Neuren Pharmaceuticals kicks off Phase 3 trial in rare syndrome

The Neuren Pharmaceuticals Ltd (ASX: NEU) share price is in focus today after the company announced its Koala Phase 3 clinical trial of NNZ-2591 for Phelan-McDermid syndrome (PMS) has dosed its first patient, marking a key milestone as the first Phase 3 trial ever in PMS.

What did Neuren Pharmaceuticals report?

• First patient has commenced dosing in the Koala Phase 3 trial of NNZ-2591 for PMS
• Trial will enrol around 160 children aged 3–12 years with PMS
• 25 families referred so far; demand strong with 37 more families on waitlists
• US FDA Fast Track, Rare Pediatric Disease, and Orphan Drug designations for NNZ-2591
• Continues as Presenting Sponsor for the 2026 PMSF Family Conference in Colorado

What else do investors need to know?

The Koala trial is a randomised, double-blind, placebo-controlled study, testing the safety and effectiveness of NNZ-2591. Right now, only two US sites are active, but over 20 more sites are expected to launch through the first half of 2026, supporting broader trial access for families.

Phelan-McDermid syndrome currently has no approved treatment options. The high level of interest from families and clinicians supports strong trial recruitment. Neuren’s commitment includes ongoing support of awareness through community initiatives like the upcoming PMS Foundation conference.

What did Neuren Pharmaceuticals management say?

Neuren CEO Jon Pilcher said:

We are excited to have started the treatment phase of our Koala Phase 3 study and are very encouraged by the level of interest in the PMS community. We are proud to be the presenting sponsor of the PMSF Family Conference in July and we anticipate strong momentum for Koala as trial sites around the US progressively activate during the first half of this year.

What’s next for Neuren Pharmaceuticals?

Looking ahead, Neuren plans to ramp up trial site activation, aiming for 20+ locations across the US by mid-year. This should pave the way for faster patient recruitment and strengthen Neuren’s position in rare paediatric neurological disorders.

Neuren’s strategy remains focused on addressing urgent unmet medical needs in childhood neurological conditions, with NNZ-2591 also being developed for other neurodevelopmental disorders besides PMS.

Neuren Pharmaceuticals share price snapshot

Over the past 12 month, Neuren Pharmaceuticals shares have declined 5%, trailing the S&P/ASX 200 Index (ASX: XJO) which has risen 4% over the same period.

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The post Neuren Pharmaceuticals kicks off Phase 3 trial in rare syndrome appeared first on The Motley Fool Australia.

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