Drug firm partners with hospitals on autism treatment trials

Drug discovery firm Evgen Pharma is working with two leading hospitals on patient trials for the treatment of Autism Spectrum Disorder (ASD).

The firm, which is focused on the treatment of cancer and neurological conditions, has offices in Wilmslow and Liverpool Science Park.

It has entered into a Memorandum of Understanding with Guy’s and St Thomas’ NHS Foundation Trust to advance its lead compound SFX-01, towards a clinical trial in patients diagnosed with ASD.

SFX-01 has previously been demonstrated in small clinical trials in the United States to have potential in the treatment of ASD.

Under the terms of the agreement, Evgen has agreed to supply SFX-01 to support a potential future trial to be sponsored by Guy’s and St Thomas’ and led by Dr Michael Absoud, consultant in paediatric neurodisability at the Evelina London Children’s Hospital, which is part of Guy’s and St Thomas’.

The trust have agreed to lead the process to secure grant funding and gain clinical trial approval for a randomised, double blind, phase II clinical trial.

Evgen will retain the option to acquire, on fair commercial terms, the clinical data to enable subsequent development, regulatory approval and commercialisation of SFX-01 in ASD.

There are currently no approved medicines for treating the three core symptoms of autism – communication difficulties, social challenges and repetitive behaviour – which have long represented a huge area of unmet clinical need for affected families.

Based on its novel mode-of-action, SFX-01 has the potential to become a first-in-class treatment for the core symptoms of ASD, disrupting the current £3bn ASD market – which includes the use of anti-depressants and anti-psychotics for the treatment of non-core symptoms – making a significant impact on the enormous economic burden to the UK.

Dr Michael Absoud, a consultant in paediatric neurodisability at Evelina, said: “We are working with patient groups to plan a grant application for a phase II clinical trial programme for children with ASD, hoping to recruit from a consortium of UK hospitals.

“Our interest, and that of the parents of some children with ASD, was first triggered when a small clinical study showed that sulforaphane helped improve quality of life in autistic young people.

“Our end goal is improving the range of treatment options available for autistic young people, in order to improve quality of life and independence.”

Dr Stephen Franklin, Evgen Pharma chief executive, said: “As a result of the successful fundraise in April of this year we are now in a position to expand the toxicology package for SFX-01, which is a prerequisite to being able to support a trial in ASD patients.

“SFX-01 works by activating the Nrf2 pathway which, in turn, activates the expression of genes that promote the body’s innate capacity to control the oxidative stress and inflammation that underpin many chronic diseases.

“Targeting the Nrf2 pathway is now a recognised therapeutic strategy for treating neurodegenerative conditions, such as multiple sclerosis, but also neuropsychiatric disorders, where there is encouraging emergent data in conditions such as autism and schizophrenia.”

He added: “Supporting investigator-led studies such as this enables us to remain focused on our in-house programmes, whilst recognising that studies of this nature provide promise to new patient groups and mitigate risk for our investor base.

“We look forward to being able to provide SFX-01 to this trial and to build on the excellent relationship we have with Dr Absoud and the trust.”