Neurizon Therapeutics Limited has announced that the US Food and Drug Administration (FDA) has lifted the clinical hold on its lead investigational drug, NUZ‑001.
This decision marks a pivotal regulatory milestone for Neurizon and the ALS community, clearing the way for Phase II/III development of NUZ-001 as part of the HEALEY ALS Platform Trial.
Dr Michael Thurn, Managing Director and CEO, commented: "The FDA's clearance of our 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 programme."
"Their efforts in raising awareness and lobbying for the urgent needs of the ALS community have been invaluable in helping us reach this point."
"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 study in ALS patients, we believe NUZ-001 has the potential to meaningfully slow the progression of this devastating disease."
Opening an IND for a platform molecule establishes a regulatory foundation that not only accelerates the development of the first candidate but also streamlines future programmes.
By creating a validated framework for safety, manufacturing and clinical design, it reduces regulatory risk, shortens timelines and enables efficient expansion into new indications.
In this case, the IND is further strengthened by the robust and comprehensive package of preclinical safety data and the detailed manufacturing and quality information secured through our licensing agreement with Elanco.
These resources enhance confidence in the platform's readiness for clinical development and reinforce its potential as a therapeutic platform with broad applicability, offering both strategic flexibility and long-term commercial value.
With IND now active, Neurizon anticipates Mass General Hospital (MGH) filing a protocol amendment to their IND for the HEALEY ALS Platform Trial to incorporate the specific protocol regimen early in the coming weeks.
Neurizon expects to initiate patient enrollment in the HEALEY ALS Platform Trial in Q4 CY2025.