Background on Neuren Pharmaceuticals and NNZ 2591

Quick Overview

Neuren Pharmaceuticals is an Australian company developing two drug candidates for neurodevelopmental disorders. Their lead program is in Phase 3 for Rett syndrome, and they are also moving into trials for Angelman, Phelan-McDermid syndrome, and Pitt-Hopkins syndrome. The drugs being developed are synthetic analogs of IGF-1 metabolites that improve the connections and signaling of neurons and dendrites. The company has orphan designation for several syndromes and is targeting synaptopathies, which are disorders characterized by deficits in synaptic plasticity. The drug NNZ-2591 has shown promising results in mouse models and is currently in Phase 1 trials. Neuren Pharmaceuticals plans to bring the drug into Phase 2 trials in pediatric patients next year.

Introduction

In this talk, Larry Glass, the Chief Science Officer of Neuren Pharmaceuticals, provides an overview of the company and its drug candidates. He discusses the science behind their programs and provides a status update on their progress.

Neuren Pharmaceuticals

Neuren Pharmaceuticals is an Australian company that focuses on developing drugs for neurodevelopmental disorders. They have two drug candidates in clinical development for five different disorders. Their lead program is in Phase 3 for Rett syndrome, and they are also moving into trials for Angelman, Phelan-McDermid syndrome, and Pitt-Hopkins syndrome.

Drug Candidates

The drug candidates being developed by Neuren Pharmaceuticals are synthetic analogs of IGF-1 metabolites that naturally occur in the brain. These analogs replicate the activity of the natural molecules but are more bioavailable and stable, making them more effective in crossing the blood-brain barrier.

Mechanism of Action

The aim of these drug candidates is to improve the connections, signaling, and structure of neurons and dendrites. They target synaptopathies, which are disorders characterized by deficits in synaptic plasticity. These disorders result in various symptoms, including behavioral, cognitive, motor, and autonomic symptoms.

NNZ-2591

NNZ-2591 is a synthetic analog of cyclic GP, which is a metabolite of IGF-1. It works by suppressing the expression of inflammatory cytokines, addressing the problem of pathologically activated microglia, and restoring the balance between synaptic protein synthesis, maintenance, and repair. In mouse models of Angelman, Pitt-Hopkins, and Phelan-McDermid syndrome, NNZ-2591 has shown promising results in fully rescuing the phenotype with six weeks of treatment.

Progress and Future Plans

Neuren Pharmaceuticals has completed safety pharmacology and toxicology studies for NNZ-2591 and is confident in its safety and toxicity profile. They have initiated a manufacturing campaign for Phase 2 and have also started a Phase 1 trial in healthy volunteers. They plan to bring together the data from these trials and their manufacturing and clinical plans to submit to the FDA next year. If all goes well, they aim to begin Phase 2 trials in pediatric patients in the same year.

Conclusion

Neuren Pharmaceuticals is making significant progress in the development of their drug candidates for neurodevelopmental disorders. With their focus on improving synaptic function and restoring balance in the brain, they aim to provide effective treatments for these challenging conditions.