In big news week Neurizon secures key orphan status for motor neurone drug in Europe

European Medicines Agency grants orphan drug designation for Neurizon’s lead drug candidate NUZ-001 to treat Amyotrophic Lateral Sclerosis.

Neurizon receives orphan drug designation for NUZ-001 to treat ALS in Europe. Pic via Getty Images

• European Medicines Agency grants orphan medicinal product designation for Neurizon’s lead drug candidate NUZ-001 to treat ALS

• Neurizon also reports positive interim eight-month update on its ongoing 12-month OLE study of NUZ-001 in patients with ALS

• Company has filed an Investigational New Drug (IND) application to the US Food and Drug Administration (FDA) for NUZ-001

Special Report: ’Twas the week before Christmas and the good news was flowing for clinical stage biotech Neurizon Therapeutics (ASX:NUZ), which had several positive announcements.

In an early Christmas present the European Medicines Agency (EMA) has granted orphan medicinal product designation (OMPD) for Neurizon’s lead drug candidate NUZ-001 – for the treatment of amyotrophic lateral sclerosis (ALS), also known as motor neurone disease.

In November clinical-stage biotech company Neurizon Therapeutics (ASX: NUZ), which is advancing treatments for neurodegenerative diseases, announced that the EMA had a positive opinion of its OPMD application and had been awaiting approval.

An OMPD provides a robust framework of benefits, including reduced regulatory fees, free protocol assistance, and market exclusivity for 10 years in the EU upon product approval.

During this exclusivity period, similar medicinal products will not be eligible for marketing authorisation in the same indication, which Neurizon noted offers a substantial commercial advantage for NUZ-001.

Significant achievement for Neurizon

Neurizon recently announced positive results of preclinical studies of NUZ-001, and noted it was getting closer to understanding the exact mode of action for the drug, which has proven to reduce aggregation (or clumping) of the TAR DNA-binding protein 43 (TDP-43). The latter builds up in patients with ALS and contributes to disease progression.

Managing director and CEO Dr Michael Thurn said not only does the OPMD bolster the company’s position in Europe but also complements the orphan drug designation previously granted by the US Food and Drug Administration (FDA), providing global market exclusivity across key territories.

“Securing Orphan Medicinal Product Designation from the European Commission is a significant achievement for Neurizon,” he said.

“Together with the positive preclinical results we’ve recently announced demonstrating its ability to reduce the aggregation of TAR DNA-binding protein 43, this milestone affirms the potential of NUZ-001 to address the substantial unmet medical needs of ALS patients.

He said with the OMPD milestone Neurizon was well positioned to advance NUZ-001 through the phase II/III HEALEY ALS Platform Trial, starting patient enrolment in early H1 CY25.

“Our goal remains steadfast – to bring innovative treatments to patients battling ALS and create value for our shareholders.”

Neurizon is continuing strategic regulatory engagement with global agencies, including the EMA, Australia’s Therapeutic Goods Administration, and FDA.

Positive interim results and filing of IND with FDA

In other good news this week, NUZ also reported a positive interim eight-month update on its ongoing 12-month open-label extension (OLE) study of NUZ-001 in patients with ALS.

The latest results confirm NUZ-001’s long-term safety and tolerability, as well as its significant potential to extend life expectancy for patients with ALS, compared to untreated matched controls.

The company also announced this week it had filed an Investigational New Drug (IND) application to the US Food and Drug Administration (FDA) to initiate the HEALEY ALS Platform trial for NUZ-001.

An IND application provides the FDA with essential information to authorise the safe testing of an investigational drug in humans or an approved drug for a new use or patient population.

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.