RARE Daily

ALS Association Grants Neuropore $500,000 to Explore Treatments that Inhibit Neuronal Damage

February 18, 2020

Rare Daily Staff

The ALS Association awarded Neuropore Therapies a $500,000 grant to support the preclinical evaluation and development of its lead therapy in development for the potential treatment of amyotrophic lateral sclerosis.

Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord that leads to the death of motor neurons and the inability of the brain to initiate and control muscle movement. People with ALS may lose the ability to speak, eat, move, and breathe. There are two different types of ALS: sporadic and familial or inherited ALS. Sporadic, the most common form of the disease in the United States, accounts for 90 to 95 percent of all cases.

Neuropore’s experimental therapy, NPT1220-312, is a toll-like receptor 2 antagonist (TLRs). TLRs are found on brain cells, functioning to trigger inflammation that activates the immune system in response to infection by viruses or bacteria. Research suggests that TLR2 over-activation plays a key role in driving chronic inflammation that leads to the degeneration of neurons in people with ALS and Parkinson’s disease. NPT1220-312 is a potential treatment to protect neurons from the damaging inflammation generated by TLR2 receptors.

“Neuropore Therapies is pursuing a very innovative and promising scientific path to tamp down overactive TLR2 receptors and reduce the kind of damage associated with the inflammation that leads to damaged and dying neurons,” said Kuldip Dave, vice president of research at The ALS Association.  “Early work in this area has shown promising results.”

Pending positive outcomes in ongoing preclinical studies, Neuropore plans to apply to the U.S. Food and Drug Administration by the first half of 2021 to begin human clinical trials.

“Targeting TLR2 is an exciting new approach to the treatment of ALS. NPT1220-312 is a potent, selective, orally-bioavailable TLR2 antagonist,” said Doug Bonhaus, CEO and chief science officer of Neuropore. “It has shown robust beneficial actions in cell-based assays and in animal models of ALS.”

The biotech also reported that it has successfully completed the phase 1 clinical trial in healthy volunteers with NPT520-34, its small molecule being developed for Parkinson’s disease and ALS. The study was designed to evaluate the safety, tolerability and pharmacokinetics of single and multiple doses of the experimental therapy.

Photo: Kuldip Dave, vice president of research at The ALS Association

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