Neurotech forms expert panel after TGA meeting

Neurotech International to establish expert PANDAS/PANS Advisory Group
Advisory group follows productive pre-submission meeting with TGA
NTI’s lead drug NTI164 is showing promise in treatment of PANDAS/PANS

Special Report: Neurotech International will establish an internationally recognised expert advisory group to provide strategic advice on children diagnosed with Paediatric Autoimmune Neuropsychiatric Disorders Associated with Streptococcal Infections (PANDAS) and Paediatric Acute Onset Neuropsychiatric Syndrome (PANS).

Neurotech (ASX:NTI) says establishment of the advisory group follows a productive pre-submission meeting with the Therapeutic Goods Administration.

At the meeting the clinical-stage biotech received non-binding guidance on its planned clinical and regulatory development for its lead drug NTI164 in PANDAS/PANS.

PANDAS/PANS is a rare neurological disorder predominantly in children characterised by an infection triggered autoimmune response and associated neuroinflammation.

The condition results in a sudden, dramatic change in personality, displayed as obsessive-compulsive disorder, anxiety, tics or other abnormal movements and personality changes.

There are no approved therapies for PANDAS/PANS globally with clinical-stage biotech NTI estimating the annual market for PANDAS/PANS is worth US$1.2bn ($1.8bn).

Strong results for NTI164 in PANDAS/PANS

NTI has reported strong initial Phase 1/2 clinical trial results at 12 weeks (primary endpoint) along with 24 and 52-week data in 15 paediatric patients in use of its lead drug NTI164 to treat PANDAS/PANS.

The company recently reported improvements in PANDAS/PANS patients after 52 weeks of NTI164 treatment, highlighting the safety profile of NTI164 and its potential as a therapy for the disease.

NTI164 is a proprietary drug formulation derived from a unique cannabis strain with low THC (M<0.3%) and a novel combination of cannabinoids including CBDA, CBC, CBDP, CBDB and CBN, which has been licensed for neurological applications globally.

Pre-clinical studies of the drug have demonstrated potent anti-proliferative, anti-oxidative, anti-inflammatory and neuro-protective effects in human neuronal and microglial cells.

NTI164 is being developed as a therapeutic drug product for a range of neurological disorders in children where neuroinflammation is involved.

Productive meeting with TGA

NTI says the company received non-binding guidance on its planned clinical and regulatory development for NTI164 in PANDAS/PANS at the pre-submission meeting with the TGA.

The company says the meeting included further insights into potential provisional application criteria for rare/orphan paediatric disorders such as PANDAS/PANS, along with clinical and non-clinical requirements.

NTI says Professor of Paediatric Neurology, University of Sydney and Children’s Hospital at Westmead and Co-Principal investigator of the PANDAS/PANS clinical trial Russell Dale provided expert input into the diagnostic and treatment criteria for PANDAS/PANS to the TGA as part of the meeting.

A provisional approval pathway allows sponsors to apply for time-limited provisional registration on the Australian Register of Therapeutic Goods.

The pathway also provides access to certain promising new medicines where the TGA has made an assessment that early availability of the treatment outweighs the risk inherent in the fact that additional data are still required.

NTI says the pathway provides a formal and transparent mechanism for expediting registration of promising new medicines in Australia with preliminary clinical data for sponsors and TGA business areas.

If granted, a provisional registration can save up to two years of development and a provisionally registered prescription medicine may be able to receive reimbursement via the Pharmaceutical Benefits Scheme through a Category 1 filing and based on a positive recommendation from the Pharmaceutical Benefits Advisory Committee.

Expert advisory group to expand

Dale will coordinate a team of global experts in the field forming the advisory group. So far the experts include Stanford Medicine Department of Pediatrics – Division of Allergy, Immunology & Rheumatology Professor Jennifer Frankovich.

In 2012 the Stanford Immune Behavioral Health Clinic was established, the first multi-disciplinary PANS clinic in the world.

Head neurology service and senior consultant head at KK’s Women’s and Children’s Hospital and Singapore General Hospital Adj Assoc Prof Terrence Thomas will also join the new advisory group.

NTI says the group is expected to expand to include a leading European-based key opinion leader in PANDAS/PANS.

NTI says the group of PANDAS/PANS experts will start a Delphi process, widely used to achieve expert consensus during development of rare or orphan drugs.

The process includes input into regulatory processes including clinical trials and guidelines for decision making in clinical practice.

It’s been a month of positive announcements for NTI. Earlier this month NTI reported ASD patients in its Phase 1/2 trial of NTI164 have now been on treatment for more than two years.

Initial positive results of the Phase 1/2 ASD trial have been a catalyst for the Phase 2/3 ASD study NTI164 is a proprietary drug formulation derived from a unique cannabis strain with low THC.

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.