Spinogenix awarded $1M to advance clinical testing of SPG302

Funding will help complete more studies of therapy's safety profile

Margarida Maia, PhD avatar

by Margarida Maia, PhD |

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Spinogenix 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 amyotrophic lateral sclerosis (ALS).

This is DoD’s Congressionally Directed Medical Research Programs’ second grant to the company, following a 2021 grant to advance SPG302 studies in ALS animal models and patient-derived cells. Spinogenix will use this grant to complete additional studies on SPG302’s safety profile, as required by the U.S. Food and Drug Administration (FDA).

The small molecule is being tested in a Phase 1 clinical study (NCT05882695) in Australia that’s still recruiting 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 company press release.

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What is SPG302 and what should it do?

ALS causes certain nerve cells in the brain and spinal cord, called motor neurons, to become damaged and die, leading to symptoms such as trouble swallowing, speaking, and breathing. While treatments are available that help slow the disease’s progression, they don’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 motor and cognitive function.

“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 Alzheimer’s disease,” 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 diagnosis of ALS, 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 orphan drug status for ALS in 2021. The designation provides a number of benefits to accelerate a drug’s development, including seven years of market exclusivity if it’s ultimately approved.