Peptron, NIH Establish Agreement to Develop SR-Exenatide for Neurodegenerative Diseases

Peptron recently obtained an exclusive worldwide license of intellectual property from the National Institute on Aging (NIA), a National Institutes of Health (NIH) center, covering the delivery and use of sustained-release (SR) exenatide in the treatment of Parkinson’s disease and other neurodegenerative disorders.

The intellectual property was developed as part of a cooperative research and development agreement between Korea-based Peptron and the NIH to create a form of exenatide that effectively crosses the blood-brain barrier and provides sustained release of the neuroprotective peptide to treat Parkinson’s and other degenerative diseases of the central nervous system.

“As experienced leaders in developing sustained-released therapeutics, the scientists at Peptron have enhanced the ability of SR-exenatide to cross the blood-brain barrier and deliver long-acting therapeutic effects of the neuroprotective peptide,” Ho-Il Choi, PhD, CEO and director of Peptron, said in a press release. “Neurodegenerative disorders, such as Parkinson’s and Alzheimer’s diseases, remain a great unmet medical need for millions of people around the world, and we are dedicated to innovating and advancing therapeutics for these life-altering diseases.”

The blood–brain barrier is a highly selective semipermeable membrane that separates circulating blood from the brain and extracellular fluid in the central nervous system, and allows the passage of water, some gases, and molecules.

However, large-molecule and most small-molecule drugs are not able to cross this barrier, making it challenging to deliver therapeutic agents to specific regions of the brain and treat most brain disorders.

SR exenatide is a glucagon-like peptide 1 (GLP-1) receptor agonist, which means it binds to the GLP-1 receptor, involved in controlling blood sugar levels, and promotes its action by enhancing insulin secretion.  GLP-1 receptors are found in the pancreas but also in the brain, and are used to treat Type 2 diabetes by stimulating insulin release.

According to the Cure Parkinson’s Trust, GLP-1 agonists are safe and well-tolerated medications that can mimic the action of human gut hormones, with a more stable profile than the actual hormones.

The systemic insulin resistance typically associated with Type 2 diabetes is also thought to somehow be associated with the onset of Parkinson’s disease, as patients often display impaired glucose tolerance — which then leads to brain insulin resistance.

Because of this link, there has been a growing interest in GLP-1 receptors and exenatide treatment for neurodegenerative diseases, and preclinical studies have shown evidence that exenatide may have beneficial disease-modifying effects.

However, delivering exenatide across the blood-brain barrier to the central nervous system is a major challenge.

Peptron is using part of the proceeds from a convertible bond that raised $24 million to establish a new manufacturing facility in Osang, Korea, equipped to produce SR-exenatide. Funds will also be used to support preparations for a Phase 2 clinical trial of SR-exenatide in Parkinson’s disease and evaluate the peptide in the treatment of Alzheimer’s disease.

“The support we have received from our NIH collaborators as well as our investors has allowed us to make significant progress in advancing a novel proprietary formulation of SR-exenatide to GMP manufacturing and Phase 2 clinical studies in Parkinson’s disease,” Choi said. “Moreover, our new facility enables us to manufacture additional sustained-release formulations of peptide and biologic drugs under GMP conditions to deliver further value in our collaborations with leading pharmaceutical and biotechnology companies.”