Clinical trial of oral ALS therapy SPG302 is enrolling in Australia

First in-human study is recruiting up to 112 patients, healthy volunteers

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by Andrea Lobo |

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Spinogenix has launched a clinical trial in Australia to investigate its amyotrophic lateral sclerosis (ALS) treatment candidate SPG302 in patients and healthy volunteers.

The first-in-human Phase 1 clinical trial (NCT05882695), which is recruiting up to 112 participants, is being conducted at the Nucleus Network Melbourne, Australia. Its opening follows the approval recently by the Human Research Ethics Committee (HREC) to let Spinogenix initiate clinical studies of SPG302 in the country.

“We are extremely pleased to receive approval from the HREC to proceed with human clinical trials,” Stella Sarraf, PhD, Spinogenix’s founder and chief executive officer, said in a company press release. “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.”

A feature of ALS is the progressive dysfunction and death of motor neurons, the nerve cells that direct voluntary movements, rendering the brain unable to control them. In early disease stages, even before symptoms become evident, motor neurons exhibit a loss of synapses, sites of contact between two nerve cells that enable communication via chemical signals.

The transmission of signals from one cell to the other through synapses is what lets people maintain cognitive and motor functions, which are reduced with ALS and other neurodegenerative diseases.

“There remains an unmet need for new innovative therapeutics for ALS which is almost invariably fatal within [three to five] 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,” Sarraf said.

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What is SPG302 and what does it do?

SPG302 is an orally available small molecule designed to increase the number of synapses in nerve cells. Called a spinogenic compound due to its mechanism of action, it’s led to improvements in motor and cognitive function in animal models of neurodegenerative diseases and spinal cord injury.

The therapy can penetrate the blood-brain barrier, a highly selective membrane that prevents most molecules in the blood from reaching the brain, limiting the effectiveness of many potential therapies.

The ongoing Phase 1 trial will evaluate the safety, tolerability, and pharmacological properties of SPG302 in healthy volunteers and people with ALS.

The trial will be conducted in three parts. Part 1 will include healthy volunteers and examine the effects of a single administration of five ascending doses. A group of patients will also be studied to see if food can impact the medication’s effects.

In Part 2, five groups of healthy volunteers will receive an ascending SPG302 dose daily over five days. In Part 3, patients will receive a once daily dose for about four weeks, after which they may choose to enter an open-label extension study to probe the long-term effects of treatment.

The trial will be placebo-controlled and double-blind, meaning all the participants will be randomly assigned to receive a placebo or SPG302 and neither researchers nor participants will know who receives which.

The therapy’s efficacy, namely its effect on the brain’s electrical activity, biomarkers, respiratory measures, and functional outcomes, will also be assessed.

“We are happy to see that Spinogenix is progressing into human trials. To my knowledge, this is the first clinical trial in ALS focused on regenerating synapses with a small molecule,” Merit Cudkowicz, MD, director of the Sean M. Healey and AMG Center for ALS at Massachusetts General Hospital, said. “It has the potential to be used in combination with many other treatments approved and in development.”

SPG302 received orphan drug status by the U.S. Food and Drug Administration (FDA) for treating ALS in 2021. The designation provides certain benefits to accelerate drug development, including tax credits and seven years of market exclusivity if the therapy is approved.