Neurotech Secures FDA Orphan Drug Status for Rett Syndrome Therapy NTI164

Clinical Milestones in Rett Syndrome

Neurotech International Limited (ASX: NTI) has marked a significant regulatory achievement with the US Food and Drug Administration granting orphan drug designation (ODD) for its lead compound NTI164 in treating Rett Syndrome. This progressive neurological disorder, predominantly affecting girls, currently lacks effective therapies despite a substantial market opportunity estimated at over US$2 billion annually. The ODD status not only underscores the promising safety and efficacy profile demonstrated in Neurotech’s Phase I/II clinical trial but also positions the company to benefit from incentives such as tax credits, fee waivers, and seven years of market exclusivity upon approval.

Associate Professor Carolyn Ellaway, principal investigator of the Rett Syndrome trial, recently presented compelling clinical data at the 9th World Rett Syndrome Congress, highlighting NTI164’s full-spectrum medicinal cannabis-derived formulation and its potential to address this orphan disease’s unmet needs.

PANDAS/PANS: Scientific Validation Amid Regulatory Setback

While Neurotech’s orphan drug designation request for NTI164 in Paediatric Autoimmune Neuropsychiatric Disorders Associated with Streptococcal Infections (PANDAS) and Paediatric Acute-Onset Neuropsychiatric Syndrome (PANS) was not granted by the FDA, the agency acknowledged the robustness of the company’s scientific rationale and clinical evidence. The FDA’s 12-month abeyance allows Neurotech to continue development without immediate regulatory hurdles.

Recent genomic analyses led by Professor Russell Dale’s group have reinforced NTI164’s ability to reverse immune dysregulation at both gene expression and protein levels in affected children. These findings, consistent with prior proteomic data, suggest a novel immunomodulatory mechanism that could redefine treatment paradigms for these complex neuropsychiatric conditions.

Sustained Progress in Autism Spectrum Disorder Trials

Neurotech continues to advance its clinical program in Autism Spectrum Disorder (ASD), with 54 patients receiving NTI164 in ongoing extension phases. Notably, 43 patients have surpassed 52 weeks of daily treatment, and some have been on therapy for over two years without any reported safety or toxicity issues. This long-term data strengthens the case for NTI164’s tolerability and potential clinical benefit.

Given the rising prevalence of ASD in Australia, now exceeding 250,000 individuals enrolled in the National Disability Insurance Scheme, Neurotech’s therapy could address a significant unmet need if approved by the Therapeutic Goods Administration (TGA). The company anticipates further regulatory engagement following completion of FDA IND-enabling studies.

Advancing Regulatory and Pre-Clinical Programs

Neurotech is actively progressing its pre-clinical pharmacology and toxicology studies in certified US laboratories, essential for FDA and TGA regulatory submissions. The recent Human Research Ethics Committee approval for a Phase I pharmacokinetic study in healthy adults marks a critical step toward Investigational New Drug (IND) application readiness.

The company expects to complete these IND-enabling studies by the end of Q1 2025, alongside anticipated orphan drug designation from the European Medicines Agency for Rett Syndrome.

Financial Position and Leadership Update

Neurotech closed the quarter with $5.9 million in cash and cash equivalents, supported by a forthcoming $2.4 million R&D tax incentive rebate. Operating expenses have moderated slightly, reflecting focused investment in clinical and regulatory activities.

In a strategic move to accelerate growth, Neurotech appointed Dr Anthony Filippis as Managing Director and CEO effective February 2025. With 25 years of biotech leadership and a track record of deal-making and partnerships, Dr Filippis is expected to drive the company’s next phase of development and commercialisation.