Neuraly Announces First Patient Dosed in Phase 2 Clinical Trial of NLY01 for Patients with Parkinson’s Disease
Novel Mechanism of Action Inhibits Pathological Activation of Microglial Cells and Prevents Neuronal Cell Death
GERMANTOWN, MD. March 3, 2020 – Neuraly, a clinical stage biotechnology company pioneering disease-modifying agents for neurodegenerative disorders, today announced the dosing of the first patient in its Phase 2 clinical trial of NLY01 for Parkinson’s disease.
“Current therapies for Parkinson’s disease provide only temporary symptomatic improvement and fail to slow or halt the relentless progression of the disease,” said Seulki Lee, Ph.D., President and Chief Executive Officer of Neuraly. “The initiation of this Phase 2 study is an important milestone in the ongoing advancement of NLY01 which we believe has the potential to be a ground-breaking therapy for Parkinson’s patients based on results we have seen to date in preclinical trials.”
NLY01 is a glucagon-like peptide 1 receptor (GLP-1R) agonist. In a paper published in Nature Medicine, it was shown that NLY01 binds upregulated GLP-1R, blocking pathological activation of microglia in animal models of neurodegenerative diseases including Parkinson’s. The study also showed that by inhibiting glial activation and subsequent pro-inflammatory cytokine products, NLY01 prevented neuronal cell death and protected against the motor function decline that was observed in the absence of treatment.
In the Phase 1 study, NLY01 was well-tolerated, providing near continuous exposure to therapeutic doses with once-weekly dosing. The half-life of NLY01 was approximately 12.5 days and when administered weekly, achieved levels three times higher than shorter-acting GLP-1R agonists which are often limited by side effects.
“Inhibition of pathological glial activation has the potential to prevent neuronal cell death and this has the potential to lead to novel treatments for Parkinson’s and other neurodegenerative diseases,” added Viktor Roschke, Ph.D., Chief Scientific Officer of Neuraly. “Numerous attempts to develop therapies for Parkinson’s have failed, partly due to poorly understood mechanisms of neurodegeneration. Our knowledge of how and why susceptible neuronal cells in the brain die in this disease provided us with the foundation for developing NLY01. We are excited to initiate this Phase 2 study and continue to explore this new opportunity to treat Parkinson’s disease and potentially forestall the progressive decline in function that occurs with available therapies.”
The Phase 2 study is a multicenter, randomized, double-blind, placebo-controlled trial that is expected to enroll approximately 240 patients in the US and Canada. The study is designed to assess the safety, tolerability and efficacy of NLY01 in subjects with early Parkinson's disease.
More information about the study is available at www.clinicaltrials.gov under the identifier NCT04154072. Topline results are expected within 24 months.
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 disease. 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.
Neuraly is a clinical-stage company whose mission is to translate scientific discoveries in neurology into revolutionary 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 in advancing a risk-diversified product portfolio for PD and AD. Neuraly is a subsidiary of D&D Pharmatech, a clinical-stage global biotech 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