FDA Lifts Clinical Hold on Neurizon Therapeutics Lead Investigational Drug NUZ-001

FDA’s lift clears a path for NUZ-001 to enter into Phase II and Phase III development as part of the Healey ALS Platform Trial.
Stock.Adobe.com

Neurizon Therapeutics announced FDA lifted its clinical hold on NUZ-001, Neurizon’s leading investigational therapy treatment for ALS. The lift marks a regulatory milestone and will allow for NUZ-001 to enter Phase II and III development as part of the Healey ALS Platform Trial, which is expected to commence later this year in Q4.¹

“The FDA’s clearance of our investigational new drug (IND) application marks a significant milestone in our mission to bring NUZ-001 closer to patients living with ALS. This achievement reflects both our unwavering commitment to rigorous scientific and clinical development and the tireless dedication of our team. We are deeply grateful to the key opinion leaders (KOLs) and patient advocacy groups whose steadfast support and engagement were instrumental in shaping and advancing this program. Their efforts in raising awareness and lobbying for the urgent needs of the ALS community have been invaluable in helping us reach this point,” Dr Michael Thurn, managing director and CEO, said. “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.”

With NUZ-001’s IND now active, Neurizon is anticipating Mass General Hospital (MGH) to file a protocol amendment for NUZ-001’s IND for the HEALEY ALS Platform Trial and incorporate its specific protocol regimen early in the coming weeks.¹ Additionally, Neurizon is expected to begin patient enrollment for the HEALEY ALS Platform Trial and continue to advance Neurizon’s mission aiming to accelerate patient access to innovative therapies and establishing NUZ-001 as a potential effective treatment for ALS.¹

Back in July of this year, Neurizon and Elanco entered into an exclusive global licensing agreement for NUZ-001, aiming to strengthen the treatments development, manufacturing, and future commercialization.² The agreement also provides Neurizon support in the regulatory foundations, including access to critical animal safety data and manufacturing data, key pillars required to support future clinical trials, potential regulatory approvals, and global market entry.² Now that FDA has lifted the clinical hold on NUZ-001, the IND for a platform molecule provides support while also creating a regulatory foundation that accelerates the development of its first candidate. It also establishes a streamlined approach for future programs. Through its validated framework for safety, manufacturing, and clinical design, it reduces regulatory risk, shortens timelines, and enables efficient expansion into new indications.¹

NUZ-001’s IND includes preclinical safety data and the detailed manufacturing and quality information, which was acquired through its licensing agreement with Elanco. Neurizon employes these resources to enhance confidence in the platform’s readiness for clinical development, and to reinforce its potential as a therapeutic platform offering both strategic flexibility and long-term commercial value.¹