Spinogenix Receives Approval from the Australia Human Research Ethics Committee to Initiate a Phase 1 Human Clinical Trial of SPG302, a Novel Regenerative Drug for the Treatment of Amyotrophic Lateral Sclerosis (ALS)

SAN DIEGO, July 10, 2023 (GLOBE NEWSWIRE) — Spinogenix, Inc., a clinical-stage biopharmaceutical company developing novel small molecule drugs for neurological conditions, today announced that it is open to enrollment having received approval from Australia’s Human Research Ethics Committee (HREC) on June 26^th to start human clinical trials with its novel drug SPG302 for the treatment of Amyotrophic Lateral Sclerosis (ALS).

SPG302 is an orally bioavailable, blood-brain barrier penetrating synthetic small molecule. It has a unique mode of action; it induces an increase in synapses, the key connections between neurons that allow people to think, plan, remember and control motor functions, faculties that are diminished in neurodegenerative diseases like ALS. The drug has demonstrated improved cognition and motor behaviors in multiple animal models of neurodegenerative disorders.

“We are extremely pleased to receive approval from the HREC to proceed with human clinical trials,” said Spinogenix Founder and Chief Executive Officer Dr. Stella Sarraf. “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. 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.”

Dr. Merit Cudkowicz, Director of the Sean M. Healey and AMG Center for ALS at Massachusetts General Hospital, commented, “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. It has the potential to be used in combination with many other treatments approved and in development.”

Phase 1 Study Overview

The first-in-human Phase 1 study will evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of SPG302 in healthy volunteers and ALS participants. The ALS cohort will include electrophysiological, biomarker, respiratory and behavioral assessments to evaluate efficacy. This study is a randomized, double-blind, placebo-controlled, single and multiple ascending dose study in healthy volunteers with food effect cohort, and a repeat dose expansion cohort(s) in participants with ALS. Additional information on the Phase 1 trial (NCT05882695), may be found on ClinicalTrials.gov.

About Spinogenix

Spinogenix was founded in 2016 with the mission to develop transformative therapeutics for diseases involving synaptic loss and dysfunction. Our drugs are designed to regenerate synapses to reverse declines in cognitive and motor function and fundamentally change treatment paradigms by restoring neuronal connections regardless of the underlying cause of synapse loss. Synapse loss is associated with a variety of neurological and psychiatric diseases, such as ALS, Alzheimer’s disease, Parkinson’s disease, schizophrenia, and depression. More information on Spinogenix can be found at www.spinogenix.com.

About ALS

Amyotrophic lateral sclerosis (ALS), also called Lou Gehrig’s disease, is a neurodegenerative disease that results in the progressive loss of motor neurons that control voluntary muscles. ALS is the most common form of motor neuron diseases. Early symptoms of ALS include stiff muscles, muscle twitches, and gradual increasing weakness and muscle wasting. Around half of people with ALS develop at least mild difficulties with thinking and behavior, and about 15% develop frontotemporal dementia. Motor neuron loss continues until the ability to eat, speak, move, and finally the ability to breathe is lost. There is no known cure for ALS. Death is usually caused by respiratory failure. The disease can affect people of any age, but usually starts around the age of 60. 

Spinogenix contact: Vincent F. Simmon, PhD, Chief Operating Officer, vince@spinogenix.com