Neurotech scores US FDA rare disease win for NTI164 in Rett syndrome

• US FDA grants Rare Paediatric Disease Designation for Neurotech’s NTI164 for treatment of Rett syndrome
• Designation helps facilitate innovative treatments for rare paediatric diseases with enhanced regulatory support and potential priority review
• Complements orphan drug designation previously granted by the US FDA

Special Report: Neurotech International has passed an important regulatory milestone with the US Food and Drug Administration (FDA) granting Rare Paediatric Disease Designation (RPDD) for its lead drug candidate NTI164 for the treatment of Rett syndrome.

The RPDD complements the orphan drug designation (ODD) previously granted by the FDA for NTI164 in Rett syndrome, further strengthening Neurotech International’s (ASX:NTI)  position in developing novel therapies for rare and underserved neurological disorders.

FDA’s RPDD program recognises promising therapies being developed for conditions affecting fewer than 200,000 people in the US.

The designation received was based on its finding that Rett syndrome is a serious or life-threatening disease and that Rett syndrome meets the definition of “rare disease or condition” under section 526 of the FD&C Act.

An RPDD helps facilitate the development of innovative treatments by providing regulatory support, including enhanced FDA guidance during the drug development process and potential priority review.

Combined with its existing ODD, the recognition may provide Neurotech with valuable financial incentives, including tax credits for clinical trials, fee exemptions and seven years of US market exclusivity if NTI164 is approved.

Rare paediatric disease affecting girls

Rett syndrome is a rare genetic neurological and developmental disorder and is almost exclusively the result of mutation(s) in the methyl CpG binding protein 2 (MECP2) gene located on the X chromosome, which is required for normal brain development and function.

Rett syndrome occurs almost exclusively in girls, with an incidence of one in 10,000 female live births.The prevalence is ~15,000 girls and women in the US and 350,000 globally.

Another ASX listed biotech Neuren Pharmaceuticals (ASX:NEU) became the first company in the world to develop a treatment for Rett Syndrome with trofinetide (marketed as DAYBUE) approved by the FDA in March 2023.

In the past five years Neuren’s stock has surged almost 1800%, and the company now commands a $2.68 billion market cap.

‘Reinforces the significance of our program’

NTI164 is a unique anti-inflammatory and neuroprotective compound derived from a proprietary Cannabis sativa strain with a distinct profile of cannabinoids.

It is currently being evaluated in multiple clinical programs targeting neurodevelopmental disorders.

Neurotech published results from its Phase I/II study in the Journal of Paediatrics and Child Health.

This report showed the therapy was well tolerated and offered signs of clinical improvement across neurological, behavioural and functional domains.

NTI recently announced it had started an Authorised Prescriber (AP) program NTI164 across a range of neurodevelopmental conditions for paediatric patients in Australia.

CEO and managing director Dr Anthony Filippis said the granting of a RPDD by the FDA marks another major milestone for NTI164 and underscores its therapeutic potential for Rett syndrome.

“Combined with the existing orphan drug designation, this recognition reinforces the significance of our program and our commitment to developing much-needed treatments for children and families affected by these challenging conditions.”

This article was developed in collaboration with Neurotech International, 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.