New Synapse-regenerating ALS Pill Turns to Phase 2 Trials on Humans in the US

By: | June 19th, 2024

A pioneer pill that helps to regenerate nerve cell connections damaged by ALS has received approval from the FDA for clinical trials after finishing the 1st Phase of safety in Australia. The drug is now being given to those with ALS and could mark an important point in the treatment of this fatal disease.

Amyotrophic lateral sclerosis (ALS), or Lou Gehrig’s disease, affects nerve cells in the brain and spinal cord, called motor neurons, that control muscle movement like walking, talking, and breathing. In case the neurons die and cannot send messages to the muscles, it causes loss of muscle control and this situation can worsen over time and is eventually fatal.

To date, the US Food and Drug Administration (FDA) has approved several drugs that aim to help manage symptoms or slow the disease’s progress, but no treatment reverses the progression of ALS.

In the midst of no cure for this fatal disease, a clinical-stage biopharmaceutical company named Spinogenix has developed a unique once-a-day pill called SPG302, that regenerates synapses between neurons to restore communication.

According to Stella Sarraf, the CEO and founder at Spinogenix, SPG302’s approach offers a different treatment from other existing ones. The drug focuses on synapse loss, which is also central to ALS, rather than only slowing disease progression alone.

Recently, SPG302 has completed its early-stage clinical trials in Australia with healthy adults and has proven that it’s well-tolerated.

After promising results from the Phase 1 safety study in healthy subjects in Australia, the FDA has approved the company’s Investigational New Drug (IND) application to pave the way for further trials.

According to US Federal law, if a drug wants to be transported across states, it must have an approved application. Therefore, if a drug manufacturer wants to make clinical tests in many states, they must apply to the FDA for an exception to that law, that also is how Spinogenix does it.

If SPG302 is proven effective in this next round of clinical trials, it could be an important step toward determining whether the drug helps recover lost functions in motor and cognitive symptom domains.

Ashton Henning

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