Neuraly Announces Strategic Sponsored Research Agreement with the University of Pennsylvania to Explore use of NLY01 to Therapeutically Target a Neuroinflammatory Mechanism of Glaucoma

Nov. 10, 2020 12:00 UTC

Study recently published by Penn team in Cell Reports provides evidence that NLY01 has the potential for clinical use in the treatment of glaucoma and potentially other retinal diseases

GAITHERSBURG, Md.--(BUSINESS WIRE)-- Neuraly, Inc, a clinical-stage biotechnology company pioneering disease-modifying agents for neurodegenerative disorders, today announced a strategic sponsored research agreement with the University of Pennsylvania to study the use of NLY01 to therapeutically target a neuroinflammatory mechanism of glaucoma.

Qi N. Cui, M.D., Ph.D., an assistant professor of ophthalmology in the Perelman School of Medicine at the University of Pennsylvania, will serve as the principal investigator for this project.

“We are excited to collaborate with Dr. Cui and her team to explore the role of NLY01 in the inhibition of pathological glial activation,” said Seulki Lee, Ph.D., President and Chief Executive Officer of Neuraly. “Use of NLY01 to reduce inflammation and prevent neuronal cell death offers a novel, potential strategy for the treatment of a number of diseases involving neurodegeneration. This preclinical study in glaucoma, in combination with Neuraly’s clinical trials assessing NLY01 in Parkinson’s and Alzheimer’s reflects the growing evidence that NLY01 can deliver a neuroprotective effect across a number of challenging diseases.”

Preclinical data recently published by Dr. Cui’s lab — along with collaborators Joshua Dunaief, M.D., Ph.D., a professor of ophthalmology at Penn, and MD/PhD candidate Jacob Sterling — in the peer-reviewed journal Cell Reports highlight a neuroinflammatory mechanism of glaucoma that could be therapeutically targeted by NLY01, a glucagon-like peptide 1 receptor (GLP-1R) agonist. NLY01 is being developed by Neuraly as a disease-modifying agent for neurodegenerative disorders, including Parkinson’s and Alzheimer’s diseases. A Phase 2 study of tolerability and efficacy in patients with early Parkinson's disease is ongoing.

“We hope this research will continue to advance the strong body of preclinical evidence implicating neuroinflammation in the pathogenesis of neurodegenerative diseases affecting both the eye and the brain, including glaucoma,” said Dr. Cui, the study’s lead author. “Our Cell Reports data implicate neuroinflammation as a mechanism behind neuronal damage in glaucoma and demonstrated the ability of NLY01 to reduce macrophage- and microglial-mediated conversion of astrocytes to an A1 neurotoxic phenotype in mice, thereby preventing the death of retinal ganglion cells.”

Data reported in the Cell Reports publication provide preliminary evidence that NLY01 has the potential for clinical use in the treatment of glaucoma and potentially other retinal diseases characterized by deleterious glial cell activity.

Similarly, NLY01 has been shown to reduce microglial production of C1q, TNF-α, and IL-1α in mouse models of Parkinson’s disease where A1 astrocytes contribute to dopaminergic cell death and poor motor phenotypes. The GLP-1R agonist is neuroprotective through the direct prevention of microglia-mediated A1 astrocyte transformation which results in a reduction in dopaminergic cell death and improvement of motor symptoms in a Parkinson’s disease model (Nature Medicine, Yun et al., 2018).

About NLY01

NLY01 is a proprietary long-acting analogue of exendin-4, a glucagon-like peptide-1 receptor (GLP-1R) agonist. NLY01 penetrates the blood-brain barrier (BBB) in animal models and its receptor (GLP-1R) is highly expressed on glial cells. NLY01 is being developed as a disease-modifying agent for neurodegenerative disorders including Parkinson’s and Alzheimer’s diseases. In clinically relevant animal models, NLY01 was found to prevent neuronal cell death by inhibition of microglial activation and formation of A1 neurotoxic astroglial cells. Treatment with NLY01 slowed disease progression, improved motor and cognitive functions and extended the lifespan in mice with Parkinson’s disease.

About Neuraly, Inc.

Neuraly is a clinical-stage biotechnology company whose mission is to translate scientific discoveries in neurology into new drugs that can radically improve and prolong the lives of people suffering from the devastating consequences of diseases such as Parkinson’s disease (PD), Alzheimer’s disease (AD) and other neurodegenerative disorders. The company is leveraging a deep understanding of the role of glia biology in neuroinflammation and neuroprotection to advance a risk-diversified product portfolio for PD and AD. Neuraly is a subsidiary of D&D Pharmatech, Inc., a clinical-stage global biotechnology company that funds the development of revolutionary medicines through disease-specific subsidiary companies founded and guided by top-tier medical research faculty and biotechnology veterans. Please visit

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Christine Quern
CBQ Communications


Source: Neuraly, Inc.

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