Neurotech focuses on global partnerships and Aussie registration

Neurotech to secure one or more strategic partnerships for NTI164 in the US, Europe and Asia
Partnerships aim to support clinical, regulatory development, manufacturing and future market launches of NTI164
Neurotech to pursue multiple registrations of NTI164 in Australia, including leveraging provisional registration pathway via the TGA

Special report: Neurotech (ASX:NTI) has unveiled its corporate strategy, focused on securing global partnering initiatives for NTI164 along with securing multiple registrations in Australia for the broad-spectrum cannabinoid drug.

Neurotech (ASX:NTI) says it’s working on securing one or more strategic partnerships for NTI164 in the US, Europe and certain Asian territories to support clinical, regulatory development, manufacturing and future market launches as part of its new corporate strategy.

The clinical-stage biotech, focused predominantly on rare paediatric neurological disorders, says such partners will have the necessary financial resources and experience in late-stage drug development, clinical trials, and commercialisation.

NTI says prospective partners will be responsible for all costs of development and commercialisation of NTI164 outside of Australia.

The company says partnerships may take the form of licensing transactions, equity-based investment(s), or M&A.

NTI is operating in an exciting area of development for pharma companies seeking exposure to rare neurological diseases.

The company says a partnership will minimise the financial, clinical, and regulatory risks for NTI shareholders in these markets.

Large opportunity for NTI164

NTI believes there is a large market opportunity for NTI164 for all three neurological disorders it is targeting, including PANDAS/PANS, Rett Syndrome and autism spectrum disorder (ASD).

Paediatric Autoimmune Neuropsychiatric Disorders Associated with Streptococcal Infections (PANDAS) and Paediatric Acute-Onset Neuropsychiatric Syndrome (PANS) is characterised by an infection-triggered autoimmune response and neuro-inflammation, leading to sudden and drastic changes in behaviour and personality.

Currently, there are no approved therapies for PANDAS/PANS globally. NTI estimates the annual market for PANDAS/PANS to be valued at US$1.2bn.

In June NTI released an update on its ongoing open-label Phase 1/2 trial of NTI164 to treat PANDAS/PANS, which showed continued improvement after 52 weeks of treatment.

In July the company released positive secondary endpoint data analysis from its Phase 2/3 trial of NTI164 in autism patients, along with positive results from the extension phase of its Phase 1/2 trial of NTI164 to treat Rett Syndrome.

Rett Syndrome is a rare genetic neurological and developmental disorder, which occurs almost exclusively in girls, with an incidence of one in 10,000 female live births with the prevalence of ~15,000 girls and women in the US and 350,000 globally.

NTI says safety and efficacy to 20 weeks are trending favourably when compared to the current FDA-approved treatment, DAYBUE – developed by $2bn capped Neuren Pharmaceuticals (ASX:NEU).

NTI estimates the annual market opportunity for NTI164 to treat Rett Syndrome at ~US$2bn.

NTI164 is also proving to be an attractive long-term therapy for ASD, the company said, a condition which is rising in prevalence.

The incidence of ASD is estimated at 1 in 50 across the population in Australia, which represents a 40-fold increase in the last 20 years.

Strong asset moat

Since mid-2022, NTI has focused on building a strong asset moat around NTI164 by generating clinical evidence of its safety and efficacy and securing PCT patent applications.

The company says the patents, if granted, will provide protection until at least 2041. NTI has also developed the ability to manufacture NTI164 year-round in a controlled semi-outdoor environment, ensuring continuous supply for clinical and future commercial needs.

The company has established stable plant genetics and a robust standard operating protocol, which it says is fully transferable to new manufacturing partners.

NTI is in the process of creating a fully integrated, GMP-compliant process for NTI164 and has developed proprietary methods for extraction, fractionation, and analytical control.

Recently, NTI submitted its first Orphan Drug Designation (ODD) request to the US FDA for PANDAS/PANS, with further ODD filings planned in the US and Europe for Rett Syndrome and PANDAS/PANS.

If granted, ODD will provide seven years of market exclusivity in the US and 10 years in Europe.

NTI says it aims to complete the necessary US FDA investigational new drug application enabling studies to help define the pharmacological and toxicological properties of NTI164 in significant detail by the end of Q1 CY25.

Subject to completion of the IND-enabling studies, NTI aims to file for and secure an FDA IND, as well as EMA approval in H1 CY25 for at least one paediatric clinical trial.

NTI says it aims to have partnered the NTI164 program before the start of a Phase 3 clinical trial in the US/Europe.

Fast-tracking commercialisation in Australia

After a pre-submission meeting with the TGA, NTI has been exploring ways to fast-track NTI164’s commercialisation in Australia.

Pending successful pre-clinical toxicology and PK studies, the company plans to pursue provisional registration for NTI164, focusing on PANDAS/PANS or Rett Syndrome.

The company says these disorders support an accelerated approach, leveraging the strong clinical evidence already gathered.

Provisional registration could potentially reduce development time by up to two years and may allow for reimbursement through the Pharmaceutical Benefits Scheme.

NTI aims to formalise a pre-submission meeting with the TGA in Q2 CY25, with the goal of filing for provisional registration in H2 CY25.

The company has also analysed the significant market opportunity in Australia for all three indications, particularly for autism patients where a significant amount of money is spent each year under the NDIS.

Although the NDIS does not fund or subsidise medicines, NTI says in the 12 months to March 2024, the company believes the NDIS has paid $5.9bn to Level 2/Level 3 ASD children.

The company says this reflects the necessary cost of support services for ASD children to be more independent and engage socially and economically.

NTI believes NTI164 has, via its multiple clinical trials, been shown to improve ASD children’s lives in a similar manner.

Leverage ‘robust clinical data’

Executive director Dr Thomas Duthy says NTI’s latest corporate strategy reflects its core focus on generating conclusive clinical evidence to the efficacy and safety of NTI164 for paediatric neurological patients with persistent or progressive neuroinflammation, where standard therapies are urgently needed or lacking altogether.

“We believe Neurotech is now in a strong position to leverage our robust clinical data, accompanying intellectual property, manufacturing expertise, and ongoing regulatory initiatives to deliver significant value to our shareholders moving forward,” he said.

“We believe now is the right time to pursue an active strategy for our unique paediatric neurological franchise, which consists of two rare (orphan) diseases with limited or no treatment options, and autism, where many patients face restrictive approved therapies that have negative side effects and are therefore not generally considered effective front-line treatments.”

Duthy says that during 2023, at least 49 deals were announced involving rare neurological diseases, with disclosed values totalling US$13.2bn.

“As a board, we are fully committed to this strategy and are positioning the business to achieve these stated goals as quickly as possible and within our balance sheet capability, with $11.6 million in cash as of 30 June 2024,” he says.

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.