Neurizon’s NUZ-001 trial reveals heightened survival rate for patients with ALS

Neurizon’s NUZ-001 treatment has demonstrated increased survival in patients with ALS, reducing risk of death by 78.5pc.

Neurizon’s NUZ-001 treatment has offered ALS patients some relief, decreasing risk of death by 78.5pc. Pic: Getty Images

Special Report: The NUZ-001 open label extension has completed its 12-month treatment period, demonstrating both good tolerability and promising indications of extended life expectancy in patients with motor neurone disease/amyotrophic lateral sclerosis.

• NUZ-001 increased survival rates compared to untreated matched controls
• Treatment significantly reduced risk of death by 78.5pc
• Patients now in 31st month of continuous treatment

Of the 12 patients originally enrolled in Neurizon Therapeutics’ (ASX:NUZ) Phase 1 MEND study, seven remain alive, with six patients continuing to receive treatment with NUZ-001 under a compassionate use program.

The study, now complete after the last open label extension patient completed the 12-month treatment period, reported no adverse effects related to treatment.

Compared to untreated matched controls from the PRO-ACT Historical Database, patients treated with NUZ-001 significantly increased their survival rates and reduced risk of death by 78.5%.

NUZ’s analysis of its study results indicate treatment with NUZ-001 may extend median survival by about 11 months, with a possibility for an extended benefit.

This result is particularly notable, as current treatments typically extend life expectancy by just three to six months.

Aiming to make ‘a meaningful difference’ 

Neurizon managing director and CEO Dr Michael Thurn said reaching this milestone in the open label extension study was a significant moment for both the company and the MND/ALS community.

“We are encouraged that some patients have now received NUZ-001 for 2.5 years and have chosen to continue treatment under a compassionate use program,” Dr Thurn said.

“The latest survival data reinforce our confidence in the clinical development strategy for NUZ-001 and further validate dose selection for the upcoming HEALEY ALS Platform Trial. 

“As we look ahead, our focus remains on advancing NUZ-001 as a potential treatment that could make a meaningful difference for people living with MND/ALS.”

Extension trial investigated long-term outcomes

Neurizon’s open label extension study was designed to investigate the long-term safety, tolerability and efficacy of NUZ-001 in patients with ALS who completed the Phase 1 MEND Study, which began in October 2022. 

Eligible patients received a daily dose of 10mg per kilogram of body weight of NUZ-001 for 12 months.

The study was conducted at two trial sites – the Calvary Health Care Bethlehem, led by Associate Professor Susan Mathers, and Macquarie University, led by Professor Dominic Rowe

“We are deeply grateful to all of our participants and their families for collaborating with us on the OLE study to explore the long-term effects and potential benefits of NUZ-001 in MND/ALS,” Associate Professor Mathers said.

“This study has demonstrated that NUZ-001 continues to be safe and well tolerated. 

“Based on the promising results previously observed in preliminary efficacy markers, we look forward to the commencement of the next study phase.”

What’s next?

Neurizon anticipates the FDA will lift the clinical hold on NUZ-001 in the third quarter of the 2025 calendar year.

The company intends to begin the HEALEY ALS Platform Trial in the fourth quarter, which NUZ believes will be a pivotal Phase 2/3 study that could deliver meaningful results for the MND/ALS community.

This article was developed in collaboration with Neurizon Therapeutics, a Stockhead advertiser at the time of publishing. 

This article does not constitute financial product advice. You should consider obtaining independent advice before making any financial decisions.

Originally published as Neurizon’s NUZ-001 trial reveals heightened survival rate for patients with ALS