Oral therapy NUZ-001 cleared by FDA to join platform trial for ALS
Study will evaluate multiple experimental drugs simultaneously
- NUZ-001, an oral therapy for ALS, has been cleared by FDA to join the HEALEY ALS platform trial.
- The drug aims to slow ALS progression by boosting autophagy to clear toxic proteins.
- Prior studies suggest NUZ-001 slowed disease progression in ALS patients.
NUZ-001, an investigational oral therapy being developed by Neurizon Therapeutics for the treatment of amyotrophic lateral sclerosis (ALS), is officially the ninth compound to be included in the HEALEY ALS platform trial.
The announcement follows clearance by the U.S. Food and Drug Administration (FDA) of an amended trial protocol, allowing the study’s sponsor, the Sean M. Healey & AMG Center for ALS at Mass General Brigham, to proceed with the regimen for NUZ-001.
“We are pleased to welcome Neurizon’s NUZ-001 regimen into the HEALEY ALS Platform Trial,” Merit Cudkowicz, MD, principal investigator of HEALEY ALS and director of the Healey Center for ALS, said in a press release from Neurizon.
With the FDA’s decision, Neurizon and the HEALEY trial team can now proceed with the remaining startup steps, including ethical reviews by the Institutional Review Board, activation of clinical trial sites, and other preparatory activities. Enrollment is expected to begin in early 2026.
“We look forward to working closely with the HEALEY team as we move toward site initiations and study [startup], bringing us one step closer to addressing this significant unmet medical need,” said Michael Thurn, PhD, Neurizon’s CEO and managing director.
FDA lifted clinical hold on NUZ-001 in October
ALS is a progressive neurological disorder that damages motor neurons, the nerve cells that control voluntary movements, causing them to die.
The HEALEY ALS platform trial (NCT04297683) is designed to evaluate multiple experimental ALS therapies simultaneously using a shared study design and placebo group. This approach aims to reduce costs, speed clinical development, and expand patient access to investigational treatments.
NUZ-001 was selected in 2024 by a group of ALS experts and members of the Healey & AMG Center Science Advisory Committee for inclusion in the study. Following that selection, Neurizon submitted an application to the FDA seeking approval to begin testing the therapy within HEALEY’s Phase 2/3 framework.
Earlier this year, however, regulators placed the application on clinical hold, citing the need for additional preclinical studies. After the requested data were submitted and reviewed, the FDA recently lifted the hold, clearing NUZ-001 to move forward in the HEALEY trial.
“Receiving the FDA’s clearance is a major milestone for Neurizon and a critical step in advancing NUZ-001 as a potential new treatment option for people living with ALS,” Thurn said.
Thurn added that the HEALEY trial “represents the gold standard for efficient and collaborative clinical development in this field,” noting that the company is “proud to progress into the next phase of activation.”
NUZ-001, previously known as monepantel, is an oral small-molecule therapy used initially as a veterinary deworming agent. However, it has an off-target effect of inhibiting the mTOR signaling pathway that normally regulates autophagy, the process by which cells break down and recycle damaged or unneeded components. By inhibiting mTOR, NUZ-001 is believed to enhance autophagy and aid in clearing toxic proteins that are thought to contribute to ALS progression.
Receiving the FDA’s clearance is a major milestone for Neurizon and a critical step in advancing NUZ-001 as a potential new treatment option for people living with ALS.
Neurizon’s application was supported by results from the Phase 1 MEND trial (NCT04894240) and its open-label extension study (NCT06177431), both of which are now complete.
Data from these studies showed that daily treatment with NUZ-001 slowed disease progression and extended survival compared with an external control group of untreated participants from a large ALS database. The therapy was also linked to slower declines in lung function.
“The FDA’s clearance to proceed with the NUZ-001 regimen is an important next step,” Cudkowicz added. “We look forward to initiating clinical sites and beginning enrollment so we can evaluate the potential of NUZ-001 to benefit people with ALS.”
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