ADNP
Syndrome.
Phase 3

Clinical Trials Overview: Pioneering Phase 3 Study for ADNP Syndrome

Introduction

Exonavis Therapeutics is at the forefront of innovative treatments for rare genetic disorders, and we are proud to announce the commencement of a groundbreaking Phase 3 clinical trial targeting ADNP Syndrome. This disorder, characterized by significant neurodevelopmental delays, presents a profound challenge to the affected children and their families. Our trial is set to explore the therapeutic potential of NAP/Davunetide, a novel peptide discovered by the esteemed Professor Illana Gozes.

Trial Overview

Study Focus and Design
This Phase 3 study is designed to evaluate the efficacy and safety of NAP/Davunetide in a population of 97 children diagnosed with ADNP Syndrome. Spanning across five countries, the trial will commence in October and is scheduled to run for one year. To ensure rigorous monitoring and transparency, interim results will be shared after six months, providing valuable insights into the drug’s performance and safety profile.

Primary and Secondary Objectives
The trial’s primary objective is to assess the ability of NAP/Davunetide to improve cognitive function, social behavior, and developmental milestones in children with ADNP Syndrome. Secondary objectives include detailed evaluations of safety, tolerability, and broader impacts on quality of life. The study will also explore potential biomarkers that could help predict and monitor treatment responses.

Significance of NAP/Davunetide in ADNP Syndrome

Mechanism of Action
NAP/Davunetide has shown promise in stabilizing microtubules, essential components of cellular structure and function, particularly within the central nervous system. This stabilization is crucial for maintaining the integrity of neurons, which are often compromised in ADNP Syndrome. By targeting these fundamental cellular processes, NAP/Davunetide holds the potential to alter the disease’s progression, offering hope for a condition currently lacking effective treatments.

Why This Trial Matters
ADNP Syndrome is a rare and debilitating condition with no approved treatments, making this trial not just a research milestone but a critical step toward offering relief and hope to affected families. Success in this trial could lead to the first-ever approved therapy specifically for ADNP Syndrome, setting a new standard in the treatment of neurodevelopmental disorders. Moreover, it would solidify Exonavis Therapeutics’ role as a leader in rare disease research and therapeutic innovation.

Clinical Trial Methodology

Patient Enrollment and Geographic Scope
The trial will enroll 97 children across five countries, ensuring a diverse and representative sample of the global ADNP patient population. This international scope will help validate the efficacy of NAP/Davunetide across different genetic backgrounds and healthcare environments, providing robust data for regulatory submissions worldwide.

Looking Ahead: The Future of ADNP Syndrome Treatment

This Phase 3 trial marks a pivotal moment in the journey toward a treatment for ADNP Syndrome. Should NAP/Davunetide prove successful, it will not only provide a much-needed therapeutic option for patients but also pave the way for further research into related neurodevelopmental and neurodegenerative conditions. At Exonavis Therapeutics, our mission is to transform the lives of those affected by rare genetic disorders, and this trial is a significant step toward that goal.