Spinogenix has received approval from Australia’s Human Research Ethics Committee (HREC) to start enrolling patients for its Phase I trial of SPG302 drug for the treatment of amyotrophic lateral sclerosis (ALS).

The single and multiple ascending dose, placebo-controlled, double-blind, randomised study will assess the pharmacokinetics, pharmacodynamics, tolerability and safety of SPG302 in healthy volunteers and ALS participants.

Healthy volunteers include the food effect cohort while ALS participants are the repeat dose expansion cohort.

Electrophysiological, biomarker, respiratory and behavioural assessments will be taken into consideration for the ALS cohort to evaluate efficacy.

Spinogenix founder and CEO Dr Stella Sarraf said: “There remains an unmet need for new innovative therapeutics for ALS which is almost invariably fatal within 3-5 years of diagnosis.

“The therapies that are currently approved for ALS provide only a modest extension of life and are not well tolerated by all patients.

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“These human clinical trials will help us determine safety and tolerability in healthy volunteers and provide early signals of efficacy of our novel, first-in-class drug to help improve the lives of people with ALS.”

SPG302 has also shown improvements in cognition and motor behaviours in several animal models of neurodegenerative disorders.

An orally bioavailable, blood-brain barrier penetrating synthetic small molecule, SPG302 induces an increase in synapses, the important connections between neurons that allow people to plan, think, remember as well as control motor functions.

Massachusetts General Hospital’s Sean M Healey and AMG Center for ALS director Dr Merit Cudkowicz said: “To my knowledge, this is the first clinical trial in ALS focused on regenerating synapses with a small molecule.

“It has the potential to be used in combination with many other treatments approved and in development.”

In addition to ALS, synapse loss is associated with schizophrenia, depression, Alzheimer’s disease and Parkinson’s disease.