The U.S. FDA has lifted the clinical hold on Neurizon Therapeutics’ lead investigational ALS drug, NUZ-001, clearing the way for phase 2/3 development.
NUZ-001 can now be part of the HEALEY ALS Platform Trial, expected to commence Q4 of this year.
“We are now partnering with the HEALEY ALS Platform Trial team to take the next critical steps in advancing NUZ-001. Supported by our compelling TDP-43 preclinical data and encouraging survival outcomes from the Open-Label Extension (OLE) study in ALS patients, we believe NUZ-001 has the potential to meaningfully slow the progression of this devastating disease,” said Michael Thurn, Neurizon CEO.
The FDA placed NUZ-001 on a clinical hold in January 2025, requesting more information about the dosing regime and trial risks. Then, in August, the agency — hit with restructuring and staffing reductions — delayed the review period on Neurizon’s response to the hold.
The active ingredient in NUZ-001 is monepantel, an oral deworming agent commonly used in veterinary medicine. Monepantel inhibits the mTOR signaling pathway that normally regulates autophagy, a cellular process through which unneeded or damaged components are broken down and recycled. By boosting autophagy, NUZ-001 may help clear the toxic proteins that are believed to contribute to ALS progression, according to Neurizon