A SUPER-powered WP Engine Atlas Test Site

Joe&#039;s Atlas Experimentation Site

<a href="https://spinogenix.com">Spinogenix</a> has won a nearly $1 million grant from the U.S. Department of Defense (DoD) to move ahead with clinical testing of SPG302, a small molecule candidate for <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS).
This is DoD’s Congressionally Directed Medical Research Programs’ second grant to the company, following a <a href="https://alsnewstoday.com/news/spinogenix-given-defense-department-grant-advance-oral-als-therapy-tests/">2021 grant</a> to advance SPG302 studies in ALS animal models and patient-derived cells. Spinogenix will use this grant to complete additional studies on SPG302&#8217;s safety profile, as required by the U.S. Food and Drug Administration (FDA).
The small molecule is being tested in a <a href="https://www.clinicaltrials.gov/study/NCT05882695">Phase 1 clinical study (NCT05882695)</a> in Australia that&#8217;s <a href="https://alsnewstoday.com/news/clinical-trial-of-oral-als-therapy-spg302-is-enrolling-in-australia/">still recruiting</a> up to 112 participants, including both healthy volunteers and people with ALS.
“We are extremely pleased to receive this new grant from the DoD to help us advance the development of our potentially groundbreaking ALS treatment,” Stella Sarraf, PhD, founder and CEO of Spinogenix, said in a <a href="https://www.globenewswire.com/news-release/2023/09/26/2749329/0/en/Spinogenix-Announces-Second-Grant-Award-from-U-S-Department-of-Defense-to-Further-Advance-SPG302-the-First-Synaptic-Regenerative-Drug-to-Treat-Amyotrophic-Lateral-Sclerosis-ALS.html">company press release</a>.
[recommended-reading id=81519]
<h3>What is SPG302 and what should it do?</h3>
ALS causes certain nerve cells in the brain and spinal cord, called motor neurons, to become damaged and die, leading to <a href="https://alsnewstoday.com/als-symptoms/">symptoms</a> such as trouble swallowing, speaking, and breathing. While <a href="https://alsnewstoday.com/als-treatment/">treatments are available</a> that help slow the disease&#8217;s progression, they don&#8217;t reverse it.
Nerve cells communicate with each other via specialized junctions called synapses, structures that allow neurons to pass an electrical or chemical signal to another neuron.
People with ALS experience loss of synapses very early in the course of their disease, however, which is thought to contribute to motor and cognitive symptoms.
An orally available small molecule, SPG302 is designed to increase the number of synapses in neurons. In animal models of spinal cord injury and Alzheimer’s, daily treatment with it led to improvements in <a href="https://physoc.onlinelibrary.wiley.com/doi/10.1113/JP284004">motor</a> and <a href="https://pubmed.ncbi.nlm.nih.gov/34738197/">cognitive function</a>.
“Spinogenix’s novel therapeutic SPG302 regenerates dendritic spine synapses and thus has the potential to slow and reverse the fundamental process of synaptic degeneration at work in ALS and other neurodegenerative diseases like <a href="https://alzheimersnewstoday.com/what-is-alzheimers-disease/">Alzheimer’s disease</a>,” Sarraf said.
The three-part Phase 1 clinical study is testing how safe and well tolerated SPG302 is in healthy volunteers and people with ALS. It’s also evaluating its’s effectiveness and pharmacokinetics, or how it moves into, through, and out of the body.
The first two parts include healthy volunteers, ages 18-55. In part 1, there will be five groups of eight participants, each receiving a greater dose than the group before. The participants in each group will be randomly assigned to a single dose of SPG302 (six) or a placebo (two). This part is also studying the effect of food on SPG302.
Part 2 will have a similar design, also with five groups of eight participants who each receive a greater dose than the previous group. The participants here will receive the therapy or a placebo for five consecutive days.
Part 3 includes people with a <a href="https://alsnewstoday.com/als-diagnosis/">diagnosis of ALS</a>, ages 18-80, who are randomly assigned to a placebo or SPG302 given once daily for 28 days (about four weeks), after which they may choose to enter an open-label extension where they will all receive SPG302.
The FDA granted SPG302 <a href="https://alsnewstoday.com/news/spinogenix-oral-therapy-spg302-als-awarded-fda-orphan-drug-designation/">orphan drug status</a> for ALS in 2021. The designation provides a number of benefits to accelerate a drug&#8217;s development, including seven years of market exclusivity if it&#8217;s ultimately approved.

Spinogenix awarded $1M to advance clinical testing of SPG302

<a href="https://spinogenix.com/">Spinogenix</a> has launched a clinical trial in Australia to investigate its <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS) treatment candidate SPG302 in patients and healthy volunteers.
The first-in-human <a href="https://www.clinicaltrials.gov/study/NCT05882695?term=spinogenix&amp;rank=1">Phase 1 clinical trial (NCT05882695)</a>, 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&#8217;s founder and chief executive officer, said in a <a href="https://www.globenewswire.com/news-release/2023/07/10/2701531/0/en/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-.html">company press release</a>. “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.&#8221;
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.
[recommended-reading id=81519]
<h3>What is SPG302 and what does it do?</h3>
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&#8217;s led to improvements in <a href="https://physoc.onlinelibrary.wiley.com/doi/10.1113/JP284004">motor</a> and <a href="https://pubmed.ncbi.nlm.nih.gov/34738197/">cognitive function</a> 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&#8217;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.&#8221;
SPG302 received <a href="https://alsnewstoday.com/news/spinogenix-oral-therapy-spg302-als-awarded-fda-orphan-drug-designation/">orphan drug status</a> 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.

Tag: SPG302

Spinogenix awarded $1M to advance clinical testing of SPG302

Clinical trial of oral ALS therapy SPG302 is enrolling in Australia