ALS Association Awards Neuropore $500K to Advance Potential Anti-inflammatory Therapy

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by Catarina Silva |

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Neuropore Therapies was awarded a $500,000 grant from The ALS Association to advance in preclinical work its neural protective candidate, NPT1220-312, as a potential treatment of amyotrophic lateral sclerosis (ALS).

The company is aiming to bring its molecule into clinical testing late next year.

NPT1220-312 is a potent and selective Toll-like receptor 2 (TLR2) antagonist (inhibitor) that works to lower markers of inflammation and neurotoxic protein burden in various cells, including neurons. NPT1220-312 is one of multiple structurally diverse, small molecule TLR2 antagonists being developed to treat neurodegenerative and inflammatory disorders.

Toll-like receptors (TLRs) are best known for recognizing harmful molecular agents like viruses or bacteria, and initiating an immune response to protect the body. These receptors are found in unusually high numbers in the brains of people with neurodegenerative diseases, and participate in inflammation and motor nerve cell degeneration.

This is especially true for the TLR2 receptor, which is seen to be overactivated in people with ALS, and to mediate ALS-linked SOD1 toxicity in animal models of the disorder.

“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,” Kuldip Dave, PhD, vice president of research at The ALS Association, said in a press release.

NPT1220-312 has a good preclinical safety profile, and has shown promising effects and potent target binding properties in animals, Neuropore reports. Clinical testing, if approved, will open with safety studies in healthy volunteers and include blood markers of TLR2 binding. Later trials in patients are expected to incorporate brain imaging and possibly blood markers of inflammation.

The grant was funded the association’s Lawrence and Isabel Barnett Drug Development Program.

“We are very pleased to receive this grant from The ALS Association to facilitate the development of NPT1220-312 for the treatment of ALS. Targeting TLR2 is an exciting new approach to the treatment of ALS,” said Doug Bonhaus, PhD, chief executive officer and chief science officer of Neuropore Therapies.

“NPT1220-312 is a potent, selective, orally-bioavailable TLR2 antagonist. It has shown robust beneficial actions in cell based assays and in an animal models of ALS,” he added. “Pending positive outcomes in ongoing studies, our goal is to submit an IND [investigational new drug] application for NPT1220-312 to FDA by first half of 2021.”

Neuropore recently completed a Phase 1 safety study of NPT520-34, another small molecule thought to have protective and anti-inflammatory actions that might treat ALS. Results from this single-center trial (NCT03954600) in 49 healthy adults found the treatment safe and well-tolerated at all doses.