanding enrollment from 160 to as many as 240 participants.
The decision follows enrollment that exceeded original expectations and the absence of another HEALEY regimen recruiting participants at the same time. According to Neurizon, the larger study population is expected to strengthen the analysis and may allow top-line results to be reported earlier than previously anticipated.
“The strength of recruitment and engagement across the HEALEY ALS Platform Trial network has positioned Neurizon to proactively optimise the study design at an important point in the trial,” Sergio Duchini, the interim executive chairman of Neurizon, said in a company press release.
Regimen I enrollment target grows
The Phase 2/3 study arm, called Regimen I (NCT07410806), recently became the ninth treatment regimen added to the HEALEY trial (NCT04297683), an ongoing study designed to evaluate multiple experimental ALS therapies under a shared trial protocol, with each regimen testing an active treatment against a matching placebo.
According to Neurizon, as of May 22, 113 participants had been assigned to Regimen I and 74 had been dosed. Sabrina Paganoni, MD, PhD, co-principal investigator of the trial, described the pace as “our most rapid enrollment to date.”
Like other HEALEY ALS trial arms, Regimen I was expected to enroll about 160 participants, with 120 assigned to receive the active treatment and 40 given a placebo. With target enrollment increasing to 240 participants, the planned allocation is 180 receiving NUZ-001 and 60 receiving a placebo.
“We believe the expanded dataset will strengthen the statistical robustness, interpretability and strategic value of the program as we advance toward topline results,” Duchini said.
ALS is a neurodegenerative disease with limited treatment options and no existing cure. NUZ-001 originated from a compound initially developed to treat parasitic worm infections in livestock. Researchers later identified its ability to inhibit signaling through the mTOR pathway.
The mTOR pathway regulates several cellular processes, including autophagy — the system cells use to break down and recycle unwanted components.
Researchers believe that impaired autophagy may contribute to the accumulation of toxic protein clumps in ALS, leading to nerve cell damage. By inhibiting mTOR signaling, NUZ-001 is designed to restore autophagy and reduce this buildup, potentially slowing disease progression.
Trial tests daily oral NUZ-001
In HEALEY’s Regimen I, participants will receive oral NUZ-001 or a placebo daily for 36 weeks, or about eight months. The trial’s main goal is to measure changes in disease severity, as assessed by scores on the ALS Functional Rating Scale-Revised (ALSFRS-R), a standard measure of physical function in ALS, and survival.
In addition to supporting the trial’s planned primary analysis, the expansion may enable more detailed evaluations of biological markers and treatment responses in specific patient subgroups, according to the company.
Based on current enrollment rates, Neurizon now anticipates dosing the last participant in the second quarter of 2027, with top-line data expected early in the third quarter of 2027.
“The expansion of Regimen I to 240 participants reflects our considerable confidence in NUZ-001 and is underpinned by strong momentum being delivered across the HEALEY ALS Platform Trial study,” Duchini said. “Importantly, this decision maintains the statistical power for the analysis of the primary endpoint and the potential for topline results ahead of our previously stated schedule.”
“The larger and more internally consistent dataset is expected to enhance future regulatory, partnering and commercial opportunities for NUZ-001, while also strengthening our ability to generate valuable biomarker and translational insights relevant to the broader neurodegenerative disease landscape,” Duchini said.
A contribution of philanthropic funds from the Sean M. Healey & AMG Center for ALS at Mass General Brigham is expected to materially offset the cost of the expanded cohort, according to Neurizon.
“This partnership with Neurizon reflects a shared commitment to advancing meaningful science while keeping people living with ALS at the center of everything we do,” said Merit Cudkowicz, MD, director of the Healey & AMG Center and sponsor of the HEALEY ALS trial. “By working collaboratively, we can accelerate progress, strengthen the research ecosystem, and ultimately deliver solutions that better serve the ALS community.”
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